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Fanconi anemia is an inherited disorder that presents in childhood with congenital anomalies and progressive bone-marrow failure. Hematopoietic stem-cell transplantation (HSCT) provides hematologic improvement, but survival is adversely affected by the associated risks for graft failure, graft-versus-host disease (GvHD) and a predisposition to leukemia, squamous-cell carcinoma, and other malignancies.
To examine treatment outcomes associated with this rare condition, European investigators conducted a retrospective, multicenter study involving 795 consecutive patients who received transplanted stem cells from the bone marrow (93%) or peripheral blood (7%) from 1972 to 2009.
At median follow-up of 6 years, results were as follows:
Graft failure…