Effectiveness and safety of asfotase alfa for people with hypophosphatasia: a plain language summary of three studies.

WHAT IS THIS SUMMARY ABOUT?: Hypophosphatasia (HPP for short) is a rare inherited condition that can present at any stage of life, with symptoms typically being more severe in cases that manifest earlier, such as during infancy or childhood. This article summarizes three published studies that looked at the safety and effectiveness of asfotase alfa for people with HPP. The aim of this article is to present the findings of three studies clearly and engagingly to make them accessible to readers without a scientific background. The first study (called study 1) discussed in this article looked at how safe and effective asfotase alfa was for children with life-threatening HPP. The second study (called study 2) looked at how safe and effective asfotase alfa was for children or adolescents who had showed the first signs of HPP at birth or in childhood. They received treatment in clinical practice in Japan, rather than in a clinical research study. The third study (called study 3) looked at how safe and effective asfotase alfa was for adults with HPP who had showed the first signs of the condition in childhood. They received treatment in clinical practice, rather than in a clinical research study.

WHAT WERE THE RESULTS?: In study 1, asfotase alfa improved bone health, the ability to breathe, and growth in children with life-threatening HPP. The researchers saw these benefits over 6 years of follow-up by HPP care teams. The treatment was generally well-tolerated. In study 2, asfotase alfa relieved symptoms such as pain, improved growth, and improved the ability to breathe in children and adolescents with HPP in Japan. The treatment was generally well-tolerated, with no serious side effects. Some participants had mild reactions at injection sites. In study 3, asfotase alfa improved physical function and quality of life for adults with HPP. People were able to walk further and faster and had improved grip strength. The researchers saw these benefits as early as 3 months, and they continued over 2 years. The treatment was welltolerated. Some people had mild reactions at injection sites.

WHAT DO THE RESULTS OF THE STUDIES MEAN?: The combined results from these studies indicate that asfotase alfa is effective and safe to treat people with HPP of different ages and levels of symptom severity. Asfotase alfa improves bone health, mobility, growth, and breathing ability, and relieves pain. Treatment can improve quality of life for people with HPP. The findings from these studies support the continued use and further development of asfotase alfa as a treatment for people with HPP.