Low-dose (550 cGy), single-exposure total body irradiation and cyclophosphamide: consistent, durable engraftment of related-donor peripheral blood stem cells with low treatment-related mortality and fatal organ toxicity

Biol Blood Marrow Transplant. 2002;8(11):608-18. doi: 10.1053/bbmt.2002.v8.abbmt080608.

Abstract

On the basis of observations of dog models and from earlier studies with humans, we hypothesized that a low-dose (550 cGy) TBI-based conditioning regimen would result in sustained engraftment of HLA-matched sibling peripheral blood stem cells (PBSC) with low treatment-related mortality (TRM) and low serious organ toxicity if the TBI was given as a single dose and at a high dose rate. The regimen included 550 cGy TBI administered as a single dose at 30 cGy/min and cyclophosphamide. Cyclosporine was given as GVHD prophylaxis. Twenty-seven good-risk (acute leukemia in first remission and chronic-phase chronic myelogenous leukemia) and 53 poor-risk (other) patients were accrued. Complete donor engraftment occurred in 93% to 100% of evaluable patients at each scheduled assessment and was durable through 4 years. Mixed chimerism (50% to 98% donor) was observed in 9 patients (11%). Without further intervention, all patients had complete donor engraftment on subsequent assessments. Graft failure did not occur. TRM through at least 2 years was 7% in the good-risk and 19% in the poor-risk diagnostic groups. Grade 4 (fatal) organ toxicity occurred in only 2 patients (2.5%). Other causes of TRM included infection and GVHD. Median follow-up for the surviving patients was 1234 days (range, 780-1632 days). Current status includes 39 patients (49%) alive and in complete remission, 2 alive in relapse, and 39 dead. Relapse occurred in 15% of the good-risk group and 45% of the poor-risk group. The Kaplan-Meier estimates of 3-year disease-free and overall survival of the good-risk group were 77% and 85%, respectively, and of the poor-risk group were 34% and 36%, respectively. Low-dose (550 cGy), single-exposure TBI given at a high dose rate with cyclophosphamide resulted in consistent durable engraftment of HLA-matched sibling PBSC with a low risk of fatal organ toxicity and TRM.

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Cyclophosphamide / administration & dosage*
  • Cyclophosphamide / toxicity
  • Graft Survival*
  • Hematologic Neoplasms / complications
  • Hematologic Neoplasms / mortality
  • Hematologic Neoplasms / therapy
  • Histocompatibility
  • Humans
  • Middle Aged
  • Peripheral Blood Stem Cell Transplantation / adverse effects
  • Peripheral Blood Stem Cell Transplantation / methods*
  • Peripheral Blood Stem Cell Transplantation / mortality
  • Radiation Dosage
  • Tissue Donors
  • Transplantation Conditioning / adverse effects
  • Transplantation Conditioning / methods*
  • Transplantation Conditioning / mortality
  • Transplantation, Homologous
  • Treatment Outcome
  • Whole-Body Irradiation / adverse effects*
  • Whole-Body Irradiation / mortality

Substances

  • Cyclophosphamide