Trial Outcomes & Findings for Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation (NCT NCT01643668)

Last Updated: 2017-07-13

Results Overview

Patients are considered to have achieved donor cell engraftment if they have an absolute neutrophil count (ANC) of at least 500 cells/uL of blood for 3 consecutive measurements and at least 75% of hematopoietic elements are donor-derived as determined by chimerism assays from peripheral blood prior to day +40 after Busulfan/Clofarabine (BuClo) reduced intensity allogeneic stem cell transplantation

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

34 participants

Primary outcome timeframe

1, 2, 3, and 4 weeks after transplantation

Results posted on

2017-07-13

Participant Flow

Participant milestones

Participant milestones
Measure	BuClo RIC + SCT BuClo RIC SCT Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Overall Study STARTED	34
Overall Study COMPLETED	33
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	BuClo RIC + SCT BuClo RIC SCT Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Overall Study Death	1

Baseline Characteristics

The 30 participants diagnosed with ALL or MDS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	BuClo RIC + SCT n=34 Participants BuClo RIC SCT Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Age, Continuous	63.5 years n=34 Participants
Sex: Female, Male Female	9 Participants n=34 Participants
Sex: Female, Male Male	25 Participants n=34 Participants
Region of Enrollment United States	34 Participants n=34 Participants
Diagnosis Acute Myeloid Leukemia (AML)	25 Participants n=34 Participants
Diagnosis Myelodysplastic Syndrome (MDS)	5 Participants n=34 Participants
Diagnosis Acute Lymphoblastic Leukemia (ALL)	4 Participants n=34 Participants
Hematopoietic Cell Transplantation (HCT) Refined Disease Risk Index (DRI) Low	1 Participants n=34 Participants
Hematopoietic Cell Transplantation (HCT) Refined Disease Risk Index (DRI) Intermediate	27 Participants n=34 Participants
Hematopoietic Cell Transplantation (HCT) Refined Disease Risk Index (DRI) High	6 Participants n=34 Participants
Disease Stage First Complete Remission (CR1)	25 Participants n=34 Participants
Disease Stage Second Complete Remission (CR2)	6 Participants n=34 Participants
Disease Stage Partial remission/active disease/blasts >5%	3 Participants n=34 Participants
Cytogenics for patients with AML/MDS Favorable	1 Participants n=30 Participants • The 30 participants diagnosed with ALL or MDS
Cytogenics for patients with AML/MDS Intermediate	24 Participants n=30 Participants • The 30 participants diagnosed with ALL or MDS
Cytogenics for patients with AML/MDS Adverse	5 Participants n=30 Participants • The 30 participants diagnosed with ALL or MDS
ALL Cytogenics Philadelphia Chromosome	2 Participants n=4 Participants • The four participants diagnosed with ALL
ALL Cytogenics Other	2 Participants n=4 Participants • The four participants diagnosed with ALL
Hematopoietic cell transplantation specific comorbidity index (HCT-CI)	1 units on a scale n=34 Participants
Donor Type Matched Related	11 Participants n=34 Participants
Donor Type Matched Unrelated	23 Participants n=34 Participants
Cytomegalovirus Serostatus Either donor or host positive for virus	22 Participants n=34 Participants
Cytomegalovirus Serostatus Both negative for virus	12 Participants n=34 Participants

PRIMARY outcome

Timeframe: 1, 2, 3, and 4 weeks after transplantation

Population: One participant experienced early death before engraftment. Outcome measure was assessed among the remaining 33 evaluable participants.

Outcome measures

Outcome measures
Measure	Treatment Arm n=33 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Assessment of Donor Stem Cell Engraftment: ANC Count Neutrophil Engraftment Achieved	33 Participants
Assessment of Donor Stem Cell Engraftment: ANC Count Neutrophil Engraftment Not Achieved	0 Participants

PRIMARY outcome

Timeframe: 1, 2, 3, 4, 8, and 14 weeks post transplant

Population: One participant experienced early death before engraftment. Outcome measure was assessed among the remaining 33 evaluable participants.

Platelet recovery was defined as having a platelet count of at least 20,000 platelets/uL of blood on 2 consecutive measurements without transfusional support prior to day +100 after BuClo RIC HSCT.

