Late effects in patients with Fanconi anemia following allogeneic hematopoietic stem cell transplantation from alternative donors.
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2016-07
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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.
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Anur, P, DN Friedman, C Sklar, K Oeffinger, M Castiel, J Kearney, B Singh, SE Prockop, et al. (2016). Late effects in patients with Fanconi anemia following allogeneic hematopoietic stem cell transplantation from alternative donors. Bone Marrow Transplant, 51(7). pp. 938–944. 10.1038/bmt.2016.32 Retrieved from https://hdl.handle.net/10161/14590.
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Kevin Charles Oeffinger
Kevin Oeffinger, MD, is a family physician, Professor in the Department of Medicine, and a member of the Duke Cancer Institute (DCI). He is founding Director of the DCI Center for Onco-Primary Care, and Director of the DCI Supportive Care and Survivorship Center. He has a long-standing track record of NIH-supported research in cancer screening and survivorship and has served in a leadership capacity in various cancer-focused and primary care-focused national committees and organizations, including the American Society of Clinical Oncology, the American Cancer Society, and the American Academy of Family Physicians. He is currently an Associate Editor for the Journal of the National Cancer Institute.
The three-fold mission of the DCI Center for Onco-Primary Care are are to: (1) deliver evidence-based, patient-centered, personalized health care across the cancer continuum by enhancing the interface between cancer specialists and primary care clinicians; (2) conduct innovative research with cutting-edge technology that can be translated to the community setting; and (3) train and educate the next generation of clinicians and researchers to extend this mission.
Dr. Oeffinger's clinical expertise is managing survivors of pediatric and young adult cancer.
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