Adverse events during long-term use of alpha asphotase in patients with hypophosphatasia

Hypophosphatasia is an orphan hereditary disease caused by decreased alkaline phosphatase activity, which leads to impaired bone mineralization. To date, more than 550 pathologic variants in the ALPL gene are known, which explains the pronounced polymorphism of symptoms and variability in the severity of the disease. The only pathogenetic therapy is continuous enzyme replacement therapy with the drug asphotase alpha, which is a human recombinant tissue-specific chimeric Fc-deca-aspartate glycoprotein alkaline phosphate glycoprotein. Asphotase alpha therapy for a long time proves its efficacy and safety in the treatment of any forms of hypophosphatasia, which is described in many clinical studies. The article presents three clinical cases describing the experience of long-term treatment with enzyme replacement therapy. Despite a rather extensive list of possible adverse events on the background of therapy, the main and the only side effects in the described cases were reactions at the site of drug administration. Key words: alpha asphotase, hypophosphatasia, adverse events, enzyme replacement therapy, effectiveness of therapy