アブストラクト
Japanese
| Title | 小児難治性ITP治療ガイド2019 |
|---|---|
| Subtitle | 委員会報告 日本小児血液・がん学会 血小板委員会報告 |
| Authors | 日本小児血液・がん学会血小板委員会, 高橋幸博1, 宮川義隆2, 森麻希子3, 國島伸治4,14, 東川正宗5, 小林尚明6, 笹原洋二7, 前田尚子8, 中舘尚也9, 別所文雄10, 白幡聡11, 今泉益栄12, 石黒精13 |
| Authors (kana) | |
| Organization | 1奈良県赤十字血液センター(2017年度委員長), 2埼玉医科大学病院総合診療内科・血栓止血センター, 3埼玉県立小児医療センター血液・腫瘍科, 4名古屋医療センター臨床研究センター高度診療研究部分子診断研究室, 5日本赤十字社伊勢赤十字病院小児科, 6康心会汐見台病院小児科, 7東北大学大学院医学系研究科小児病態学分野, 8国立病院機構名古屋医療センター小児科, 9国立成育医療センター総合診療部, 10日本医療科学大学, 11北九州八幡東病院, 12宮城県立こども病院, 13国立成育医療研究センター, 教育研修センター・血液内科(2018年度委員長), 14現:岐阜医療科学大学保健科学部 |
| Journal | 日本小児血液・がん学会雑誌 |
| Volume | 56 |
| Number | 1 |
| Page | 61-68 |
| Year/Month | 2019 / |
| Article | 報告 |
| Publisher | 日本小児血液・がん学会 |
| Abstract | 「要旨」免疫性血小板減少症(ITP)は, 血小板に対する自己抗体により血小板減少症をきたす自己免疫性疾患で, 小児では感染症やワクチン接種を契機に発症することが多い. 約8割は自然寛解するが, 副腎皮質ステロイドや免疫グロブリンなどの薬物療法に抵抗性を示す難治例が存在し, 時に重篤な出血症状を起こす. この難治性ITPの治療は, 従来から有効性が高い脾臓摘出術(脾摘)が成人例に行われてきたが, 侵襲的な治療法で, 術後感染のリスクもあり小児ではその選択は限られていた. 近年, 成人の慢性ITPの治療に抗体医薬リツキシマブとトロンボポエチン受容体作動薬が承認され, 脾摘と並ぶセカンドライン治療として広く使われている. 日本小児血液・がん学会の血小板委員会では, これら薬剤の小児難治性ITPに向けて, 現時点までに得られている国内外の知見をもとに, 科学的根拠に基づく小児難治性ITP治療ガイド2019を策定したので報告する. |
| Practice | 臨床医学：一般 |
| Keywords | 難治性ITP, 治療ガイド, リツキシマブ, トロンボポエチン受容体作動薬, refractory immune thrombocytopenia, treatment guide, rituximab, thrombopoietin receptor agonists |
English
| Title | Treatment Guide for Refractory Immune Thrombocytopenia in Children for 2019 |
|---|---|
| Subtitle | Report of the Platelet Committee, Japanese Society of Pediatric Hepatology / Oncology |
| Authors | Yukihiro Takahashi1, Yoshitaka Miyakawa2, Makiko Mori3, Shinji Kunishima4,14, Masamune Higashigawa5, Kobayashi Naoaki6, Youji Sasahara7, Naoko Maeda8, Hisaya Nakadate9, Fumio Bessho10, Akira Shirahata11, Masue Imaizumi12, Akira Ishiguro13 |
| Authors (kana) | |
| Organization | 1Japanese Red Cross Nara Blood Center (Former Chair of the Platelet Committee in 2017), 2Saitama Medical University Hospital, Department of General Medicine and Thrombosis and Hemostasis Center, 3Saitama Children's Medical Center, Department of Hematology & Oncology, 4Nagoya Medical Center, Clinical Research Center, 5Japanese Red Cross Ise Hospital, Department of Pediatrics, 6Koshinkai Shiomidai Hospital, Department of Pediatrics, 7Tohoku University Graduate School of Medicine, Department of Pediatrics, 8National Hospital Organization Nagoya Medical Center, Department of Pediatrics, 9National Center for Child Health and Development, Department of General Pediatrics & Interdisciplinary Medicine, 10Nihon Institute of Medical Science, 11Kitakyushu Yahaha Higashi Hospital, 12Miyagi Children's Hospital, 13National Center for Child Health and Development, Center for Postgraduate Education and Training / Division of Hematology (Current Chair of the Platelet Committee in 2018), 14Present: Gifu University of Medical Science, Department of Medical Technology |
| Journal | The Japanese Journal of Pediatric Hematology/Oncology |
| Volume | 56 |
| Number | 1 |
| Page | 61-68 |
| Year/Month | 2019 / |
| Article | Report |
| Publisher | The Japanese Society of Pediatric Hematology/Oncology |
