Division of Hematology, Children's National Medical Center, Washington, DC, USA.
Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, TX, USA.
Eur J Haematol. 2020 Sep;105(3):237-246. doi: 10.1111/ejh.13430. Epub 2020 May 19.
Early diagnosis, treatment, and prevention of a vaso-occlusive crisis (VOC) are critical to the management of patients with sickle cell disease. It is essential to differentiate between VOC-associated pain and chronic pain, hyperalgesia, neuropathy, and neuropathic pain. The pathophysiology of VOCs includes polymerization of abnormal sickle hemoglobin, inflammation, and adhesion. Hydroxyurea, L-glutamine, crizanlizumab, and voxelotor have been approved by the US Food and Drug Administration for reducing the frequency of VOCs; the European Medicines Agency has approved only hydroxyurea. Other novel treatments are in late-stage clinical development in both the United States and the European Union. The development of agents for prevention and treatment of VOCs should be driven by our understanding of its pathophysiology.
早期诊断、治疗和预防血管阻塞性危象(VOC)对于镰状细胞病患者的管理至关重要。区分与 VOC 相关的疼痛与慢性疼痛、痛觉过敏、神经病和神经性疼痛非常重要。VOC 的病理生理学包括异常镰状血红蛋白的聚合、炎症和黏附。羟基脲、L-谷氨酰胺、crizanlizumab 和 voxelotor 已被美国食品和药物管理局批准用于降低 VOC 的频率;欧洲药品管理局仅批准了羟基脲。其他新型治疗方法正在美国和欧盟的后期临床开发中。我们对其病理生理学的理解应推动对 VOC 预防和治疗药物的研发。