New results show similar outcomes for Fanconi anemia patients, regardless of stem cell source
Historically, outcomes after HLA matched sibling donor transplant for Fanconi anemia (FA) have been superior to those observed after alternative donor (AD) (i.e., unrelated or HLA mismatched related donor) transplant. Alternative transplantation has most often been complicated by high rates of graft failure, graft-versus-host-disease (GVHD), organ toxicity and infection, resulting in poor survival rates. Over the past 2 decades, the transplant approach has been modified leading to marked improvements in outcomes.
Since 2006, FA patients with marrow failure and low to moderate risk of myelodysplasia (MDS) have been treated with low dose total body irradiation (300 cGy) with thymic shielding, fludarabine (FLU), cyclophosphamide (CY) and antithymocyte globulin (ATG). Since 1999, patients with an HLA matched sibling donor received FLU, CY and ATG (without any radiation). All patients with a marrow donor (related or unrelated) had most of the T cells removed prior to transplantation to reduce the risk of the life threatening complication of, GVHD which is also a known risk factor for later cancers.
As shown in Table 1, the incidence of neutrophil recovery, acute and chronic GVHD are similar between patients who received a matched sibling donor and those who received an alternative donor. As shown in Figure 1, the probability of survival at 3 years after transplantation is the same in recipients of a matched sibling donor and alternative donor transplant.
To our knowledge, this is the first demonstration that survival and other transplant outcomes are comparable after alternate and matched sibling donor transplantation for patients with FA demonstrating the importance of research to optimize outcomes for patients.
To refer a patient and learn more our Fanconi Anemia Comprehensive Care Program, contact us at 612-273-2800.