Department of Internal Medicine, Division of Hematology and Oncology, Faculty of Medicine, Jazan University, Jazan, Saudi Arabia.
Medicine (Baltimore). 2024 Jan 19;103(3):e36936. doi: 10.1097/MD.0000000000036936.
Autoimmune disorders place a substantial burden on the healthcare system all over the world affecting almost 3% to 8% of the population. Immune thrombocytopenic purpura (ITP), also known as idiopathic thrombocytopenic purpura, is a blood disorder in which the body immune system destroys platelets, leading to low platelet counts in the blood (peripheral blood platelet count < 150 × 109/L). Although the pathophysiology of ITP is not fully understood, it is believed to result from a complex interplay between hereditary and environmental variables. Certain factors, such as a low platelet count, history of bleeding, and certain comorbidities can increase the risk of severe bleeding in patients with ITP. Corticosteroids, intravenous immunoglobulin (IVIG), immunosuppressants, rituximab, and thrombopoietin receptor agonists (TPO-RAs) are some of the advanced treatments for ITP. Although these therapies may be successful, they also carry the risk of negative effects. Recently, significant advancements have been made in the understanding and treatment of ITP. There is still much to learn about the disease, and new, more effective treatments are needed. This comprehensive review offers a comprehensive assessment of recent advancements in ITP management, with a focus on active research projects, novel therapeutic targets, new treatment modalities, and areas of uncertainty and unmet needs. According to research, it is crucial to develop individualized treatment plans for ITP patients based on their age, platelet count, risk of bleeding, and comorbidities. The article also looks at how future developments in gene editing, bispecific antibody therapies, and cellular therapy may completely change the treatment of ITP.
自身免疫性疾病在全球范围内给医疗保健系统带来了巨大负担，几乎影响了 3%至 8%的人口。免疫性血小板减少性紫癜（ITP），也称为特发性血小板减少性紫癜，是一种身体免疫系统破坏血小板的血液疾病，导致血液中血小板计数低（外周血血小板计数<150×109/L）。尽管 ITP 的病理生理学尚未完全了解，但据信它是由遗传和环境变量之间的复杂相互作用引起的。某些因素，如血小板计数低、出血史和某些合并症，会增加 ITP 患者发生严重出血的风险。皮质类固醇、静脉注射免疫球蛋白（IVIG）、免疫抑制剂、利妥昔单抗和血小板生成素受体激动剂（TPO-RAs）是 ITP 的一些高级治疗方法。尽管这些疗法可能有效，但它们也存在副作用的风险。最近，人们对 ITP 的理解和治疗取得了重大进展。尽管人们对这种疾病还有很多需要了解的地方，并且需要新的、更有效的治疗方法，但目前仍有许多研究在进行中。这篇全面的综述对 ITP 管理的最新进展进行了全面评估，重点介绍了正在进行的研究项目、新的治疗靶点、新的治疗方法以及不确定性和未满足的需求领域。根据研究，根据患者的年龄、血小板计数、出血风险和合并症，为 ITP 患者制定个体化的治疗计划至关重要。本文还探讨了基因编辑、双特异性抗体疗法和细胞疗法的未来发展如何可能彻底改变 ITP 的治疗。
