Skip to main content

Table 5 Guidance statements for HSCT

From: Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance

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%
  1. Post-consensus comments by the SC to be taken into consideration
  2. 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