Management of acquired aplastic anemia in children

Abstract

on behalf of the SAA-WP of the EBMT The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The gold standard of therapy remains hematopoietic SCT with a graft of BM cells for those children with matched sibling donors. Conversely for children without a sibling donor the high response and markedly improved overall survival rates of combined immunosuppressive therapy have proven robust, especially when horse derived anti-thymocyte globuline plus ciclosporine A are used. Incomplete response, relapse and progression to myelodysplasia/ leukemia however have emerged as significant long-term issues. Improvements in outcome of alternative donor transplantation and the use of established and novel immunosuppressive agents provide multiple alternatives for treating refractory or relapsed patients. Regardless of the type of therapeutic approach, patients require centralized treatment in a center of excellence, ongoing monitoring for recurrence of disease and/or therapy-related immediate side effects and long-term effects.

Second allogeneic hematopoietic stem cell transplantation in children with severe aplastic anemia

“…Among the 55 patients, 16 and 38 patients suffered from 1 Department of Pediatrics, Fujita Health University School of Medicine, Toyoake, Japan; primary and secondary GF, respectively. Information on the time of GF was not available for one patient.…”

“…[1][2][3] HSCT from an HLA-matched unrelated donor (MUD) is indicated as a salvage treatment for children who are nonresponsive to immunosuppressive therapy. [4][5][6] Although the overall survival (OS) following HSCT from both MRD and MUD has markedly improved and exceeds 90%, graft failure (GF) remains the most frequent cause of HSCT failure in children aplastic anemia (AA).…”

Correlation analysis of severe aplastic anemia immunosuppressive therapy and human leukocyte antigen alleles in pediatric patients

“…In addition to failure of the bone marrow's blood-producing function, AA exhibits autoimmune disease characteristics, and an immune-mediated pathogenesis is crucially involved in the occurrence of AA (1)(2)(3)(4)(5). Allogeneic hematopoietic stem cell transplantation and immunosuppressive therapy (IST) are the two primary treatment methods for severe AA (SAA) (6)(7)(8). Stem cell transplantation is the treatment of choice for children with a human leukocyte antigen (HLA)-matched sibling donor (1).…”

Outcome of patients activating an unrelated donor search for severe acquired aplastic anemia

“…In addition to consolidate current treatment recommendations in children [29], this adds evidence that unrelated donor HSCT is, also in adults, the best option for ATG-refractory SAA. Prospective trials comparing unrelated HSCT with further immunosuppressive therapy are certainly warranted in this population of patients.…”