[Vosoritide in achondroplasia]
Mengarelli C, Bardach A, Alfie V, Fernandez S, Pichon Riviere A, Garcia Martí S, Ciapponi A, Alcaraz A, Augustovski F
Record ID 32018013572
Spanish
Original Title:
Vosoritide en niños con acondroplasia versus cuidados estándar
Authors' recommendations:
Moderate-quality evidence shows that the use of vosoritide in achondroplastic patients with active growth plates probably yields a considerable net benefit as its use was associated with an increase in measurable annual growth velocity in children 5 to 18 years old, compared to standard care, which maintained up to 104 weeks. Recent information shows that its use increased growth (measured in height Z-scores) and the proportion of upper and lower limb growth over a four-year follow-up period, enhancing quality of life in the physical domain. It has been well-tolerated, with no serious adverse events reported over seven years of follow-up.
No clinical practice guidelines have been found that mention or recommend the use of vosoritide.
Health funders from some high-income countries and from the Latin American region cover it in patients over five years old.
Economic evaluations from Ireland and Nordic countries have been identified; they do not consider it cost-effective. The estimated budget impact for Argentina is high exceeding the payment threshold.
Details
Project Status:
Completed
Year Published:
2024
URL for published report:
https://ets.iecs.org.ar/publication/2230
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Argentina
MeSH Terms
- Achondroplasia
- Child
- Adolescent
- Natriuretic Peptide, C-Type
Contact
Organisation Name:
Institute for Clinical Effectiveness and Health Policy
Contact Address:
Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA
Contact Name:
info@iecs.org.ar
Contact Email:
info@iecs.org.ar
Copyright:
<p>Institute for Clinical Effectiveness and Health Policy (IECS)</p>
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.