阿扎胞苷对低危骨髓增生异常综合症没有明显的生存获益：长崎的一项回顾性研究。,Cancer Science

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阿扎胞苷对低危骨髓增生异常综合症没有明显的生存获益：长崎的一项回顾性研究。
Cancer Science ( IF 4.5 ) Pub Date : 2020-09-17 , DOI: 10.1111/cas.14653
Eo Toriyama ₁ , Tomoko Hata ₂ , Ken-Ichi Yokota ₃ , Masahiko Chiwata ₄ , Rena Kamijo ₅ , Miki Hashimoto ₁ , Masataka Taguchi ₆ , Makiko Horai ₆ , Masatoshi Matsuo ₇ , Emi Matsuo ₈ , Yumi Takasaki ₉ , Yasuhisa Kawaguchi ₁₀ , Hidehiro Itonaga ₆ , Shinya Sato ₆ , Koji Ando ₂ , Yasushi Sawayama ₆ , Jun Taguchi ₁₁ , Yoshitaka Imaizumi ₆ , Hideki Tsushima ₇ , Tatsuro Jo ₁₁ , Shinichiro Yoshida ₄ , Yukiyoshi Moriuchi ₅ , Yasushi Miyazaki _{1,

2}

Affiliation

Department of Hematology, Atomic Bomb Disease and Hibakusha Medicine Unit, Atomic Bomb Disease Institute, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan.
Department of Hematology, Atomic Bomb Disease and Hibakusha Medicine Unit, Atomic Bomb Disease Institute, Nagasaki University, Nagasaki, Japan.
Biostatistics Section, Division of Scientific Data Registry, Atomic Bomb Disease Institute, Nagasaki University, Nagasaki, Japan.
Department of Hematology, National Hospital Organization Nagasaki Medical Center, Ohmura, Japan.
Department of Hematology, Sasebo City General Medical Center, Sasebo, Japan.
Department of Hematology, Nagasaki University Hospital, Nagasaki, Japan.
Department of Hematology, Nagasaki Harbor Medical Center, Nagasaki, Japan.
Department of Hematology, Japan Community Health Care Organization Isahaya General Hospital, Isahaya, Japan.
Department of Internal Medicine, Saint Francis Hospital, Nagasaki, Japan.
Department of Internal Medicine, National Hospital Organization Nagasaki Hospital, Nagasaki, Japan.
Department of Hematology, Japanese Red Cross Nagasaki Genbaku Hospital, Nagasaki, Japan.

阿扎胞苷（AZA）对低危（LR）骨髓增生异常综合症（MDS）生存的疗效存在争议。为解决此问题，我们回顾了国际预后评分系统（IPSS）定义的AZA对LR-MDS患者的长期生存获益。利用长崎县489例LR‐MDS患者的数据，比较了接受国际支持小组（BSC），免疫抑制治疗（IST），促红细胞生成促进剂的患者根据国际工作组2006年的血液学反应和总生存期（OS）（ ESA）和AZA。接受AZA治疗的患者显示完全缓解（CR）率为11.3％，骨髓CR为1.9％，血液学改善率为34.0％，其中27.3％的患者具有红细胞输血独立性（TI）。和血小板分别占20％的患者。接受IST，ESA，BSC和AZA的患者的中位OS（分别未达到91个月，58个月和29个月）差异显着（P <0.001）。在超过20％的接受AZA治疗的患者中观察到了与感染相关的严重不良事件。多因素分析显示，OS的年龄，性别，诊断时的IPSS评分和输血依赖性均显着，但AZA治疗则无统计学意义，对AZA的反应仍然均匀，并且添加了AZA给药时的IPSS危险状况。对于LR‐MDS患者，我们没有发现AZA治疗具有明显的生存获益。

"点击查看英文标题和摘要"

No clear survival benefit of azacitidine for lower-risk myelodysplastic syndromes: a retrospective study of Nagasaki.

The efficacy of azacitidine (AZA) on survival of lower risk (LR) ‐ myelodysplastic syndromes (MDS) is controversial. To address this issue, we retrospectively evaluated the long‐term survival benefit of AZA for patients with LR‐MDS defined by International Prognostic Scoring System (IPSS). Using data from 489 patients with LR‐MDS in Nagasaki, hematologic responses according to International Working Group 2006 and overall survival (OS) were compared among patients that received best supportive care (BSC), immunosuppressive therapy (IST), erythropoiesis‐stimulating agents (ESA), and AZA. Patients treated with AZA showed complete remission (CR) rate at 11.3%, marrow CR at 1.9%, and any hematologic improvement at 34.0%, with transfusion independence (TI) of red blood cells in 27.3% of patients. and platelet in 20% of patients, respectively. Median OS for patients received IST, ESA, BSC, and AZA (not reached, 91 months, 58 months, and 29 months, respectively) differed significantly (P < .001). Infection‐related severe adverse events were observed in more than 20% of patients treated with AZA. Multivariate analysis showed age, sex, IPSS score at diagnosis, and transfusion dependence were significant for OS, but AZA treatment was not, which maintained even response to AZA, and IPSS risk status at AZA administration was added as factors. We could not find significant survival benefit of AZA treatment for LR‐MDS patients.

更新日期：2020-09-17

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