Abstract：
BACKGROUND: Hypophosphatasia (HPP) is a rare genetic disorder characterized by defective bone mineralization, leading to skeletal abnormalities and systemic complications. Asfotase alfa, a recombinant human tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy, has emerged as a promising treatment for HPP. However, a comprehensive evaluation of its efficacy and safety is warranted to guide clinical practice effectively.
METHODS: The study followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines and was registered in Prospective Register of Systematic Reviews (PROSPERO). A search strategy across databases found studies on asfotase alfa for HPP. Two researchers independently extracted and assessed data. This systematic review examined how the drug impacted clinical outcomes such as survival rates, musculoskeletal symptoms, respiratory function, growth measurements, dental health, quality of life, and laboratory results.
RESULTS: This systematic review included 15 articles with a total of 455 HPP patients. Asfotase alfa was predominantly administered at a dose of 6 mg per kg per week among the reviewed studies. Notable findings included enhanced survival rates, relief from musculoskeletal pain, improvements in respiratory outcomes, growth parameters, dental health, and quality of life. Changes in laboratory variables indicated positive responses to treatment, including changes such as increase in alkaline phosphatase (ALP), decline in pyridoxal 5\'-phosphate (PLP) and inorganic pyrophosphate (PPi) levels.
CONCLUSIONS: Asfotase alfa demonstrates efficacy in improving clinical outcomes and safety in patients with HPP. Its therapeutic benefits extend across various domains. However, Larger, age-stratified comparative studies are needed to further investigate the drug\'s effects in HPP patients.
METHODS: The study followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines and was registered in Prospective Register of Systematic Reviews (PROSPERO). A search strategy across databases found studies on asfotase alfa for HPP. Two researchers independently extracted and assessed data. This systematic review examined how the drug impacted clinical outcomes such as survival rates, musculoskeletal symptoms, respiratory function, growth measurements, dental health, quality of life, and laboratory results.
RESULTS: This systematic review included 15 articles with a total of 455 HPP patients. Asfotase alfa was predominantly administered at a dose of 6 mg per kg per week among the reviewed studies. Notable findings included enhanced survival rates, relief from musculoskeletal pain, improvements in respiratory outcomes, growth parameters, dental health, and quality of life. Changes in laboratory variables indicated positive responses to treatment, including changes such as increase in alkaline phosphatase (ALP), decline in pyridoxal 5\'-phosphate (PLP) and inorganic pyrophosphate (PPi) levels.
CONCLUSIONS: Asfotase alfa demonstrates efficacy in improving clinical outcomes and safety in patients with HPP. Its therapeutic benefits extend across various domains. However, Larger, age-stratified comparative studies are needed to further investigate the drug\'s effects in HPP patients.
摘要：
背景：低磷酸盐症（HPP）是一种罕见的遗传性疾病，以骨矿化缺陷为特征，导致骨骼异常和全身并发症。Asfotasealfa,重组人组织非特异性碱性磷酸酶（TNSALP）酶替代疗法，已成为HPP的有希望的治疗方法。然而,有必要对其疗效和安全性进行全面评估，以有效指导临床实践。
方法：本研究遵循系统评价和荟萃分析（PRISMA）指南的首选报告项目，并在前瞻性系统评价登记册（PROSPERO）中注册。跨数据库的搜索策略发现了对HPP的asfotasealfa的研究。两名研究人员独立提取和评估数据。这项系统评价研究了该药物如何影响临床结果，如生存率，肌肉骨骼症状,呼吸功能,生长测量，牙齿健康,生活质量,和实验室结果。
结果：Asfotasealfa显着提高了生存率，疼痛程度,肌肉力量,功能尺度，矿化,射线照相变化，呼吸功能,生长参数，牙齿健康,生活质量,HPP患者的实验室变量。值得注意的发现包括提高生存率，缓解肌肉骨骼疼痛，呼吸结果的改善，生长参数，牙齿健康,和生活质量。实验室变量的变化表明对治疗的积极反应，包括碱性磷酸酶（ALP）增加等变化，吡哆醛5'-磷酸（PLP）和无机焦磷酸盐（PPi）水平下降。
结论：Asfotasealfa在改善HPP患者的临床结局和安全性方面具有疗效。它的治疗益处延伸到各个领域。然而,较大,需要进行年龄分层的对比研究,以进一步研究该药物在HPP患者中的作用.
方法：本研究遵循系统评价和荟萃分析（PRISMA）指南的首选报告项目，并在前瞻性系统评价登记册（PROSPERO）中注册。跨数据库的搜索策略发现了对HPP的asfotasealfa的研究。两名研究人员独立提取和评估数据。这项系统评价研究了该药物如何影响临床结果，如生存率，肌肉骨骼症状,呼吸功能,生长测量，牙齿健康,生活质量,和实验室结果。
结果：Asfotasealfa显着提高了生存率，疼痛程度,肌肉力量,功能尺度，矿化,射线照相变化，呼吸功能,生长参数，牙齿健康,生活质量,HPP患者的实验室变量。值得注意的发现包括提高生存率，缓解肌肉骨骼疼痛，呼吸结果的改善，生长参数，牙齿健康,和生活质量。实验室变量的变化表明对治疗的积极反应，包括碱性磷酸酶（ALP）增加等变化，吡哆醛5'-磷酸（PLP）和无机焦磷酸盐（PPi）水平下降。
结论：Asfotasealfa在改善HPP患者的临床结局和安全性方面具有疗效。它的治疗益处延伸到各个领域。然而,较大,需要进行年龄分层的对比研究,以进一步研究该药物在HPP患者中的作用.
