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
Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients With X-Linked Hypophosphatemia.
作者信息
Ali Dalal S, Mirza Reza D, Alsarraf Farah, Hussein Salma, Abu Alrob Hajar, Appelman-Dijkstra Natasha M, Beck-Nielsen Signe Sparre, Biosse-Duplan Martin, Brandi Maria Luisa, Carpenter Thomas O, Chaussain Catherine, Cohen-Solal Martine, Crowley Rachel K, Dandurand Karel, Florenzano Pablo, Fukumoto Seiji, Gagnon Claudia, Goodyer Paul, Grasemann Corinna, Imel Erik A, Jan de Beur Suzanne M, Lehman Anna, Lewiecki E Michael, Morgante Emmett, Ward Leanne M, Khan Aliya A, Guyatt Gordon
机构信息
Division of Endocrinology and Metabolism, McMaster University, Hamilton, ON, Canada L8S 4L8.
Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada L8S 4L8.
出版信息
J Clin Endocrinol Metab. 2025 May 19;110(6):1767-1780. doi: 10.1210/clinem/dgae890.
CONTEXT
Understanding the effects of burosumab compared to conventional therapy or no treatment on patient-important outcomes in adults with X-linked hypophosphatemia (XLH) is essential to guide evidence-based treatment recommendations.
OBJECTIVE
To examine the highest certainty evidence addressing the management of 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 randomized controlled trials (RCTs) and observational studies of individuals aged 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 4114 records, after removing duplicates, and assessed 254 full texts. One RCT and 2 observational studies were eligible. The RCT of burosumab vs 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 vs conventional therapy. Two observational studies on conventional therapy vs 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 vs 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.
背景
了解布罗索尤单抗与传统疗法或不治疗相比，对成人X连锁低磷血症（XLH）患者重要结局的影响，对于指导基于证据的治疗建议至关重要。
目的
审查关于成人XLH管理的最高确定性证据，以为治疗建议提供信息。
方法
我们检索了截至2023年5月的Embase、MEDLINE、Web of Science和Cochrane Central。符合条件的研究包括随机对照试验（RCT）以及对18岁及以上临床或基因确诊XLH个体的观察性研究。纳入了比较布罗索尤单抗与不治疗或传统疗法（磷酸盐和活性维生素D）以及传统疗法与不治疗的手稿。两名审查员独立确定 eligibility、提取数据并评估偏倚风险（RoB）。采用GRADE方法评估证据确定性。
结果
我们筛选了4114条记录，去除重复项后，评估了254篇全文。1项RCT和2项观察性研究符合条件。布罗索尤单抗与不治疗的RCT的RoB较低。布罗索尤单抗可能改善骨折/假性骨折愈合引起的疼痛（中等确定性），但对直接疼痛测量几乎没有影响（中等确定性）。布罗索尤单抗可能减少甲状旁腺切除术的需求（低确定性），但在24周内对疲劳（高确定性）、僵硬（中等确定性）和活动能力（低确定性）几乎没有影响。布罗索尤单抗可能增加牙脓肿风险（低确定性）。比较布罗索尤单抗与传统疗法的间接证据关于布罗索尤单抗与传统疗法的确定性较低。两项关于传统疗法与不治疗的观察性研究的RoB较高，关于传统疗法对患者重要结局的影响的确定性非常低。
结论
目前尚无成人布罗索尤单抗与传统疗法之间的正式比较。传统疗法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索尤单抗和传统疗法对成人XLH患者重要结局的长期影响的数据。
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