Table 4.

MPS II with HSCT

Author	Details	Results
Tanaka et al. 2012 [49]	26 MPS II patients Treated between 1990 and 2003 in Japan 21 questionnaires collected Mean age at transplantation - 64.2±30.2 months Average follow-up period - 115.7±41.4	88.5% 5-year survival rate Category I and III brain lesions positively altered Attenuated phenotype improved ADL, IQ/DQ, FIM, and brain MRI questionnaires 32% less valve regurgitation 56% valves stabilized Category IV lesions continued to progress in 6 patients
Tanjuakio et al. 2015 [45]		Average ADL - 27.9 ± 11.4. Early ERT mean ADL - 21.6 ± 9.0 Late ERT reported a mean ADL score of 9.5 ± 10.5 HSCT before the age of 5 reported an average ADL of 33 ± 7.0 HSCT after the age of 5 reported a mean ADL score of 21.2 ± 13.0
Yasuda et al. 2015 [3, 137]	Patient received HSCT at the age of 2 years	Normal blood GAGs ADL kept normal Present height and weight: 162 cm and 55 kg at 15 years of age Milder symptoms compared to untreated patients Ten years after HSCT, several manifestations still present At 13.5 years of age: no vacuolization in chondrocytes, the chondrocytes were of a normal size in spine
Kubaski et al.2017 [13, 27, 49]	146 MPS II patients Compared results to 51 ERT patients and 15 untreated Mean age at transplantation −5.5 years of age	From the previously published cases: 8 (9%) of 85 cases had GVHD 9 (8%) died from transplantation-related complications Of the 27 new patients: 76% under the age of 6 at the transplantation 3 developed GVHD 100% survival All had ERT before HSCT Improvements in somatic features, joint movements, and ADL, compared to those treated with ERT Improvements or stabilization of lesions located in categories III and IV Donor cells found in the brain of one patient, as well as positive hydrocephalic changes and perivascular enlargement improvements
Barth et al. 2017 [10]	Seven-year old patient Received transplanation at 2 months UCB from unrelated donor Busulfan, cyclophosphamide, and ATG regimen	Improved cognitive function and skeletal manifestations Improved ADL 80% chimerism Slowed speech development Moderate hearing loss No coarse facial features, corneal clouding, or hepatosplenomegaly Normal cardiac function Mild dysostosis multiplex