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Biol Blood Marrow Transplant. 2014 Apr;20(4):571-6. doi: 10.1016/j.bbmt.2014.01.015. Epub 2014 Jan 22.

HLA-haploidentical T cell-depleted allogeneic hematopoietic stem cell transplantation in children with Fanconi anemia.

Author information

1
Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
2
Dipartimento di Oncoematologia Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
3
Servizio di Immunoematologia e Medicina Trasfusionale, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
4
Struttura Complessa di Radioterapia Oncologica, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
5
Dipartimento di Oncoematologia Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy; Department of Pediatric Science, Università degli Studi di Pavia, Italy. Electronic address: franco.locatelli@opbg.net.

Abstract

We report the outcome of 12 consecutive pediatric patients with Fanconi anemia (FA) who had neither an HLA-identical sibling nor an HLA-matched unrelated donor and who were given T cell-depleted, CD34(+) positively selected cells from a haploidentical related donor after a reduced-intensity, fludarabine-based conditioning regimen. Engraftment was achieved in 9 of 12 patients (75%), and the cumulative incidence of graft rejection was 17% (95% confidence interval [CI], 5% to 59%). Cumulative incidences of grades II to IV acute and chronic graft-versus-host disease were 17% (95% CI, 5% to 59%) and 35% (95% CI, 14% to 89%), respectively. The conditioning regimen was well tolerated, with no fatal regimen-related toxicity and 3 cases of grade III regimen-related toxicity. The cumulative incidence of transplant-related mortality was 17% (95% CI, 5% to 59%). The 5-year overall survival, event-free survival, and disease-free survival were 83% (95% CI, 62% to 100%), 67% (95% CI, 40% to 93%), and 83% (95% CI, 62% to 100%), respectively. These data demonstrate that a fludarabine-based conditioning regimen, followed by infusion of high doses of T cell-depleted stem cells, is able to ensure engraftment with good overall survival and disease-free survival, confirming the feasibility of haploidentical hematopoietic stem cell transplantation in FA. To the best of our knowledge, this is the largest series of hematopoietic stem cell transplantation from a haploidentical related donor in FA patients reported to date.

KEYWORDS:

Fanconi anemia; Haploidentical stem cell transplantation; Reduced-intensity conditioning; T cell depletion

PMID:
24462983
DOI:
10.1016/j.bbmt.2014.01.015
[Indexed for MEDLINE]
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