What question did this study set out to answer?

To explain the pathophysiology and treatment strategies for paroxysmal nocturnal hemoglobinuria and cold agglutinin disease.

May 12, 2026Nihon Naika Gakkai Zasshi0 citationsOpen Access

Pathophysiology and Treatment of Hemolytic Anemia

JNJunichi NishimuraOsaka City University

Key Points

To explain the pathophysiology and treatment strategies for paroxysmal nocturnal hemoglobinuria and cold agglutinin disease.
Review of recent approvals and developments in hemolytic anemia treatment.
Focus on anti-C5 antibody eculizumab for PNH and anti-C1s antibody sutimlimab for CAD.
Eculizumab significantly improved quality of life and survival rates in PNH patients after its 2007 approval.
Sutimlimab was approved in 2022 for cold agglutinin disease, enhancing treatment options for AIHA.

Abstract

本邦成人における溶血性貧血の約1/4を占める発作性夜間ヘモグロビン尿症（paroxysmal nocturnal hemoglobinuria：PNH）に対する溶血治療薬として，補体を標的とした抗C5抗体Eculizumabが2007年に承認された．患者QOLと生命予後が劇的に改善したことは言うに及ばず，新たに見えた病態に即した新規抗補体薬が，次々と開発され承認に至っている．また2022年，本邦成人溶血性貧血の約半数を占める自己免疫性溶血性貧血（autoimmune hemolytic anemia：AIHA）の一病型である寒冷凝集素症（cold agglutinin disease：CAD）の溶血治療薬として，抗C1s抗体Sutimlimabが承認された．本稿では，新規治療薬開発から新たに見えてきたPNHとCADの病態とその治療戦略に焦点を当てて概説する．

Perguntar à IA

Bookmark

View Full Paper