Outcome measures

Outcome measures
Measure	Treatment Arm n=33 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Donor Stem Cell Engraftment: Platelet Count Platelet Engraftmnet Achieved	33 Participants
Donor Stem Cell Engraftment: Platelet Count Platelet Engraftmnet Not Achieved	0 Participants

SECONDARY outcome

Timeframe: 100 days, 1 year

The percentage of participants that experienced non-relapse mortality (NRM) at day 100 and 1 year after BuClo RIC SCT. Non-relapse mortality is any mortality that is not associated with or proceeded by disease progression of prior cancers.

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Cumulative Incidence of Non-relapse Mortality 100 Days	5.9 percentage of participants who died Interval 1.0 to 17.4
Cumulative Incidence of Non-relapse Mortality 1 Year	24 percentage of participants who died Interval 13.0 to 39.0

SECONDARY outcome

Timeframe: 1 year, 2 years

The 1-year and 2-year progression-free and overall survival measured from the time of stem cell transplantation. Progression is the recurrence or increase in the number of cancer cells found in the body.

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Progression-Free and Overall Survival Progression Free Survival at 1 year	50 percentage of participants Interval 32.0 to 65.0
Progression-Free and Overall Survival Progression Free Survival at 2 years	50 percentage of participants Interval 32.0 to 65.0
Progression-Free and Overall Survival Overall Survival at 1 year	56 percentage of participants Interval 38.0 to 71.0
Progression-Free and Overall Survival Overall Survival at 2 years	56 percentage of participants Interval 38.0 to 71.0

SECONDARY outcome

Timeframe: 100 days

The percentage of participants who experienced grades 2-4 and grades 3-4 acute graft-versus-host disease (GVHD) by 100 days post transplantation. GVHD is a condition that can occur following an allogenic stem cell transplantation when the donated bone marrow or peripheral stem cells view the recipients body as foreign and the donated cell/marrow attack the body. Acute GVHD is generally observed within the first 100 days post transplant. Acute GVHD is associated with increased treatment related morbidity and mortality. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. The grade of the GVHD is determined by grading GHVD associated adverse events. Associated adverse events were graded using Common Terminology Criteria for Adverse Events (CTCAE) version 4 which uses the same mild, moderate, severe, life threatening grading system as the overall GHVD assessment.

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Cumulative Incidence and Severity of Acute GVHD Within 100 Days Post Transplant Grades II-IV acute GVHD	21 percentage of Participants Interval 8.9 to 35.6
Cumulative Incidence and Severity of Acute GVHD Within 100 Days Post Transplant Grades III-IV acute GVHD	12 percentage of Participants Interval 3.6 to 25.1

SECONDARY outcome

Timeframe: 1 year

The percentage of participants who experienced chronic Graft Versus Host Disease (GVHD) by one year. GVHD is a condition that can occur following an allogenic stem cell transplantation when the donated bone marrow or peripheral stem cells view the recipients body as foreign and the donated cell/marrow attack the body. Chronic GVHD normally occurs after the first 100 days post transplantation. Chronic GVHD can adversely influence long term survival.

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Cumulative Incidence of Chronic GVHD at One Year	44 percentage of participants Interval 27.0 to 60.0

SECONDARY outcome

Timeframe: 2 years

The number of participants that experienced hepatic veno-occlusive disease (VOD). VOD is a condition in which some of the small veins in the liver are obstructed.

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Incidence of Hepatic Veno-occlusive Disease	0 Participants

SECONDARY outcome

Timeframe: 2 years

The number of participants that experienced the specified grade 3 and 4 non-hematological toxicities during treatment and follow-up as assessed by Common Terminology Criteria for Adverse Events version 4(CTAE v 4.0). Grade 3 toxicity is considered to be severe and grade 4 is considered to be life threatening.

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Grade 3 or 4 Toxicities Bacterial Infection	10 Participants
Grade 3 or 4 Toxicities Viral Infection	1 Participants
Grade 3 or 4 Toxicities Fungal Infection	2 Participants
Grade 3 or 4 Toxicities Mucositis	1 Participants
Grade 3 or 4 Toxicities Increased bilirubin	1 Participants
Grade 3 or 4 Toxicities Increased alanine aminotransferase	1 Participants
Grade 3 or 4 Toxicities Increased aspartate aminotransferase	1 Participants
Grade 3 or 4 Toxicities Engraftment Syndrome	0 Participants
Grade 3 or 4 Toxicities Febrile Neutropenia	2 Participants
Grade 3 or 4 Toxicities Sepsis	1 Participants
Grade 3 or 4 Toxicities Acute Renal Failure	1 Participants
Grade 3 or 4 Toxicities Diffuse alveolar hemorrhage	0 Participants
Grade 3 or 4 Toxicities Idiopathic pneumonia syndrome	0 Participants

