Study of montelukast in children with sickle cell disease (SMILES): a study protocol for a randomised controlled trial.
Study of montelukast in children with sickle cell disease (SMILES): a study protocol for a randomised controlled trial.
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DOI：
10.1186/s13063-021-05626-6
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发表时间：
2021-10-10
期刊：
影响因子：
2.5
通讯作者：
Kirkham FJ
中科院分区：
文献类型：
--
作者：
Hood AM;Stotesbury H;Kölbel M;DeHaan M;Downes M;Kawadler JM;Sahota S;Dimitriou D;Inusa B;Wilkey O;Pelidis M;Trompeter S;Leigh A;Younis J;Drasar E;Chakravorty S;Rees DC;Height S;Lawson S;Gavlak J;Gupta A;Ridout D;Clark CA;Kirkham FJ
患有镰状细胞性贫血（SCA）的幼儿通常具有与脑白色物质完整性降低、低氧饱和度和睡眠呼吸障碍（SDB）相关的处理速度减慢，部分与腺样体和扁桃体增大有关。SDB的常见治疗包括腺样体切除术和夜间持续气道正压通气（CPAP），但腺样体切除术是一种侵入性外科手术，CPAP很少耐受良好。此外，目前还没有关于这些治疗改善认知功能的能力的共识。几项双盲、随机对照试验（RCT）已经证明了孟鲁司特（一种安全、耐受性良好的抗炎药）作为治疗气道阻塞和减少无SCA儿童腺样体大小的有效性。然而，我们还不知道孟鲁司特是否能减少SCA患儿的腺样体大小并改善其认知功能。孟鲁司特在镰状细胞病儿童中的研究（SMILES）是一项为期12周的多中心，双盲随机对照试验。SMILES旨在招募200名3-7.99岁的SCA和SDB儿科患者，以评估与安慰剂相比，孟鲁司特可改善认知功能（即处理速度）和睡眠并减少腺样体大小和白色物质损伤的程度。患者将随机接受孟鲁司特或安慰剂治疗12周。SMILES试验的主要目的是评估孟鲁司特对SCA幼儿处理速度的影响。在基线和治疗后，我们将进行认知评估;护理人员将完成问卷调查（例如睡眠，疼痛）和人口统计学指标。将从作为标准治疗一部分收集的病历中获得实验室检查值。如果家属同意，患者将在基线和治疗后接受腺样体大小和其他结构和血液动力学定量测量的脑部MRI，我们将获得过夜血氧饱和度。这项研究的结果将增加我们对孟鲁司特是否是一种有效治疗SCA幼儿的理解。使用认知测试和MRI，SMILES试验希望获得关键知识，以帮助制定有针对性的干预措施，以改善SCA幼儿的结果。ClinicalTrials.govNCT04351698.于2020年4月17日注册。欧洲临床试验数据库（EudraCT编号2017-004539-36）。注册日期：2020年5月19日
Young children with sickle cell anaemia (SCA) often have slowed processing speed associated with reduced brain white matter integrity, low oxygen saturation, and sleep-disordered breathing (SDB), related in part to enlarged adenoids and tonsils. Common treatments for SDB include adenotonsillectomy and nocturnal continuous positive airway pressure (CPAP), but adenotonsillectomy is an invasive surgical procedure, and CPAP is rarely well-tolerated. Further, there is no current consensus on the ability of these treatments to improve cognitive function. Several double-blind, randomised controlled trials (RCTs) have demonstrated the efficacy of montelukast, a safe, well-tolerated anti-inflammatory agent, as a treatment for airway obstruction and reducing adenoid size for children who do not have SCA. However, we do not yet know whether montelukast reduces adenoid size and improves cognition function in young children with SCA. The Study of Montelukast In Children with Sickle Cell Disease (SMILES) is a 12-week multicentre, double-blind, RCT. SMILES aims to recruit 200 paediatric patients with SCA and SDB aged 3–7.99 years to assess the extent to which montelukast can improve cognitive function (i.e. processing speed) and sleep and reduce adenoidal size and white matter damage compared to placebo. Patients will be randomised to either montelukast or placebo for 12 weeks. The primary objective of the SMILES trial is to assess the effect of montelukast on processing speed in young children with SCA. At baseline and post-treatment, we will administer a cognitive evaluation; caregivers will complete questionnaires (e.g. sleep, pain) and measures of demographics. Laboratory values will be obtained from medical records collected as part of standard care. If a family agrees, patients will undergo brain MRIs for adenoid size and other structural and haemodynamic quantitative measures at baseline and post-treatment, and we will obtain overnight oximetry. Findings from this study will increase our understanding of whether montelukast is an effective treatment for young children with SCA. Using cognitive testing and MRI, the SMILES trial hopes to gain critical knowledge to help develop targeted interventions to improve the outcomes of young children with SCA. ClinicalTrials.govNCT04351698. Registered on April 17, 2020. European Clinical Trials Database (EudraCT No. 2017-004539-36). Registered on May 19, 2020
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DOI：
10.1164/rccm.200407-894oc
发表时间：
2005-02-15
影响因子：
24.7
作者：
Bisgaard, H;Zielen, S;Polos, P
通讯作者：
Polos, P
影响因子：
3.2
作者：
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影响因子：
8
作者：
Goldbart, Aviv D.;Greenberg-Dotan, Sari;Tal, Asher
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影响因子：
3.8
作者：
Crawford, Regina D.;Jonassaint, Charles R.
通讯作者：
Jonassaint, Charles R.
影响因子：
11.5
作者：
Becker, A
通讯作者：
Becker, A