TransIT, Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia PBMTC NMD1601
Study ID: STU 052018-057
This trial will address the feasibility of randomization, test whether patients can be evaluated in a timely fashion and safely begin therapy with MuD HSCT or iST, and give a preliminary assessment of the safety of up-front MuD HSCT. if successful, this trial will lead to a randomized prospective phase iii trial comparing directly iST to MuD HSCT in this disease.
Primary objective: To determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (iST) versus matched unrelated donor (MuD) hematopoietic stem cell transplant (HSCT) for pediatric acquired severe aplastic anemia. Secondary objectives: 1) To measure the time from screening consent and randomization of patients to initiation of the preparative regimen of those randomized to HSCT. 2) in patients who fail to receive their primary assigned therapy (HSCT or iST), to define reasons for this failure. 3) To obtain preliminary estimates of the incidence of bacteremia, viremia, and invasive fungal infection in both HSCT and iST arms, along with time to immune reconstitution in the HSCT arm. 4) To obtain preliminary estimates of treatment-related mortality and overall survival at one year from randomization in both arms. 5) To obtain preliminary estimates of the time from randomization to neutrophil recovery, platelet recovery, re[See protocol for complete text]