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Background <p>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).</p> Methods <p>Patients were enrolled between September 2014 and February 2022; urinary keratan sulfate (uKS), 6-minute walk test (6MWT) distance, forced expiratory volume in 1&#xa0;s (FEV<sub>1</sub>), forced vital capacity (FVC), EuroQoL-5D-5L (EQ-5D-5L) score, and safety were assessed during routine care.</p> Results <p>As of February 13, 2022, 90 patients who initiated ERT had enrolled (median exposure: 5.6 years; median age at first ERT: 27.8 years). Reductions from baseline in uKS levels were sustained over mean follow-up of 5.4 years (mean percent change: -52.9%; <i>p</i> &lt; 0.0001). In patients with available data, mean change in 6MWT distance was + 15.8&#xa0;m (<i>p</i> = 0.3627) over a mean follow-up of 5.8 years. FEV<sub>1</sub> and FVC remained stable over mean follow-up of 5.3 years (mean change: 0.0&#xa0;L for both). The mean change from baseline in EQ-5D-5L index score was + 0.1 after 1 year of treatment. Thirty-four patients (39.5%) had ≥ 1 adverse event (AE), 23 patients (26.7%) had ≥ 1 serious AE, and 10 (11.6%) had ≥ 1 drug-related AE (infusion-related reactions [<i>n</i> = 3; 3.5%], pyrexia [<i>n</i> = 2; 2.3%]). Eight deaths occurred; none were deemed treatment related.</p> Conclusions <p>Real-world data collected from MARS suggest that patients with MPS IVA who initiated ERT in adulthood remained stable over 7 years of follow-up. No new safety signals were identified.</p>

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

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  • 发表日期：

    2025年10月30日

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  • Karolina M. Stepien,
  • Barbara K. Burton,
  • Michael B. Bober,
  • Philippe M. Campeau,
  • Carolyn Ellaway,
  • Kaustuv Bhattacharya,
  • Nathalie Guffon,
  • David Hinds,
  • Abigail Hunt,
  • Alice Lail,
  • Shuan-Pei Lin,
  • Martin Magner,
  • Elaine Murphy,
  • Pascal Reisewitz,
  • John J. Mitchell

摘要

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: 27.8 years). Reductions from baseline in uKS levels were sustained over mean follow-up of 5.4 years (mean percent change: -52.9%; p < 0.0001). In patients with available data, mean change in 6MWT distance was + 15.8 m (p = 0.3627) over a mean follow-up of 5.8 years. FEV1 and FVC remained stable over mean follow-up of 5.3 years (mean change: 0.0 L for both). The mean change from baseline in EQ-5D-5L index score was + 0.1 after 1 year of treatment. Thirty-four patients (39.5%) had ≥ 1 adverse event (AE), 23 patients (26.7%) had ≥ 1 serious AE, and 10 (11.6%) had ≥ 1 drug-related AE (infusion-related reactions [n = 3; 3.5%], pyrexia [n = 2; 2.3%]). Eight deaths occurred; none were deemed treatment related.

Conclusions

Real-world data collected from MARS suggest that patients with MPS IVA who initiated ERT in adulthood remained stable over 7 years of follow-up. No new safety signals were identified.