V．発作性夜間血色素尿症：病態と治療の最前線
発作性夜間血色素尿症は，後天的なPIGA遺伝子変異を持つ造血幹細胞クローンの拡大によって引き起こされる，造血幹細胞疾患である．血管内溶血，血栓症，造血不全を3主徴とし，根治には造血幹細胞移植が必要だが，明確な適応基準がない．多くの症例で対症療法が中心となるが，エクリズマブの登場により血管内溶血が劇的に抑制され，造血不全を除く諸症状も改善され，さらに生命予後の改善も期待されている．...
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| Published in | 日本内科学会雑誌 Vol. 104; no. 7; pp. 1397 - 1404 |
|---|---|
| Main Authors | , |
| Format | Journal Article |
| Language | Japanese |
| Published |
一般社団法人 日本内科学会
2015
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| Subjects | |
| Online Access | Get full text |
| ISSN | 0021-5384 1883-2083 |
| DOI | 10.2169/naika.104.1397 |
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| Abstract | 発作性夜間血色素尿症は，後天的なPIGA遺伝子変異を持つ造血幹細胞クローンの拡大によって引き起こされる，造血幹細胞疾患である．血管内溶血，血栓症，造血不全を3主徴とし，根治には造血幹細胞移植が必要だが，明確な適応基準がない．多くの症例で対症療法が中心となるが，エクリズマブの登場により血管内溶血が劇的に抑制され，造血不全を除く諸症状も改善され，さらに生命予後の改善も期待されている． |
|---|---|
| AbstractList | 発作性夜間血色素尿症は，後天的なPIGA遺伝子変異を持つ造血幹細胞クローンの拡大によって引き起こされる，造血幹細胞疾患である．血管内溶血，血栓症，造血不全を3主徴とし，根治には造血幹細胞移植が必要だが，明確な適応基準がない．多くの症例で対症療法が中心となるが，エクリズマブの登場により血管内溶血が劇的に抑制され，造血不全を除く諸症状も改善され，さらに生命予後の改善も期待されている． |
| Author | 西村, 純一 植田, 康敬 |
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| Copyright | 2015 一般社団法人 日本内科学会 |
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| DOI | 10.2169/naika.104.1397 |
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| References | 3) Murakami Y, et al: Deregulated expression of HMGA2 is implicated in clonal expansion of PIGA deficient cells in paroxysmal nocturnal haemoglobinuria. Br J Haematol 156: 383-287, 2012. 7) Nishimura J, et al: Genetic variants in C5 and poor response to eculizumab. N Engl J Med 370: 632-639, 2014. 9) 発作性夜間ヘモグロビン尿症診療の参照ガイド 平成26年度改訂版/PNH妊娠の参照ガイド (付記). 2015. http://zoketsushogaihan.com/file/guideline_H26/PNH2015.pdf http://zoketsushogaihan.com/file/guideline_H26/PNH-ninpu.pdf 10) 西村純一, 金倉 譲: 発作性夜間ヘモグロビン尿症, 難治性貧血の診療ガイド. 「難治性貧血の診療ガイド」編集委員会編. 南江堂, 東京, 2011, 93-130. 1) Parker C, et al: Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 106: 3699-3709, 2005. 6) Rother RP, et al: Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 25: 1256-1264, 2007. 5) Wang H, et al: Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood 100: 3897-3902, 2002. 2) Kelly RJ, et al: Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood 117: 6786-6792, 2011. 4) Sugimori C, et al: Minor population of CD55-CD59-blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anemia. Blood 107: 1308-1314, 2006. 8) Risitano AM, et al: Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood 113: 4094-4100, 2009. |
| References_xml | – reference: 6) Rother RP, et al: Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 25: 1256-1264, 2007. – reference: 4) Sugimori C, et al: Minor population of CD55-CD59-blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anemia. Blood 107: 1308-1314, 2006. – reference: 9) 発作性夜間ヘモグロビン尿症診療の参照ガイド 平成26年度改訂版/PNH妊娠の参照ガイド (付記). 2015. http://zoketsushogaihan.com/file/guideline_H26/PNH2015.pdf http://zoketsushogaihan.com/file/guideline_H26/PNH-ninpu.pdf – reference: 7) Nishimura J, et al: Genetic variants in C5 and poor response to eculizumab. N Engl J Med 370: 632-639, 2014. – reference: 2) Kelly RJ, et al: Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood 117: 6786-6792, 2011. – reference: 10) 西村純一, 金倉 譲: 発作性夜間ヘモグロビン尿症, 難治性貧血の診療ガイド. 「難治性貧血の診療ガイド」編集委員会編. 南江堂, 東京, 2011, 93-130. – reference: 3) Murakami Y, et al: Deregulated expression of HMGA2 is implicated in clonal expansion of PIGA deficient cells in paroxysmal nocturnal haemoglobinuria. Br J Haematol 156: 383-287, 2012. – reference: 8) Risitano AM, et al: Complement fraction 3 binding on erythrocytes as additional mechanism of disease in paroxysmal nocturnal hemoglobinuria patients treated by eculizumab. Blood 113: 4094-4100, 2009. – reference: 1) Parker C, et al: Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 106: 3699-3709, 2005. – reference: 5) Wang H, et al: Clinical significance of a minor population of paroxysmal nocturnal hemoglobinuria-type cells in bone marrow failure syndrome. Blood 100: 3897-3902, 2002. |
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| SubjectTerms | PNH エクリズマブ 抗補体薬 発作性夜間血色素尿症 血栓症 血管内溶血 造血不全疾患 |
| Title | V．発作性夜間血色素尿症：病態と治療の最前線 |
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