Faculty Collaboration Database

Long-term Survival, Organ Function, and Malignancy after Hematopoietic Stem Cell Transplantation for Fanconi Anemia. Biol Blood Marrow Transplant 2016 Jul;22(7):1257-1263

Date

03/16/2016

Pubmed ID

26976241

DOI

10.1016/j.bbmt.2016.03.007

Scopus ID

2-s2.0-84964339368 (requires institutional sign-in at Scopus site)   54 Citations

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.

Author List

Bonfim C, Ribeiro L, Nichele S, Bitencourt M, Loth G, Koliski A, Funke VAM, Pilonetto DV, Pereira NF, Flowers MED, Velleuer E, Dietrich R, Fasth A, Torres-Pereira CC, Pedruzzi P, Eapen M, Pasquini R

Author

Mary Eapen MBBS, DCh, MRCPI, MS Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Carcinoma, Squamous Cell
Child
Child, Preschool
Chimerism
Fanconi Anemia
Female
Follow-Up Studies
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Humans
Infant
Longitudinal Studies
Male
Risk Factors
Survival Analysis
Young Adult