Trial Outcomes & Findings for Modified Stem Cell Transplant Procedure to Treat Patients With Blood and Immune System Cancers (NCT NCT00051311)

Last Updated: 2021-09-09

Results Overview

Here is the number of recipients who developed grades I-IV acute graft versus host disease (GVHD) following a combination of cyclosporine and a mini-dose methotrexate regimen for graft-versus-host disease (GVHD) prophylaxis. Grades I-IV acute GVHD clinical staging was assessed by the Glucksberg Criteria. Grade I-IV acute GVHD can occur in the skin, liver, and/or gut. Grade 1: rash \< 25% body surface area (BSA), total bilirubin 2-3 mg/dl, and diarrhea 500-1000 ml/d. Grade 2: 25-50% BSA, total bilirubin 3-6 mg/dl, and diarrhea 1000-1500 ml/d. Grade 3: generalized erythroderma, total bilirubin 6-15 mg/dl, and diarrhea \>1500 ml/d. Grade 4: desquamation and bullae, total bilirubin \>15 mg/dl, and pain +/- ileus.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

62 participants

Primary outcome timeframe

At least 100 days of follow-up post reduced-intensity stem cell transplantation (RIST)

Results posted on

2021-09-09

Participant Flow

Participant milestones

Participant milestones
Measure	Donor Apheresis: Donors will undergo apheresis to collect stem cells for a stem cell transplant for the recipient.	Recipient Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Overall Study STARTED	31	31
Overall Study COMPLETED	19	17
Overall Study NOT COMPLETED	12	14

Reasons for withdrawal

Reasons for withdrawal
Measure	Donor Apheresis: Donors will undergo apheresis to collect stem cells for a stem cell transplant for the recipient.	Recipient Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Overall Study Starting new study	0	1
Overall Study Pt. to pursue treatment for progressive disease	0	1
Overall Study Death of recipient	8	8
Overall Study Recipient taken off study	4	4

Baseline Characteristics

Modified Stem Cell Transplant Procedure to Treat Patients With Blood and Immune System Cancers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Donor n=31 Participants Apheresis: Donors will undergo apheresis to collect stem cells for a stem cell transplant for the recipient.	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).	Total n=62 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	25 Participants n=5 Participants	27 Participants n=7 Participants	52 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants	4 Participants n=7 Participants	10 Participants n=5 Participants
Age, Continuous	51.98 years STANDARD_DEVIATION 12.01 • n=5 Participants	52.27 years STANDARD_DEVIATION 12.33 • n=7 Participants	52.12 years STANDARD_DEVIATION 12.07 • n=5 Participants
Sex/Gender, Customized Female	19 Participants n=5 Participants	6 Participants n=7 Participants	25 Participants n=5 Participants
Sex/Gender, Customized Male	11 Participants n=5 Participants	23 Participants n=7 Participants	34 Participants n=5 Participants
Sex/Gender, Customized Missing	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized White	26 Participants n=5 Participants	29 Participants n=7 Participants	55 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Missing	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	31 participants n=5 Participants	31 participants n=7 Participants	62 participants n=5 Participants

PRIMARY outcome

Timeframe: At least 100 days of follow-up post reduced-intensity stem cell transplantation (RIST)

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis Grade I GVHD	1 Participants
Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis Grade II GVHD	7 Participants
Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis Grade III GVHD	6 Participants
Number of Recipients Who Developed Grades I-IV Acute Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis Grade IV GVHD	0 Participants

PRIMARY outcome

Timeframe: At least 100 days of follow-up post reduced-intensity stem cell transplantation (RIST)

Here is the number of recipients who developed chronic graft versus host disease (GVHD) following a combination of cyclosporine and a mini-dose methotrexate regimen for graft-versus-host disease (GVHD) prophylaxis. Grades I-IV chronic GVHD clinical staging was assessed by the Glucksberg Criteria. Grade I-IV chronic GVHD can occur in the skin, liver, and/or gut. Grade 1: rash \< 25% body surface area (BSA), total bilirubin 2-3 mg/dl, and diarrhea 500-1000 ml/d. Grade 2: 25-50% BSA, total bilirubin 3-6 mg/dl, and diarrhea 1000-1500 ml/d. Grade 3: generalized erythroderma, total bilirubin 6-15 mg/dl, and diarrhea \>1500 ml/d. Grade 4: desquamation and bullae, total bilirubin \>15 mg/dl, and pain +/- ileus.

