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[慢性髓性白血病的诊断、治疗及监测建议]

[Recommendations for diagnosis, treatment and monitoring of chronic myeloid leukemia].

作者信息

Almeida António, Castro Isabel, Coutinho Jorge, Guerra Lurdes, Marques Herlander, Pereira Ana Marques

机构信息

Departamento de Hematologia. Instituto Português de Oncologia Francisco Gentil. Lisboa.

出版信息

Acta Med Port. 2009 Sep-Oct;22(5):537-44. Epub 2009 Oct 22.

PMID:19944036
Abstract

Chronic Myeloid Leukemia (CML) is a clonal stem cell disease characterized by the expression of the fusion protein bcr-abl1, which has deregulated tirosine-kinase activity. Tyrosine kinase inhibitors (TKIs), and in particular imatinib, introduced fundamental changes in the treatment of CML, becoming, in most cases, the first-line treatment of choice in the chronic phase of this disease. Compared to other available therapies imatinib results in a marked increase in overall survival, tolerability and quality of life. The introduction of second generation TKI, with increased potency against bcr-abl1, expanded the number of therapeutic options for this disease and offers an alternative for patients resistant or intolerant to imatinib or who have progressed to the accelerated phase under this therapy. In order to achieve optimal outcomes, TKI therapy must be managed rigorously, requiring a careful monitoring of treatment response in pre-established time periods, thus permitting disease evaluation and safe decision of the most adequate option. Despite the definition of the criteria for imatinib treatment response, the therapeutic strategies to adopt according to the responses obtained are less clear. The objective of this paper is to review the criteria for CML diagnosis, treatment and monitoring, with recommendations as to the most adequate therapeutic choice according to the response to TKI therapy. The paper also focuses the current lines of investigation and debate areas that in the short term can significantly change the therapeutic scenario in this disease. These recommendations, supported by published scientific evidence and by the clinical practice of the expert panel involved in their elaboration, may constitute an important instrument for a better understanding and standardisation of the treatment and monitoring of CML in Portugal.

摘要

慢性髓性白血病(CML)是一种克隆性干细胞疾病,其特征是融合蛋白bcr-abl1的表达,该蛋白具有失调的酪氨酸激酶活性。酪氨酸激酶抑制剂(TKIs),尤其是伊马替尼,给CML的治疗带来了根本性变化,在大多数情况下成为这种疾病慢性期的一线治疗选择。与其他可用疗法相比,伊马替尼可显著提高总生存率、耐受性和生活质量。第二代TKI的引入,增强了对bcr-abl1的抑制效力,扩大了该疾病的治疗选择范围,并为对伊马替尼耐药或不耐受或在该疗法下进展至加速期的患者提供了替代方案。为了实现最佳疗效,必须严格管理TKI治疗,需要在预先确定的时间段内仔细监测治疗反应,从而进行疾病评估并安全地决定最合适的治疗方案。尽管已经明确了伊马替尼治疗反应的标准,但根据所获得的反应应采取的治疗策略尚不太明确。本文的目的是回顾CML的诊断、治疗和监测标准,并就根据TKI治疗反应选择最合适的治疗方案提出建议。本文还重点介绍了当前的研究方向和短期内可能显著改变该疾病治疗现状的争议领域。这些建议得到了已发表的科学证据以及参与制定的专家小组临床实践的支持,可能成为更好地理解和规范葡萄牙CML治疗与监测的重要工具。

相似文献

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[Recommendations for diagnosis, treatment and monitoring of chronic myeloid leukemia].[慢性髓性白血病的诊断、治疗及监测建议]
Acta Med Port. 2009 Sep-Oct;22(5):537-44. Epub 2009 Oct 22.
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Important therapeutic targets in chronic myelogenous leukemia.慢性髓性白血病的重要治疗靶点。
Clin Cancer Res. 2007 Feb 15;13(4):1089-97. doi: 10.1158/1078-0432.CCR-06-2147.
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Advanced CML: therapeutic options for patients in accelerated and blast phases.晚期慢性粒细胞白血病:加速期和急变期患者的治疗选择
J Natl Compr Canc Netw. 2008 Mar;6 Suppl 2:S31-S36.
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Treatment strategies for CML.CML 的治疗策略。
Best Pract Res Clin Haematol. 2009 Sep;22(3):303-13. doi: 10.1016/j.beha.2009.08.001.
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Response definitions and European Leukemianet Management recommendations.反应定义和欧洲白血病网管理建议。
Best Pract Res Clin Haematol. 2009 Sep;22(3):331-41. doi: 10.1016/j.beha.2009.10.001.
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Technical aspects and clinical applications of measuring BCR-ABL1 transcripts number in chronic myeloid leukemia.慢性髓性白血病中BCR-ABL1转录本数量检测的技术方面及临床应用
Am J Hematol. 2009 Aug;84(8):517-22. doi: 10.1002/ajh.21457.

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