Summary

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• This summary is based on the review of three systematic review(s)/meta-analysis(es). ^[1-3]
• Asfotase alfa is effective in treating Hypophosphatasia (HPP) across various age groups, including infants, children, and adults, contributing to increased patient lifespan.
• In a study involving adolescents and adults with HPP, asfotase alfa normalized TNSALP (Tissue nonspecific isoenzyme of alkaline phosphatase) substrate concentrations and improved functional outcomes, with similar clinical improvements observed in both biallelic and monoallelic variant groups, despite biallelic patients having higher baseline levels of inorganic pyrophosphate [PPi] and pyridoxal 5'-phosphate [PLP].
• Overall, asfotase alfa demonstrates consistent efficacy in managing HPP, with effectiveness noted in various populations and no significant differences in outcomes among different genetic subgroups, as reported in the studies.
• The safety profile of asfotase alfa in patients with HPP indicates no significant safety concerns or adverse effects reported in the available literature.
• Safety outcomes were evaluated across different doses of asfotase alfa, with no specific safety concerns highlighted for adolescents and adults with HPP, regardless of ALPL (alkaline phosphatase) variant state (biallelic or monoallelic).
• Asfotase alfa is effective for treating HPP across various age groups, including infants, children, and adults, with no specific differences in effectiveness among population types; both biallelic and monoallelic patients showed similar clinical improvements, despite biallelic patients having higher baseline levels of PPi and PLP.
• The studies specifically focus on adolescents and adults with HPP categorized by ALPL variant state, providing evidence that both groups benefit from asfotase alfa treatment without notable differences in treatment response.