| Abstract | [Abstract] Immune thrombocytopenia (ITP) is an autoimmune disease that causes thrombocytopenia due to the production of autoantibodies to platelets. ITP often develops in children as a result of infections and vaccinations. Most patients with ITP improve spontaneously; however, refractory ITP resistant to glucocorticoid and immunoglobulin treatments may cause severe bleeding. Splenectomy has been performed on adult patients with refractory ITP; however, this treatment option is not highly recommended for children because of its invasiveness and the risk of postoperative infections. In recent years, new drugs have been developed for refractory ITP, including rituximab and thrombopoietin receptor agonists. These drugs are considered to be second-line treatments for adult refractory ITP in addition to splenectomy. The Platelet Committee of the Japan Society of Pediatric Hematology/Oncology provides here a therapeutic guide for refractory ITP in children for 2019 based on current scientific evidence. |
| Practice | Clinical medicine |
| Keywords | refractory immune thrombocytopenia, treatment guide, rituximab, thrombopoietin receptor agonists |
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参考文献
- 1) 白幡聡,石井榮一,江口春彦,他:小児特発性血小板減少性紫斑病-診断・治療・管理ガイドライン-日本小児血液学会ITP委員会.日小血会誌 18: 210-218, 2004.
- 2) British Committee for Standards in Haematology General Haematology Task Force: Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol 120: 574-596, 2003.
- 3) Neunert C, Lim W, Crowther M, et al: The American Society of Hematology 2011 evidenced-based practice guideline for immune thrombocytopenia. Blood 117: 4190-4207, 2011.
- 4) Iyori H, Bessho F, Ookawa H, et al: Intracranial hemorrhage in children with immune thrombocytopenic purpura. Japanese Study Group on Childhood ITP. Ann Hematol 79: 691-695, 2000.
- 5) Lilleyman JS: Intracranial haemorrhage in idiopathic thrombocytopenic purpura. Paediatric Haematology Forum of the British Society of Haematology. Arch Dis Child 71: 251-253, 1994.
残りの70件を表示する
- 6) Guyatt GH, Cook DJ, Jaeschke R, et al: Grades of recommendation for antithrombotic agents: American College of Chest Physicians Evidence-Based Clinical Practice Guidelines (8th Edition). Chest 133: 123S-131S, 2008.
- 7) Bengtson KL, Skinner MA, Ware RE: Successful use of anti-CD20 (rituximab) in severe, life-threatening childhood immune thrombocytopenic purpura. J Pediatr 143: 670-673, 2003.
- 8) Pusiol A, Cesaro S, Nocerino A, et al; Successful treatment with monoclonal antibody rituximab in two children with refractory immune thrombocytopenia. Eur J Pediatr 163: 305-307, 2004.
- 9) Trelinski J, Chojnowski K, Kurenko-Deptuch M, et al: Successful treatment of refractory autoimmune thrombocytopenia with rituximab and cyclosporine A in a patient with chronic granulomatous disease. Ann Hematol 84: 835-836, 2005.
- 10) Taube T, Schmid H, Reihard H, et al: Effect of a single dose of rituximab in chronic immune thrombocytopenic purpura in children. Haematologica 90: 281-283, 2005.
- 11) Wang J, Wiley JM, Luddy R, et al: Chronic immune thrombocytopenic purpura in children: assessment of rituximab treatment. J Pediatr 146: 217-221, 2005.
- 12) Bennett CM, Rogers ZR, Kinnamon DD, et al: Prospective phase 1/2 study of rituximab in childhood and adolescent chronic immune thrombocytopenic purpura. Blood 107: 2639-2642, 2006.
