Late effects in patients with Fanconi anemia following allogeneic hematopoietic stem cell transplantation from alternative donors. Academic Article uri icon

Overview

abstract

  • Hematopoietic stem cell transplantation (HSCT) is curative for hematological manifestations of Fanconi anemia (FA). We performed a retrospective analysis of 22 patients with FA and aplastic anemia, myelodysplastic syndrome or acute myelogenous leukemia who underwent a HSCT at Memorial Sloan Kettering Cancer Center and survived at least 1 year post HSCT. Patients underwent either a TBI- (N=18) or busulfan- (N=4) based cytoreduction followed by T-cell-depleted transplants from alternative donors. Twenty patients were alive at time of the study with a 5- and 10-year overall survival of 100 and 84% and no evidence of chronic GvHD. Among the 18 patients receiving a TBI-based regimen, 11 (61%) had persistent hemochromatosis, 4 (22%) developed hypothyroidism, 7 (39%) had insulin resistance and 5 (27%) developed hypertriglyceridemia after transplant. Eleven of 16 evaluable patients (68%), receiving TBI, developed gonadal dysfunction. Two patients who received a TBI-based regimen died of squamous cell carcinoma. One patient developed hemochromatosis, hypothyroidism and gonadal dysfunction after busulfan-based cytoreduction. TBI appears to be a risk factor for malignant and endocrine late effects in the FA host. Multidisciplinary follow-up of patients with FA (including cancer screening) is essential for early detection and management of late complications, and improving long-term outcomes.

publication date

  • March 21, 2016

Research

keywords

  • Fanconi Anemia
  • Hematopoietic Stem Cell Transplantation

Identity

PubMed Central ID

  • PMC4968886

Scopus Document Identifier

  • 84961390970

Digital Object Identifier (DOI)

  • 10.1016/j.bbmt.2010.05.016

PubMed ID

  • 26999465

Additional Document Info

volume

  • 51

issue

  • 7