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我们确实仍在进行 CML 的移植,不是吗?

We do still transplant CML, don't we?

机构信息

Centre for Clinical Haematology, Queen Elizabeth Hospital, Birmingham, United Kingdom.

出版信息

Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):177-184. doi: 10.1182/asheducation-2018.1.177.

DOI:10.1182/asheducation-2018.1.177
PMID:30504307
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6246013/
Abstract

The remarkable clinical activity of tyrosine kinase inhibitors (TKIs) in chronic myeloid leukemia (CML) has transformed patient outcome. Consequently, allogeneic stem cell transplantation (allo-SCT) is no longer the only treatment modality with the ability to deliver long-term survival. In contrast to the central position it held in the treatment algorithm 20 years ago, allografting is now largely reserved for patients with either chronic-phase disease resistant to TKI therapy or advanced-phase disease. Over the same period, progress in transplant technology, principally the introduction of reduced intensity conditioning regimens coupled with increased donor availability, has extended transplant options in patients with CML whose outcome can be predicted to be poor if they are treated with TKIs alone. Consequently, transplantation is still a vitally important, potentially curative therapeutic modality in selected patients with either chronic- or advanced-phase CML. The major causes of transplant failure in patients allografted for CML are transplant toxicity and disease relapse. A greater understanding of the distinct contributions made by various factors such as patient fitness, patient-donor HLA disparity, conditioning regimen intensity, and transplant toxicity increasingly permits personalized transplant decision making. At the same time, advances in the design of conditioning regimens coupled with the use of adjunctive posttransplant cellular and pharmacologic therapies provide opportunities for reducing the risk of disease relapse. The role of SCT in the management of CML will grow in the future because of an increase in disease prevalence and because of continued improvements in transplant outcome.

摘要

酪氨酸激酶抑制剂 (TKI) 在慢性髓性白血病 (CML) 中的显著临床活性改变了患者的预后。因此,异基因造血干细胞移植 (allo-SCT) 不再是唯一能够实现长期生存的治疗方式。与 20 年前在治疗方案中占据中心地位相比,同种异体移植现在主要保留给对 TKI 治疗有耐药性的慢性期疾病或晚期疾病的患者。在此期间,移植技术的进步,主要是引入强度降低的调理方案,加上供体可用性的增加,为 CML 患者的移植选择提供了扩展,对于这些患者,如果仅用 TKI 治疗,其预后可能较差。因此,在选择的慢性期或晚期 CML 患者中,移植仍然是一种至关重要的、潜在的治愈性治疗方式。同种异体移植治疗 CML 患者失败的主要原因是移植毒性和疾病复发。对各种因素(如患者健康状况、患者-供体 HLA 差异、调理方案强度和移植毒性)的不同贡献有了更深入的了解,越来越能够实现个体化的移植决策。同时,调理方案的设计进步以及辅助性移植后细胞和药物治疗的应用,为降低疾病复发风险提供了机会。由于疾病流行率的增加以及移植结果的持续改善,SCT 在 CML 管理中的作用将在未来增长。

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Life after ponatinib failure: outcomes of chronic and accelerated phase CML patients who discontinued ponatinib in the salvage setting.泊那替尼治疗失败后的生存情况:在挽救治疗中停用泊那替尼的慢性期和加速期慢性髓性白血病患者的结局
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Cancer. 2017 Nov 15;123(22):4391-4402. doi: 10.1002/cncr.30864. Epub 2017 Jul 25.
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Transplantation of Ex Vivo Expanded Umbilical Cord Blood (NiCord) Decreases Early Infection and Hospitalization.体外扩增脐带血移植(NiCord)可降低早期感染率和住院率。
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Overall survival with ponatinib versus allogeneic stem cell transplantation in Philadelphia chromosome-positive leukemias with the T315I mutation.在伴有T315I突变的费城染色体阳性白血病中,泊那替尼与异基因干细胞移植的总生存期比较。
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