Low risk of graft-versus-host disease with transplantation of CD34 selected peripheral blood progenitor cells from alternative donors for Fanconi anemia.

Objectives: Transplant results for Fanconi anemia with alternative-donor bone marrow transplantation currently entail a high incidence of graft failure and graft-versus-host disease (GVHD). The authors sought to improve outcome in this disease category with alternative donors with a 5-6/6 antigen match by transplantation of highly purified peripheral blood progenitor cells (PBPC) using the Isolex 300i v2.5 device as a means of T-cell depletion to lessen the risk of GVHD. Methods: All Fanconi anemia patients (n = 8) received the same preparative regimen that included total body irradiation (450 cGy), Cytoxan (20 mg/kg), ATGAM, and fludarabine (120 mg/m 2 ). The cell dose of CD34+ cells was a median of 11.4 × 10 6 /kg; the cell dose of CD3 + cells was a median of 1.9 × 10 4 /kg. Primary engraftment was rapid in all patients, with neutrophil recovery occurring at a median of day 10 and platelet count more than 50,000 on day 27. Two patients subsequently had secondary graft failure. Despite lack of cyclosporine GVHD prophylaxis, only two patients developed acute GVHD (both grade I), and no patients developed chronic GVHD. Three patients died: one at day 59 secondary to disseminated fungal infection, the second at day 196 during a second transplant, and the third at day 202 due to graft failure. With a median follow-up of 12 months, the overall survival was 58 ± 18%. Conclusions: Transplantation of CD34-selected PBPCs from alternative donors results in a very low risk of GVHD in patients with Fanconi anemia.