Targeting low-risk myelodysplastic syndrome with novel therapeutic strategies.
Targeting low-risk myelodysplastic syndrome with novel therapeutic strategies.
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DOI：
10.1016/j.molmed.2021.06.013
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发表时间：
2021-10
影响因子：
13.6
通讯作者：
Zhang L
中科院分区：
文献类型：
--
作者：
Trivedi G;Inoue D;Zhang L
骨髓增生异常综合征(MDS)是一组治疗选择有限的造血系统疾病。贫血是MDS的常见症状，尽管促红细胞生成素、来那度胺和卢帕西普等促红细胞生成剂可用于治疗贫血，但许多MDS患者对这些一线治疗没有反应。因此，需要采用替代药物开发策略来提高治疗效果。用于纠正剪接相关缺陷的剪接调节剂在临床试验中显示出良好的效果。在MDS中靶向分化早期红系祖细胞以增加红系输出是另一种新的方法，在临床前阶段显示了令人鼓舞的结果。总之，这些治疗策略为针对以前无法治疗的MDS症状提供了新的途径。
Myelodysplastic syndrome (MDS) is a group of hematopoietic disorders with limited treatment options. Anemia is a common symptom in MDS, and although erythropoiesis-stimulating agents such as erythropoietin, lenalidomide, and luspatercept are available to treat anemia, many MDS patients do not respond to these first-line therapies. Therefore, alternative drug development strategies are needed to improve therapeutic efficacy. Splicing modulators to correct splicing-related defects have shown promising results in clinical trials. Targeting differentiation of early erythroid progenitors to increase the erythroid output in MDS is another novel approach, which has shown encouraging results at the pre-clinical stage. Together, these therapeutic strategies provide new avenues to target MDS symptoms untreatable previously.
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影响因子：
82.9
作者：
Bernard E;Nannya Y;Hasserjian RP;Devlin SM;Tuechler H;Medina-Martinez JS;Yoshizato T;Shiozawa Y;Saiki R;Malcovati L;Levine MF;Arango JE;Zhou Y;Solé F;Cargo CA;Haase D;Creignou M;Germing U;Zhang Y;Gundem G;Sarian A;van de Loosdrecht AA;Jädersten M;Tobiasson M;Kosmider O;Follo MY;Thol F;Pinheiro RF;Santini V;Kotsianidis I;Boultwood J;Santos FPS;Schanz J;Kasahara S;Ishikawa T;Tsurumi H;Takaori-Kondo A;Kiguchi T;Polprasert C;Bennett JM;Klimek VM;Savona MR;Belickova M;Ganster C;Palomo L;Sanz G;Ades L;Della Porta MG;Elias HK;Smith AG;Werner Y;Patel M;Viale A;Vanness K;Neuberg DS;Stevenson KE;Menghrajani K;Bolton KL;Fenaux P;Pellagatti A;Platzbecker U;Heuser M;Valent P;Chiba S;Miyazaki Y;Finelli C;Voso MT;Shih LY;Fontenay M;Jansen JH;Cervera J;Atsuta Y;Gattermann N;Ebert BL;Bejar R;Greenberg PL;Cazzola M;Hellström-Lindberg E;Ogawa S;Papaemmanuil E
通讯作者：
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影响因子：
16.6
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通讯作者：
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影响因子：
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通讯作者：
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作者：
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通讯作者：
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