title: Treatment of children with refractory anemia: the Japanese Childhood MDS Study Group trial (MDS99).
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.
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  IST is likely to be a safe and effective modality for childhood RA.
authors.affiliation: Department of Pediatrics, St. Luke's International Hospital, Tokyo, Japan. hase-dai@umin.net
authors.forename: Daisuke
authors.initials: D
authors.lastname: Hasegawa
authors.forename: Atsushi
authors.initials: A
authors.lastname: Manabe
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Yagasaki
authors.forename: Yoshitoshi
authors.initials: Y
authors.lastname: Ohtsuka
authors.forename: Masami
authors.initials: M
authors.lastname: Inoue
authors.forename: Akira
authors.initials: A
authors.lastname: Kikuchi
authors.forename: Akira
authors.initials: A
authors.lastname: Ohara
authors.forename: Masahiro
authors.initials: M
authors.lastname: Tsuchida
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
authors.forename: Tatsutoshi
authors.initials: T
authors.lastname: Nakahata
mesh_terms: D000293:Adolescent; D000753:Anemia, Refractory / Q000150:complications / Q000401:mortality / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D002869:Chromosome Aberrations; D016572:Cyclosporine / Q000008:administration & dosage; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000009:adverse effects; D007223:Infant; D008297:Male; D008775:Methylprednisolone / Q000008:administration & dosage; D009503:Neutropenia / Q000188:drug therapy / Q000209:etiology; D012074:Remission Induction; D016019:Survival Analysis
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D008775:Methylprednisolone