TY - JOUR
T1 - A two-step approach to myeloablative haploidentical transplantation
T2 - Low nonrelapse mortality and high survival confirmed in patients with earlier stage disease
AU - Grosso, Dolores
AU - Gaballa, Sameh
AU - Alpdogan, Onder
AU - Carabasi, Matthew
AU - Filicko-O'Hara, Joanne
AU - Kasner, Margaret
AU - Martinez-Outschoorn, Ubaldo
AU - Wagner, John L.
AU - O'Hara, William
AU - Rudolph, Shannon
AU - Chervoneva, Inna
AU - Colombe, Beth
AU - Farley, Patricia Cornett
AU - Flomenberg, Phyllis
AU - Pro, Barbara
AU - Sharma, Manish
AU - Shi, Wenyin
AU - Weiss, Mark
AU - Flomenberg, Neal
N1 - Publisher Copyright:
© 2015 American Society for Blood and Marrow Transplantation.
Copyright:
Copyright 2016 Elsevier B.V., All rights reserved.
PY - 2015/4/1
Y1 - 2015/4/1
N2 - 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.
AB - 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.
KW - Disease-free survival
KW - Donor availability
KW - Haploidentical stem cell transplantation
KW - Nonrelapse mortality
KW - T cell dosing
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UR - http://www.scopus.com/inward/citedby.url?scp=84924225098&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2014.12.019
DO - 10.1016/j.bbmt.2014.12.019
M3 - Article
C2 - 25542159
AN - SCOPUS:84924225098
VL - 21
SP - 646
EP - 652
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 4
ER -