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.
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Authors | G Kriván, L Timár, V Goda, M Réti, P Reményi, T Masszi |
Journal | Bone marrow transplantation
(Bone Marrow Transplant)
Vol. 22 Suppl 4
Pg. S80-3
(Dec 1998)
ISSN: 0268-3369 [Print] England |
PMID | 9916644
(Publication Type: Journal Article)
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Topics |
- 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
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