SECONDARY outcome

Timeframe: 2 years

The number of patients with infection-related complications

Outcome measures

Outcome measures
Measure	Treatment Arm n=34 Participants Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Infection-related Complications	0 Participants

Adverse Events

Treatment Arm

Serious events: 14 serious events

Other events: 24 other events

Deaths: 15 deaths

Serious adverse events

Serious adverse events
Measure	Treatment Arm n=34 participants at risk Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Renal and urinary disorders Acute Kidney Injury	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Alanine aminotransferase increased	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Blood and lymphatic system disorders Anemia	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Blood bilirubin increased	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Skin and subcutaneous tissue disorders Erythroderma	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Vascular disorders Hypertension	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Metabolism and nutrition disorders Hypertriglyceridemia	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Infections and infestations Lung Infection	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Neutrophil Count Decreased	8.8% 3/34 • Number of events 3 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Platelet count decreased	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	5.9% 2/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Infections and infestations Sepsis	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Respiratory, thoracic and mediastinal disorders Viral Pneumonitis	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.

Other adverse events

Other adverse events
Measure	Treatment Arm n=34 participants at risk Reduced Intensity Conditioning with Busulfan/Clofarabine followed by Allogeneic Stem Cell Transplantation (BuClo RIC SCT) Busulfan: Busulfan as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Clofarabine: Clofarabine as part of reduced intensity conditioning prior to allogeneic stem cell transplantation Allogeneic Stem Cell Infusion: Allogeneic stem cell transplantation after reduced intensity conditioning with busulfan / clofarabine chemotherapy
Gastrointestinal disorders Abdominal pain	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Activated partial thromboplastin time prolonged	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Renal and urinary disorders Acute kidney injury	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Alanine aminotransferase increased	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Gastrointestinal disorders Anal mucositis	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Blood and lymphatic system disorders Anemia	11.8% 4/34 • Number of events 7 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Metabolism and nutrition disorders Anorexia	5.9% 2/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Musculoskeletal and connective tissue disorders Arthralgia	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Aspartate aminotransferase increased	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Musculoskeletal and connective tissue disorders Back pain	5.9% 2/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Blood bilirubin increased	8.8% 3/34 • Number of events 3 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Psychiatric disorders Depression	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Gastrointestinal disorders Diarrhea	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Respiratory, thoracic and mediastinal disorders Dyspnea	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Skin and subcutaneous tissue disorders Erythroderma	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
General disorders Fatigue	2.9% 1/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Blood and lymphatic system disorders Febrile neutropenia	5.9% 2/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	11.8% 4/34 • Number of events 5 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Metabolism and nutrition disorders Glucose intolerance	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Hemoglobin increased	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Injury, poisoning and procedural complications Hip fracture	5.9% 2/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Metabolism and nutrition disorders Hyperglycemia	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Vascular disorders Hypertension	8.8% 3/34 • Number of events 3 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Metabolism and nutrition disorders Hyponatremia	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Infections and infestations Lung infection	8.8% 3/34 • Number of events 3 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Gastrointestinal disorders Mucositis oral	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Musculoskeletal and connective tissue disorders Muscle weakness lower limb	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Musculoskeletal and connective tissue disorders Myalgia	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Gastrointestinal disorders Nausea	8.8% 3/34 • Number of events 3 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Neutrophil count decreased	14.7% 5/34 • Number of events 5 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	2.9% 1/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Platelet count decreased	11.8% 4/34 • Number of events 5 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Renal and urinary disorders Renal and urinary disorders - Other, specify	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Infections and infestations Skin infection	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Vascular disorders Thromboembolic event	2.9% 1/34 • Number of events 1 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.
Investigations Weight loss	5.9% 2/34 • Number of events 2 • Duration of the study, median duration of 20 months Adverse events were assessed from the start of reduced intensity conditioning with Busulfan/Clofarabine until the participant died, relapsed/ had progressive disease, was taken off the study, or withdrew consent (median duration of 20 months). Adverse events were assessed at every study visit. Adverse events were assessed with the use of physicals/review of systems and routine laboratory tests.

Additional Information

Yi-Bin Chen, MD

Massachusetts General Hospital

Phone: 617-726-0187

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place