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Number of Recipients Who Developed Chronic Graft Versus Host Disease (GVHD) Following a Combination of Cyclosporine and a Mini-dose Methotrexate Regimen for Graft Versus Host Disease (GVHD) Prophylaxis	23 Participants

SECONDARY outcome

Timeframe: Day+14

Recipients who achieved engraftment with donor chimerism. Engraftment is defined as a target peripheral blood cluster of differentiation 4 (CD4) count of 100 cells/ul after induction therapy.

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Percentage of Recipients Who Achieved Donor Chimerism at Day +14	90 percentage of recipients

SECONDARY outcome

Timeframe: Day+100

Recipients who achieved engraftment with donor chimerism. Engraftment is defined as a target peripheral blood cluster of differentiation 4 (CD4) count of 100 cells/ul after induction therapy.

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Number of Recipients Evaluable Who Achieved Full Donor Chimerism at Day+100	29 Participants

SECONDARY outcome

Timeframe: Up to cycle 3

Median cycles of induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone given to recipients.

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Median Cycles of Induction Chemotherapy With Fludarabine, Cyclophosphamide, Etoposide, Doxorubicin, Vincristine, and Prednisone Given to Recipients	2 Cycles Interval 1.0 to 3.0

SECONDARY outcome

Timeframe: Up to 2 months after stem cell transplant

Population: The time to neutrophil recovery could not be determined precisely for 1 recipient who received granulocyte transfusions for an active infection following transplantation.

Neutrophil recovery is defined as a neutrophil count ≥5000 µl for three consecutive days.

Outcome measures

Outcome measures
Measure	Recipient n=30 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Median Time to Neutrophil Recovery	14 Days Interval 7.0 to 21.0

SECONDARY outcome

Timeframe: Up to 2 months after stem cell transplant

Population: Six recipients were not evaluable for platelet recovery \> 20K because their platelet count never fell below that level (n=2) or because they were receiving aggressive platelet transfusions (n=4).

Platelet recovery is defined as \>50,000 mm3 platelet cell count after transfusion.

Outcome measures

Outcome measures
Measure	Recipient n=25 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Median Time to Platelet Recovery	13 Days Interval 9.0 to 34.0

SECONDARY outcome

Timeframe: At least 100 days after post reduced-intensity stem cell transplantation (RIST).

Complete remission (CR) is complete disappearance of all detectable signs and symptoms of lymphoma for a period of at least one month. Complete remission unconfirmed (CRu) is a residual lymph node mass \> 1.5 cm in greatest transverse diameter will be considered CRu if it has regressed by more than 75% in the SPD, does not change over at least one month, is negative by PET or gallium, and is negative on any biopsies obtained (biopsy not required). Progressive disease (PD) is a 25% or greater increase in SPD of all measured lesions compared to the smallest previous measurements, or appearance of any new lesion(s).

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Number of Recipients With a Response to Reduced-intensity Stem Cell Transplantation (RIST) Complete Remission or Complete Remission Unconfirmed	15 Participants
Number of Recipients With a Response to Reduced-intensity Stem Cell Transplantation (RIST) Progressive Disease	16 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Date treatment consent signed to date off study, approximately 58 months and 13 days.

Population: Adverse events data were only collected for the recipients.

Here is the number of recipients with non-serious adverse events assessed by the Common Toxicity Criteria version 2.0. A non-serious adverse event is any untoward medical occurrence.

