Trial reportPediatric research2015
Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y.
Trial report in Pediatric research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
22 citing papers in PubMed, 41 citations in OpenAlex.
- Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study.American journal of medical genetics. Part A · 2015Trial
- Long-term follow-up and response to elosulfase alfa in mucopolysaccharidosis type IVA: a single-center cohort from the Czech Republic.Orphanet journal of rare diseases · 2026Article
- Growth Patterns in MPS IVA and MPS IIIA: A Longitudinal Single-Center Study.Journal of clinical medicine · 2026Article
- Long-term Natural History and Elosulfase Alfa Treatment for Mucopolysaccharidosis Type IVA: A Single-Center Study in the Czech Republic.Research square · 2026Article
- Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time.Genetics in medicine open · 2025Article
- Consensus-based expert recommendations on the management of MPS IVa and VI in Saudi Arabia.Orphanet journal of rare diseases · 2024Article
- Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome.Drug design, development and therapy · 2022Review
- Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement.Orphanet journal of rare diseases · 2021Article
- Morquio A syndrome and effect of enzyme replacement therapy in different age groups of Turkish patients: a case series.Orphanet journal of rare diseases · 2021Article
- Diagnostic journey and impact of enzyme replacement therapy for mucopolysaccharidosis IVA: a sibling control study.Orphanet journal of rare diseases · 2020Article
- Oral immunotherapy tolerizes mice to enzyme replacement therapy for Morquio A syndrome.The Journal of clinical investigation · 2020Article
- Long-Term Outcomes of Early Enzyme Replacement Therapy for Mucopolysaccharidosis IV: Clinical Case Studies of Two Siblings.Diagnostics (Basel, Switzerland) · 2020Article
- A Case Report of a Japanese Boy with Morquio A Syndrome: Effects of Enzyme Replacement Therapy Initiated at the Age of 24 Months.International journal of molecular sciences · 2020Article
- Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future.Journal of human genetics · 2019Review
- Effect of enzyme replacement therapy on the growth of patients with Morquio A.Journal of human genetics · 2019Article
- Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance.Orphanet journal of rare diseases · 2019Article
- Enzyme replacement therapy: efficacy and limitations.Italian journal of pediatrics · 2018Review
- 30 months follow-up of an early enzyme replacement therapy in a severe Morquio A patient: About one case.Molecular genetics and metabolism reports · 2016Article
- Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues.Orphanet journal of rare diseases · 2016Review
- Activity of daily living for Morquio A syndrome.Molecular genetics and metabolism · 2016Article
Corrections and comments
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Authors and funding
8 authors at 5 institutions in 3 countries.
Funding
Abstract
backgroundPrevious studies have shown that elosulfase alfa has a favorable efficacy/safety profile in Morquio A patients aged ≥5 y. This study evaluated safety and impact on urine keratan sulfate (uKS) levels and growth velocity in younger patients.
methodsFifteen Morquio A patients aged <5 y received elosulfase alfa 2.0 mg/kg/week for 52 wk during the primary treatment phase of a phase II, open-label, multinational study. Primary endpoint was safety and tolerability; secondary endpoints were change in uKS and growth velocity over 52 wk.
resultsAll 15 patients completed the primary treatment phase. Six of 743 infusions (0.8%) administered led to adverse events (AEs) requiring infusion interruption and medical intervention. Eleven patients (73.3%) had ≥1 study drug-related AE, mostly infusion-associated reactions. Mean z-score growth rate per year numerically improved from -0.6 at baseline to -0.4 at week 52. Comparison to untreated subjects of similar age in the Morquio A Clinical Assessment Program study showed a smaller decrease in height z-scores for treated than for untreated patients. Mean percent change from baseline in uKS was -30.2% at 2 wk and -43.5% at 52 wk.
conclusionEarly intervention with elosulfase alfa is well-tolerated and produces a decrease in uKS and a trend toward improvement in growth.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.