ArticleAdvances in therapy2025
Mobility and Quality of Life in Children with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement.
Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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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
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Who cites it
3 citing papers in PubMed.
- Evolving Therapeutic Paradigms in Pediatric Hypophosphatasia: From Survival-Driven Care to Integrated Precision Management.International journal of molecular sciences · 2026Review
- Natural history of hypophosphatasia in Chinese patients younger than 18 years: a single-center retrospective observational study.Frontiers in endocrinology · 2026Observational
- Key Learnings from Clinical Research and Real-World Evidence on Asfotase Alfa Effectiveness in Hypophosphatasia: 10 Years Post-Approval.Advances in therapy · 2025Review
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionHypophosphatasia (HPP) is a rare, inherited metabolic bone disease with a high degree of morbidity and mortality in children. Asfotase alfa is an enzyme replacement therapy for HPP reimbursed in the UK since 2017 under a Managed Access Agreement (MAA). This analysis assessed the effectiveness and safety of asfotase alfa in children < 18 years of age.
methodsThe MAA was a prospective, longitudinal data collection in children with paediatric-onset HPP. Effectiveness outcomes were evaluated in children who were treated with asfotase alfa for ≥ 6 months. Data were collected on respiratory support, growth, mobility, motor development, analgesic use, quality of life, and safety at enrolment and throughout the 5-year MAA.
resultsTwenty-four children enrolled in the MAA and 20 were included in the analysis. Twelve children had received asfotase alfa before enrolment through a clinical trial or compassionate use program. From baseline to month 60, the median (minimum, maximum) change in height and weight Z-scores were 0.20 (- 0.9, 1.2; n = 6) and - 0.5 (- 1.9, 1.5; n = 6), respectively. The median (minimum, maximum) percent of predicted distance walked in the 6-Minute Walk Test increased by 3.8% (- 8.6, 4.3; n = 5) at month 3 and was sustained through follow-up. Median (minimum, maximum) child- and parent-reported Pediatric Quality of Life Inventory scores were 59.2 (15.2, 91.3; n = 11) and 53.4 (16.3, 100.0; n = 18) at baseline and increased by 21.7 (5.4, 37.0; n = 3) and 16.3 (9.8, 45.7; n = 4) at month 60, respectively. Treatment-naïve children had a greater clinical response than treatment-experienced participants, who maintained their status. No deaths occurred in the study. The most common adverse events were injection site reactions, reported in 8/24 participants (33.3%).
conclusionThis analysis confirmed the clinical benefit of asfotase alfa in children with HPP. Asfotase alfa was well tolerated, with no new safety signals identified.
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