Invasive urodynamic investigations in the management of women with refractory overactive bladder symptoms: FUTURE, a superiority RCT and economic evaluation.

IF 3.5 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES
Mohamed Abdel-Fattah, Christopher Chapple, Suzanne Breeman, David Cooper, Helen Bell-Gorrod, Preksha Kuppanda, Karen Guerrero, Simon Dixon, Nikki Cotterill, Karen Ward, Hashim Hashim, Ash Monga, Karen Brown, Marcus Drake, Andrew Gammie, Alyaa Mostafa, Rebecca Bruce, Victoria Bell, Christine Kennedy, Suzanne Evans, Graeme MacLennan, John Norrie
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引用次数: 0

Abstract

Background: Overactive bladder is a common problem affecting the United Kingdom adult female population. Symptoms include urinary urgency, with or without urgency incontinence, increased daytime urinary frequency and nocturia. Initial conservative treatments for overactive bladder are unsuccessful in 25-40% of women (refractory overactive bladder). Before considering invasive treatments, such as botulinum toxin injection-A or sacral neuromodulation, guidelines recommend urodynamics to confirm diagnosis of detrusor overactivity. However, the clinical and cost effectiveness of urodynamics has never been robustly assessed.

Objectives: To compare the clinical and cost effectiveness of urodynamics plus comprehensive clinical assessment versus comprehensive clinical assessment only in the management of refractory overactive bladder in women.

Design: Parallel-group, multicentre, superiority, open-label, randomised controlled trial. Allocation by remote web-based randomisation (1 : 1 ratio). The cost-effectiveness analysis took the National Health Service perspective with a model-based lifetime time horizon, as informed by a within-trial analysis.

Setting: Sixty-three United Kingdom secondary and tertiary hospitals.

Participants: Women aged ≥ 18 years with refractory overactive bladder or urgency-predominant mixed urinary incontinence who had failed conservative management and pharmacological treatment and were being considered for invasive treatment. Women were excluded if any of the following criteria were met: predominant stress urinary incontinence; previous urodynamics in last 12 months; current pelvic malignancy or clinically significant pelvic mass; bladder pain syndrome; neurogenic bladder; urogenital fistulae; previous treatment with botulinum toxin injection-A or sacral neuromodulation for urinary incontinence; previous pelvic radiotherapy; prolapse beyond introitus; pregnant or planning pregnancy; recurrent urinary tract infection where a significant pathology has not been excluded; and inability to give an informed consent.

Interventions: Urodynamics plus comprehensive clinical assessment (urodynamics arm) versus comprehensive clinical assessment only.

Main outcome measures: Participant-reported success at the last follow-up time point as measured by the Patient Global Impression of Improvement. Primary economic outcome was incremental cost per quality-adjusted life-year gained as modelled over the lifetime of participants.

Results: A total of 1099 participants were included: 550 randomised to the urodynamics arm and 549 to the comprehensive clinical assessment only arm. At the final follow-up time point, participant-reported success rates of 'very much improved' and 'much improved' were not superior in the urodynamics arm (117 participants; 23.6%) compared to the comprehensive clinical assessment only arm (114 participants; 22.7%) [adjusted odds ratio 1.12 (95% confidence interval 0.73 to 1.74); p = 0.601]. Serious adverse events were low and similar between groups. Based on the estimated incremental costs and quality-adjusted life-years of urodynamics (£463 and 0.011, respectively), the incremental cost-effectiveness ratio was £42,643 per quality-adjusted life-year gained. The cost-effectiveness acceptability curve shows that urodynamics has a 34% probability of being cost-effective at a willingness-to-pay threshold of £20,000 per quality-adjusted life-year gained. This probability reduced further when the results were extrapolated over the patient's lifetime. Limitations include: only short-term outcomes were available, and as most participants underwent botulinum toxin injection-A treatment, pre-planned secondary analyses for some outcomes such as sacral neuromodulation were not possible.

Conclusion: Participant-reported success in the urodynamics arm was not superior to the comprehensive clinical assessment only arm at 15-months follow-up. Urodynamics is not cost-effective at a threshold of £20,000 per quality-adjusted life-year gained. Longer-term follow-up is required to explore need for further interventions and treatments and their effect on the clinical and cost-effectiveness analyses.

