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asfotase alfa (STRENSIQo) and hypophosphatasia

机译：Ashotase Alfa（strensiqo）和次磷酸盐

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Hypophosphatasia is a rare inherited disorder for which there is no known curative treatment as of 2017. Its manifestations are highly variable, including in particular disorders of defective bone mineralisation with fractures, growth and gait disorders, and respiratory problems. Its severity usually declines with the age of onset of the first signs and symptoms, with mortality approaching 100% in the weeks following birth for perinatal-onset forms of the disease, and over 50% during the first year of life when signs and symptoms develop before the age of 6 months (infantile-onset forms).The juvenile-onset forms, which develop after the age of 6 months, are often less severe but can have considerable functional consequences.Asfotase alfa (Strensiq0, Alexion) is a recombinant protein that reproduces the activity of tissue non-specific alkaline phosphatase, the enzyme which is deficient in hypophosphatasia. This drug has been authorised in the European Union for the treatment of paediatric-onset hypophosphatasia, i.e. patients in whom manifestations developed before the age of 18 years.Clinical evaluation mainly consists of three non-comparative trials: two trials in 11 and 59 children under the age of 5 years with perinatal or infantile hypophosphatasia, and one trial in 8 children aged 6 years to 12 years with hypophosphatasia of later onset.In the two trials in children with perinatal- or infantile-onset hypophosphatasia, the estimated mortality after 5 years was 9%. Comparisons versus historical controls, which provide a low level of evidence, showed lower mortality in children treated with asfotase alfa.In children with juvenile-onset hypophosphatasia, the data suggest some improvements in height and weight with asfotase alfa.The main identified adverse effects of asfotase alfa are injection site reactions, lipodystrophy, hypersensitivity reactions, and probably ectopic calcifications, especially in the eyes.
机译：次磷酸盐是一种罕见的遗传障碍，其截至2017年没有已知的治疗方法。其表现是高度可变的，包括特定的骨矿化与骨折，生长和步态疾病的缺陷症，以及呼吸问题。其严重程度通常随着第一个迹象和症状的发病年龄而下降，死亡率在出生后的遗传疾病形式后的几周内接近100％，并且在征兆和症状发展的第一年的生命的第一年超过50％以上超过50％在6个月（婴儿发病形式）之前。幼年发病形式，在6个月之后发展，通常不那么严重，但可以具有相当的功能性后果。传染酶Alfa（strensiq0，alexion）是重组蛋白质再现组织非特异性碱性磷酸酶的活性，缺血性缺乏的酶。该药物已在欧盟授权用于治疗儿科发病次磷酸盐，即患者在18岁之前制定的表现。临床评价主要包括三项非比较试验：在11和59名儿童中进行两项试验围产期或婴儿次磷酸的年龄5年，以及8名6岁儿童的一项试验，患有后来的次磷酸盐萎缩的次磷酸盐。在围产期或婴儿发病次磷酸的儿童的两项试验中，5年后的估计死亡率是9％。与历史对照进行比较，这提供了低级别的证据，表明，在患有青少年发作的次磷酸盐的儿童治疗儿童的儿童中表现出较低的死亡率，该数据表明Asfotase Alfa的高度和体重增加有一些改善。主要确定的副作用Ashotase Alfa是注射部位反应，脂肪蓄水池，过敏反应，以及可能异位钙化，尤其是眼睛。

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    《Prescrire international》 |2017年第187期|共2页
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