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Coming soon: New trial to reduce immunosuppression in Fanconi Anemia patients following transplant

Fanconi anemia (FA) is a rare, genetically and phenotypically heterogeneous, inherited disorder that affects roughly 1 in 130,000 people worldwide. Typically diagnosed in childhood, FA is characterized by congenital abnormalities, bone marrow failure and a propensity to develop malignancies, especially myelodysplastic syndrome and acute myeloid leukemia. To date, researchers have identified 16 genes related to FA, all of which encode proteins that are critical for DNA repair. Allogeneic hematopoietic cell transplant (HCT) is the only curative option presently available for the hematological manifestations of FA. Over the past two decades, significant advances in this field have allowed for dramatic improvements in patient survival after HSCT. Achievement of successful transplantation largely hinges on striking a delicate balance between immune suppression, which allows transplanted cells to take and grow, and immune surveillance, necessary for protecting against potentially lethal infections.

Prior to transplant, a patient’s immune system is ‘prepared,’ in order to prevent graft failure, by chemotherapeutic conditioning regimens, with and without radiation. In addition to graft failure, graft-versus-host-disease (GVHD), wherein donor-derived T cells attack recipient tissue, is a common complication in transplanted patients. The risk of GVHD is significantly reduced, however, by depleting the T cells from donor marrow prior to transplantation, though at the cost of significant weakening of the immune system. University of Minnesota researchers found that shielding of the thymus, the organ responsible for educating immature immune cells, during total body irradiation appreciably sped up immune recovery after transplant. Though these advances have increased survival from 25 percent to greater than 90 percent in children under 18 years of age, patients are required to take potent immunosuppressive drugs for a period of six to nine months following transplant, resulting in considerable susceptibility to life-threatening infections.

Margaret MacMillan, MD, MSc, and John Wagner, MD, Co-Directors of the Fanconi Anemia Comprehensive Care Program at the University of Minnesota are now focusing efforts to reduce the need for prolonged immune suppression after transplantation. With support from Jimbo & Candi Fisher’s Kidz1stFund they are designing new methods to manipulate donor cells before giving them to patients such that prolonged immune suppression after transplant will no longer be required. It is anticipated that this will greatly reduce the risk of life-threatening infections after transplantation.

The vast improvement in outcomes after transplantation for patients with Fanconi anemia highlights the strengths of an academic setting, taking a translational, interdisciplinary approach that bridges the gap between bench science and the clinic. Using step-wise changes to protocols, and learning from what has already been done, researchers hope to significantly improve outcomes and patients’ quality of life.

To refer a patient or learn more about our Fanconi Anemia Comprehensive Care Program, contact us at 612-273-2800.

Journey Clinic - Pediatric Blood and Marrow Transplantation Center

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