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.
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.
Authors: Angela R Smith; Ellen C Christiansen; John E Wagner; Qing Cao; Margaret L MacMillan; Heather E Stefanski; Barbara A Trotz; Michael J Burke; Michael R Verneris Journal: Pediatr Blood Cancer Date: 2012-11-14 Impact factor: 3.167
Authors: Ayami Yoshimi; Marry M van den Heuvel-Eibrink; Irith Baumann; Stephan Schwarz; Ingrid Simonitsch-Klupp; Pascale de Paepe; Vit Campr; Gitte Birk Kerndrup; Maureen O'Sullivan; Rita Devito; Roos Leguit; Miguel Hernandez; Michael Dworzak; Barbara de Moerloose; Jan Stary; Henrik Hasle; Owen P Smith; Marco Zecca; Albert Catala; Markus Schmugge; Franco Locatelli; Monika Führer; Alexandra Fischer; Anne Guderle; Peter Nöllke; Brigitte Strahm; Charlotte M Niemeyer Journal: Haematologica Date: 2013-10-25 Impact factor: 9.941