Bone marrow transplantation in non-malignant disorders

From January 1992 to December 1997, 21 consecutive patients (14 SAA, 3 SCID , 1 Fanconi anemia, 1 Diamond-Blackfan anemia, 1 mucolipidosis and 1. mucop olysaccharidosis type I,) were transplanted (16 HLA-id, family, 2 MUD and 3 haploidentical family donors) in a single center. The median follow up per iod is 41 months (range 7-76), The probability of 3.5 year overall disease free survival is 14/21 (67%), the transplant related mortality is 4/21 (19% ), All the SCID patients are alive and disease free. 3 SAP patients had sig ns of fungal infection prior to transplant. They died in spite of intensive antifungal treatment resulting reduced DFS for SAA to 71%, Two patients wi th lysosomal storage disorders (mucolipidosis and MPS I.) rejected the hapl oidentical T-cell depleted graft 1 and 11 months after transplant, respecti vely. In 2 cases non-engraftment occured, both patients were retranslplante d succesfully.