Table 9.

Summary of clinical effect by HSCT

Type	CNS	Bone growth	Air way	Joint mobility; rigidity	Heart	Cornea	Liver, Spleen	Overall clinical effect	Recommendation	Condition	Remarks
MPS T	++ ~ +++	++	+++	Upper joints: ++ ~ +++ Lower joints: + ~++	++ ~ +++	− ~+	+++	++ ~ +++	Yes	Patients under 2.5 years of ag. Elimination of immune response against infused enzyme with ERT	Approved and/or conducted as standard of care
ref	[38]	[136]	[142–144]	[5,8, 21,64,87, 122, 124, 200, 201]	[5,8, 21, 87, 202–204]	[136]	[5,8, 21, 87, 202–204]	Better than ERT
MPS II	+ ~ ++	++	++	++	++	NA	+++	++	Yes	Early stage. Elimination of immune response against infused enzyme with ERT	Approved and/or conducted in Japan, China, Brazil, Europe, and USA. Reported impact on CNS although less than MPS I
ref	[49]	[3, 12]	[18]	[13]	[149]		[149]	Better than ERT		[13,37, 45, 49, 53, 109]
MPS III	-	NA	NA	NA	NA	NA	NA	-	No	No significant positive effect; insufficient information
ref	[155]									[158]
MPS IVA	NA	+	++	hypermobile joint; + ~ ++	++	unknown	+++	++	Yes	Optional treatment at an early stage. Elimination of immune response against infused enzyme with ERT	Approved and/or conducted in Japan and China
ref		[12, 19]	[12, 18, 19]	[12,18, 19]	[12, 18, 19]		[12, 18, 19]	Better than ERT		No criteria
MPS VI	NA	+ ~ ++	++	+ ~ ++	++	+ ~ ++	+++	++	Yes	Optional treatment. Elimination of immune response against infused enzyme with ERT	Approved and/or conducted in Japan, China, Europe, and USA.
ref		[172]	[17, 170,172, 175]	[6,7, 61, 164]	[164]	[97,127, 172, 173]	[3]	Similar to ERT		No criteria
MPS VII	++	++	++	+ ~++	++	unknown	+++	++	Yes	Optional treatment. Elimination of immune response against infused enzyme with ERT	A few reports with significant improvements
ref	[20, 180, 182]	[20, 180, 182]	[20]	[20, 180, 182]	[20, 180, 182]		[20, 180, 182]	[20, 180, 182]		No criteria

Note; The most effective+++, no improvement -; NA, not applicable