Bone marrow transplantation from matched siblings in patients with Fanconianemia utilizing low-dose cyclophosphamide, thoracoabdominal radiation andantithymocyte globulin

Nineteen patients with Fanconi anemia (FA) and bone marrow failure underwen t bone marrow transplantation (BMT) from matched siblings. Median age at BM T was 8.7 years, Conditioning consisted of low-dose cyclophosphamide (CY 5 mg/kg x 4 days) and thoracoabdominal irradiation (TAI 400 cGy), Graft-versu s-host disease (GVHD) prophylaxis was cyclosporin A (CsA) in 13 patients an d CsA plus methotrexate in 6 patients. Antithymocyte globulin (ATG) was add ed in the pretransplant as well as the post-transplant period. All patients received high-dose acyclovir from day 2 pre-BMT to day 28 post BMT, and in travenous immunoglobulins (IVIG), 500 mg/kg weekly from day 7 pre-BMT to da y 90 post BMT, No fungal prophylaxis was given. All patients engrafted, (me dian, 14 days for an absolute neutrophil count greater than or equal to0.5 x 10(9)/l; median, 37 days for platelet count greater than or equal to 20 x 10(9)/1), Fourteen (74%) patients are alive with sustained engraftment and are transfusion independent. Three (16.6%) patients developed acute GVHD; none developed chronic GVHD, Five (26%) patients developed invasive fungal infections, and two (10%) developed fatal CMV disease. We believe the addit ion of ATG may have contributed to the increased incidence of severe life-t hreatening fungal and viral infections in our series.