title: Allogeneic bone marrow transplantation in juvenile myelomonocytic leukemia without total body irradiation.
abstract: Allogeneic bone marrow transplantation (BMT) without a total body irradiation (TBI) conditioning regimen was investigated in children with juvenile myelomonocytic leukemia (JMML). Eight consecutive patients with JMML (n = 6) or monosomy 7 (n = 2) underwent BMT at a median age of 20 months. Donor source included fully matched related (n = 3), mismatched related (n = 2), or fully matched unrelated (n = 3). The conditioning regimen included busulfan, cyclophosphamide, and etoposide (VP16) (melphalan was substituted for VP16 in one patient). The first patient in the series underwent TBI. Graft-versus-host disease prophylaxis was with cyclosporin and methotrexate and in vivo T-cell depletion (Campath 1 g) for mismatched and unrelated transplants. Seven and two patients, respectively, received chemotherapy and splenectomy before BMT. At a median follow-up of 48 months after BMT, five patients remained in remission. The overall survival rate was 63% at 5 years. All deaths occurred in patients with refractory disease at the time of BMT. Allogeneic BMT without TBI appears to be effective therapy for JMML and avoids some of the potential late sequelae of TBI in preschool children.
authors.affiliation: Department of Hematology, Princess Margaret Hospital, GPO Box D184, Perth, Western Australia 6001. David.Baker@health.wa.gov.au
authors.forename: David
authors.initials: D
authors.lastname: Baker
authors.forename: Catherine
authors.initials: C
authors.lastname: Cole
authors.forename: Jamie
authors.initials: J
authors.lastname: Price
authors.forename: Marianne
authors.initials: M
authors.lastname: Phillips
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation / Q000379:methods*; D002675:Child, Preschool; D003131:Combined Modality Therapy; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D015479:Leukemia, Myelomonocytic, Acute / Q000188:drug therapy / Q000401:mortality / Q000601:surgery*; D013156:Splenectomy; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents