糖皮质激素治疗阵发性睡眠性血红蛋白尿症的局限性与挑战
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。...
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| Published in | Zhōnghuá xuèyèxué zázhì Vol. 46; no. 3; pp. 193 - 197 |
|---|---|
| Format | Journal Article |
| Language | Chinese English |
| Published |
No. 288, Nanjing road, Heping district, Tianjin
Editorial office of Chinese Journal of Hematology
14.03.2025
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| Subjects | |
| Online Access | Get full text |
| ISSN | 0253-2727 2707-9740 |
| DOI | 10.3760/cma.j.cn121090-20241213-00568 |
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| 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。 |
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| AbstractList | 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。 |
| AuthorAffiliation | 中国医学科学院北京协和医院血液科，北京 100730 Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China |
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| Title | 糖皮质激素治疗阵发性睡眠性血红蛋白尿症的局限性与挑战 |
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