Nyeko Richard, Geriga Fadhil, Angom Racheal, Kambugu Joyce Balagadde, van Heerden Jaques
Department of Paediatrics and Child Health, Lira University, P.O. Box 1035, Lira, Uganda.
Division of Paediatric Oncology, Uganda Cancer Institute, P.O. Box 3935, Kampala, Uganda.
BMC Cancer. 2025 Aug 11;25(1):1299. doi: 10.1186/s12885-025-14735-3.
The treatment outcomes for children and adolescents with rhabdomyosarcoma (RMS) in low-income countries are poor. However, there is a paucity of literature on RMS and its management outcomes in low-resource settings. We evaluated the treatment of RMS with the aim of identifying prognostic factors during management to improve outcomes.
We sourced data on children under 18 years treated for rhabdomyosarcoma at the Uganda Cancer Institute between January 2016 and December 2020. Kaplan-Meier survival analysis and Cox's proportional hazards model were used for five-year survival analysis.
One hundred twenty-eight RMS cases were identified, with a median age of 6.0 years (IQR 3.6-10.0). The most common primary tumour site was the head and neck region, comprising non-parameingeal sites, 37 (28.9%); parameingeal sites, 32 (25.0%); and orbital tumours, 17 (13.3%). Overall, 68 (53.1%) of the primary tumour sites were unfavourable sites. Seventeen (13.3%) patients had metastatic disease at diagnosis, primarily to the lungs, 11 (64.8%). Embryonal and alveolar RMS accounted for 50.0% and 20.3% of the cases, respectively. Only 31 (24.2%) of the patients underwent surgery, and 36 (28.1%) were irradiated. The treatment completion rate was 33.6%, while 46.1% abandoned treatment. Only 25 (19.5%) patients were alive at the time of the study, 65 (50.8%) had died, and 38 (29.7%) had an unknown status. The five-year overall and event-free survival rates were 35% and 30%, respectively. Orbital primary tumour site (HR = 2.86; 95% CI 1.12-7.31; p = 0.028), metastatic disease (HR = 4.09; 95% CI 2.01-8.31; p < 0.001), elevated serum lactate dehydrogenase at diagnosis above 400 U/L (HR = 2.80; 95% CI 1.46-5.33; p = 0.002), and lack of local control (HR = 3.33; 95% CI 1.34-8.29; p = 0.010) were significant factors for poor survival.
Rhabdomyosarcoma outcomes in Ugandan children are largely poor, with high treatment abandonment and mortality. Concerted, multidisciplinary efforts are needed to improve outcomes in this setting.
低收入国家儿童和青少年横纹肌肉瘤（RMS）的治疗效果较差。然而，关于资源匮乏地区RMS及其管理结果的文献较少。我们评估了RMS的治疗情况，旨在确定管理过程中的预后因素以改善治疗结果。
我们收集了2016年1月至2020年12月期间在乌干达癌症研究所接受横纹肌肉瘤治疗的18岁以下儿童的数据。采用Kaplan-Meier生存分析和Cox比例风险模型进行五年生存分析。
共识别出128例RMS病例，中位年龄为6.0岁（四分位间距3.6 - 10.0岁）。最常见的原发肿瘤部位是头颈部区域，包括非脑膜旁部位37例（28.9%）；脑膜旁部位32例（25.0%）；眼眶肿瘤17例（13.3%）。总体而言，68例（53.1%）原发肿瘤部位为不良部位。17例（13.3%）患者在诊断时已有转移性疾病，主要转移至肺部，11例（64.8%）。胚胎型和肺泡型RMS分别占病例的50.0%和20.3%。仅31例（24.2%）患者接受了手术，36例（28.1%）接受了放疗。治疗完成率为33.6%，而46.1%的患者放弃了治疗。在研究时，仅25例（19.5%）患者存活，65例（50.8%）患者死亡，38例（29.7%）患者状态不明。五年总生存率和无事件生存率分别为35%和30%。眼眶原发肿瘤部位（风险比[HR]=2.86；95%置信区间[CI] 1.12 - 7.31；P = 0.028）、转移性疾病（HR = 4.09；95% CI 2.01 - 8.31；P < 0.001）、诊断时血清乳酸脱氢酶升高超过400 U/L（HR = 2.80；95% CI 1.46 - 5.33；P = 0.002）以及缺乏局部控制（HR = 3.33；95% CI 1.34 - 8.29；P = 0.010）是生存不良的重要因素。
乌干达儿童横纹肌肉瘤的治疗结果总体较差，治疗放弃率和死亡率较高。需要协同进行多学科努力以改善这种情况下的治疗结果。
