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

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064165%3A_____%2F25%3A10505043" target="_blank" >RIV/00064165:_____/25:10505043 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/00216208:11110/25:10505043

Výsledek na webu

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=Uw.-qNuMKK" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=Uw.-qNuMKK</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1186/s13023-025-04064-w" target="_blank" >10.1186/s13023-025-04064-w</a>

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

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 &gt;= 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 &lt; 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 &gt;= 1 adverse event (AE), 23 patients (26.7%) had &gt;= 1 serious AE, and 10 (11.6%) had &gt;= 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.

Název v anglickém jazyce

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

Popis výsledku anglicky

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 &gt;= 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 &lt; 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 &gt;= 1 adverse event (AE), 23 patients (26.7%) had &gt;= 1 serious AE, and 10 (11.6%) had &gt;= 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.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30209 - Paediatrics

Projekt

—

Návaznosti

N - Vyzkumna aktivita podporovana z neverejnych zdroju

Rok uplatnění

2025

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Orphanet Journal of Rare Diseases

ISSN

1750-1172

e-ISSN

1750-1172

Svazek periodika

20

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

10

Strana od-do

548

Kód UT WoS článku

001606629300002

EID výsledku v databázi Scopus

2-s2.0-105020457377