Al-Ahmari et al. (2004) performed stem cell transplantation (SCT) from HLA-matched related donors in five patients with confirmed congenital amegakaryocytic thrombocytopenia (CAT). The median age at SCT was 3.2 years (range, 0.4-5 years). Conditioning regimen consisted of busulfan (BU) and cyclophosphamide (CY). Antithymocyte globulin (ATG) was also given; graft-versus-host disease (GVHD) prophylaxis was with cyclosporine and methotrexate. Four of the patients engrafted survived and became transfusion independent with a median follow up time of 30 months (range, 16-45 months). One patient failed to engraft and underwent a second SCT four months later but died of respiratory failure.

Revesz et al. (1993) reviewed the occurrence of rare congenital forms of aplastic anemias in the United Arab Emirates and concluded that amegakaryocytic thrombocytopenia occurs exceptionally rarely in the population.