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复发/难治性急性髓系白血病新型治疗药物的管理和进展更新:一个伊比利亚专家小组的共识。

Update on management and progress of novel therapeutics for R/R AML: an Iberian expert panel consensus.

机构信息

Hematology Department, Hospital Universitari I Politècnic La Fe, Av. Fernando Abril Martorell, 106, 46026, Valencia, Spain.

CIBERONC, Instituto de Salud Carlos III, Madrid, Spain.

出版信息

Ann Hematol. 2019 Nov;98(11):2467-2483. doi: 10.1007/s00277-019-03820-w. Epub 2019 Oct 30.

DOI:10.1007/s00277-019-03820-w
PMID:31667544
Abstract

A significant proportion of adult patients with acute myeloid leukemia (AML) fail to achieve complete remission or will relapse later on after achieving it. Prognosis for relapsed or refractory (R/R) AML patients remains discouraging, with the main curative option still relying on hematopoietic stem cell transplant (HSCT) for those who are eligible. Beyond morphological bone marrow and peripheral blood assessment, evaluation of patient performance status and comorbidities, as well as genetic/molecular characterization, is crucial to make an accurate diagnosis and prognosis, which will be useful to select the most appropriate treatment. Emerging strategies are mainly focusing on the development of immune- and molecular-based approaches. Novel targeted therapies are generally well tolerated, potentially allowing them to be administered alone or in combination with classical chemotherapy agents. Enrolment in clinical trials should be considered first option for R/R AML patients, either as a bridge to HSCT or to benefit from novel therapies that eventually may prolong survival and improve quality of life. An Iberian expert panel has reviewed the recent advances in the management of R/R AML with the aim to develop updated evidence and expert opinion-based recommendations.

摘要

相当比例的急性髓系白血病(AML)成年患者未能达到完全缓解,或在达到缓解后会复发。复发/难治(R/R)AML 患者的预后仍然令人沮丧,主要的治疗选择仍然依赖于造血干细胞移植(HSCT),但仅适用于符合条件的患者。除了形态学骨髓和外周血评估外,对患者的身体状况和合并症的评估,以及遗传/分子特征的评估,对于做出准确的诊断和预后至关重要,这将有助于选择最合适的治疗方法。新兴策略主要集中在免疫和基于分子的方法的发展上。新型靶向治疗通常具有良好的耐受性,有可能单独使用或与经典化疗药物联合使用。对于 R/R AML 患者,应首先考虑参加临床试验,作为 HSCT 的桥梁,或受益于新的治疗方法,这些方法最终可能延长生存时间并提高生活质量。一个伊比利亚专家小组审查了 R/R AML 治疗的最新进展,旨在制定更新的证据和专家意见为基础的建议。

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