文献

J-GLOBAL ID：201902229143537608   整理番号：19A0234752

活性化ホスファチジルイノシトール-3-OHキナーゼδ症候群1型患者における進行性複合免疫不全とリンパ増殖のための造血幹細胞移植【JST・京大機械翻訳】

Hematopoietic stem cell transplantation for progressive combined immunodeficiency and lymphoproliferation in patients with activated phosphatidylinositol-3-OH kinase δ syndrome type 1

著者 (56件)： Okano Tsubasa (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Imai Kohsuke (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Tsujita Yuki (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Mitsuiki Noriko (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Yoshida Kenichi (Department of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan) ,  Kamae Chikako (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Honma Kenichi (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Mitsui-Sekinaka Kanako (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Sekinaka Yujin (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Kato Tamaki (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Hanabusa Katsuyuki (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Endo Eri (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Takashima Takehiro (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Hiroki Haruka (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Yeh Tzu-Wen (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Tanaka Keisuke (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Nagahori Masakazu (Department of Gastroenterology and Hepatology, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Tsuge Ikuya (Department of Pediatrics, School of Medicine, Fujita Health University, Toyoake, Japan) ,  Bando Yuki (Department of Pediatrics, Kitasato University School of Medicine, Sagamihara, Japan) ,  Iwasaki Fuminori (Division of Hemato-Oncology Regenerative Medicine, Yokohama, Japan) ,  Shikama Yoshiaki (Division of Infectious Disease and Immunology, Kanagawa Children’s Medical Center, Yokohama, Japan) ,  Inoue Masami (Department of Hematology/Oncology, Osaka Women’s and Children’s Hospital, Osaka, Japan) ,  Kimoto Tomiko (Department of Hematology/Oncology, Osaka Women’s and Children’s Hospital, Osaka, Japan) ,  Moriguchi Naohiko (Department of Pediatrics, Kindai University Sakai Hospital, Sakai, Japan) ,  Yuza Yuki (Department of Hematology/Oncology, Tokyo Metropolitan Children’s Medical Center, Tokyo, Japan) ,  Kaneko Takashi (Department of Hematology/Oncology, Tokyo Metropolitan Children’s Medical Center, Tokyo, Japan) ,  Suzuki Kyoko (Department of Pediatrics, Juntendo University, Urayasu Hospital, Chiba, Japan) ,  Matsubara Tomoyo (Department of Pediatrics, Juntendo University, Urayasu Hospital, Chiba, Japan) ,  Maruo Yoshihiro (Department of Pediatrics, Shiga University of Medical Science, Shiga, Japan) ,  Kunitsu Tomoaki (Department of Pediatrics, Shiga University of Medical Science, Shiga, Japan) ,  Waragai Tomoko (Department of Pediatrics, Fukushima Medical University, Fukushima, Japan) ,  Sano Hideki (Department of Pediatric Oncology, Fukushima Medical University, Fukushima, Japan) ,  Hashimoto Yuko (Department of Diagnostic Pathology, Fukushima Medical University School of Medicine, Fukushima, Japan) ,  Tasaki Kazuhiro (Department of Diagnostic Pathology, Fukushima Medical University School of Medicine, Fukushima, Japan) ,  Suzuki Osamu (Department of Diagnostic Pathology, Fukushima Medical University School of Medicine, Fukushima, Japan) ,  Shirakawa Toshihiko (Department of Pediatrics, Nagasaki University Hospital, Nagasaki, Japan) ,  Kato Motohiro (Division of Transplantation and Cell Therapy, Children’s Cancer Center, National Center for Child Health and Development, Tokyo, Japan) ,  Uchiyama Toru (Department of Human Genetics, National Center for Child Health and Development, Tokyo, Japan) ,  Ishimura Masataka (Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan) ,  Tauchi Tetsuzo (Department of Hematology, Tokyo Medical University, Tokyo, Japan) ,  Yagasaki Hiroshi (Department of Pediatrics, Nihon University Itabashi Hospital, Tokyo, Japan) ,  Jou Shiann-Tarng (Department of Pediatrics, National Taiwan University Children’s Hospital, National Taiwan University College of Medicine, Taipei, Taiwan) ,  Yu Hsin-Hui (Department of Pediatrics, National Taiwan University Children’s Hospital, National Taiwan University College of Medicine, Taipei, Taiwan) ,  Kanegane Hirokazu (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan) ,  Kracker Sven (Universite Paris Descartes, Sorbonne Paris Cite, Imagine Institute, INSERM UMR 1163, Human Lymphohematopoiesis Laboratory, Paris, France) ,  Durandy Anne (Universite Paris Descartes, Sorbonne Paris Cite, Imagine Institute, INSERM UMR 1163, Human Lymphohematopoiesis Laboratory, Paris, France) ,  Kojima Daiei (Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan) ,  Muramatsu Hideki (Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan) ,  Wada Taizo (Department of Pediatrics, Graduate School of Medical Science, Kanazawa University, Kanazawa, Japan) ,  Inoue Yuzaburo (Department of Pediatrics, Eastern Chiba Medical Center, Togane, Chiba, Japan) ,  Takada Hidetoshi (Department of Perinatal and Pediatric Medicine, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan) ,  Kojima Seiji (Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan) ,  Ogawa Seishi (Department of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan) ,  Ohara Osamu (Department of Technology Development, Kazusa DNA Research Institute, Kisarazu, Japan) ,  Nonoyama Shigeaki (Department of Pediatrics, National Defense Medical College, Saitama, Japan) ,  Morio Tomohiro (Department of Pediatrics, Tokyo Medical and Dental University (TMDU), Tokyo, Japan)
資料名： Journal of Allergy and Clinical Immunology  (Journal of Allergy and Clinical Immunology)
巻： 143  号： 1  ページ： 266-275  発行年： 2019年 
JST資料番号： A0182B  ISSN： 0091-6749  資料種別： 逐次刊行物 (A)
記事区分： 原著論文  発行国： オランダ (NLD)  言語： 英語 (EN)

