The HAVEN study-hydroxychloroquine in ANCA vasculitis evaluation-a multicentre, randomised, double-blind, placebo-controlled trial: study protocol and statistical analysis plan.
The HAVEN study-hydroxychloroquine in ANCA vasculitis evaluation-a multicentre, randomised, double-blind, placebo-controlled trial: study protocol and statistical analysis plan.
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非严重 ANCA 相关性血管炎 (AAV) 患者通常会服用免疫抑制药物，这些药物会带来严重的副作用和生活质量下降。这些患者对更安全有效的治疗的需求尚未得到满足。羟氯喹因其对类似自身免疫性疾病（如系统性红斑狼疮）的作用而正在被探索。双盲、安慰剂对照多中心试验在 20 个地点招募了 76 名患者。在 52 周的过程中，除了标准免疫抑制治疗之外，参与者还将按 1:1 的比例随机接受羟氯喹或安慰剂。 II 期选择设计将用于确定羟氯喹的疗效，使用泼尼松龙剂量和伯明翰血管炎活动评分作为疾病活动的衡量标准。次要结局将探讨 AAV 进展的其他因素，包括疾病发作和缓解时间。该试验旨在探索羟氯喹作为 AAV 患者的治疗方法。如果有效，可以减少对泼尼松龙等免疫抑制治疗的需求。与传统的免疫抑制治疗相比，羟氯喹更安全、更便宜且副作用更少。这可以改善患者的治疗效果，同时为 NHS 节省资金。 ISRCTN：ISRCTN79334891。注册日期：2021 年 6 月 7 日。EudraCT：2018-001268-40。注册日期：2019 年 9 月 13 日。ClinicalTrials.gov：NCT04316494。注册日期为 2020 年 3 月 20 日。在线版本包含可在 10.1186/s13063-023-07108-3 获取的补充材料。
Patients with non-severe ANCA-associated vasculitis (AAV) are often prescribed immunosuppressive medications that are associated with severe side effects and a reduced quality of life. There is an unmet need for safer effective treatments for these patients. Hydroxychloroquine is being explored due to its effect in similar autoimmune conditions such as systemic lupus erythematosus. Double-blind, placebo-controlled multicentre trial recruiting 76 patients across 20 sites. Participants will be randomised 1:1 to hydroxychloroquine or placebo in addition to standard of care immunosuppressive therapies over the course of 52 weeks. A phase II selection design will be used to determine hdroxychloroquine’s efficacy, using prednisolone dosage and Birmingham Vasculitis Activity Score as a measure of disease activity. Secondary outcomes will explore other elements of AAV progression, including disease flares and time to remission. This trial aims to explore Hydroxychloroquine as a treatment for patients with AAV. If effective, the need for immunosuppressive treatments such as prednisolone could be reduced. Hydroxychloroquine is safer, cheaper and has fewer adverse effects than conventional immunosuppressive treatments. This could improve patient outcomes while saving money for the NHS. ISRCTN: ISRCTN79334891. Registered 07 June 2021. EudraCT: 2018-001268-40. Registered 13 September 2019. Clinicaltrials.gov: NCT04316494. Registered 20 March 2020. The online version contains supplementary material available at 10.1186/s13063-023-07108-3.
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影响因子：
13.6
作者：
Kronbichler A;Geetha D;Smith RM;Egan AC;Bajema IM;Schönermarck U;Mahr A;Anders HJ;Bruchfeld A;Cid MC;Jayne DRW
通讯作者：
Jayne DRW
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27.4
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Flossmann, Oliver;Berden, Annelies;Westman, Kerstin
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Westman, Kerstin
影响因子：
13.3
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Feldman, Candace H.;Hiraki, Linda T.;Winkelmayer, Wolfgang C.;Marty, Francisco M.;Franklin, Jessica M.;Kim, Seoyoung C.;Costenbader, Karen H.
通讯作者：
Costenbader, Karen H.
影响因子：
158.5
作者：
Jayne, D;Rasmussen, N;Pusey, C
通讯作者：
Pusey, C
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27.4
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Mukhtyar, C.;Lee, R.;Luqmani, R. A.
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Luqmani, R. A.