Trial Outcomes & Findings for Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis (NCT NCT01818492)

NCT ID: NCT01818492

Last Updated: 2023-02-21

Results Overview

Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm. CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin \<2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen \>150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25. PR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology. HI: improvement (\>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 45 participants
• Primary outcome timeframe: End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)
• Results posted on: 2023-02-21

Participant Flow

Patients were screened within 1 week prior to the first administration of emapalumab (NI-0501). 66 patients were screened, and 45 were enrolled and treated.

Participant milestones

Measure	NI-0501 All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Study STARTED	45
Overall Study COMPLETED	35
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Measure	NI-0501 All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Study Adverse Event	2
Overall Study Death	3
Overall Study Withdrawal by Subject	1
Overall Study Additional therapy when not allowed PP	4

Baseline Characteristics

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

Baseline characteristics by cohort

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Age, Continuous	1 years n=5 Participants
Age, Customized <2 years	32 Participants n=5 Participants
Age, Customized ≥ 2 - < 12 years	12 Participants n=5 Participants
Age, Customized ≥ 12 - <18 years	1 Participants n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Race/Ethnicity, Customized White	30 Participants n=5 Participants
Race/Ethnicity, Customized Asian	7 Participants n=5 Participants
Race/Ethnicity, Customized African Descent	3 Participants n=5 Participants
Race/Ethnicity, Customized Mixed/multi-racial	0 Participants n=5 Participants
Race/Ethnicity, Customized LOther	5 Participants n=5 Participants
Region of Enrollment United States	23 Participants n=5 Participants
Region of Enrollment Europe	22 Participants n=5 Participants
HLH disease chracteristics at baseline Fever	6 Participants n=5 Participants
HLH disease chracteristics at baseline Splenomegaly	27 Participants n=5 Participants
HLH disease chracteristics at baseline Platelet counts < 100 x10^9/L	31 Participants n=5 Participants
HLH disease chracteristics at baseline Neutrophil counts < 1 x10^9/L	25 Participants n=5 Participants
HLH disease chracteristics at baseline Ferritin > 500 μg/L	38 Participants n=5 Participants
HLH disease chracteristics at baseline Ferritin > 2000 μg/L	30 Participants n=5 Participants
HLH disease chracteristics at baseline Fibrinogen < 1.5 g/L	20 Participants n=5 Participants
HLH disease chracteristics at baseline Triglycerides > 3 mmol/L	29 Participants n=5 Participants
HLH disease chracteristics at baseline D-Dimers > 500 μg/L	33 Participants n=5 Participants
HLH disease chracteristics at baseline ALT > 125 IU/L	21 Participants n=5 Participants
HLH disease chracteristics at baseline Total bilirubin > 25 μmol/L	13 Participants n=5 Participants
HLH disease chracteristics at baseline LDH > 1000 IU/L	12 Participants n=5 Participants
HLH disease chracteristics at baseline CNS involvement	15 Participants n=5 Participants

PRIMARY outcome

Timeframe: End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)

Population: Primary analysis set: Second Line (27 patients who had previously received conventional HLH therapy before enrollment, data collected by regulatory cut-off: 20 July 2017)

Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.

CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.

PR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.

HI: improvement (>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).

Outcome measures

Measure	NI-0501 n=27 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Response Rate (ORR) Second Line	63.0 percentage of participants Interval 42.4 to 80.6

PRIMARY outcome

Timeframe: End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)

Population: Primary analysis set: All Treated (all patients who received any part of an emapalumab infusion, data collected by regulatory cut-off: 20 July 2017)

Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.

CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.

PR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.

HI: improvement (>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).

Outcome measures

Measure	NI-0501 n=34 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Response Rate (ORR) All Treated	64.7 percentage of participants Interval 46.5 to 80.3

PRIMARY outcome

Timeframe: End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)

Population: Follow-on analysis set: Second Line (34 patients who had previously received conventional HLH therapy before enrollment, totality of the data collected in the NI-0501-04 study)

Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.

CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.

PR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.

HI: improvement (>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).

Outcome measures

Measure	NI-0501 n=34 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Response Rate (ORR) Follow-on Analysis Set:	58.8 percentage of participants Interval 40.7 to 75.4

PRIMARY outcome

Timeframe: End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)

Population: Follow-on analysis set: All Treated (45 patients who received any part of an emapalumab infusion, totality of the data collected in the NI-0501-04)

Achievement of either Complete (CR) or Partial Response (PR), or HLH Improvement (HI) at End of Treatment of Study NI 0501-04 (EOT 04), based on pre-specified algorithm.

CR: no fever, normal spleen size, no cytopenia (ANC ≥ 1.0x109/L and platelet count ≥ 100x109/L), no hyperferritinemia (serum ferritin <2000 μg), no coagulopathy (normal D-dimer and/or fibrinogen >150 mg/dL), no neurological and CSF abnormalities attributed to HLH, no sustained worsening of sCD25.

PR: at least 3 HLH clinical and laboratory criteria (including CNS abnormalities) met the CR criteria, no progression of other aspects of HLH disease pathology.

HI: improvement (>50% change from baseline) of at least 3 HLH clinical and laboratory criteria (including CNS involvement).

