Trial Outcomes & Findings for Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant (NCT NCT00343785)

NCT ID: NCT00343785

Last Updated: 2017-04-13

Results Overview

Analyzed using cumulative incidence estimates, treating death or rejection as competing risk events.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2017-04-13

Participant Flow

Participant milestones

Measure	Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis) Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant

Baseline characteristics by cohort

Measure	Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis) n=21 Participants Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Age, Continuous	15 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants
Race (NIH/OMB) More than one race	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: All Patients

Analyzed using cumulative incidence estimates, treating death or rejection as competing risk events.

Outcome measures

Measure

Patients Receive a Conditioning Regimen Comprising Cyclophosph n=21 Participants Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.

Incidence of Chronic GVHD

5 number participants with chronic GVHD

SECONDARY outcome

Timeframe: 100 days post-transplant

Population: All patients

First of 3 consecutive days of neutrophils >500/uL

Outcome measures

Measure

Patients Receive a Conditioning Regimen Comprising Cyclophosph n=21 Participants Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.

Number of Days to Neutrophil Recovery to >500/uL

26 days Interval 21.0 to 36.0

SECONDARY outcome

Timeframe: From the time of enrollment until death from any cause up to one year

Population: All Patients

Number of patients alive at one year

Outcome measures

Measure

Patients Receive a Conditioning Regimen Comprising Cyclophosph n=21 Participants Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.

Overall Survival

21 Participants

Adverse Events

Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis)

Serious events: 5 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Measure	Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis) n=21 participants at risk Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Gastrointestinal disorders parental nutrition	14.3% 3/21 • Number of events 3
Cardiac disorders hypertension	9.5% 2/21 • Number of events 2
Hepatobiliary disorders hyperbilirubinemia	4.8% 1/21 • Number of events 1
Ear and labyrinth disorders Tinnitus	4.8% 1/21 • Number of events 1
Nervous system disorders hypoxia	4.8% 1/21 • Number of events 1

Other adverse events

Measure	Treatment (Conditioning Regimen, Transplant, GVHD Prophylaxis) n=21 participants at risk Patients receive a conditioning regimen comprising cyclophosphamide IV on days -5 to -2 and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2. Patients undergo allogeneic bone marrow transplantation on day 0. Patients then receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1 hour or PO twice daily on days -1 to 50, followed by a taper until 6 months after grafting.
Gastrointestinal disorders nausea	100.0% 21/21 • Number of events 21
Gastrointestinal disorders vomiting	100.0% 21/21 • Number of events 21
Gastrointestinal disorders mucositits	100.0% 21/21 • Number of events 21

Additional Information

Dr. Rainer Storb, Director Transplantation Biology

Fred Hutchinson Cancer Research Center

Phone: 2066676839

Email: rstrob@fredhutch.org

Results disclosure agreements

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