抄録/ポイント： 活性化ホスファチジルイノシトール-3-OHキナーゼδ症候群1型(APDS1)は,PIK3CDの生殖系列獲得機能突然変異により引き起こされる再発性呼吸器感染,リンパ性過形成およびHerpesviridae感染により特徴付けられる最近報告された原発性免疫不全症候群である。造血幹細胞移植(HSCT)は進行性免疫不全および関連悪性腫瘍を改善すると考えられるが,APDS1に対するHSCTの適切な適応,方法および転帰は未定義のままである。著者らの目的は,APDS1の臨床症状,検査所見,予後および治療を分析し,HSCTの適切な適応および方法を調査することであった。協力施設でHSCTを受けているコホートの医療記録を遡及的にレビューした。全生存率は86.1%であったが,無病生存率は39.6%であった。重篤な感染またはリンパ増殖のような生命を脅かすイベントは,小児期および青年期に多く,HSCTに対する一般的な徴候であった。9人の患者は,フルダラビンに基づく低強度コンディショニングでHSCTを受けた。7人の患者は,頻繁な有害合併症と生着不全の後に生存した。ほとんどの症状はHSCT後に改善した。APDS1を有する患者は,様々な臨床症状を示した。生命を脅かす進行性複合免疫不全と大量のリンパ増殖は,HSCTのための一般的適応であった。フルダラビンに基づく低強度コンディショニング-HSCTは臨床症状を改善したが,移植片不全を含む移植関連合併症は頻繁であった。Copyright 2019 Elsevier B.V., Amsterdam. All rights reserved. Translated from English into Japanese by JST.【JST・京大機械翻訳】

シソーラス用語： *免疫不全症候群 ,  *リンパ ,  増生 ,  *造血幹細胞移植 ,  移植片 ,  再発 ,  予後 ,  合併症 ,  突然変異 ,  プリンヌクレオシド ,  リボヌクレオシド
準シソーラス用語： 生着不全 ,  全生存率 ,  臨床症状 ,  転帰 ,  悪性腫瘍 , 【Automatic Indexing@JST】

著者キーワード (7件)： 活性化ホスファチジルイノシトール-3-OHキナーゼδ症候群1型 ,  PIK3CD ,  造血幹細胞移植 ,  複合免疫不全症 ,  T細胞受容体切除サークル ,  カッパ-削除再結合切除円 ,  IgG2

分類 (2件)： 血液の疾患  (GK03000F) ,  免疫性疾患・アレルギー性疾患一般  (GD04010C)

物質索引 (1件)： フルダラビン

タイトルに関連する用語 (9件)： 活性化 ,  ホスファチジルイノシトール ,  キナーゼ ,  症候群 ,  患者 ,  複合免疫不全 ,  リンパ ,  増殖 ,  造血幹細胞移植