■急性淋巴细胞白血病(ALL)是最常见的儿科恶性肿瘤。虽然在高收入国家,儿童ALL的存活率超过90%。低收入和中等收入国家的估计生存率为22-79%,取决于地区和当地资源。
■本研究回顾性回顾了人口学,生物,以及2013-2017年间在南美4个国家的5个儿科中心出现的18岁以下新诊断ALL儿童的临床参数.使用Kaplan-Meier方法估计生存分析。
■跨越五个中心,对752例患者进行了分析(玻利维亚[N=9],厄瓜多尔[N=221],巴拉圭[N=197],秘鲁[N=325])和92.1%(n=690)的患者被诊断为B细胞和7.5%(n=56)的T细胞ALL。中位年龄为5.5岁(IQR7.29)。诊断时,根据他们的机构方案,47.8%的患者被归类为标准和51.9%的患者被归类为高风险。先进的诊断可用性因模式而异。对69.1%的患者进行了MRD评估;ETV6-RUNX可进行分子检测,BCR-ABL1、TCF3-PBX1和KMT2A在75-81%的患者中重排ALL;然而,iAMP21的核型分析和评估仅在42-61%的患者中进行.57.3%(n=429)的患者在诊断时评估了中枢神经系统(CNS)受累;其中,93.7%(n=402)为CNS1,1.6%(n=7)为CNS2,0.7%(n=11)为CNS3,1.9%(n=8)为颅神经麻痹,2.1%(n=9)的结果不可用。在治疗期间,有56.0%(n=421)的患者报告化疗延迟>2周。延迟归因于63.2%(n=265)的感染,药物相关毒性占47.3%(n=198),和资源限制,包括23.2%(n=97)的患者缺乏床位。3年放弃敏感性EFS和OS分别为61.0±1.9%和67.2±1.8%,分别。3年EFS和OS分别为71.0±1.8%和79.6±1.7%,分别。
■这项工作揭示了改善生存的机会,包括解决严重感染,治疗中断,和药物短缺导致的修改。2018年,南美洲的医疗保健专业人员与圣裘德儿童研究医院合作成立了拉丁美洲儿科肿瘤学(POLA)小组。POLA合作者开发了一种基于证据的,达成共识,适应治疗指南,根据这项评估的初步结果,作为参与机构中儿科ALL的新护理标准。
UNASSIGNED: Acute lymphoblastic Leukemia (ALL) is the most common pediatric malignancy. While the survival rate for childhood ALL exceeds 90% in high-income countries, the estimated survival in low-and middle-income countries ranges from 22-79%, depending on the region and local resources.
UNASSIGNED: This study retrospectively reviewed demographic, biological, and clinical parameters of children under 18 years of age with newly diagnosed ALL presenting between 2013-2017 across five pediatric centers in 4 countries in South America. Survival analyses were estimated using the Kaplan-Meier method.
UNASSIGNED: Across the five centers, 752 patients were analyzed (Bolivia [N=9], Ecuador [N=221], Paraguay [N=197], Peru [N=325]) and 92.1% (n=690) patients were diagnosed with B-cell and 7.5% (n= 56) with T-cell ALL. The median age was 5.5 years old (IQR 7.29). At diagnosis, 47.8% of patients were categorized as standard and 51.9% as high risk per their institutional regimen. Advanced diagnostics availability varied between modalities. MRD was evaluated in 69.1% of patients; molecular testing was available for ETV6-RUNX, BCR-ABL1, TCF3-PBX1, and KMT2A-rearranged ALL in 75-81% of patients; however, karyotyping and evaluation for iAMP21 were only performed in 42-61% of patients. Central nervous system (CNS) involvement was evaluated at diagnosis in 57.3% (n=429) patients; of these, 93.7% (n=402) were CNS 1, 1.6% (n=7) were CNS 2, 0.7% (n=11) were CNS3, 1.9% (n=8) had cranial nerve palsy, and 2.1% (n=9) results unavailable. Chemotherapy delays >2 weeks were reported in 56.0% (n=421) patients during treatment. Delays were attributed to infection in 63.2% (n=265), drug-related toxicities in 47.3% (n=198), and resource constraints, including lack of bed availability in 23.2% (n=97) of patients. The 3-year Abandonment-sensitive EFS and OS were 61.0±1.9% and 67.2±1.8%, respectively. The 3-year EFS and OS were 71.0±1.8% and 79.6±1.7%, respectively.
UNASSIGNED: This work reveals opportunities to improve survival, including addressing severe infections, treatment interruptions, and modifications due to drug shortages. In 2018, healthcare professionals across South America established the Pediatric Oncology Latin America (POLA) group in collaboration with St. Jude Children\'s Research Hospital. POLA collaborators developed an evidence-based, consensus-derived, adapted treatment guideline, informed by preliminary results of this evaluation, to serve as the new standard of care for pediatric ALL in participating institutions.