Late toxicity of a novel allogeneic stem cell transplant using single fraction total body irradiation for hematologic malignancies in children

J Pediatr Hematol Oncol. 2015 Mar;37(2):e94-e101. doi: 10.1097/MPH.0000000000000272.

Abstract

Single fraction total body irradiation (SFTBI) as part of a myeloablative preparative regimen in allogeneic hematopoietic stem cell transplantation (HSCT) for hematopoietic malignancies was shown to have similar survival compared with fractionated total body irradiation (FTBI)-containing regimens, with less acute toxicity. The objective of this study was to determine long-term toxicity >2 years following SFTBI-based HSCT. Twenty-one patients were evaluated at a median follow-up of 6.8 years. Thyroid dysfunction was found in 21% of patients, 1 of whom (5.2%) was symptomatic; 23% had gonadal failure; 50% of patients with growth potential had linear growth disturbance; 27% had mild to moderate pulmonary disease; and 25% had cataracts. Intelligence quotient was stable. cGVHD was present in 28%, and 4 patients (19%) were on immune suppression 2 years posttransplant. Overall survival subsequent to 2 years posttransplant was 76% in this cohort of patients. No secondary malignancies were observed. In conclusion, the toxicities of SFTBI occurred at similar or reduced frequency compared with FTBI. SFTBI should be considered for patients who may benefit from a radiation-containing HSCT preparative regimen.

Publication types

  • Comparative Study

MeSH terms

  • Adolescent
  • Adult
  • Antineoplastic Agents, Alkylating / adverse effects
  • Child
  • Child, Preschool
  • Combined Modality Therapy
  • Cyclophosphamide / adverse effects*
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease / diagnosis
  • Graft vs Host Disease / etiology*
  • Graft vs Host Disease / mortality
  • Hematologic Neoplasms / complications
  • Hematologic Neoplasms / mortality
  • Hematologic Neoplasms / therapy*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Humans
  • Infant
  • Male
  • Prognosis
  • Survival Rate
  • Transplantation Conditioning
  • Transplantation, Homologous
  • Whole-Body Irradiation / adverse effects*
  • Young Adult

Substances

  • Antineoplastic Agents, Alkylating
  • Cyclophosphamide