Also see RELATED-DONOR CORD BLOOD TRANSPLANTS and REVIEWS

iii. ACQUIRED NON-MALIGNANT DISORDERS

1. Successful unrelated cord blood transplantation in a 7-year-old boy with Evans syndrome refractory to immunosuppression and double autologous stem cell transplantation. Urban C, Lackner H, Sovinz P, Benesch M, Schwinger W, Dornbusch HJ, Moser A. Eur J Haematol. 2006 Jun;76(6):526-30.

Evans syndrome is characterized by thrombocytopenia and autoimmune hemolytic anemia, often with poor response to immunosuppression. The authors describe a 2-yr-old boy with Evans syndrome whose course was remarkable in that he showed only short-lasting responses to multiple aggressive immunosuppressive treatments. His initial therapy was with IVIG and corticosteroids. Thrombocytopenia recurred and the patient was treated with combined immunosuppressive therapy including IVIG, corticosteroids, vincristine and danazole. Partial remission was achieved but he developed extremely disfiguring hypercorticism and polyneuropathy. He was next treated with cyclophosphamide (Cy) followed by mobilization, collection and immnomagnetic selection of peripheral blood stem cells. A short remission was achieved but when severe pancytopenia recurred an autologous peripheral stem cell transplant (auto-PSCT) was performed following Cy and ATG. The patient engrafted but on day +12 thrombocytopenia recurred for which a second auto-PSCT was performed following fludarabine and campath conditioning. Four months later a massive intracranial bleed occurred in the left parietal lobe for which emergency splenectomy and subsequent bilateral ventricular drainage were performed. The patient recovered surprisingly well without neurological deficits. However, thrombocytopenia recurred once again 3 weeks after the splenectomy, and this led to the decision to perform an allogeneic hematopoietic cell transplant. No matched sibling or unrelated adult donor could be found so an unrelated umbilical cord blood transplant (UCBT) was performed following conditioning of busulfan, ATG and etoposide. The UCBT was performed using a 7/10 HLA-matched female cord blood specimen which had a total of 11.7 x 10⁷ TNC/kg and 0.6 x 10⁶ CD34+ cells/kg. He engrafted promptly and platelets have subsequently remained in a range above 500 x 10⁹/L. He developed grade II aGVHD which resolved, but suffers from long-lasting feeding difficulties and prolonged immobility due to general muscular atrophy. One and one-half years after the allo-UCBT the patient is in good clinical condition although still undergoes intensive rehabilitation because of persistent muscular atrophy and immobility. Blood counts are normal.

In this case the decision for allo-SCT was made 6 months following a second auto-PSCT after a life-threatening intracranial bleeding had occurred. The authors comment that allo-SCT including UCBT might be considered earlier in children with refractory Evans syndrome to prevent further life-threatening complications. They further indicate that, to the best of their knowledge, this is the first report of a child with refractory Evans syndrome who was successfully treated with an unrelated, HLA-mismatched UCBT.