Humanistic burden of mucopolysaccharidoses: a systematic literature review

CONCLUSIONS: This systematic literature review revealed the substantial humanistic burden of individuals affected by MPS as well as caregivers. Significant variation in HRQoL scores was observed, however studies indicate that quality of life of MPS patients is influenced primarily by severity of disease (MPS type and phenotype), and then by time of diagnosis, pain, cognitive involvement, mobility, dependence and time of treatment initiation. Further studies are needed to assess the global humanistic burden of MPS, particularly in MPS III, VI, VII, and IX subtypes, in adults, and for a longer follow-up period. Considering the vast array of HRQoL assessment tools available and used in this study, researchers should also consider using scales with condition specific measures to ensure appropriate estimates of effectiveness.PMID:38328952 | DOI:10.1080/03007995.2024.2316213
Source: Current Medical Research and Opinion - Category: Research Authors: Source Type: research