Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).

ABSTRACT:

Background

Mucopolysaccharidosis (MPS) IVA is a rare disease with substantial, multisystemic morbidity. We assessed real-world safety and effectiveness of the enzyme replacement therapy (ERT) elosulfase alfa in patients with MPS IVA in the multinational, observational Morquio A Registry Study (MARS) who initiated ERT in adulthood (aged ≥ 18 years).

Methods

Patients were enrolled between September 2014 and February 2022; urinary keratan sulfate (uKS), 6-minute walk test (6MWT) distance, forced expiratory volume in 1 s (FEV₁), forced vital capacity (FVC), EuroQoL-5D-5L (EQ-5D-5L) score, and safety were assessed during routine care.

Results

As of February 13, 2022, 90 patients who initiated ERT had enrolled (median exposure: 5.6 years; median age at first ERT:

SUBMITTER: Stepien KM 

PROVIDER: S-EPMC12574230 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature