SynthesisOrphanet journal of rare diseases2026
Investigating the therapeutic profile of velaglucerase alfa in paediatric patients with Gaucher disease: a systematic review across all paediatric age groups.
Synthesis in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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.
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5 authors.
Funding
Abstract
backgroundGaucher disease (GD) is a rare autosomal recessive genetic disorder. The clinical manifestations can be adequately managed with enzyme replacement therapy (ERT). The aim of this systematic literature review was to explore the safety and efficacy or effectiveness (depending on the type of evidence) profile of velaglucerase alfa in the treatment of paediatric patients with type 1 (GD1) and type 3 (GD3) GD across all paediatric ages.
methodsA systematic review of the PubMed/Medline and Embase databases, along with communications from international conferences, was conducted. The inclusion criteria comprised clinical studies published in either English or Spanish that assessed the therapeutic profile of velaglucerase alfa in patients with GD1 (primarily) and GD3 (exploratorily) of all paediatric ages (0–18 years). For each of the selected publications, data regarding the safety and efficacy/effectiveness of this treatment were extracted.
resultsA total of 539 publications were identified, of which 23 studies encompassing data from 159 paediatric patients were included. Nine studies (71 patients) provided information about the safety in paediatric patients with GD1, describing it as well tolerated. Regarding the efficacy/effectiveness, 14 articles (113 patients) reported relevant data for the same subpopulation. Overall, improvements in haematological, visceral, skeletal, biomarker and health-related quality-of-life outcomes have been described in treatment-naïve paediatric patients with GD1 who were initially treated with velaglucerase alfa, as well as maintained stability in patients previously treated with imiglucerase. Furthermore, it has been reported that the safety and efficacy/effectiveness profile administered as home therapy enhances the quality of life for both patients and caregivers. The use of velaglucerase alfa in paediatric patients with GD3 was described in 7 publications (26 patients), suggesting a favourable safety profile, whereas its efficacy/effectiveness was reported in 5 articles (16 patients). Improvements in the non-neurological manifestations of the disease were recorded in patients with GD3.
conclusionThis systematic review summarizes the limited evidence on velaglucerase alfa in paediatric patients with GD. Findings suggest that velaglucerase alfa may be a beneficial option for GD1 across all paediatric age groups (0–18 years). Additionally, it might be considered a therapeutic option for non-neurological GD3 symptoms, although evidence is scarce and exploratory, highlighting the need for further research in those patients.
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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 Socratic graph.