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JCO：口服阿扎胞苷对低风险骨髓增生异常综合征患者的影响

2021-03-29 宁咸襄 MedSci原创

阿扎胞苷骨髓增生异常综合征
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Journal of Clinical Oncology近日发表了一篇研究。结果显示，CC-486虽然可以增加LR-MDS患者不依赖红细胞输血比例，但可能会增加患者早死风险。

近日，Journal of Clinical Oncology在线发表了一篇研究，研究显示，CC-486虽然可以增加LR-MDS患者不依赖红细胞输血比例，但可能会增加患者早死风险。

论文封面截图

骨髓增生异常综合症（MDS）涵盖了一组以骨髓异常增生和外周血细胞减少症为特征的血液系统疾病。国际预后评分系统（IPSS）根据细胞遗传学特征，血细胞减少症的数量和胚细胞百分比将患者分为四个风险组：低风险和中风险-1型被认为是较低风险的MDS（LR-MDS），而中风险-2型和高风险被认为是较高风险的MDS。

该实验于2013年4月至2018年2月在20个国家/地区的101个地点招募了患者，研究一共纳入216例LR-MDS患者，患者入组后按照1:1比例被随机分入CC-486组（107例）和安慰剂组（109例），两组患者分别接受300mgCC-486/安慰剂治疗，21天/28天为一个治疗周期。

技术路线


入组患者中位血小板计数为25×109/L。在CC-486组和安慰剂组中，分别有31%和11％的患者接受了非红细胞依赖输注治疗（RBC-TI）（P=0.0002），两组的中位持续治疗时间分别为11.1个月和5个月。相比基线，CC-486组血红蛋白升高≥1.5g/dL的患者比例更高（23.4%vs 4.6%），血液学的改善情况亦更好（24.3%vs 6.5%）。虽然两组的总死亡率相似，但是在研究开始的56天内，CC-486组患者死亡率更高（16例vs 6例），大多数死亡原因为中性粒细胞减少导致感染。


虽然CC-486可以增加具备高风险临床特征的LR-MDS患者的RBC-TI治疗比例并改善此类患者血液学情况，但在CC-486组中更多的患者出现因中性粒细胞减少导致感染的早死情况。

这些结果表明，CC-486可以在具有症状性疾病和难治性血细胞减少症的LR-MDS患者中减少RBC的输血负担并改善血小板减少症，但是CC-486组的早期死亡发生率较高，这与治疗前中性粒细胞减少症患者的感染密切相关。正在进行的分子和HRQoL分析可能为这些临床结果提供更多背景。有必要对MDS患者进行CC-486的进一步评估，这可能有助于确定可能从该疗法中受益的患者。

 

参考文献：

Garcia-Manero G, Santini V, Almeida A, Platzbecker U, Jonasova A, Silverman LR, Falantes J, Reda G, Buccisano F, Fenaux P, Buckstein R, Diez Campelo M, Larsen S, Valcarcel D, Vyas P, Giai V, Olíva EN, Shortt J, Niederwieser D, Mittelman M, Fianchi L, La Torre I, Zhong J, Laille E, Lopes de Menezes D, Skikne B, Beach CL, Giagounidis A. Phase III, Randomized, Placebo-Controlled Trial of CC-486 (Oral Azacitidine) in Patients With Lower-Risk Myelodysplastic Syndromes. J Clin Oncol. 2021 Mar 25:JCO2002619. doi: 10.1200/JCO.20.02619. Epub ahead of print. PMID: 33764805.

https://ascopubs.org/doi/10.1200/JCO.20.02619?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed

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    2022-09-02 小小医者

    口服#阿扎胞苷#对低风险#骨髓增生异常综合征#患者的影响

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    2021-11-22 huangshifeng

    #骨髓增生异常#

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    2021-10-19 xinmeili

    #骨髓增生异常综合#

    0

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    2021-11-02 fengyqf

    #骨髓增生#

    0

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    2021-09-14 lidong40

    #JCO#

    0

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    2021-03-31 zhouqu_8

    #综合征#

    0

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    2021-03-30 carrotlyl

    认真

    0

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