Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

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  • sponsor
    Michael Pulsipher, MD
Updated on 8 August 2021
stem cell transplantation
total body irradiation
antithymocyte globulin
neutrophil count
aplastic anemia
reticulocyte count
horse anti-thymocyte globulin
rabbit atg


The purpose of this study is 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.


A major challenge in treating pediatric Severe Aplastic Anemia (SAA) is the determination of best primary therapy for patients who lack a fully matched related donor for HSCT. Good survival outcomes have been seen with IST, but initial and late failures, CSA dependence, persistent cytopenias and secondary Myelodysplastic Syndrome (MDS) / Acute Myeloid Leukemia (AML) in a portion of patients leave considerable room for improvement. MUD HSCT survival in SAA has markedly improved, but a direct comparison of this approach with IST is necessary to determine whether this approach is feasible and will lead to better Event Free Survival. 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 future prospective trial comparing directly IST to MUD HSCT in this disease.

Condition Severe Aplastic Anemia
Treatment cyclophosphamide, cyclosporine, methotrexate, Fludarabine, Horse Anti-Thymocyte Globulin (ATG), Matched Unrelated Donor Hematopoietic Stem Cell Transplant, rabbit anti-thymocyte globulin (ATG), low-dose total body irradiation (TBI), Immunosuppressive Therapy (IST)
Clinical Study IdentifierNCT02845596
SponsorMichael Pulsipher, MD
Last Modified on8 August 2021


Yes No Not Sure

Inclusion Criteria

Confirmed diagnosis of idiopathic SAA, defined as
Bone marrow cellularity <25%, or <30% hematopoietic cells
Two out of three of the following (in peripheral blood): neutrophils <0.5 x109/L, platelets <20 x109/L, reticulocyte count <60 x109/L with hemoglobin <8g/dL
Age 25 years old
No suitable fully matched related donor available (minimum 6/6 match for Human Leukocyte antigen (HLA) -A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing)
At least two unrelated donors noted on National Marrow Donor Program (NMDP) search who are well matched (9/10 or 10/10 for HLA-A, B, C, DRB1, and DQB1 using high resolution)
Signed informed consent for the randomized trial by patient and/or legal guardian
Adequate organ function defined as in the judgment of the investigator, there is not irreversible organ damage that would preclude the patient from meeting the organ function inclusion criteria for HSCT listed in section 2.3.4 by the intended time of HSCT (6-8 weeks after randomization) or preclude patients from receiving horse ATG

Exclusion Criteria

Inherited bone marrow failure syndromes (IBMFS). The diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow. Telomere length testing should be sent on all patients to exclude Dyskeratosis congenita, but if results are delayed or unavailable and there are no clinical manifestations of DC, patients may enroll. If patients have clinical characteristics suspicious for Shwachman Diamond syndrome, this syndrome must be excluded by pancreatic isoamylase testing or gene mutation analysis. Note: pancreatic isoamylase testing is not accurate in children less than 3 years
Clonal cytogenetic abnormalities or fluorescence In Situ Hybridization (FISH) pattern consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (see section [](telnet:// for details of the required MDS FISH panel)
Known severe allergy to horse ATG
Prior allogeneic stem cell transplant
Prior solid organ transplant
Infection with human immunodeficiency virus (HIV)
Active Hepatitis B or C. This should be excluded in patients where there is clinical suspicion of hepatitis (e.g. elevated LFTs)
Female patients who are pregnant or breast-feeding
Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ
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