シクロフォスファミドとプレドニゾンを併用したイプタコパンに反応するプラスの補完成分C3dを持つ耐火性自己免疫性血液溶解性貧血の重症発症:症例報告

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Yawen Zhang¹, Yuemin Gong¹, Guangsheng He¹

¹Department of Hematology, The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province Hospital, Key Laboratory of Hematology of Nanjing Medical University, Collaborative Innovation Center for Cancer Personalized Medicine, Nanjing, People's Republic of China.

Hematology (Amsterdam, Netherlands)

|August 25, 2025

まとめ

Ptacopanは,経口で投与されるB因子阻害剤で,サイクロフォスファミドとプレドニゾンを併用して,耐火性自己免疫性溶血性貧血 (AIHA) の3人の患者を効果的に治療しました. この併用療法により,ヘモグロビン濃度が急速に改善され,血液溶解が減少し,重症患者の新しい選択肢が提供されました.

科学分野:

血液学
免疫学
薬理学について

背景:

自己免疫性溶解性貧血 (AIHA) は,自己免疫性による赤血球の破壊を伴う.
AIHAとエヴァンズ症候群は稀で重症で再発率が高く致死する可能性もあります
耐火性AIHAは標準的な治療に弱い反応を示し,重度の溶血エピソードに対する新しい治療戦略を必要とします.

研究の目的:

耐火性AIHAの治療におけるプタコパンとサイクロフォスファミドおよびプレドニゾンの組み合わせの有効性を評価する.
血液学的パラメータと血液溶解マーカーに対するこの治療法の影響を評価する.

主な方法:

補足成分C3dに陽性で,血解性発作を経験した3人の患者さんを治療した.
治療には,経口によるB因子阻害剤であるプタコパン,サイクロフォスファミド,プレドニゾンが含まれていた.

主要な成果:

3人の患者全員のヘモグロビン (Hb) 濃度が治療後に急速に上昇した.
網膜細胞の割合の減少と,血液溶解マーカー (LDH,非結合ビリルビン) の有意な減少が観察されました.
耐火性AIHA患者の治療効果は良好でした.

結論:

Ptacopanは,サイクロフォスファミドとプレドニゾンの併用で,耐火性AIHAにおける重度の溶血エピソードに対する効果的な治療法として有望であることが示されています.
この治療アプローチは,治療への反応が限られている患者にとって潜在的に価値ある選択肢です.

キーワード:

自己免疫性溶解性貧血イプタコパン症例報告コンプリメントコンポーネントC3d コンプリメント阻害剤

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