TBI and melphalan followed by allogeneic hematopoietic SCT in children with advanced hematological malignancies

Bone Marrow Transplant. 2011 Aug;46(8):1057-62. doi: 10.1038/bmt.2010.253. Epub 2010 Nov 1.

Abstract

We evaluated the efficacy and safety of the conditioning regimen that consisted of TBI and melphalan (L-PAM), followed by hematopoietic SCT (HSCT) in 23 children with advanced hematological malignancies. The median age at HSCT was 9 (range, 2-15) years. The underlying diseases were ALL in 16 patients (5 in CR2, 3 in CR3, 6 in relapse (RP) and 2 in induction failure (IF)), AML in 4 patients (3 in RP and 1 in IF) and non-Hodgkin's lymphoma in 3 patients (1 in CR3, 1 in CR4 and 1 in RP). The stem cell sources were BM for 19 patients and cord blood for 4 patients. All patients received the conditioning regimen that consisted of TBI 12 or 13.2 Gy and L-PAM 210 mg/m(2). In all, 22 patients engrafted on the median of day 16 (range, 10-23). The regimen was well tolerated and common regimen-related toxicities (RRTs) included grade II stomatitis and grade I hepatic toxicity. The cumulative incidences of RP and TRM were 47.6 and 21.5%, respectively. At a median follow-up of 24.4 months, the probability of disease-free survival was 41.0%. The regimen may provide sufficient anti-leukemic effect without increased RRT for advanced pediatric hematological malignancies.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Combined Modality Therapy
  • Disease-Free Survival
  • Female
  • Hematologic Neoplasms / drug therapy
  • Hematologic Neoplasms / radiotherapy
  • Hematologic Neoplasms / surgery
  • Hematologic Neoplasms / therapy*
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Male
  • Melphalan / therapeutic use*
  • Retrospective Studies
  • Transplantation Conditioning / adverse effects
  • Transplantation Conditioning / methods*
  • Whole-Body Irradiation

Substances

  • Melphalan