Patients with Fanconi's anemia (FA) tolerate poorly standard preconditioning used for acquired aplastic anemia and severe GvHD has been reported following allogeneic BMT. T-Cell depletion has been infrequently utilised for this disorder.

PATIENTS: Five children with cytogenetically proven FA, aged 4 to 11, received HLA identical marrow, 4 from siblings and one from the mother. Conditioning consisted of low dose cyclophosphamide (CFU) (5mg/kg, 10mg/kg and 3 patients at 20mg/Kg for 4 days) and fractionated total nodal irradiation(TNI) of 600cGy. T-Cell depletion was with CAMPATH-1G (CDw 52 murine monoclonal anti human T-Cell antibody) in vitro and in 4 patients, in addition, it was also administered in vivo (5 mg daily for 10 days). No further post BMT immunosuppression was given. G-CSF (5 ugm/kg) was administered until the ANC recovered.

RESULTS: 4 patients engrafted reaching an ANC > 500 at 15, 24, 27 and 35 days post BMT, respectively. None developed GvHD. One patient who received 10 mg/kg CFU failed to engraft and on day +42 was transplanted with a second unfractionated graft from the same donor. Preconditioning was with cyclophosphamide 10 mg/Kg for four consecutive days and ALG for three days. This patient engrafted rapidly but developed grade 2 acute GvHD which responded to prednisone. The patient who only received 5 mg/Kg has a chimeric bone marrow. Engraftment was quickest in the patient who did not receive in vivo CAMPATH. All patients remain well with normal blood counts at 3, 17, 18, 25 and 28 months post BMT.

CONCLUSION: CAMPATH 1G is effective in preventing GvHD and does not lead to excessive rates of graft failure. Conditioning with cyclophosphamide 20mg/kg and TNI is an adequate immunosuppressive strategy in patients with FA who will receive a T-Cell depleted graft.