Velaglucerase alfa
Sources réglementaires consultées
Indications approuvées
- Traitement enzymatique substitutif au long cours de la maladie de Gaucher de type 1.
Mises en garde cliniques
- Mise en garde encadrée · Avertissement encadre: des reactions d'hypersensibilite potentiellement mortelles, y compris l'anaphylaxie, peuvent survenir. Commencer et administrer dans un etablissement de sante disposant d'un soutien de reanimation. — FDA VPRIV prescribing information
Effets indésirables
Communs (≥1%)
Reactions d'hypersensibilite · céphalée · vertiges · douleur abdominale · nausées · pyrexie
Grossesse et allaitement
Les donnees disponibles de pharmacovigilance et de cohortes n'ont pas identifie d'association avec des issues defavorables, mais la decision pendant la grossesse ou l'allaitement doit etre individualisee avec l'equipe specialisee.
Bibliographie récente (PubMed)
Gaucher disease (GD) is a rare disorder linked to the absence/deficiency of glucocerebrosidase. GD can be treated by enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). The aim of this systematic review (SR) is to assess the effectiveness of drugs used for GD treatment. Searches were conducted in PubMed and Scopus, in April 2021. The search strategies encompassed the name of the disease and of the drug treatments. Manual search was also conducted. Observational and interventional longitudinal studies evaluating ERT and SRT for GD were included. Single mean meta-analyses were conducted for each drug using R. The initial search retrieved 2246 articles after duplicates were removed. Following screening and eligibility assessment, 68 reports were included. The studies evaluated imiglucerase, velaglucerase alfa, taliglucerase alfa, miglustat, and eliglustat. The results showed that ERT is effective as a treatment in both naïve and experienced patients. Miglustat did not significantly improve blood outcomes in naïve patients and resulted in a decrease in the platelet levels of experienced patients. Eliglustat was mainly assessed for experienced patients and resulted in stable outcome values. This extensive SR confirms the effectiveness of GD treatments in short- and long-term follow-ups. The results were favorable for all ERTs and for eliglustat. Based on the assessed evidence, miglustat did not achieved expressive results. However, all evidence should be interpreted considering its limitations and does not replace well-conducted randomized trials.
BACKGROUND: Gaucher 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. METHODS: A 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. RESULTS: A 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