Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

Exclusion Criteria

• •1. 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.
• •2. Clonal cytogenetic abnormalities or fluorescence In Situ Hybridization (FISH) pattern consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (see section 4.2.3.1 for details of the required MDS FISH panel).
• •3. Known severe allergy to horse ATG.
• •4. Prior allogeneic stem cell transplant.
• •5. Prior solid organ transplant.
• •6. Infection with human immunodeficiency virus (HIV).
• •7. Active Hepatitis B or C. This should be excluded in patients where there is clinical suspicion of hepatitis (e.g. elevated LFTs).
• •8. Female patients who are pregnant or breast-feeding.
• •9. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ.