Bone marrow transplantation in non-malignant disorders

Bone Marrow Transplant. 1998 Dec:22 Suppl 4:S80-3.

Abstract

From January 1992 to December 1997, 21 consecutive patients (14 SAA, 3 SCID, 1 Fanconi anemia, 1 Diamond-Blackfan anemia, 1 mucolipidosis and 1 mucopolysaccharidosis type I.) were transplanted (16 HLA-id. family, 2 MUD and 3 haploidentical family donors) in a single center. The median follow up period 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 SAA patients had signs of fungal infection prior to transplant. They died in spite of intensive antifungal treatment resulting reduced DFS for SAA to 71%. Two patients with lysosomal storage disorders (mucolipidosis and MPS I.) rejected the haploidentical T-cell depleted graft 1 and 11 months after transplant, respectively. In 2 cases non-engraftment occured, both patients were retransplanted successfully.

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / therapy*
  • Bone Marrow Transplantation* / adverse effects
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Fanconi Anemia / therapy
  • Female
  • Graft Rejection
  • Graft Survival
  • Humans
  • Infant
  • Lysosomal Storage Diseases / therapy*
  • Male
  • Mucolipidoses / therapy
  • Mucopolysaccharidosis I / therapy
  • Severe Combined Immunodeficiency / therapy*
  • Treatment Outcome