Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia.

IF 5 2区 医学 Q1 ENDOCRINOLOGY & METABOLISM
Dalal S Ali, Reza D Mirza, Farah Alsarraf, Salma Hussein, Hajar Abu Alrob, Natasha M Appelman-Dijkstra, Signe Sparre Beck-Nielsen, Martin Biosse-Duplan, Maria Luisa Brandi, Thomas O Carpenter, Catherine Chaussain, Martine Cohen-Solal, Rachel K Crowley, Karel Dandurand, Pablo Florenzano, Seiji Fukumoto, Claudia Gagnon, Paul Goodyer, Corinna Grasemann, Erik A Imel, Suzanne M Jan de Beur, Anna Lehman, E Michael Lewiecki, Emmett Morgante, Leanne M Ward, Aliya A Khan, Gordon Guyatt
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引用次数: 0

Abstract

Objective: To examine the highest certainty evidence addressing the management of X-linked hypophosphatemia (XLH) in adults to inform treatment recommendations.

Methods: We searched Embase, MEDLINE, Web of Science, and Cochrane Central up to May 2023. Eligible studies included RCTs and observational studies of individuals 18+ with clinically or genetically confirmed XLH. Manuscripts comparing burosumab to no treatment or conventional therapy (phosphate and active vitamin D) and conventional therapy to no treatment were included. Two reviewers independently determined eligibility, extracted data, and assessed risk of bias (RoB). GRADE methodology was used to assess evidence certainty.

Results: We screened 4,114 records, after removing duplicates, and assessed 254 full texts. One RCT and two observational studies were eligible. The RCT of burosumab versus no treatment had low RoB. Burosumab probably improves pain from fracture/pseudofracture healing (moderate certainty) but has little or no impact on direct pain measures (moderate certainty). Burosumab may reduce the need for parathyroidectomy (low certainty) but has little or no impact on fatigue (high certainty), stiffness (moderate certainty), and mobility (low certainty) over 24 weeks. Burosumab may increase dental abscess risk (low certainty). Indirect evidence comparing burosumab to conventional therapy provided low certainty regarding burosumab versus conventional therapy. Two observational studies on conventional therapy versus no treatment had high RoB and very low certainty regarding the impact of conventional therapy on patient-important outcomes.

Conclusion: No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy versus no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH.

系统评价:药物治疗对x连锁低磷血症成年患者重要结局的疗效。
目的:研究成人x连锁低磷血症(XLH)管理的最高确定性证据,以告知治疗建议。方法:检索Embase、MEDLINE、Web of Science和Cochrane Central,检索时间截止到2023年5月。符合条件的研究包括临床或遗传证实的18岁以上XLH患者的随机对照试验和观察性研究。文献中包括了比较布罗单抗与无治疗或常规治疗(磷酸盐和活性维生素D)以及常规治疗与无治疗的文献。两名审稿人独立确定入选资格、提取数据并评估偏倚风险(RoB)。GRADE方法用于评估证据的确定性。结果:我们筛选了4,114条记录,剔除重复,评估了254篇全文。一项随机对照试验和两项观察性研究符合条件。布罗单抗与未治疗的RCT结果显示,RoB较低。Burosumab可能改善骨折/假性骨折愈合的疼痛(中等确定性),但对直接疼痛测量的影响很小或没有影响(中等确定性)。Burosumab可能减少甲状旁腺切除术的需要(低确定性),但在24周内对疲劳(高确定性)、僵硬(中等确定性)和活动(低确定性)几乎没有影响。布若单抗可能增加牙脓肿风险(低确定性)。比较布罗单抗与常规治疗的间接证据提供了布罗单抗与常规治疗的低确定性。两项关于常规治疗与不治疗的观察性研究的RoB较高,而常规治疗对患者重要结局影响的确定性非常低。结论:在成人中不存在布罗单抗和常规治疗之间的正式比较。常规治疗与不治疗的证据非常不确定。我们的综述强调,需要更多关于布罗单抗和常规治疗对成年XLH患者重要结局的长期影响的数据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Journal of Clinical Endocrinology & Metabolism
Journal of Clinical Endocrinology & Metabolism 医学-内分泌学与代谢
CiteScore
11.40
自引率
5.20%
发文量
673
审稿时长
1 months
期刊介绍: The Journal of Clinical Endocrinology & Metabolism is the world"s leading peer-reviewed journal for endocrine clinical research and cutting edge clinical practice reviews. Each issue provides the latest in-depth coverage of new developments enhancing our understanding, diagnosis and treatment of endocrine and metabolic disorders. Regular features of special interest to endocrine consultants include clinical trials, clinical reviews, clinical practice guidelines, case seminars, and controversies in clinical endocrinology, as well as original reports of the most important advances in patient-oriented endocrine and metabolic research. According to the latest Thomson Reuters Journal Citation Report, JCE&M articles were cited 64,185 times in 2008.
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