[Limitations and challenges of glucocorticoids in the treatment of paroxysmal nocturnal hemoglobinuria].

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.