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全球儿童癌症生存估计和重点设定:基于模拟的分析。

Global childhood cancer survival estimates and priority-setting: a simulation-based analysis.

机构信息

Center for Health Decision Science, Harvard Medical School, Harvard University, Boston, MA, USA.

Harvard T H Chan School of Public Health, and Department of Pediatrics, Harvard Medical School, Harvard University, Boston, MA, USA; Division of General Pediatrics, Boston Children's Hospital, Boston, MA, USA.

出版信息

Lancet Oncol. 2019 Jul;20(7):972-983. doi: 10.1016/S1470-2045(19)30273-6. Epub 2019 May 22.

Abstract

BACKGROUND

Accurate childhood cancer survival estimates are crucial for policy makers and clinicians for priority-setting and planning decisions. However, observed survival estimates are lacking for many countries, and when available, wide variation in outcomes is reported. Understanding the barriers to optimising survival can help improve childhood cancer outcomes. We aimed to provide estimates of global childhood cancer survival, accounting for the impact of multiple factors that affect cancer outcomes in children.

METHODS

We developed a microsimulation model to simulate childhood cancer survival for 200 countries and territories worldwide, accounting for clinical and epidemiologic factors, including country-specific treatment variables, such as availability of chemotherapy, radiation, and surgery. To ensure model results were consistent with reported survival data, we calibrated the model to estimates from the CONCORD-2 and CONCORD-3 studies using an Approximate Bayesian Computation approach. We estimated 5-year net survival for diagnosed cases of childhood cancer in each country and territory and estimated potential survival gains of seven policy interventions focused on improving treatment availability and delivery (ie, increasing the availability of chemotherapy, radiation, general surgery, neurosurgery, or ophthalmic surgery, reducing treatment abandonment, and improving the quality of care to the mean of high-income countries) implemented in isolation or as packages.

FINDINGS

Our model estimated that, for diagnosed cases, global 5-year net childhood cancer survival is currently 37·4% (95% uncertainty interval 34·7-39·8), with large variation by region, ranging from 8·1% (4·4-13·7) in eastern Africa to 83·0% (81·6-84·4) in North America. Among the seven policy interventions modelled, each individually provided small gains, increasing global 5-year net survival to between 38·4% (35·8-40·9) and 44·6% (41·7-47·4). 5-year net survival increased more substantially when policy interventions were bundled into packages that improved service delivery (5-year net survival 50·2% [47·3-53·0]) or that expanded treatment access (54·1% [50·1-58·5]). A comprehensive systems approach consisting of all policy interventions yielded superadditive gains with a global 5-year net survival of 53·6% (51·5-55·6) at 50% scale-up and 80·8% (79·5-82·1) at full implementation.

INTERPRETATION

Childhood cancer survival varies widely by region, with especially poor survival in Africa. Although expanding access to treatment (chemotherapy, radiation, and surgery) and addressing financial toxicity are essential, investments that improve the quality of care, at both the health-system and facility level, are needed to improve childhood cancer outcomes globally.

FUNDING

Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard TH Chan School of Public Health, Harvard Medical School, National Cancer Institute, SickKids, St Jude Children's Research Hospital, Union for International Cancer Control, Children with Cancer UK Davidson and O'Gorman Fellowship.

摘要

背景

准确的儿童癌症生存估计对于政策制定者和临床医生在优先事项设定和规划决策方面至关重要。然而,许多国家缺乏观察到的生存估计,并且即使有可用的估计,也报告了结果的广泛差异。了解优化生存的障碍可以帮助改善儿童癌症的结果。我们旨在提供全球儿童癌症生存的估计数,同时考虑到影响儿童癌症结果的多种因素。

方法

我们开发了一种微观模拟模型,用于模拟全球 200 个国家和地区的儿童癌症生存情况,同时考虑了临床和流行病学因素,包括国家特定的治疗变量,如化疗、放疗和手术的可用性。为了确保模型结果与报告的生存数据一致,我们使用近似贝叶斯计算方法对模型进行了校准,以符合 CONCORD-2 和 CONCORD-3 研究的估计值。我们估计了每个国家和地区诊断出的儿童癌症病例的 5 年净生存率,并估计了 7 项政策干预措施的潜在生存获益,这些措施侧重于提高治疗的可用性和提供(即增加化疗、放疗、普通外科、神经外科或眼科手术的可用性、减少治疗放弃以及提高护理质量以达到高收入国家的平均水平),这些措施是单独实施还是作为一揽子措施实施。

结果

我们的模型估计,对于诊断出的病例,全球 5 年净儿童癌症生存率目前为 37.4%(95%置信区间为 34.7-39.8%),区域差异很大,从东非的 8.1%(4.4-13.7%)到北美的 83.0%(81.6-84.4%)。在我们建模的七种政策干预措施中,每种措施单独实施都只能带来较小的收益,将全球 5 年净生存率提高到 38.4%(35.8-40.9%)至 44.6%(41.7-47.4%)。当政策干预措施捆绑成提高服务提供(5 年净生存率 50.2%[47.3-53.0%])或扩大治疗机会(54.1%[50.1-58.5%])的一揽子计划时,5 年净生存率的增加更为显著。一个包含所有政策干预措施的全面系统方法在 50%的推广水平上产生了超加性收益,全球 5 年净生存率为 53.6%(51.5-55.6%),在全面实施时达到 80.8%(79.5-82.1%)。

解释

儿童癌症的生存情况在区域上差异很大,非洲的生存情况尤其差。尽管扩大治疗(化疗、放疗和手术)的机会和解决财务毒性问题至关重要,但还需要投资提高护理质量,包括在卫生系统和医疗机构层面上的质量,以改善全球儿童癌症的结果。

资助

波士顿儿童医院、达纳法伯癌症研究所、哈佛陈曾熙公共卫生学院、哈佛医学院、美国国立癌症研究所、 SickKids、圣裘德儿童研究医院、国际癌症控制联盟、英国儿童癌症协会 Davidson 和 O'Gorman 奖学金。

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