Asfotase alfa's effect on rare bone disorder extends beyond survival, read!

According to a novel review published in ‘Bone’ journal, Asfotase alfa emerged as a pioneering enzyme replacement therapy for people with hypophosphatasia (HPP). HPP is a rare genetic disorder that leads to severe skeletal issues and systemic complications, posing a significant challenge for patients and healthcare providers alike. This systematic review by Amirhossein Shirinezhad et al. followed rigorous PRISMA guidelines and was registered in the PROSPERO database.

It investigated the impact of Asfotase alfa (recombinant human tissue-nonspecific alkaline phosphatase enzyme replacement therapy), on clinical outcomes for HPP patients. Asfotase alfa was most commonly given at a weekly dose of 6 mg/kg in the studies reviewed. After analyzing 15 studies involving 455 patients, the research proposed noteworthy improvements in survival rates, musculoskeletal pain relief, respiratory function, growth, dental health, and overall quality of life.

The treatment also exposed favorable changes in key laboratory markers (increased alkaline phosphatase, reduced levels of pyridoxal 5'-phosphate and inorganic pyrophosphate), implying a robust response to therapy. There is a need for larger, comparative studies in different age groups further to dig deep into this therapy for HPP patients, concluded the study authors.