摘要
近年来,补体抑制剂的出现和临床应用,显著提升了阵发性睡眠性血红蛋白尿症(PNH)患者的生存和生活质量,PNH的治疗目标从控制溶血升级为脱离输血和血红蛋白正常化。由于PNH疾病的异质性、克隆演变的存在,即使补体抑制剂初始治疗有效,仍可能出现突破性溶血、血管外溶血、血栓事件和疾病演变等问题。由于PNH克隆一般不会消失,患者需要接受长期甚至终身的补体抑制剂治疗,疾病管理应遵从慢性病管理的模式。需要整合实验室数据、临床评估、患者报告,进行合理的疗效评估和规范化的随访,以实现长期的疾病控制,提高患者的生活质量。
Recently,the development and clinical application of complement inhibitors have significantly improved the survival and quality of life of patients with paroxysmal nocturnal hemoglobinuria(PNH).The treatment goal of PNH has been upgraded from hemolysis control to transfusion independence and hemoglobin normalization.Even with the initial control of hemolysis with compliment inhibitors,there is still questions like breakthrough hemolysis,extra vascular hemolysis,thrombotic events or disease evolution due to the diversities of PNH.Because PNH clones generally do not disappear after compliment inhibitors,patients require long-term follow-up and life-long time management which is similar to the management of chronic disease.Patient reports,clinical assessments,laboratory data,efficacy assessments need to be integrated to improve the quality of life of patients.
作者
韩冰
HAN Bing(Department of Hematology,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking UnionMedical College,Beijing 100032,China)
出处
《中国实用内科杂志》
北大核心
2025年第7期554-558,共5页
Chinese Journal of Practical Internal Medicine
基金
中国医学科学院临床与转化医学研究专项(2023-12M-C&T-B-013)
国家自然科学基金(82370121)
北京自然科学基金(7232109)
中央高水平医院临床科研业务费(2022-PUMCHC-026,2022-PUMCH-D-002,2022-PUMCH-B-046)。
关键词
阵发性睡眠性血红蛋白尿症
疗效评估
随访
paroxysmal nocturnal hemoglobinuria
efficacy assessment
follow-up
