对 8-17 岁伴有黏膜嗜酸性粒细胞增多症的功能性消化不良患者使用酮替芬的试点研究

IF 3.4 3区 医学 Q1 PEDIATRICS
Pediatric Drugs Pub Date : 2024-07-01 Epub Date: 2024-05-21 DOI:10.1007/s40272-024-00628-8
Chance S Friesen, Valentina Shakhnovich, Paul Toren, Brandon Retke, Jennifer Schurman, Jennifer Colombo, Amanda Deacy, Craig A Friesen, Susan Abdel-Rahman
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引用次数: 0

摘要

背景和目的:肥大细胞与腹痛相关的肠脑相互作用紊乱(如功能性消化不良)有关。因此,作为第二代抗组胺药和肥大细胞稳定剂的酮替芬可能是治疗这些疾病的可行选择。本试验研究的主要目的是评估功能性消化不良青少年对酮替芬的临床反应,并评估其药代动力学:我们对 11 名患有功能性消化不良和十二指肠粘膜嗜酸性粒细胞增多症的青少年进行了一项随机、双盲、安慰剂对照、交叉试验。根据 5 点李克特量表对总体临床反应进行评分。在稳定状态下采集单份血浆样本进行药代动力学分析:结果:就总体临床反应而言,酮替芬并不比安慰剂更有优势。只有18%的患者表现出完全或接近完全的临床反应。估计半衰期为3.3小时:虽然酮替芬的疗效并不优于安慰剂,但这项研究强调了为患有慢性腹痛的青少年开展药物试验所面临的几个重要挑战。临床试验注册:本研究已在 ClinicalTrials.gov 注册:NCT02484248。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia.

A Pilot Study of Ketotifen in Patients Aged 8-17 Years with Functional Dyspepsia Associated with Mucosal Eosinophilia.

Background and objective: Mast cells have been implicated in abdominal pain-associated disorders of gut-brain interaction, such as functional dyspepsia. As such, ketotifen, a second-generation antihistamine and mast cell stabilizer, could represent a viable treatment option in these conditions. The primary aim of the current pilot study was to assess clinical response to ketotifen and assess pharmacokinetics in youth with functional dyspepsia.

Methods: We conducted a pilot randomized, double-blind, placebo-controlled, cross-over trial of ketotifen in 11 youth with functional dyspepsia and duodenal mucosal eosinophilia with 4 weeks of active treatment at a dose of 1 mg twice daily. Global clinical response was graded on a 5-point Likert Scale. A single plasma sample was obtained at steady state for pharmacokinetic analysis.

Results: Ketotifen was not superior to placebo with regard to global clinical response. Only 18% of patients demonstrated a complete or near-complete clinical response. The estimated half-life was 3.3 h.

Conclusions: While ketotifen was not superior to placebo, this study highlights several important challenges for developing drug trials for youth with chronic abdominal pain. Recommendations are made for designing a larger treatment trial for ketotifen in this patient group.

Clinical trial registration: This study was registered at ClinicalTrials.gov: NCT02484248.

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来源期刊
Pediatric Drugs
Pediatric Drugs PEDIATRICS-PHARMACOLOGY & PHARMACY
CiteScore
7.20
自引率
0.00%
发文量
54
审稿时长
>12 weeks
期刊介绍: Pediatric Drugs promotes the optimization and advancement of all aspects of pharmacotherapy for healthcare professionals interested in pediatric drug therapy (including vaccines). The program of review and original research articles provides healthcare decision makers with clinically applicable knowledge on issues relevant to drug therapy in all areas of neonatology and the care of children and adolescents. The Journal includes: -overviews of contentious or emerging issues. -comprehensive narrative reviews of topics relating to the effective and safe management of drug therapy through all stages of pediatric development. -practical reviews covering optimum drug management of specific clinical situations. -systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. -Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in the pediatric population. -original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pediatric Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.
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