New insights into sickle cell disease: mechanisms and investigational therapies.
New insights into sickle cell disease: mechanisms and investigational therapies.
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DOI：
10.1097/moh.0000000000000241
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发表时间：
2016-05
影响因子：
3.2
通讯作者：
Kato GJ
中科院分区：
文献类型：
--
作者：
Kato GJ
镰状细胞病（SCD）困扰着全世界近数百万人。其单核苷酸突变的简单性掩盖了该疾病的生物学和心理社会复杂性。尽管只有一种专门用于治疗SCD的药物获得批准，但新的发现提供了前进的方向。过去一年为影响红细胞、溶血和血管病变、先天免疫系统激活、血细胞和内皮细胞增殖、疼痛中枢敏感化和慢性脑损伤的机制提供了大量支持。支持扩大使用羟基脲的证据继续增加。许多有前途的疗法正在进入临床试验阶段，包括治愈性疗法，还有更多的疗法即将推出。证据是令人信服的，使用的羟基脲必须扩大由临床医生获得充分的多效性的好处，这种批准的药物。临床医生必须意识到，严重的急性和慢性疼痛具有生物学和神经学基础，并且对这一基础的理解正在增长。研究人员正在SCD领域以前所未有的速度测试研究性疗法，患者、研究人员和私营部门之间的伙伴关系提供了最快速和最富有成效的前进方向。
Sickle cell disease (SCD) afflicts nearly millions worldwide. The simplicity of its single nucleotide mutation belies the biological and psychosocial complexity of the disease. Despite only a single approved drug specifically for the treatment of SCD, new findings provide the direction forward. The last year has provided a wealth of support for mechanisms affecting the red cell, hemolysis and vasculopathy, the innate immune system activation, blood cell and endothelial adhesiveness, central sensitization to pain and chronic brain injury. The evidence supporting expanded use of hydroxyurea continues to mount. Many promising therapies are reaching clinical trial, including curative therapies, with more on the horizon. Evidence is compelling that the use of hydroxyurea must be expanded by clinicians to gain the full pleiotropic benefits of this approved drug. Clinicians must become aware that severe acute and chronic pain has a biological and neurologic basis, and the understanding of this basis is growing. Researchers are testing investigational therapies at an unprecedented pace in SCD, and partnership between patients, researchers and the private sector provide the most rapid and productive way forward.