title: Early outcomes after allogeneic hematopoietic SCT in pediatric patients with hematologic malignancies following single fraction TBI.
abstract: Fractionated TBI (FTBI) followed by allogeneic hematopoietic SCT results in donor engraftment and improves survival in children with high-risk hematologic malignancies. However, acute toxicities (skin, lung and mucosa) are common after FTBI. Late complications include cataracts, endocrine dysfunction, sterility and impaired neurodevelopment. Instead of FTBI, we used low-dose single fraction TBI (550 cGy) with CY as transplant conditioning for pediatric hematologic malignancies. GVHD prophylaxis included CYA and short-course MTX; methylprednisolone was added for unrelated donor transplants. A total of 55 children in first (40%) or second remission and beyond (60%) underwent transplantation from BM (65%) or peripheral blood; 62% from unrelated donors; 22% were mismatched. Median follow-up was 18.5 months (1-68). Overall survival and disease-free survival at 1 year were 60 and 47%, respectively. Acute toxicities included grade 3-4 mucositis (18%), invasive infections (11%), multiorgan failure/shock (11%), hemolytic anemia (7%), veno-occlusive disease (4%) and renal failure (4%). TRM was 11% at 100 days. Non-relapse mortality was 6% thereafter. Graft rejection occurred in 2%. Three patients (5%) died of GVHD. The regimen was well tolerated even in heavily pretreated children and supported donor cell engraftment; long-term follow up is in progress.
authors.affiliation: Division of Pediatric Hematology and Oncology, Department of Pediatrics, Bone Marrow Transplantation and Leukemia Section, Washington University School of Medicine, One Children's Place, Saint Louis, MO 63110, USA.
authors.forename: T E
authors.initials: TE
authors.lastname: Druley
authors.forename: R
authors.initials: R
authors.lastname: Hayashi
authors.forename: D B
authors.initials: DB
authors.lastname: Mansur
authors.forename: Q Jean
authors.initials: QJ
authors.lastname: Zhang
authors.forename: Y
authors.initials: Y
authors.lastname: Barnes
authors.forename: K
authors.initials: K
authors.lastname: Trinkaus
authors.forename: S
authors.initials: S
authors.lastname: Witty
authors.forename: T
authors.initials: T
authors.lastname: Thomas
authors.forename: E E
authors.initials: EE
authors.lastname: Klein
authors.forename: J F
authors.initials: JF
authors.lastname: DiPersio
authors.forename: D
authors.initials: D
authors.lastname: Adkins
authors.forename: S
authors.initials: S
authors.lastname: Shenoy
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000276:immunology; D019337:Hematologic Neoplasms / Q000532:radiotherapy / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D009504:Neutrophils / Q000378:metabolism / Q000637:transplantation; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods; D016896:Treatment Outcome; D014916:Whole-Body Irradiation / Q000009:adverse effects / Q000379:methods*; D055815:Young Adult