Outcome measures

Outcome measures
Measure	Recipient n=31 Participants Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Number of Recipients With Non-serious Adverse Events	31 Participants

Adverse Events

Recipient

Serious events: 0 serious events

Other events: 31 other events

Deaths: 11 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Recipient n=31 participants at risk Recipients will receive induction chemotherapy with fludarabine, cyclophosphamide, etoposide, doxorubicin, vincristine, and prednisone. (one cycle is 5 days on drug therapy followed by a 16 day rest period). Prior to the transplant procedure, recipients will receive conditioning therapy followed by the infusion of donor stem cells. Methotrexate: Recipients will receive 4 doses of methotrexate by vein after the transplant to help prevent graft-versus-host disease (GVHD). Cyclosporine: Recipients will receive cyclosporine by vein or by mouth for about 6 months after the transplant to help prevent graft-versus-host disease (GVHD).
Gastrointestinal disorders Abdominal pain or cramping	25.8% 8/31 • Number of events 10 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Vascular disorders Acute vascular leak syndrome	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Alkaline phosphatase	3.2% 1/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Immune system disorders Allergic reaction/hypersensitivity (including drug fever)	6.5% 2/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Immune system disorders Allergy-Other	12.9% 4/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Amylase	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Anorexia	22.6% 7/31 • Number of events 9 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Cardiac disorders Arrhythmia - other	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Musculoskeletal and connective tissue disorders Arthralgia (joint pain)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Ascites (non-malignant)	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders Ataxia (incoordination)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Immune system disorders Autoimmune reaction	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Bilirubin	71.0% 22/31 • Number of events 47 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Bilirubin-graft versus host disease (GVHD)	6.5% 2/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Renal and urinary disorders Bladder spasms	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Musculoskeletal and connective tissue disorders Bone pain	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Carbon monoxide diffusion capacity (DLCO)	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Cardiac disorders Cardiac left ventricular function	9.7% 3/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Eye disorders Cataract	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Catheter-related infection	35.5% 11/31 • Number of events 15 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Musculoskeletal and connective tissue disorders Chest pain (non-cardiac and non-pleuritic)	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders CNS hemorrhage/bleeding	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders Cognitive disturbance/learning problems	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Colitis	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Psychiatric disorders Confusion	19.4% 6/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Eye disorders Conjunctivitis	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Constipation	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
General disorders Constitutional symptoms - Other	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Cough	9.7% 3/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations CPK (creatine phosphokinase)	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Creatinine	35.5% 11/31 • Number of events 15 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Diarrhea (without colostomy)	51.6% 16/31 • Number of events 27 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Diarrhea for BMT	29.0% 9/31 • Number of events 11 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders DIC (disseminated intravascular coagulation)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Eye disorders Dry eye	12.9% 4/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Dysphagia, esophagitis, odynophagia (painful swallowing)	9.7% 3/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Renal and urinary disorders Dysuria (painful urination)	12.9% 4/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
General disorders Edema	19.4% 6/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Endocrine disorders Endocrine-Other	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Skin and subcutaneous tissue disorders Erythema multiforme (e.g., Stevens-Johnson syndrome, toxic epidermal necrolysis	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
General disorders Fatigue (lethargy, malaise, asthenia)	19.4% 6/31 • Number of events 6 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders Febrile neutropenia	67.7% 21/31 • Number of events 30 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations FEV1	12.9% 4/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
General disorders Fever (in absence of neutropenia, where neutropenia is defined as AGC<1.0x109/L)	77.4% 24/31 • Number of events 55 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Gastritis	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders GI-Other	9.7% 3/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders Headache	9.7% 3/31 • Number of events 6 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Renal and urinary disorders Hematuria (in the absence of vaginal bleeding)	22.6% 7/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Hemoglobin (Hgb)	90.3% 28/31 • Number of events 54 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Hemoptysis	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders Hemorrhage/bleeding with grade 3 or 4 thrombocytopenia	16.1% 5/31 • Number of events 5 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders Hemorrhage/bleeding without grade 3 or 4 thrombocytopenia	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Hepatobiliary disorders Hepatic-Other	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Hyperglycemia	25.8% 8/31 • Number of events 12 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Hyperkalemia	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Vascular disorders Hypertension	19.4% 6/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Hyperuricemia	