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated	60.0 percentage of participants Interval 44.3 to 74.3

SECONDARY outcome

Timeframe: Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)

Population: All Treated (all patients who received any part of an emapalumab infusion)

Time from the date of the first dose of emapalumab to first achievement of response (at least HLH improvement)

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Time to Response	7.0 Count of days Interval 6.0 to 9.0

SECONDARY outcome

Timeframe: Assessed up to End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks)

Population: All Treated (all patients who received any part of an emapalumab infusion)

Maintenance of response achieved any time during the study

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Durability of First Response	58.0 Count of days Interval 39.0 to Upper limit of 95% CI is not estimable due to too few events.

SECONDARY outcome

Timeframe: Time from the date of first dose to last dose, or 8 weeks after first dose.

Population: All Treated (all patients who received any part of an emapalumab infusion)

Number of patients being alive at end of treatment or at week 8, pending on which comes first.

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Overall Survival	42 participants

SECONDARY outcome

Timeframe: End of Treatment (3 days after the last infusion of emapalumab in study NI-0501-04, occurring between 4 and 8 weeks.

Population: All Treated (all patients who received any part of an emapalumab infusion)

Number of patients able to reduce glucocorticoids by 50% or more and between ≥30%-<50%, of baseline dose at EOT 04.

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Number of Patients Able to Reduce Glucocorticoids Reduction ≥50%	46.7 Percentage of patients
Number of Patients Able to Reduce Glucocorticoids Reduction ≥30%-<50%	11.1 Percentage of patients

SECONDARY outcome

Timeframe: up to start of HSCT conditioning, whenever HSCT conditioning is scheduled (at least 4 weeks after treatment start), or End of Treatment 04/05 (if the patient did not have HSCT performed)

Population: All Treated (all patients who received any part of an emapalumab infusion)

Percent of treatment time in response from the first achievement of an Overall Response until HSCT conditioning, or End of Treatment 04/05 (if the patient did not have HSCT performed) Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05.

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Cumulative Duration of Response	78.6 Percentage of treatment time Interval 33.3 to 91.9

SECONDARY outcome

Timeframe: Assessed up to HSCT, whenever HSCT occurred (up to approximately 6 months after treatment initiation)

Population: All Treated (all patients who received any part of an emapalumab infusion)

Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients who receive HSCT will be censored at that date; patients who did not receive HSCT will be censored at last date of contact.

Where applicable, data were collected in both NI-0501-04 and long-term follow-up study NI-0501-05.

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Survival Pre-HSCT Month 3	0.83 Kaplan-Meier survival probability Interval 0.669 to 0.915
Survival Pre-HSCT Month 6	0.73 Kaplan-Meier survival probability Interval 0.524 to 0.859

SECONDARY outcome

Timeframe: Assessed up to Last Observation (up to 1 year after transplant, or up to approximately 18 months after treatment initiation)

Population: All Treated (all patients who received any part of an emapalumab infusion)

Time from the date of first dose to the date of death, expressed in Kaplan-Meier survival probability estimates. Patients without an event will be censored at last assessment date in either the NI-0501-04 or NI-0501-05 study. Patients who do not proceed to HSCT will be excluded from this analysis.

Outcome measures

Measure	NI-0501 n=45 Participants All patients received emapalumab at a starting dose of 1 mg/kg every 3 days with possible escalation up to 10 mg/kg, for a minimum of 4 weeks.
Survival Post-HSCT Month 6	0.82 Kaplan-Meier survival probability Interval 0.621 to 0.921
Survival Post-HSCT Month 12	0.82 Kaplan-Meier survival probability Interval 0.621 to 0.921

