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Treatment of children with refractory anemia: The Japanese Childhood MDS Study Group trial (MDS99)
Author(s) -
Hasegawa Daisuke,
Manabe Atsushi,
Yagasaki Hiroshi,
Ohtsuka Yoshitoshi,
Inoue Masami,
Kikuchi Akira,
Ohara Akira,
Tsuchida Masahiro,
Kojima Seiji,
Nakahata Tatsutoshi
Publication year - 2009
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.22121
Subject(s) - medicine , pediatrics , neutropenia , anemia , hematopoietic stem cell transplantation , adverse effect , clinical trial , transplantation , chemotherapy
Abstract Background Although hematopoietic stem cell transplantation (HSCT) is offered as a curative therapy for pediatric myelodysplastic syndrome (MDS), it may cause severe complications and mortality. Several reports have shown the efficacy of immunosuppressive therapy (IST) in adult patients with refractory anemia (RA), but its safety and efficacy remains to be fully elucidated in childhood RA. Procedure Eleven children diagnosed with RA and enrolled on a prospective multicenter trial conducted by the Japanese Childhood MDS Study Group were eligible for analysis. If patients showed transfusion dependent or suffered from infection due to neutropenia, they received IST consisting of antithymocyte globulin (ATG), cyclosporine (CyA), and methylprednisolone (mPSL). Results Eight children received IST, 2 received only supportive therapy, and one underwent HSCT without IST. Five (63%) of eight children who received IST showed hematological response. Of note, one patient showed the disappearance of monosomy 7 after IST. Responders were significantly younger than non‐responders (29 months vs. 140 months; P = 0.03). No severe adverse events related to IST were reported in this study. Of 6 children with chromosomal abnormalities who received IST, four showed hematological response. The probability of failure‐free and overall survival at 5 years was 63 ± 17% and 90 ± 9% respectively. Conclusion IST is likely to be a safe and effective modality for childhood RA. Pediatr Blood Cancer 2009;53:1011–1015. © 2009 Wiley‐Liss, Inc.