Hematopoietic stem cell transplantation in children and young adults with secondary myelodysplastic syndrome and acute myeloid leukemia following aplastic anemia | Aplastic Anemia and MDS International Foundation

Hematopoietic stem cell transplantation in children and young adults with secondary myelodysplastic syndrome and acute myeloid leukemia following aplastic anemia

Journal Title: 
Biol Blood Marrow Transplant
Author(s): 
Yoshimi A, Strahm B, Baumann I, Furlan I, Schwarz S, Teigler-Schlegel A, Walther JU, Schlegelberger B, Göhring G, Nöllke P, Führer M, Niemeyer CM
Primary Author: 
Yoshimi A
Original Publication Date: 
Wednesday, December 4, 2013

Secondary myelodysplastic syndrome and acute myelogenous leukemia (sMDS/sAML) are the most serious secondary events occurring after immunosuppressive therapy in patients with aplastic anemia. Here we evaluate the outcome of hematopoietic stem cell transplantation (HSCT) in 17 children and young adults with sMDS/sAML after childhood aplastic anemia. The median interval between the diagnosis of aplastic anemia and the development of sMDS/sAML was 2.9 years (range, 1.2 to 13.0 years). At a median age of 13.1 years (range, 4.4 to 26.7 years), patients underwent HSCT with bone marrow (n = 6) or peripheral blood stem cell (n = 11) grafts from HLA-matched sibling donors (n = 2), mismatched family donors (n = 2), or unrelated donors (n = 13). Monosomy 7 was detected in 13 patients. The preparative regimen consisted of busulfan, cyclophosphamide, and melphalan in 11 patients and other agents in 6 patients All patients achieved neutrophil engraftment. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 47%, and that of chronic GVHD was 70%. Relapse occurred in 1 patient. The major cause of death was transplant-related complication (n = 9). Overall survival and event-free survival at 5 years after HSCT were both 41%. In summary, this study indicates that HSCT is a curative therapy for some patients with sMDS/sAML after aplastic anemia. Future efforts should focus on reducing transplantation-related mortality.

Bone Marrow Diseases: