Immunosuppressive therapy with anti-thymocyte globulin and cyclosporine A in selected children with hypoplastic refractory cytopenia.
It is currently unknown whether immunosuppressive therapy or hematopoietic stem cell transplantation is the most appropriate treatment strategy for children with refractory cytopenia and normal karyotype or trisomy 8. We report on 31 children with hypoplastic refractory cytopenia treated with immunosuppressive therapy consisting of antithymocyte globulin and cyclosporine. At 6 months, 22 of 29 evaluable patients had a complete or partial response; a total of ten patients achieved a complete response at varying time points. Six patients subsequently received a transplant because of non-response, progression to advanced myelodysplastic syndrome or evolution of monosomy 7. Overall and failure-free survival rates at 3 years were 88% and 57%, respectively.
Yoshimi
Ayami
A
Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Germany.
Baumann
Irith
I
Führer
Monika
M
Bergsträsser
Eva
E
Göbel
Ulrich
U
Sykora
Karl-Walter
KW
Klingebiel
Thomas
T
Gross-Wieltsch
Ute
U
van den Heuvel-Eibrink
Marry M
MM
Fischer
Alexandra
A
Nöllke
Peter
P
Niemeyer
Charlotte
C
D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D001803:Blood Transfusion; D002648:Child; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D053208:Kaplan-Meier Estimate; D007958:Leukocyte Count; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics / Q000401:mortality / Q000601:surgery; D011241:Prednisone / Q000627:therapeutic use; D011446:Prospective Studies; D012008:Recurrence; D012074:Remission Induction; D018709:Statistics, Nonparametric; D013601:T-Lymphocytes; D016896:Treatment Outcome
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine; D011241:Prednisone