Adverse Events

Pre-conditioning

Serious events: 28 serious events

Other events: 41 other events

Deaths: 10 deaths

Post-conditioning

Serious events: 20 serious events

Other events: 28 other events

Deaths: 5 deaths

Serious adverse events

Measure	Pre-conditioning n=45 participants at risk All patients before start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation.	Post-conditioning n=30 participants at risk All patients after start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation.
Blood and lymphatic system disorders Haemolytic anaemia	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Blood and lymphatic system disorders Lymphocytosis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Blood and lymphatic system disorders Thrombotic microangiopathy	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Cardiac arrest	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Cardiac tamponade	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Cardiomyopathy	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Cardiopulmonary failure	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Right ventricular dysfunction	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Eye disorders Eye movement disorders	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Abdominal pain	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Gastrointestinal haemorrhage	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Inguinal hernia	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Pneumatosis intestinalis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Small intestinal obstruction	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Vomiting	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Condition aggravated	15.6% 7/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Multiple organ dysfunction	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Pyrexia	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Hepatobiliary disorders Autoimmune hepatitis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Acute GVHD in intestine	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Anaphylactic reaction	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Engraftment syndrome	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Septic shock	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Pneumonia	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Viral upper respiratory tract infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Enterococcal sepsis	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Gastroenteritis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Gastroenteritis norovirus	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Urosepsis	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Appendicitis perforated	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Bacterial sepsis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Gianotti-Crosti syndrome	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Histoplasmosis disseminated	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Necrotizing fasciitis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Pneumocystis jirovecii pneumonia	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Respiratory syncytial virus infection	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Staphylococcal bacteraemia	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Staphylococcal infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Clostridium difficile colitis	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Clostridium difficile infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Klebsiella sepsis	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Bronchiolitis	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Ear infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Enterococcal infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Epstein-Barr virus infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Gastroenteritis rotavirus	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Gastroenteritis viral	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Pneumonia cytomegaloviral	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Pneumonia pseudomonal	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Rhinovirus infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Sinusitis	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Injury, poisoning and procedural complications BSCT failure	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Injury, poisoning and procedural complications Engraft failure	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Injury, poisoning and procedural complications Spinal compression fracture	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Blood creatinine increased	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Failure to thrive	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hypokalaemia	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Nervous system disorders Cerebral disorders	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Nervous system disorders Haemorrhage intracranial	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Nervous system disorders Neurological decompensation	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Nervous system disorders Seizure	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Nervous system disorders Subdural hygroma	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Renal and urinary disorders Acute kidney injury	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Renal and urinary disorders Nephrolithiasis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders ARDS	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Aspiration	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Haemothorax	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Pulmonary alveolar haemorrhage	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Pulmonary artery thrombosis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Pulmonary haemorrhage	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Pulmonary hypertension	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Respiratory distress	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Respiratory failure	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Vascular disorders Circulatory collapse	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Vascular disorders Hypertension	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .

Other adverse events

Measure	Pre-conditioning n=45 participants at risk All patients before start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation.	Post-conditioning n=30 participants at risk All patients after start of conditioning. Conditioning is the treatments used to prepare a patient for stem cell transplantation.
Blood and lymphatic system disorders Lymphadenopathy	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Blood and lymphatic system disorders Lymphocytosis	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Blood and lymphatic system disorders Thrombotic microangiopathy	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	13.3% 4/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Bradycardia	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Pericardial effusion	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Cardiac disorders Tachycardia	17.8% 8/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Abdominal distension	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Abdominal pain	11.1% 5/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	13.3% 4/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Constipation	13.3% 6/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Diarrhoea	17.8% 8/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	20.0% 6/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Nausea	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Stomatitis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	20.0% 6/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Gastrointestinal disorders Vomiting	11.1% 5/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	20.0% 6/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Asthenia	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Condition aggravated	40.0% 18/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Mucosal inflammation	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	26.7% 8/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Oedema	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Oedema peripheral	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Pain	13.3% 6/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
General disorders Pyrexia	31.1% 14/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	50.0% 15/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Graft versus host disease	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Graft versus host disease in gastrointestinal tract	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Graft versus host disease in liver	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Immune system disorders Graft versus host disease in skin	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	16.7% 5/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations BK virus infection	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Clostridium difficile infection	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Cytomegalovirus infection	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	16.7% 5/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Otitis externa	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Rhinitis	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Staphylococcal bacteraemia	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Infections and infestations Upper respiratory tract infection	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Injury, poisoning and procedural complications Contusion	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Injury, poisoning and procedural complications Skin abrasion	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Adenovirus test positive	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Blood immunoglobulin G decreased	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Human rhinovirus test positive	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Polyomavirus test positive	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Transaminases increased	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Dehydration	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Fluid overload	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	13.3% 4/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hyperglycaemia	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hypoalbuminaemia	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hypocalcaemia	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hypokalaemia	13.3% 6/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	13.3% 4/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Investigations Hypomagnesaemia	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	13.3% 4/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hyponatraemia	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Metabolism and nutrition disorders Hypophosphataemia	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Musculoskeletal and connective tissue disorders Arthralgia	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Musculoskeletal and connective tissue disorders Back pain	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Psychiatric disorders Agitation	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Psychiatric disorders Irritability	11.1% 5/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Renal and urinary disorders Renal failure	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Atelectasis	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Cough	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Dyspnoea	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Epistaxis	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Hypoxia	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Pulmonary haemorrhage	0.00% 0/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Respiratory, thoracic and mediastinal disorders Tachypnoea	15.6% 7/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Drug eruption	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Erythema	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	13.3% 4/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Hyperhidrosis	8.9% 4/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	3.3% 1/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Pruritus	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Rash	15.6% 7/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	20.0% 6/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Rash erythematous	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Rash macular	6.7% 3/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	0.00% 0/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Rash maculo-papular	4.4% 2/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Skin and subcutaneous tissue disorders Skin disorder	2.2% 1/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	6.7% 2/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Vascular disorders Hypertension	42.2% 19/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	30.0% 9/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .
Vascular disorders Hypotension	11.1% 5/45 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .	10.0% 3/30 • Assessed up to Last Observation (up to 1 year after HSCT, or up to approximately 18 months after treatment initiation). Adverse Events are summarized for the NI-0501-04 and long-term follow-up NI-0501-05 studies, and analyzed separately for the pre-conditioning and post-conditioning time periods .

Additional Information

Carl Johan Treutiger

Sobi AB

Phone: 46 76 00 11 815

Email: CarlJohan.Treutiger@sobi.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place