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- W2014643503 abstract "Background: Hurler’s syndrome (HS), the most severe form of mucopolysaccharidosis type-I causes progressive deterioration of the central nervous system and death in childhood. Allogeneic-stem cell transplantation (SCT) before the age of two years halts disease progression and prolongs life. Graft-failure and mixed chimerism limits the success of SCT for HS. (Unrelated) cord blood grafts are suggested to be a good alternative source for bone marrow. Methods: Patient registered to EUROCORD-database and EBMT-Hurler syndrome database were included. Between 1991 and 2004 forty-two patients received an (unrelated)-cord blood transplant. The children were evaluated for engraftment, adverse event and effects on disease symptoms. A risk factor analyses was performed using logistic regression. Results: From 40 patients enough data was available. After a median follow up period of 14 (7-84) mths the “alive and engrafted” rate was 69% after first SCT. Donors used were: 4 HLA-identical family and 36 unrelated cord blood (23 mismatched: 18 5/6-matched and five 4/6-matched). With the exception of 2, all patients received a myeloablative conditioning regimen. Cell dose used was median 7.9 (1.5-32) *107 NC/kg and 2,6 (0,7-25) *105 CD34+/Kg. In multivariate logistic regression analyses, year of transplantation (≤2000 vs. ≥2001: A&E 40 vs 84%: OR 7,5 range 1,7-30, p=0.007) and NC-dose of less than 5*107 NC/kg (OR 6,4, range 1,7-39: p= 0.045) were found to be risk factors for graft-failure. Only 2 of the 27 patients A&E patients showed a mixed chimerism (86 and 92%: still increasing). All patients had normal enzyme levels. Acute-GvHD (grade>1) was observed in 15%, while chronic-GvHD was seen in 19% (extensive 8%) of patients at risk. SCT improved somatic features of HS. Conclusion: Outcome following cord blood SCT for Hurler’s syndrome is encouraging. (U)CB is a good alternative stem cell source and might even be preferential since cord-blood appeared to increase the likelihood of sustained engraftment resulting in full-donor chimerism and normal enzyme levels." @default.
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- W2014643503 date "2007-02-01" @default.
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- W2014643503 title "157: Outcomes of cord blood transplantation for Hurler’s syndrome. An EUROCORD-working party inborn errors EBMT survey" @default.
- W2014643503 doi "https://doi.org/10.1016/j.bbmt.2006.12.161" @default.
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