患者报告的急性淋巴细胞白血病治疗结果和不良反应:一项中低收入国家横断面研究。

Eduardo Cerello Chapchap, Nina Melo, Denise Martins, Maria Lucia Lee, Nelson Hamerschlak
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引用次数: 0

摘要

简介巴西成人急性淋巴细胞白血病患者的治疗情况尚未得到很好的描述:方法:对1981年至2019年期间在巴西淋巴瘤和白血病协会(ABRALE)登记的400名急性淋巴细胞白血病患者或其护理人员进行了电话访问,以评估患者报告的对诊断、治疗和不良反应的看法:203名患者为男性,平均年龄为15.7岁,中位随访时间为6.2年。主要症状为发热(39%)、出血/瘀斑(38%)、极度疲劳(30%)和肌肉骨骼疼痛(28%)。公立医疗机构(17.9%)和私立医疗机构(31.1%;P 值 = 0.019)的患者在发病一周内得到诊断的比例不同。此外,公立医疗机构的诊断难度更高:35% 对 22.6%(p 值 = 0.034)。只有 36 名患者能够报告他们的治疗方案;在报告的八种方案中,最常见的是巴西儿童急性淋巴细胞白血病治疗合作组(GBTLI - 10/27.8%)和柏林-法兰克福-明斯特(BFM - 8/22.2%)方案。70名患者(17.5%)需要调整治疗方案,其中37.1%的患者因严重不良反应而需要调整治疗方案;21.7%的患者接受的治疗时间较短(≤6个月),16%的患者进行了同种异体造血干细胞移植,其中17/64(27%)的患者在这一步骤中遇到困难,延迟时间超过3个月。移植指征与微小残留病和头颅放疗有关;41.7%的患者报告了与治疗有关的不良反应(范围:1-6),尤其是:情绪障碍(26.3%)、神经系统缺陷(13.8%)、认知/记忆障碍(12%)和肺部疾病(15%)。不良反应的风险因素包括年龄、移植指征和居住在大城市。诊断和移植延迟等治疗差异仍是这些患者面临的挑战:结论:需要采取紧急干预措施,优化医疗服务,减少不良反应,尤其是对青少年和年轻成人患者的不良反应。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Patient-reported outcomes of treatment and adverse effects following acute lymphoblastic leukemia: a low- and middle-income country cross-sectional study.

Introduction: The scenario of adult patients with acute lymphoblastic leukemia treated in Brazil has not been well described yet.

Methods: Four hundred patients diagnosed with acute lymphoblastic leukemia from 1981 to 2019, registered in the Brazilian lymphoma and leukemia association (ABRALE) or their caregivers were interviewed by telephone to evaluate patient-reported perceptions of diagnosis, treatment and adverse effects.

Results: Overall, 203 were male with a mean age of 15.7 years and median follow-up of 6.2 years. Main presenting symptoms were fever (39 %), bleeding/ecchymosis (38 %), intense fatigue (30 %), and musculoskeletal pain (28 %). The proportion of patients diagnosed within one week of symptoms onset differed between public (17.9 %) and private healthcare (31.1 %; p-value = 0.019). Additionally, diagnostic difficulties were higher in public care: 35 % versus 22.6 % (p-value = 0.034). Only 36 patients were able to report their treatment protocols; from a list of eight reported protocols, the most common were the Brazilian Childhood Cooperative Group for Treatment of Acute Lymphoblastic Leukemia in Children (GBTLI - 10/27.8 %) and Berlin-Frankfurt-Münster (BFM - 8/22.2 %). Seventy patients (17.5 %) required treatment modification, 37.1 % due to severe adverse effects; 21.7 % received short treatment duration (≤6 months) and 16 % proceeded to allogeneic hematopoietic stem cell transplantation with 17/64 (27 %) reporting difficulties in this step, characterized as >3 months delay. Indication for transplantation was related to minimal residual disease and cranial radiotherapy; 41.7 % reported treatment-related adverse effects (range: 1-6), in particular: mood disorders (26.3 %), neurologic deficit (13.8 %), cognitive/memory impairment (12 %), and lung disease (15 %). Risk factors for adverse effects were age, indication of transplantation and living in a large city. Treatment disparities such as diagnostic and transplantation delays remain challenges in these patients.

Conclusions: Urgent interventions are needed to optimize healthcare and reduce adverse effects, especially in adolescent and young adult patients.

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