Trial registration: This trial is registered as ISRCTN63268739.

Funding: This award was funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme (NIHR award ref: 15/150/05) and is published in full in Health Technology Assessment Vol. 29, No. 27. See the NIHR Funding and Awards website for further award information.

有创尿动力学研究在治疗难治性膀胱过度活动症状的女性中的应用:FUTURE,一项优势随机对照试验和经济评估。
背景:膀胱过度活动是影响英国成年女性人群的常见问题。症状包括尿急,伴或不伴尿急尿失禁,日间尿频增加和夜尿。25-40%的女性(难治性膀胱过度活跃)最初的保守治疗不成功。在考虑有创性治疗前,如注射a型肉毒杆菌毒素或骶骨神经调节,指南建议尿动力学检查以确认逼尿肌过度活动的诊断。然而,尿动力学的临床和成本效益从未得到过有力的评估。目的:比较尿动力学加综合临床评估与单纯综合临床评估治疗难治性膀胱过动症的临床和成本效益。设计:平行组、多中心、优势、开放标签、随机对照试验。远程网络随机分配(1:1比例)。成本效益分析采用了基于模型的终身时间范围的国家卫生服务视角,根据试验内分析提供信息。环境:63家联合王国二级和三级医院。参与者:年龄≥18岁的女性,患有难治性膀胱过动症或急性病混合性尿失禁,保守治疗和药物治疗失败,正在考虑进行侵入性治疗。如果符合以下任何标准,则排除女性:主要压力性尿失禁;过去12个月的尿动力学记录;当前盆腔恶性肿瘤或有临床意义的盆腔肿块;膀胱疼痛综合征;神经性膀胱功能障碍;泌尿生殖管状器官;既往有a型肉毒毒素注射或骶神经调节治疗尿失禁;既往盆腔放疗;脱垂超过内向质;怀孕的或计划怀孕的;复发性尿路感染,未排除明显病理;以及无法给予知情同意。干预措施:尿动力学加综合临床评估(尿动力学组)vs仅综合临床评估。主要结果测量:参与者报告的最后一个随访时间点的成功,以患者总体改善印象来衡量。主要的经济结果是参与者一生中每个质量调整生命年的增量成本。结果:共纳入1099名参与者:550名随机分配到尿动力学组,549名随机分配到综合临床评估组。在最后的随访时间点,参与者报告的“非常改善”和“非常改善”的成功率在尿动力学组中并不优越(117名参与者;23.6%)与仅综合临床评估组(114名参与者;22.7%)[校正优势比1.12(95%可信区间0.73 ~ 1.74);p = 0.601]。组间严重不良事件发生率低且相似。根据估计的尿动力学增量成本和质量调整生命年(分别为463英镑和0.011英镑),每个质量调整生命年的增量成本-效果比为42,643英镑。成本效益可接受度曲线显示,在每个质量调整生命年获得20,000英镑的支付意愿阈值下,尿动力学具有成本效益的概率为34%。当结果外推到病人的一生时,这种可能性进一步降低。局限性包括:只有短期结果,并且由于大多数参与者接受了肉毒杆菌毒素注射a治疗,因此不可能对某些结果(如骶神经调节)进行预先计划的二次分析。结论:随访15个月后,参与者报告的尿动力学组的成功率并不优于仅综合临床评估组。如果每个质量调整生命年的门槛为20,000英镑,Urodynamics就不具有成本效益。需要进行长期随访,以探讨进一步干预和治疗的必要性及其对临床和成本效益分析的影响。试验注册:该试验注册号为ISRCTN63268739。资助:该奖项由美国国家卫生与保健研究所(NIHR)卫生技术评估项目(NIHR奖励编号:15/150/05)资助,全文发表在《卫生技术评估》第29卷第27期。有关进一步的奖励信息,请参阅美国国立卫生研究院资助和奖励网站。
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来源期刊
Health technology assessment
Health technology assessment 医学-卫生保健
CiteScore
6.90
自引率
0.00%
发文量
94
审稿时长
>12 weeks
期刊介绍: Health Technology Assessment (HTA) publishes research information on the effectiveness, costs and broader impact of health technologies for those who use, manage and provide care in the NHS.
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