Mbarara University of Science and Technology, Mbarara, Uganda.
The Collaborative African Network for Childhood Cancer Care and Research, CANCaRe Africa, Blantyre, Malawi.
Pediatr Blood Cancer. 2024 Aug;71(8):e31069. doi: 10.1002/pbc.31069. Epub 2024 May 21.
The Wilms Africa studies implemented an adapted Wilm's tumor (WT) treatment protocol in sub-Saharan Africa in two phases. Phase I began with four sites and provided out-of-pocket costs. Phase II expanded the number of sites, but lost funding provision. Objective is to describe the outcomes of Phase II and compare with Phase I.
Wilms Africa Phase I (n = 4 sites; 2014-2018) and Phase II (n = 8 sites; 2021-2022) used adapted treatment protocols. Funding for families' out-of-pocket costs was provided during Phase I but not Phase II. Eligibility criteria were age less than 16 years and newly diagnosed unilateral WT. We documented patients' outcome at the end of planned first-line treatment categorized as treatment abandonment, death during treatment, and disease-related events (death before treatment, persistent disease, relapse, or progressive disease). Sensitivity analysis compared outcomes in the same four sites.
We included 431 patients in Phase I (n = 201) and Phase II (n = 230). The proportion alive without evidence of disease decreased from 69% in Phase I to 54% in Phase II at all sites (p = .002) and 58% at the original four sites (p = .04). Treatment abandonment increased overall from 12% to 26% (p < .001), and was 20% (p = .04) at the original four sites. Disease-related events (5% vs. 6% vs. 6%) and deaths during treatment (14% vs. 14% vs. 17%) were similar.
Provision of out-of-pocket costs was important to improve patient outcomes at the end of planned first-line treatment in WT. Prevention of treatment abandonment remains an important challenge.
威尔姆斯非洲研究在撒哈拉以南非洲的两个阶段实施了适应性威尔姆斯肿瘤 (WT) 治疗方案。第一阶段从四个地点开始,提供自费费用。第二阶段扩大了地点数量,但失去了资金供应。目的是描述第二阶段的结果,并与第一阶段进行比较。
威尔姆斯非洲第一阶段(n=4 个地点;2014-2018 年)和第二阶段(n=8 个地点;2021-2022 年)使用了适应性治疗方案。第一阶段为家庭自费费用提供资金,但第二阶段没有。入选标准为年龄小于 16 岁且新诊断为单侧 WT。我们记录了计划一线治疗结束时患者的结局,分为治疗放弃、治疗期间死亡和疾病相关事件(治疗前死亡、持续疾病、复发或进行性疾病)。敏感性分析比较了相同四个地点的结果。
我们纳入了第一阶段(n=201)和第二阶段(n=230)的 431 名患者。在所有地点,无疾病证据的存活率从第一阶段的 69%下降到第二阶段的 54%(p=0.002),在最初的四个地点下降到 58%(p=0.04)。治疗放弃率总体从 12%增加到 26%(p<0.001),在最初的四个地点为 20%(p=0.04)。疾病相关事件(5%对 6%对 6%)和治疗期间死亡(14%对 14%对 17%)相似。
提供自费费用对于改善 WT 计划一线治疗结束时的患者结局非常重要。预防治疗放弃仍然是一个重要的挑战。