Han B
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China.
Zhonghua Xue Ye Xue Za Zhi. 2025 Mar 14;46(3):193-197. doi: 10.3760/cma.j.cn121090-20241213-00568.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic stem cell disease that mainly occurs in young adults and is characterized by bone marrow failure, persistent intravascular hemolysis and thrombosis, all of which can cause severe end-organ damage, increase the risk of early death, and cause a severe disease burden in patients. In China, based on the historic reasons, glucocorticoids are still routinely used in many places. However, the effects of glucocorticoids on PNH hemolysis are uncertain. Evidence-based medical data and clinical benefits for glucocorticoid on PNH are missing, but the long-term use of glucocorticoids significantly increases the risk of adverse reactions in patients. Since PNH needs a lifelong follow-up and management, long-term glucocorticoid therapy will unavoidably seriously damage the health of patients. Therefore, glucocorticoids are not recommended for the treatment of PNH, either from domestic or overseas guidelines, or expert consensus. In this article, the limitations and challenges of glucocorticoids in the treatment of PNH were expounded upon, in order to encourage more effective and safe strategies to be accepted in China.
阵发性睡眠性血红蛋白尿症（PNH）是一种罕见的获得性造血干细胞疾病，主要发生于青壮年，其特征为骨髓衰竭、持续性血管内溶血和血栓形成，所有这些均可导致严重的终末器官损害，增加早期死亡风险，并给患者带来沉重的疾病负担。在中国，基于历史原因，糖皮质激素在许多地方仍被常规使用。然而，糖皮质激素对PNH溶血的作用尚不确定。目前缺乏关于糖皮质激素治疗PNH的循证医学数据和临床获益，但长期使用糖皮质激素会显著增加患者不良反应的风险。由于PNH需要终身随访和管理，长期糖皮质激素治疗将不可避免地严重损害患者健康。因此，无论是国内还是国外的指南或专家共识，均不推荐使用糖皮质激素治疗PNH。本文阐述了糖皮质激素治疗PNH的局限性和挑战，以期鼓励在中国采用更有效、安全的治疗策略。