- 13) Parodi E, Nobili B, Perrotta S, et al: Rituximab (anti-CD20 monoclonal antibody) in children with chronic refractory symptomatic immune thrombocytopenic purpura: efficacy and safety of treatment. Int J Hematol 84: 48-53, 2006.
- 14) Mahevas M, Le Page L, Salle V, et al: Efficiency of rituximab in the treatment of autoimmune thrombocytopenic purpura associated with common variable immunodeficiency. Am J Hematol 81: 645-646, 2006.
- 15) Bay A, Oner AF, Uner A, et al: Use of rituximab in chronic childhood immune thrombocytopenic purpura. Pediatr Int 48: 514-516, 2006.
- 16) Franchini M, Zaffanello M, Veneri D, et al: Rituximab for the treatment of childhood chronic idiopathic thrombocytopenic purpura and hemophilia with inhibitors. Pediatr Blood Cancer 49: 6-10, 2007.
- 17) Parodi E, Rivetti E, Amendola G, et al: Long-term follow-up analysis after rituximab therapy in children with refractory symptomatic ITP: identification of factors predictive of a sustained response. Br J Haematol 144: 552-558, 2009.
- 18) Dogan M, Oner AF, Acikgoz M, et al: Treatment of chronic immune thrombocytopenic purpura with rituximab in children. Indian J Pediatr 76: 1141-1144, 2009.
- 19) Mueller BU, Bennett CM, Feldman HA, et al: One year follow-up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab. Pediatr Blood Cancer 52: 259-262, 2009.
- 20) Goto S, Goto H, Tanoshima R, et al: Serum sickness with an elevated level of human anti-chimeric antibody following treatment with rituximab in a child with chronic immune thrombocytopenic purpura. Int J Hematol 89: 305-309, 2009.
- 21) Iesato K, Hatakeyama N, Yamamoto M, et al: Treatment of an infant with severe acute refractory immune thrombocytopenic purpura using combination therapy including rituximab. Pediatr Blood Cancer 53: 203-205, 2009.
- 22) Polido-Pereira J, Ferreira D, Rodrigues AM, et al: Rituximab use in pediatric autoimmune diseases: four case reports. Ann NY Acad Sci 1173: 712-720, 2009.
- 23) 柴田真弓,今村俊彦,吉田路子,他:難治性慢性血小板減少に対しrituximab投与を行った3例.第51回日本小児血液学会 305, 2009.
- 24) Citak EC, Citak FE: Treatment results of children with chronic immune thrombocytopenic purpura (ITP) treated with rituximab. J Trop Pediatr 57: 71-72, 2011.
- 25) Stiaki E, Prdikogianni C, Thomou C, et al: Idiopathic thrombocytopenic purpura in childhood: twenty years of experience in a single center. Pediatr Int 54: 524-527, 2012.
- 26) Patel VL, Mahevas M, Lee SY, et al: Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood 119: 5989-5995, 2012.
- 27) Gokcebay DC, Tavil B, Fettah A, et al: Evaluation of children with chronic immune thrombocytopenic purpura and Evans syndrome treated with rituximab. Clin Appl Thromb Hemost 19: 663-667, 2013.
- 28) 大内芽里,星能元,佐藤篤,他:リツキシマブが奏功した臍帯移植後免疫性血小板減少症の1例.日児誌 115: 691, 2011.
- 29) Ansari SH, Rostami T, Yousefian S, et al: Rituximab efficacy in the treatment of children with chronic immune thrombocytopenic purpura. Pediatr Hematol Oncol 31: 555-562, 2014.
- 30) Matsubara K, Takahashi Y, Hayakawa A, et al: Long-term follow-up of children with refractory immune thrombocytopenia treated with rituximab. Int J Hematol 99: 429-36, 2014.
- 31) Ay Y, Karapinar TH, Oymak Y, et al: Retrospective analysis of rituximab therapy and splenectomy in childhood chronic and refractory immune thrombocytopenic purpura. Blood Coagul Fibrinolysis 27: 431-435, 2016.
- 32) Provan D, Stasi R, Newland AC, et al: International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 115: 168-186, 2010.
- 33) ACIP General Best Guidance for Immunization-altered Immunocompetence. https://www.cdc.gov/vaccines/hcp/acip-recs/general-resc/immunocompetence.html (2018年4月現在).
- 34) Greenbook. Contraindication and special considerations-Gov. uk. https://:www.gov.uk/government/.../Greenbook_chapter_6 (2018年4月現在).
