Table 3.

MPS I with HSCT

Author	Details	Results
Yasuda et al. 2015[137]	Transplant at 2 years Unrelated cord blood transplant	10-year follow up Normal ADL and cognitive function Bone deformity continued Improved bone lesion Reduced GAG levels
Aldenhoven et al. 2015 [21, 38]	217 MPS IH patients Patients from various centers Transplantation took place between January 1985 and February 2011 Results were followed for a median time of 9.2 years	Early transplantation correlates with significant cognitive development Many achieved normal enzyme levels 26 patients’ enzyme levels below the reference limit Some patients experienced continued manifestations of the disease TBI and older age correlates with little neurodevelopmental improvements 71 % of patients with severe cognitive impairment had DQ/IQ below 70 post-transplantation 15% of patients with DQ/IQ greater than 70 developed severe cognitive impairment post-HSCT Mild developmental impairment post-transplantation
Rodgers et al. 2017 [84]	134 MPS IH patients Patients received HSCT at the University of Minnesota from 1983–2013 In 2013, follow-up data were obtained for 122 patients The median follow-up period for patients was 10.7 (interquartile range (IQR): 5.0 – 17.2) years post-transplant Longest follow-up period was 28.97 years	Survival rate after transplantation: 70% one-year after transplantation 62% 10-years after transplantation 32% 25-years after transplantation Survival rates before 2004: 65%, 1-year post-transplantation 57%, 8-years post-transplantation Survival rates after 2004: - 84%, 1-year post-transplantation 81%, 8 -years post-transplantation Preferred treatment changes led to normal IDUA enzyme levels increase from 54.8% before 2004 to 75.9% after 2004
Lum et al. 2017 [82]	240 MPS IH patients Between 1983–2015 102 received pre-transplant ERT	85.2 % survival rate 85 patients alive and engrafted 60 patients experienced graft-failure Cord blood associated with graft failure
Lum et al. 2017 [94]	54 patients 1985–2008 Median age 15.1 months	18 experienced graft failure 9 died 17 received second transplantation 12 alive and engrafted after second transplantation 73.7% survival rate 20-years post-transplantation 9 normal cardiac assessments 4 on angiotensin-converting-enzyme inhibitors 2 mild cardiomyopathy 2 aortic valvular replacement