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Chapter 10: Unrelated Donor HSCT 219 initial hospitalization for the transplant procedure, all patients are kept in a single occupancy room equipped with a high-efficiency air filtration system to reduce exposure to infectious agents. Once the marrow has recovered sufficiently, patients are allowed out of their hospital rooms unless intercurrent problems prevent this. After discharge, patients are expected to avoid crowded enclosed spaces and to wear masks in an attempt to reduce exposure to viral, bacterial, and fungal pathogens. Alternatives to Unrelated Donor HSCT Recent cloning of the FA genes has provided new insights into the molecular basis of FA and has made new opportunities available for better care of FA patients. For example, knowledge of the complementation group or mutation not only allows the physician to predict the course of the disease in some cases, 1 it permits the potential use of gene therapy and preimplantation genetic diagnosis (PGD). While gene correction of stem cells is not yet a clinical reality, PGD in combination with in vitro fertilization allows couples at high risk of having children with FA to have additional children free of the disease. In addition, PGD can be used to select those embryos that are HLA-matched with the child affected with the disease. 18-20 While there are ethical issues regarding the use of PGD and embryo selection, it is nonetheless a strategy that is being considered by many couples. Remaining Challenges Substantial improvement has been made in the survival of FA patients undergoing alternate donor HSCT, but challenges and questions remain. These include: 1) the optimal timing of alternate donor HSCT; 2) the impact
220 Fanconi Anemia: Guidelines for Diagnosis and Management of androgens on survival after HSCT; 3) the selection of stem cell source (marrow versus peripheral blood versus umbilical cord blood); 4) the optimal pretransplant and GvHD therapies; 5) the effect of the mosaic phenotype on the natural history of the disease; and 6) the role of radiation and chronic GvHD on the risk of malignancy later in life. Acknowledgements Numerous individuals contributed to the discussion that culminated in this chapter, most notably Farid Boulad, MD, Memorial Sloan-Kettering Cancer Center, New York; Alfred Gillio, MD, Hackensack University Medical Center, Hackensack, NJ; and Eva Guinan, MD, Children’s Hospital, Boston, MA. References 1. Wagner JE, MacMillan M, Auerbach AD. Hematopoietic Cell Transplantation for Fanconi Anemia. Blume KG, Forman SJ, Appelbaum FR, eds., Thomas’ Hematopoietic Cell Transplantation. Oxford, UK: Blackwell Publishing Ltd.; 2008. 2. Gluckman E, Auerbach AD, Horowitz MM, et al. Bone marrow transplantation for Fanconi anemia. Blood 1995; 86: 2856-2862. 3. Ebell W. Transplant results and observations from our transplant expert in Germany. FA Family Newsletter 2002; 32: 5. 4. de Mederios CR, Bitencourt MA, Zanis-Neto J, et al. Allogeneic hematopoietic stem cell transplantation from an alternative stem cell source in Fanconi anemia patients: analysis of 47 patients from a single institution. Brazilian Journal of Medicine and Biological Research 2006; 39: 1297-1304. 5. Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic cell transplantations in patients for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood 2000; 95: 422-429.
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Chapter 4 Gastrointestinal, Hepatic
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