Long-term Survival, Organ Function, and Malignancy after Hematopoietic Stem Cell Transplantation for Fanconi Anemia - PubMed (original) (raw)

. 2016 Jul;22(7):1257-1263.

doi: 10.1016/j.bbmt.2016.03.007. Epub 2016 Mar 11.

Lisandro Ribeiro 2, Samantha Nichele 2, Marco Bitencourt 2, Gisele Loth 2, Adriana Koliski 2, Vaneuza A M Funke 2, Daniela V Pilonetto 3, Noemi F Pereira 3, Mary E D Flowers 4, Eunike Velleuer 5, Ralf Dietrich 6, Anders Fasth 7, Cassius C Torres-Pereira 8, Paola Pedruzzi 9, Mary Eapen 10, Ricardo Pasquini 2

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Long-term Survival, Organ Function, and Malignancy after Hematopoietic Stem Cell Transplantation for Fanconi Anemia

Carmem Bonfim et al. Biol Blood Marrow Transplant. 2016 Jul.

Free article

Abstract

We report on long-term survival in 157 patients with Fanconi anemia (FA) who survived 2 years or longer after their first transplantation with a median follow-up of 9 years. Marrow failure (80%) was the most common indication for transplantation. There were 20 deaths beyond 2 years after transplantation, with 12 of the deaths occurring beyond 5 years after transplantation. Donor chimerism was available for 149 patients: 112 (76%) reported > 95% chimerism, 27 (18%) reported 90% to 95% chimerism, and 8 (5%) reported 20% to 89% donor chimerism. Two patients have < 20% donor chimerism. The 10- and 15-year probabilities of survival were 90% and 79%, respectively. Results of multivariate analysis showed higher mortality risks for transplantations before 2003 (hazard ratio [HR], 7.87; P = .001), chronic graft-versus-host disease (GVHD) (HR, 3.80; P = .004) and squamous cell carcinoma after transplantation (HR, 38.17; P < .0001). The predominant cause of late mortality was squamous cell carcinoma, with an incidence of 8% and 14% at 10 and 15 years after transplantation, respectively, and was more likely to occur in those with chronic GVHD. Other causes of late mortality included chronic GVHD, infection, graft failure, other cancers, and hemorrhage. Although most patients are disease free and functional long term, our data support aggressive surveillance for long periods to identify those at risk for late mortality.

Keywords: Allogeneic transplantation; Fanconi anemia; Late effects; Survival.

Copyright © 2016 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.

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