Table 5.

MPS IVA with HSCT

Author	Details	Results
Chinen et al. 2014 [3, 12, 58]	MPS IVA patient Allogeneic BMT at 15 years and 8 months	Five years after HSCT the patient’s GALNS enzyme activity matched donor’s Return to walking after osteotomies, the digression of a narrow airway resulting in shortness of breath Recovery in pulmonary function, no snoring, and increase of bone mineral density in the lumbar vertebrae (L2–4) Some physical activities remained restricted Mineral density in the lumbar spine increased by 50% one year after transplant Unchanged epiphyseal dysplasia in trochanter major and minor and hyperlaxity of the joints
Yabe et al. 2016 [19]	Long-term study of four MPS IVA patients 3 severe and 1 attenuated phenotype Received allogeneic BMT at the mean age of 10.5 years HLA-identical siblings or HLA-identical unrelated donors	Transplantation successful in all four cases No serious reports of GVHD All achieved full engraftment Two achieved normal enzyme activity Two achieved the same enzyme level activity as a carrier donor Improved clinical course of MPS IVA Earliest transplantation reported the highest improvement in ADL scores (59 of 60) One patient required bilateral osteotomies after transplantation Reduced the bone manifestations Lessened the impact on growth and laxity of joints for those with severe MPS IVA Three patients reported improved or stabilized walking No spinal cord compression
Wang etal. 2016 [18]	4 patients Between 2004–2015 Myeloabaltive busulfan, cyclophosphamide, fludarabine, and ATG regimen	Remission in hepatosplenomegaly Stable spinal cord compression Joint laxity and hypermobility, upper-airway, otitis media, height, and thoracic deformity improved