What happens to intolerant, relapsed or refractory chronic myeloid leukemia patients without access to clinical trials?
What happens to intolerant, relapsed or refractory chronic myeloid leukemia patients without access to clinical trials?
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DOI：
10.1016/j.htct.2018.11.005
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发表时间：
2019-09-01
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影响因子：
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通讯作者：
Silla, Lucia Mariano da Rocha
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文献类型：
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作者：
Bosi, Guilherme Rasia;Fogliatto, Laura Maria;Silla, Lucia Mariano da Rocha
目的：为了评估临床结果的不耐受，复发或难治性患者谁不能与新的酪氨酸激酶抑制剂或实验therapy.Methods治疗：回顾性队列的90例慢性粒细胞白血病患者在所有阶段的疾病治疗伊马替尼甲磺酸作为他们的第一TKI疗法，并与达沙替尼或尼洛替尼作为下一个治疗线。我们评估了这些患者的临床结果，特别关注的组，需要两个以上的治疗line.Results：百分之三十九的患者难治性或不耐受伊马替尼。与那些能够维持伊马替尼作为一线治疗的患者相比，接受三线或三线以上治疗的患者的8年总生存率明显较低（分别为83%和22%，p < 0.01）。总生存率下降与晚期疾病（p < 0.01）、一线治疗未能达到主要分子学缓解（p < 0.01）和因任何原因中断一线治疗（p = 0.023）相关。在实现完全细胞遗传学反应和主要分子反应和治疗中断失败与进展到三线treatment.Conclusion：观察复发，不耐受或难治性慢性期CML患者的关键结果反映了未满足的需要，这组患者没有替代疗法，如新药或临床试验中的实验性疗法。更广泛地获得新的治疗可能性是提高CML患者生存率的关键资产，特别是那些对一线治疗难治或不耐受的患者。(C)2019年巴西血液学协会，Hemoterapia e Terapia Celular。由爱思唯尔编辑有限公司出版。这是一个在CC BY-NC-ND许可证下的开放获取文章（http：//creativecommons.org/licenses/by-nc-nd/4.0/）。
Objective: To assess clinical outcomes of intolerant, relapsed or refractory patients who could not be treated with new tyrosine kinase inhibitors or experimental therapies.Methods: A retrospective cohort of 90 chronic myeloid leukemia patients in all phases of the disease treated with imatinib mesylate as their first TKI therapy, and with dasatinib or nilotinib as the next line of therapy. We evaluated clinical outcomes of these patients, with special focus on the group that needed more than two therapy lines.Results: Thirty-nine percent of patients were refractory or intolerant to imatinib. An 8-year overall survival rate of the patients who went through three or more lines of treatment was significantly lower, compared to those who were able to maintain imatinib as their first-line therapy (83% and 22%, respectively p < 0.01). Decreased overall survival was associated with advanced-phase disease (p < 0.01), failure to achieve major molecular response in first-line treatment (p < 0.01) and interruption of first-line treatment due to any reason (p = 0.023). Failure in achieving complete cytogenetic response and major molecular response and treatment interruption were associated with the progression to the third-line treatment.Conclusion: The critical outcome observed in relapsed, intolerant or refractory chronic phase CML patients reflects the unmet need for this group of patients without an alternative therapy, such as new drugs or experimental therapies in clinical trials. Broader access to newer treatment possibilities is a crucial asset to improve survival among CML patients, especially those refractory or intolerant to first-line therapies. (C) 2019 Associacao Brasileira de Hematologia, Hemoterapia e Terapia Celular. Published by Elsevier Editora Ltda. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).