Fanconi Anemia: 29 Years Experience in a Single Center

摘要

Hematopoietic stem cell transplantation, which is the only curative therapy for marrow failure experienced by Fanconi anemia, was not available for all patients due to lacking in adequate transplantation centers in Turkey in the past years. For this reason, although androgens side effects are well known, they have been widely used for Fanconi anemia. Here we reported the clinical characteristics and outcome of 42 Fanconi anemia cases in 29-year period in a single center. The median follow up period was 67.5 months (range: 1 -111 months) after diagnosis. Treatment modalities were as follows: Androgens with low dose corticosteroids (n= 34) (anti lymphocyte globulin were used in three of 34 patients, and five of 34 underwent to hematopoietic stem cell transplantation), high dose methylprednisolone (n= 1), hematopoietic stem cell transplantation (n= 1), supportive treatment (n= 2), no treatment required (n= 4). The uses of androgens in our patients were as follows: Oxymethalone and prednisolone (n= 21), testosterone and prednisolone (n= 8), testosterone, prednisolone, and anti lymphocyte globulin (n= 3), oxymethalone, testosterone, and prednisolone (n= 2) were given to 34 patients. In our series, one patient developed acute myeloid leukemia. None of our patients developed liver tumors and peliosis hepatitis was diagnosed in one patient. We used androgens with low dose corticosteroids. Only six (14.3%) patients underwent hematopoietic stem cell transplantation. Eight (19%) patients were lost to follow up, and 11 patients (26.2%) died.