Pre-transplant risk factors affecting outcome in Hurler syndrome.
Allogeneic transplantation remains the standard of care for patients with Hurler syndrome. As enzyme replacement therapy (ERT) has become available, controversy has emerged in regards to whether the use of enzyme in the peri-transplant period is appropriate. An analysis was performed on 74 patients with Hurler syndrome transplanted at the University of Minnesota between 1990 and 2003, before our use of ERT associated with transplant, with the intention of determining if patients at higher risk during the transplant can be identified based on evaluations and events before transplantation. Age, the presence of hydrocephalus, a history of cardiovascular issues or upper airway obstruction before transplant was not associated with significant differences in survival. In contrast, patients who had a history of lower airway disease, including reactive airway disease or bronchiolitis, or a history of pneumonia, had a significantly inferior outcome based on OS. The risk for serious respiratory complications was also assessed by evaluating the incidence of intubation. Overall, 31% of these patients were intubated. The risk of intubation was higher in older patients and in those with a history of lower airway disease. These findings have implications for the care of patients with high-risk features.
Orchard
P J
PJ
Program in Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA. orcha001@umn.edu
Milla
C
C
Braunlin
E
E
DeFor
T
T
Bjoraker
K
K
Blazar
B R
BR
Peters
C
C
Wagner
J
J
Tolar
J
J
D001988:Bronchiolitis; D002675:Child, Preschool; D056947:Enzyme Replacement Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D008910:Minnesota; D008059:Mucopolysaccharidosis I / Q000175:diagnosis* / Q000401:mortality / Q000628:therapy*; D011014:Pneumonia; D011379:Prognosis; D012189:Retrospective Studies; D012307:Risk Factors; D015996:Survival Rate; D014184:Transplantation, Homologous; D016896:Treatment Outcome