Treatment of congenital thrombotic thrombocytopenic purpura (TTP)

نویسندگان

چکیده

先天性血栓性血小板減少性紫斑病(先天性thrombotic thrombocytopenic purpura:TTP)は,von Willebrand因子の切断酵素であるA disintegrin and metalloproteinase with thrombospondin motifs 13(ADAMTS13)の先天的欠乏により,血小板減少,溶血性貧血,腎障害,動揺性精神系精神症状などの症状を発症する疾患である.先天性TTPの治療は,FFPによるADAMTS13の補充療法である.後天性TTPとは異なり血漿交換療法は不要で,FFPの輸注のみで十分症状が改善する.症状の出現を予防するために,非発作時にもFFPの定期輸注が必要な症例から,症状出現時のみにFFP輸注が必要な症例まで,有効なFFPの投与方法は症例によって異なる.FFPの定期輸注は,我が国の診療ガイドでは5~10 mL/kgを2~3週毎に輸注するとされている.現在遺伝子組み替えADAMTS13製剤の開発が進められており,これが承認されれば先天性TTPの治療は大きく変化すると考えられる.

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ژورنال

عنوان ژورنال: Japanese Journal of Thrombosis and Hemostasis

سال: 2022

ISSN: ['0915-7441', '1880-8808']

DOI: https://doi.org/10.2491/jjsth.33.408