# Limitations and challenges of glucocorticoids in the treatment of paroxysmal nocturnal hemoglobinuria

## TL;DR

This paper discusses the limitations of using glucocorticoids to treat paroxysmal nocturnal hemoglobinuria, a rare blood disorder, and highlights the need for better treatment strategies.

## Contribution

The paper highlights the lack of evidence supporting glucocorticoid use in PNH and emphasizes the need for updated treatment guidelines in China.

## Key findings

- Glucocorticoids lack clear evidence of efficacy in controlling hemolysis in PNH patients.
- Long-term glucocorticoid use increases the risk of adverse effects in PNH patients.
- Current guidelines and expert consensus do not recommend glucocorticoids for PNH treatment.

## Abstract

阵发性睡眠性血红蛋白尿症（Paroxysmal nocturnal hemoglobinuria, PNH）是一种罕见的获得性造血干细胞疾病，多起病于青壮年，特征是骨髓衰竭、持续的血管内溶血和血栓形成，均可引起严重的终末器官损伤，增加早期死亡的风险，造成患者严重的疾病负担。在我国，针对PNH患者的治疗尚未与国际同步，糖皮质激素仍然是临床常规用药。然而，糖皮质激素对于PNH溶血的控制机制并不明确，缺少支持其使用的循证医学数据和临床获益经验，且长期使用糖皮质激素显著增加患者不良反应的发生风险。鉴于PNH需要终身管理，长期糖皮质激素治疗将严重损害患者健康，国内外指南/专家共识均不建议应用糖皮质激素。本文针对糖皮质激素治疗PNH患者的局限与挑战进行论述，以促进中国PNH治疗策略的更迭。