- 35) Ramaswamy K, Hsieh L, Leven E, et al: Thrombopoietic agents for the treatment of persistent and chronic immune thrombocytopenia in children. J Pediatr 165: 600-605. e4, 2014.
- 36) Bussel JB, de Miguel PG, Despotovic JM, et al: Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicenter, placebo-controlled study. Lancet Haematol 2: e315-e325, 2015.
- 37) Grainger JD, Locatelli F, Chotsampancharoen T, et al: Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomized multicenter, placebo-controlled trial. Lancet 386: 1649-1658, 2015.
- 38) Gerrits AJ, Leven EA, Frelinger AL 3rd, et al: Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood 126: 1367-1378, 2015.
- 39) Neunert C, Despotovic J, Haley K, et al: Thrombopoietin receptor agonist use in children: Data from the pediatric ITP consortium of North American ICON 2 study. Pediatr Blood Cancer 63: 1407-1413, 2016.
- 40) 柳澤敦広,橋本みゆき,益田大幸,他:エルトロンボパグを導入した治療抵抗性慢性ITP女児の1例.日児誌 117: 175, 2013.
- 41) 森麻希子,加藤元博,康勝好,他:慢性ITPに対するエルトロンボパグの使用経験.日児誌 117: 155, 2013.
- 42) 傍示幸,飯盛美由紀,持永早希子,他:ロミプロスチムが有効であったエルトロンボパグオラミン抵抗性の特発性血小板減少性紫斑病の1例.日小児臨薬理会誌 27: 95, 2014.
- 43) 高橋俊行,小林良二,岸本健治,他:小児慢性特発性血小板減少性紫斑病に対するエルトロンボパグの使用経験. 日児誌 118: 1091-1097, 2014.
- 44) 小池和俊,小宅奈津子,吉見愛,他:1型糖尿病に合併した慢性免疫性血小板減少性紫斑病に対するeltrombopag治療.日児誌 118: 424, 2014.
- 45) 坂田謙治,高瀬浩二郎,永井攻造,他:小児難治性ITPに対しトロンボポエチン(TPO)受容体作動薬(エルトロンボパグ)で治療を行った4例.日児誌 118: 89, 2014.
- 46) 小池和俊,吉見愛,中尾朋平,他:4歳以下の重症慢性免疫性血小板減少性紫斑病(ITP)5例に対するeltrombopag治療.日児誌 119: 287, 2015.
- 47) 森麻希子,加藤元博,康勝好,他:Eltrombopagからromiplostimへの治療変更が有用であった小児難治性ITP.臨床血液 56: 511-513, 2015.
- 48) Kojima D, Murakami N, Sekiya Y, et al: Successful management of acute refractory ITP with rituximab and eltrombopag, Jpn J Clin Hematol (abstract) 56: 1662, 2015.
- 49) 野村武雅,木部哲也,横地健治,他:小児慢性ITPに対するエルトロンボパグとロミプロスチムの使用経験.日児誌 120: 419, 2016.
- 50) Bussel JB, Buchanan GR, Nugent DJ, et al: A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood 118: 28-36, 2011.
- 51) Elalfy MS, Abdelmaksoud AA, Eltonbary KY: Romiplostim in children with chronic refractory ITP: randomized placebo controlled study. Ann Hematol 90: 1341-1344, 2011.
- 52) Mokhtar GM, Tantawy AA, El Sherif NH: Romiplostim therapy in children with unresponsive chronic immune thrombocytopenia. Platelets 23: 264-273, 2012.
- 53) Escudero Vilaplana V, Aragones JH, Fernandez-Llamazares CM, et al: Use of romiplostim for primary immune thrombocytopenia in children. Pediatr Hematol Oncol 29: 197-205, 2012.
- 54) Pasquet M, Aladjidi N, Guiton C, et al: Romiplostim in children with chronic immune thrombocytopenia (ITP): the French experience. Br J Haematol 164: 266-271, 2014.
- 55) Seidel MG, Urban C, Sipurzynski J, et al: High response rate but short-term effect of romiplostim in paediatric refractory chronic immune thrombocytopenia. Br J Haematol 165: 419-421, 2014.
- 56) Minowa K, Arai K, Kasahara M, et al: Romiplostim treatment allows for platelet transfusion-free liver transplantation in pediatric thrombocytopenic patient with primary sclerosing cholangitis. Pediatr Transplant 18: E212-E215, 2014.