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Hypoalbuminemia	25.8% 8/31 • Number of events 8 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Hypokalemia	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Metabolism and nutrition disorders Hyponatremia	22.6% 7/31 • Number of events 11 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Vascular disorders Hypotension	29.0% 9/31 • Number of events 11 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Hypoxia	38.7% 12/31 • Number of events 27 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia	71.0% 22/31 • Number of events 38 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Infection with unknown ANC	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Infection without neutropenia	74.2% 23/31 • Number of events 86 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Infection, Other	45.2% 14/31 • Number of events 27 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Musculoskeletal and connective tissue disorders Joint, muscle, or bone (osseous)-Other	6.5% 2/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Eye disorders Keratitis (corneal inflammation/corneal ulceration)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Leukocytes (total WBC)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders Lymphatics	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Melena/GI bleeding	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Ear and labyrinth disorders Middle ear/hearing	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Psychiatric disorders Mood alteration-depression	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Mouth dryness	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Mucositis due to radiation	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Musculoskeletal and connective tissue disorders Muscle weakness (not due to neuropathy)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Musculoskeletal and connective tissue disorders Myalgia (muscle ache)	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Myositis (inflammation/damage of muscle)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Nausea	25.8% 8/31 • Number of events 11 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders Neurologic-Other	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders Neuropathy motor	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Neutrophils/granulocytes (ANC/AGC)	90.3% 28/31 • Number of events 58 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Neutrophils/granulocytes (ANC/AGC) for BMT	100.0% 31/31 • Number of events 36 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Eye disorders Ocular-Other	22.6% 7/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
General disorders Pain-Other	9.7% 3/31 • Number of events 3 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Platelets	77.4% 24/31 • Number of events 49 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Platelets for BMT	90.3% 28/31 • Number of events 33 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Pleural effusion (non-malignant)	22.6% 7/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Pleuritic pain	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Pneumonitis/pulmonary infiltrates	25.8% 8/31 • Number of events 12 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Cardiac disorders Pericardial effusion/pericarditis	9.7% 3/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Respiratory, thoracic and mediastinal disorders Pulmonary-Other	16.1% 5/31 • Number of events 6 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Injury, poisoning and procedural complications Radiation recall reaction	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Skin and subcutaneous tissue disorders Rash/desquamation	87.1% 27/31 • Number of events 49 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Skin and subcutaneous tissue disorders Rash-desquamation for BMT	41.9% 13/31 • Number of events 19 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Renal and urinary disorders Renal/GU-Other	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Secondary Malignancy-Other	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Nervous system disorders Seizure(s)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations SGOT (AST)	54.8% 17/31 • Number of events 36 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations SGPT (ALT)	58.1% 18/31 • Number of events 37 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Skin and subcutaneous tissue disorders Skin-Other	9.7% 3/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Stomatitis/pharyngitis (oral/pharyngeal mucositis)	29.0% 9/31 • Number of events 13 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Infections and infestations Stomatitis/pharyngitis (oral/pharyngeal mucositis) for BMT	22.6% 7/31 • Number of events 9 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Cardiac disorders Supraventricular arrhythmias (SVT/atrial fibrillation/flutter)	16.1% 5/31 • Number of events 5 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Vascular disorders Thrombosis/embolism	12.9% 4/31 • Number of events 6 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders Transfusion: Platelets	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Blood and lymphatic system disorders Transfusion: pRBCs	16.1% 5/31 • Number of events 7 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Renal and urinary disorders Ureteral obstruction	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Renal and urinary disorders Urinary frequency/urgency	12.9% 4/31 • Number of events 4 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Cardiac disorders Ventricular arrhythmia (PVCs/bigeminy/trigeminy/ventricular tachycardia)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Ear and labyrinth disorders Vertigo	6.5% 2/31 • Number of events 2 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Eye disorders Vision-blurred vision	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Gastrointestinal disorders Vomiting	22.6% 7/31 • Number of events 9 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Weight gain	19.4% 6/31 • Number of events 6 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Weight gain-veno-occlusive disease (VOD)	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.
Investigations Weight loss	3.2% 1/31 • Number of events 1 • Date treatment consent signed to date off study, approximately 58 months and 13 days. Adverse events data were only collected for the recipients.

Additional Information

Dr. Ronald Gress

National Cancer Institute

Phone: 240-760-6167

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place