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. 2019 May 29;14:118. doi: 10.1186/s13023-019-1080-y

Table 5.

Guidance statements for HSCT

Statement Percentage consensus

With consideration of the associated risks of morbidity and mortality associated with this procedure, HSCT may be an option for patients with MPS VI who have a matched related donor (or unrelated donor), or cord blood grafta

Evidence Grade: C (consistent with level 4 studies and extrapolations from level 3 studies)

86%

Due to the risk of mortality, it is critical that HSCT is only performed in an institution with a multidisciplinary team experienced in the care of patients with MPS VI

Evidence Grade: D (level 3/4 studies with inconsistent risk/benefit results)

91%

Post-consensus comments by the SC to be taken into consideration

aHSCT may be an option for patients with MPS VI who have a matched related donor (preferably not a carrier) or a well-matched adult unrelated donor