Hypophosphatasia: presentation and response to asfotase alfa
Authors' conclusion
HPP treatment with AA in individuals with both pediatric and adult-onset forms resulted in significant subjective improvement in musculoskeletal and cognitive manifestations in addition to patients' quality of life. The drug was well tolerated in 6 patients. 1 patient discontinued therapy because of minor adverse effects with myalgias.
Study context
- Population
- Patients with hypophosphatasia (HPP) confirmed by DNA analysis, aged 19–68 years (4 females, 3 males); 5 pediatric-onset, 2 adult-onset
- Participants
- 7 patients
- Outcome
- Subjective clinical improvement with AA
- Follow-up
- Mean 6 months (SD= 5)
Main finding
As reported in the abstract
- Muscle strength and pain improvement from baseline (subjective)
- up to 70%
- Walking distance improvement
- up to 100%
- Mood improvement
- up to 90%
- Energy level improvement
- 75%
- First clinical signs of improvement after 3 months of therapy
- 4 out of 6 patients
Observational data: associations, not effects.
Safety
Adverse events
- Toe fracture 10 months on Asfotase Alfa (AA)
- 1 out of the 7 patients
- AA well tolerated; injection site reactions most reported adverse effect
Original publication
Hypophosphatasia: presentation and response to asfotase alfa. Osteoporos Int. Published 23 November 2023. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →