中国实用内科杂志2025，Vol.45Issue(7)：554-558,5.DOI:10.19538/j.nk2025070103
阵发性睡眠性血红蛋白尿症补体抑制剂疗效评估与随访
Efficacy evaluation of complement inhibitors and clinical follow-up of paroxysmal nocturnal hemoglobinuria
摘要
Abstract
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.关键词
阵发性睡眠性血红蛋白尿症/疗效评估/随访Key words
paroxysmal nocturnal hemoglobinuria/efficacy assessment/follow-up分类
医药卫生引用本文复制引用
韩冰..阵发性睡眠性血红蛋白尿症补体抑制剂疗效评估与随访[J].中国实用内科杂志,2025,45(7):554-558,5.基金项目
中国医学科学院临床与转化医学研究专项(2023-12M-C&T-B-013) （2023-12M-C&T-B-013）
国家自然科学基金(82370121) （82370121）
北京自然科学基金(7232109) （7232109）
中央高水平医院临床科研业务费(2022-PUMCH-C-026,2022-PUMCH-D-002,2022-PUMCH-B-046) （2022-PUMCH-C-026,2022-PUMCH-D-002,2022-PUMCH-B-046）
