Summary

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• Asfotase alfa (Strensiq) is indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP). It has proven effective across all ages, including infants, children, and adults suffering from HPP.
• Three systematic reviews/meta-analyses were examined to gather information on the safety and effectiveness of asfotase alfa in treating HPP.
• No other treatments for HPP were directly compared to asfotase alfa, highlighting its unique position as an effective treatment option for this condition where alternative treatments are absent.
• The long-term data over 5 years suggests that asfotase alfa has an acceptable safety profile given its chronic administration context. However, explicit details on adverse effects or specific safety concerns associated with it were not provided in the studies.
• Asfotase Alfa's effectiveness does not appear to be markedly affected by genetic heterogeneity of HPP, such as biallelic vs monoallelic ALPL variants, making it a broadly applicable treatment across a diverse spectrum of presentations within this disease group.
• Pediatric use is notably important due to significant improvement seen in lifespan quality and management of disease symptoms, but data on adults also expands its therapeutic relevance beyond childhood, addressing the wide age spectrum presented by HPP.