- 57) Bussel JB, Hsieh L, Buchanan GR, et al: Long-term use of the thrombopoetin-mimetic romiplostim in children with severe chronic immune thrombocytopenia (ITP). Pediatr Blood Cancer 62: 208-213, 2015.
- 58) Mathias SD, Li X, Eisen M, et al: A phase 3, randomized, double-blind, placebo-controlled study to determine the effect of romiplostim on health-related quality of life in children with primary immune thrombocytopenia and associated burden in their parents. Pediatr Blood Cancer 63: 1232-1237, 2016.
- 59) Tarantino MD, Bussel JB, Blanchette VS, et al: Romiplostim in children with immune thrombocytopenia: a phase 3, randomized, double-blind, placebo-controlled study. Lancet 388: 45-54, 2016.
- 60) Graiger JD, Bussel JB, Cooper N, et al: A single-arm, open-label, long-term efficacy and safety study of subcutaneous (SC) romiplostim in children with immune thrombocytopenia, AHA 58 Annual Meeting & Exposition 868, 2016.
- 61) Bussel JB, Wang X, Lopez A, et al: Case study of remission in adults with immune thrombocytopenia following cessation of treatment with the thrombopoietin mimetic romiplostim. Hematology 21: 257-262, 2016.
- 62) Suntsova EV, Demina IM, Ignatova AA, et al: Bleeding tendency and platelet function during treatment with romiplostim in children with severe immune thrombocytopenic purpura. Int J Hematol 105: 841-848, 2017.
- 63) Gernsheimer TB, George JM, Aledort LM, et al: Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). J Thromb Haemst 8: 1372-1382, 2010.
- 64) Vishnu P, Aboulafia DM: Long-term safety and efficacy of romiplostim for treatment of immune thrombocytopenia. J Blood Med 7: 99-106, 2016.
- 65) Tarantino MD, Despotovic JM, Roy J, et al: Safety and efficacy of romiplostim in over 200 children with immune thrombocytopenia (ITP): Results of integrated database of 5 clinical trials. ASH 60th Annual Meeting & Exposition 2428, 2018.
- 66) Wong RSM, Saleh MN, Khelif A, et al: Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood 130: 2527-2536, 2017.
- 67) Mantadakis E, Buchanan GR: Elective splenectomy in children with idiopathic thrombocytopenic purpura. J Pediatr Hematol Oncol 22: 148-153, 2000.
- 68) Bhatt NS, Bhatt P, Donda K, et al: Trends of splenectomy in pediatric patients with immune thrombocytopenia before and after the ASH 2011 Evidence-based Practice Guidelines Blood 130: 676, 2017.
- 69) Mithoowani S, Gregory-Miller K, Goy J, et al: High-dose dexamethasone compared with prednisone for previously untreated primary immune thrombocytopenia: a systematic review and meta-analysis. Lancet Haematol 3: e489-e496, 2016.
- 70) Fujisawa K, Iyori H, Ohkawa H, et al: A prospective, randomized trial of conventional, dose-accelerated corticosteroids and intravenous immunoglobulin in children with newly diagnosed idiopathic thrombocytopenic purpura. Int J Hematol 72: 376-383, 2000.
- 71) Yamazaki T, Shibuta A, Ishii S, et al: High-dose cepharanthin for pediatric chronic immune thrombocytopenia in Japan. Pediatr Int 59: 303-308, 2017.
- 72) 藤沢康司,伊従秀章:血小板減少症の治療-小児の血小板減少症とセファランチンR-.新薬と臨床 47: 97-103, 1998.
- 73) 太田茂,鈴木淳史,杉浦康夫,他:小児慢性特発性血小板減少性紫斑病に対する大量ビタミンCおよびセファランチン投与の経験.日小児血液会誌 7: 452-456, 1993.
- 74) 桜井信男,安永幸二郎,野村武夫,他:特発性血小板減少性紫斑病(ITP)に対するTJ-137ツムラ加味帰脾湯の多施設臨床評価.臨床と研究 70: 345-352, 1993.
- 75) 和田和夫,小林正行,南川光三,他:特発性血小板減少性紫斑病に対する加味帰脾湯の使用経験.漢方医学 17: 383-386, 1993.
