title: Clinical characteristics and treatment outcome in 65 cases with refractory cytopenia of childhood defined according to the WHO 2008 classification.
abstract: This study analysed 65 children who were prospectively registered between 1999 and 2008 and fulfilled the World Health Organization 2008 criteria of refractory cytopenia of childhood (RCC). First-line therapy was determined by the treating physicians: 25 patients received immunosuppressive therapy (IST), 12 patients received haematopoietic stem cell transplantation (HSCT) and one patient received intensive chemotherapy. The remaining 27 patients were followed without treatment for more than 2 years (watch and wait; WW). In the WW group, 18 patients had stable disease without further intervention. Thirteen of 29 patients (45%) who ended up receiving IST showed response. The combination of ciclosporin and antithymocyte globulin was not shown to be superior to ciclosporin alone with regard to response rate or survival. Of 28 patients who ended up undergoing HSCT, 17 patients are alive in complete remission, whereas nine patients died mostly due to transplantation-related mortality. The 5-year overall survival for all patients was 82 ± 5%. Eight patients suffered from disease progression. Patients with monosomy 7 or multilineage-dysplasia had a significantly higher incidence of disease progression. This analysis revealed heterogeneity in the clinical course of RCC, varying from those who remained stable for long periods to those who progressed to advanced disease.
authors.affiliation: Department of Paediatrics, St. Luke's International Hospital, Tokyo, Japan.
authors.forename: Daisuke
authors.initials: D
authors.lastname: Hasegawa
authors.forename: Xiaojuan
authors.initials: X
authors.lastname: Chen
authors.forename: Shinsuke
authors.initials: S
authors.lastname: Hirabayashi
authors.forename: Yasushi
authors.initials: Y
authors.lastname: Ishida
authors.forename: Shizuka
authors.initials: S
authors.lastname: Watanabe
authors.forename: Yuji
authors.initials: Y
authors.lastname: Zaike
authors.forename: Masahiro
authors.initials: M
authors.lastname: Tsuchida
authors.forename: Atsuko
authors.initials: A
authors.lastname: Masunaga
authors.forename: Ayami
authors.initials: A
authors.lastname: Yoshimi
authors.forename: Asahito
authors.initials: A
authors.lastname: Hama
authors.forename: Seiji
authors.initials: S
authors.lastname: Kojima
authors.forename: Masafumi
authors.initials: M
authors.lastname: Ito
authors.forename: Tatsutoshi
authors.initials: T
authors.lastname: Nakahata
authors.forename: Atsushi
authors.initials: A
authors.lastname: Manabe
mesh_terms: D000293:Adolescent; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D018450:Disease Progression; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009190:Myelodysplastic Syndromes / Q000628:therapy*; D010198:Pancytopenia / Q000628:therapy*; D011446:Prospective Studies; D016896:Treatment Outcome; D014944:World Health Organization
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016572:Cyclosporine
keywords: disease progression; haematopoietic stem cell transplantation; immunosuppressive therapy; myelodysplastic syndrome; refractory cytopenia of childhood