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关于高危骨髓增生异常综合征使用去甲基化药物进行桥接治疗后行异基因移植的专家意见及相关建议调查

Survey of expert opinions and related recommendations regarding bridging therapy using hypomethylating agents followed by allogeneic transplantation for high-risk MDS.

作者信息

Sohn Sang Kyun, Moon Joon Ho

机构信息

Dept. of Hematology, Kyungpook National University Hospital, 50 Samduck-2Ka, Jung-Ku, Daegu, South Korea.

Dept. of Hematology, Kyungpook National University Hospital, 50 Samduck-2Ka, Jung-Ku, Daegu, South Korea.

出版信息

Crit Rev Oncol Hematol. 2015 Aug;95(2):243-50. doi: 10.1016/j.critrevonc.2015.03.004. Epub 2015 Mar 18.

DOI:10.1016/j.critrevonc.2015.03.004
PMID:25840907
Abstract

According to current guidelines on therapeutic strategies for myelodysplastic syndrome (MDS), cytoreductive therapies before allogeneic stem cell transplantation (SCT) are not widely recommended for patients with high-risk MDS or refractory anemia with excess blasts (RAEB) who are eligible for allogeneic SCT because of controversial evidence on the role of such therapies. Yet, while treatment with hypomethylating agents (HMAs) has a critical limitation in eradicating MDS clones, the use of HMA treatment as a bridge to allogeneic SCT has become a focus with the hope of improving the SCT outcome based on the chance of achieving complete remission or reducing the blast percentage safely and effectively before allogeneic SCT. However, a consensus needs to be established on the use of HMAs as a bridging therapy for high-risk MDS or RAEB. Thus, the Korean AML/MDS working party group surveyed 34 Korean MDS experts on their bridging therapies for high-risk MDS. Accordingly, this paper presents the survey questionnaire and resulting data, along with a summary of the consensus and related recommendations regarding strategies using HMA treatment and allogeneic SCT based on reported studies and the current survey results.

摘要

根据目前关于骨髓增生异常综合征(MDS)治疗策略的指南,对于高危MDS或有过多原始细胞的难治性贫血(RAEB)且符合异基因干细胞移植(SCT)条件的患者,在异基因SCT前进行细胞减灭治疗未被广泛推荐,因为此类治疗作用的证据存在争议。然而,虽然使用去甲基化药物(HMA)治疗在根除MDS克隆方面存在关键局限性,但将HMA治疗用作异基因SCT的桥梁已成为一个焦点,人们希望基于在异基因SCT前实现完全缓解或安全有效地降低原始细胞百分比的机会来改善SCT结果。然而,对于将HMA用作高危MDS或RAEB的桥接治疗,需要达成共识。因此,韩国急性髓系白血病/骨髓增生异常综合征工作组对34位韩国MDS专家进行了关于高危MDS桥接治疗的调查。据此,本文展示了调查问卷及所得数据,以及基于已发表研究和当前调查结果的关于使用HMA治疗和异基因SCT策略的共识及相关建议的总结。

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Survey of expert opinions and related recommendations regarding bridging therapy using hypomethylating agents followed by allogeneic transplantation for high-risk MDS.关于高危骨髓增生异常综合征使用去甲基化药物进行桥接治疗后行异基因移植的专家意见及相关建议调查
Crit Rev Oncol Hematol. 2015 Aug;95(2):243-50. doi: 10.1016/j.critrevonc.2015.03.004. Epub 2015 Mar 18.
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Survey of experts on therapeutic policies and proposals for the optimal timing for allogeneic peripheral blood stem cell transplantation in transfusion-dependent patients with myelodysplastic syndrome-refractory anemia.输血依赖型骨髓增生异常综合征难治性贫血患者异基因外周血干细胞移植最佳时机的治疗策略专家调查及建议
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[The role of pre-transplant debulking treatment in patients undergoing allogeneic stem cell transplantation for high-risk myelodysplastic syndrome].[移植前减瘤治疗在高危骨髓增生异常综合征患者异基因干细胞移植中的作用]
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When is the optimal timing for allogeneic transplantation in the case of MDS patients treated with hypomethylating agents?对于接受低甲基化剂治疗的 MDS 患者,何时是异体移植的最佳时机?
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Comparison of conditioning regimens of various intensities for allogeneic hematopoietic SCT using HLA-identical sibling donors in AML and MDS with <10% BM blasts: a report from EBMT.比较不同强度预处理方案在 HLA 相合同胞供者异基因造血干细胞移植治疗 AML 和 MDS 中<10%BM 原始细胞患者中的疗效:来自 EBMT 的报告。
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Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology.原发性骨髓增生异常综合征治疗的循证与共识性实践指南。来自意大利血液学会的声明。
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Thiotepa, busulfan, and cyclophosphamide as a preparative regimen for allogeneic transplantation for advanced myelodysplastic syndrome and acute myelogenous leukemia.噻替派、白消安和环磷酰胺作为晚期骨髓增生异常综合征和急性髓性白血病同种异体移植的预处理方案。
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Children with myelodysplastic syndrome (MDS) and increasing mixed chimaerism after allogeneic stem cell transplantation have a poor outcome which can be improved by pre-emptive immunotherapy.患有骨髓增生异常综合征(MDS)且异基因干细胞移植后混合嵌合体比例不断增加的儿童预后较差,而抢先免疫疗法可改善这种情况。
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引用本文的文献

1
No benefit of hypomethylating agents compared to supportive care for higher risk myelodysplastic syndrome.与支持性治疗相比,低甲基化药物治疗高危骨髓增生异常综合征没有获益。
Korean J Intern Med. 2018 Nov;33(6):1194-1202. doi: 10.3904/kjim.2016.426. Epub 2017 Dec 15.