A retrospective analysis of azacitidine treatment for juvenile myelomonocytic leukemia
A retrospective analysis of azacitidine treatment for juvenile myelomonocytic leukemia
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阿扎胞苷治疗幼年粒单核细胞白血病的回顾性分析
DOI：
10.1007/s12185-021-03248-x
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发表时间：
2021
影响因子：
2.1
通讯作者：
Sakashita Kazuo
中科院分区：
文献类型：
--
作者：
Honda Yuko;Muramatsu Hideki;Nanjo Yuka;Hirabayashi Shinsuke;Meguro Toru;Yoshida Nao;Kakuda Harumi;Ozono Shuichi;Wakamatsu Manabu;Moritake Hiroshi;Yasui Masahiro;Sano Hideki;Manabe Atsushi;Sakashita Kazuo
小儿髓单细胞白血病（JMML）是一种预后不良的儿童血液系统恶性肿瘤。虽然已经发表了几个病例系列，描述了JMML患者对阿扎胞苷（AZA）治疗的血液学和分子反应，但AZA的有效性和安全性并没有得到很好的研究，特别是在亚洲儿童和接受造血干细胞移植（HSCT）的儿童中。我们回顾性分析了日本5例接受了12个周期（中位2个周期）AZA治疗的患者。5例患者均为男孩，治疗时年龄分别为21、23、24、26、46个月。所有5例患者均耐受AZA治疗，包括4例HSCT后接受AZA治疗的患者。AZA的治疗毒性主要局限于血液毒性。唯一严重的非血液学不良事件是在第二次HSCT后接受AZA治疗的患者中观察到的高胆红素血症（III-IV级）。5名接受AZA治疗的患者中有2名获得了部分缓解（PR），而3名接受移植后复发治疗的患者没有客观缓解。未来应开展前瞻性研究，开发与AZA等分子靶向药物联合治疗高危患者的方法。
Juvenile myelomonocytic leukemia (JMML) is a pediatric hematological malignancy with a poor prognosis. Although several case series have been published describing hematological and molecular responses to azacitidine (AZA) treatment in patients with JMML, the efficacy and safety profile of AZA is not well investigated, especially in Asian children and children undergoing hematopoietic stem cell transplantation (HSCT). We retrospectively analyzed 5 patients who received a total of 12 cycles (median 2 cycles) of AZA treatment in Japan. All five patients were boys and their ages at the time of treatment were 21, 23, 24, 26, and 46 months, respectively. All five patients tolerated AZA treatment, including four patients who received AZA after HSCT. Therapeutic toxicity with AZA was mostly limited to hematological toxicity. The only serious non-hematological adverse event was hyperbilirubinemia (grades III–IV) observed in a patient who received AZA after a second HSCT. Two out of five patients treated with AZA achieved a partial response (PR), while three patients treated for post-transplant relapse did not have an objective response. Future prospective studies should be conducted to develop combination therapies with AZA and other molecular targeted drugs for high-risk patients.
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影响因子：
20.3
作者：
Locatelli, F;Nöllke, P;Niemeyer, CM
通讯作者：
Niemeyer, CM
儿童时期患有慢性粒单核细胞白血病。
影响因子：
20.3
作者：
C. Niemeyer;M. Aricò;G. Basso;A. Biondi;A. C. Rajnoldi;U. Creutzig;O. Haas;J. Harbott;H. Hasle;G. Kerndrup;F. Locatelli;G. Mann;B. Stollmann;E. T. Veer;E. Wering;M. Zimmermann
通讯作者：
M. Zimmermann
与常规护理方案相比，阿扎西丁氨酸的疗效在治疗高风险的髓质发育异常综合征中：一项随机，开放标签的III期研究。
影响因子：
51.1
作者：
Fenaux, Pierre;Mufti, Ghulam J.;Hellstrom-Lindberg, Eva;Santini, Valeria;Finelli, Carlo;Giagounidis, Aristoteles;Schoch, Robert;Gattermann, Norbert;Sanz, Guillermo;List, Alan;Gore, Steven D.;Seymour, John F.;Bennett, John M.;Byrd, John;Backstrom, Jay;Zimmerman, Linda;McKenzie, David;Beach, C. L.;Silverman, Lewis R.
通讯作者：
Silverman, Lewis R.
影响因子：
45.3
作者：
Silverman, LR;Demakos, EP;Holland, JF
通讯作者：
Holland, JF