A two-step approach to myeloablative haploidentical transplantation: Low nonrelapse mortality and high survival confirmed in patients with earlier stage disease

Dolores Grosso*, Sameh Gaballa, Onder Alpdogan, Matthew Carabasi, Joanne Filicko-O'Hara, Margaret Kasner, Ubaldo Martinez-Outschoorn, John L. Wagner, William O'Hara, Shannon Rudolph, Inna Chervoneva, Beth Colombe, Patricia Cornett Farley, Phyllis Flomenberg, Barbara Pro, Manish Sharma, Wenyin Shi, Mark Weiss, Neal Flomenberg

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

31 Scopus citations

Abstract

Haploidentical hematopoietic stem cell transplantation (HSCT) is an attractive alternative donor option based on the rapid availability of an acceptable donor for most patients and decreased cost compared with costs of other alternative donor strategies. The safety of haploidentical HSCT has increased in recent years, making it ethically feasible to offer to patients with earlier stage disease. We developed a 2-step approach to haploidentical HSCT that separates the lymphoid and myeloid portions of the graft, allowing fixed T cell dosing to improve consistency in outcome comparisons. In the initial 2-step trial, the subset of patients without morphologic disease at HSCT had high rates of disease-free survival. To confirm these results, 28 additional patients without evidence of their disease were treated and are now 15 to 45 (median, 31) months past HSCT. To date, the 2-year cumulative incidence of nonrelapse mortality is 3.6%, with only 1 patient dying of nonrelapse causes, confirming the safety of this approach. Based on low regimen toxicity, the probabilities of disease-free and overall survival at 2years are 74% and 77%, respectively, consistent with the findings in the initial trial and supporting the use of this approach in earlier stage patients lacking a matched related donor.

Original languageEnglish (US)
Pages (from-to)646-652
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Volume21
Issue number4
DOIs
StatePublished - Apr 1 2015

Keywords

  • Disease-free survival
  • Donor availability
  • Haploidentical stem cell transplantation
  • Nonrelapse mortality
  • T cell dosing

ASJC Scopus subject areas

  • Hematology
  • Transplantation

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