BACKGROUND AND AIM: @entity11126 ( @entity11126 , @entity12751 ) naturally leads to @entity204 within the first decade of life, primarily from cardiac and pulmonary causes. To determine how hematopoietic stem cell transplantation (HSCT) has altered mortality, we analyzed our institution's 30-year experience of @entity1 with @entity11126 undergoing HSCT. METHODS: Using chart review and the National Death Index, we determined survival status of 134 @entity1 (males = 69) with @entity11126 transplanted between 9/16/1983 and 7/25/2013 on 12/31/2013. Analysis included descriptive statistics, Kaplan-Meier curves, and regression analysis by Cox proportional hazards model. RESULTS: Overall survival (95% CI) at one- and 25-years was 70% (62-78%) and 37% (19-55%), respectively. From 2004 onward, overall survival at one- and 8-years was 84% (73-96%) and 81% (69-94%), respectively, compared to 65% (55-74%) and 57% (47-67%) prior to 2004 (Log-rank p   =   0.032). Regardless of era, male survival was significantly better than female (HR 0.40, [95% CI: 0.21-0.74], p   =   0.004). The cumulative incidence of @entity204 (95% CI) at 25  years was 63% (45-81%); incidence of @entity204 was the highest at 27% (10-41%) compared to 8% (0.3-16%) for cardiac, 12% (6-17%) for @entity166 , and 16% (3-27%) from other complications. CONCLUSIONS: HSCT has increased survival in @entity11126 beyond the third decade of life and decreased the incidence of @entity299 , but deaths after the third year post-HSCT occur in excess of expected US mortality. It is important to determine if improved transplant strategies since 2004 result in better long-term survival in the current @entity1 population.

Mortality after hematopoietic stem cell transplantation for severe XXXX : the 30-year University of Minnesota experience.

@entity1 :: ('9606', 'Species') :: ['patients', 'patient'] @entity11126 :: ('MESH:C565524', 'Disease') :: ['MPS IH', 'Mucopolysaccharidosis IH'] @entity12751 :: ('MESH:D008059', 'Disease') :: ['Hurler syndrome'] @entity299 :: ('MESH:D006331', 'Disease') :: ['cardiac mortality'] @entity204 :: ('MESH:D003643', 'Disease') :: ['pulmonary-related death', 'death'] @entity166 :: ('MESH:D003141', 'Disease') :: ['infectious disease']
@entity12751 :: (MESH:D008059,Disease) :: ['mucopolysaccharidosis type I']
