Treatment of children with refractory anemia: the Japanese Childhood MDS Study Group trial (MDS99).
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.
Hasegawa
Daisuke
D
Department of Pediatrics, St. Luke's International Hospital, Tokyo, Japan. hase-dai@umin.net
Manabe
Atsushi
A
Yagasaki
Hiroshi
H
Ohtsuka
Yoshitoshi
Y
Inoue
Masami
M
Kikuchi
Akira
A
Ohara
Akira
A
Tsuchida
Masahiro
M
Kojima
Seiji
S
Nakahata
Tatsutoshi
T
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
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D008775:Methylprednisolone