Taizo A Nakano, Amanda B Grimes, Robert J Klaassen, Michele P Lambert, Cindy Neunert, Jennifer A Rothman, Kristin A Shimano, Christina Amend, Megan Askew, Sherif M Badawy, Jillian M Baker, Vicky Breakey, Shelley Crary, Monica Davini, Stephanie Fritch Lilla, Megan Gilbert, Taru Hays, Kerry Hege, Kirsty Hillier, Amanda Jacobson-Kelly, Shipra Kaicker, Taylor O Kim, Manpreet Kochhar, Thierry Leblanc, Marie Martinelli, Mara Nunez, Allison Remiker, Corrina Schultz, Ruchika Sharma, Rachael F Grace
{"title":"名字的含义：定义小儿难治性 ITP。","authors":"Taizo A Nakano, Amanda B Grimes, Robert J Klaassen, Michele P Lambert, Cindy Neunert, Jennifer A Rothman, Kristin A Shimano, Christina Amend, Megan Askew, Sherif M Badawy, Jillian M Baker, Vicky Breakey, Shelley Crary, Monica Davini, Stephanie Fritch Lilla, Megan Gilbert, Taru Hays, Kerry Hege, Kirsty Hillier, Amanda Jacobson-Kelly, Shipra Kaicker, Taylor O Kim, Manpreet Kochhar, Thierry Leblanc, Marie Martinelli, Mara Nunez, Allison Remiker, Corrina Schultz, Ruchika Sharma, Rachael F Grace","doi":"10.1182/bloodadvances.2024012707","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>There are no agreed upon terminology to define \"refractory\" pediatric immune thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease-modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4000,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11460440/pdf/","citationCount":"0","resultStr":"{\"title\":\"What is in a name: defining pediatric refractory ITP.\",\"authors\":\"Taizo A Nakano, Amanda B Grimes, Robert J Klaassen, Michele P Lambert, Cindy Neunert, Jennifer A Rothman, Kristin A Shimano, Christina Amend, Megan Askew, Sherif M Badawy, Jillian M Baker, Vicky Breakey, Shelley Crary, Monica Davini, Stephanie Fritch Lilla, Megan Gilbert, Taru Hays, Kerry Hege, Kirsty Hillier, Amanda Jacobson-Kelly, Shipra Kaicker, Taylor O Kim, Manpreet Kochhar, Thierry Leblanc, Marie Martinelli, Mara Nunez, Allison Remiker, Corrina Schultz, Ruchika Sharma, Rachael F Grace\",\"doi\":\"10.1182/bloodadvances.2024012707\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Abstract: </strong>There are no agreed upon terminology to define \\\"refractory\\\" pediatric immune thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease-modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.</p>\",\"PeriodicalId\":9228,\"journal\":{\"name\":\"Blood advances\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":7.4000,\"publicationDate\":\"2024-10-08\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11460440/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Blood advances\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1182/bloodadvances.2024012707\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood advances","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1182/bloodadvances.2024012707","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
What is in a name: defining pediatric refractory ITP.
Abstract: There are no agreed upon terminology to define "refractory" pediatric immune thrombocytopenia (ITP). Guidelines are therefore limited to arbitrary and outdated definitions. The Pediatric ITP Consortium of North America held a meeting in 2023 to define this entity. With 100% agreement, the faculty established that pediatric ITP that is refractory to emergent therapy could be defined as no platelet response after treatment with all eligible emergent pharmacotherapies. With 100% agreement, the working group established that pediatric patients with ITP that continue to demonstrate high disease burden and/or no platelet response despite treatment with multiple classes of disease-modifying therapies represent a challenging subset of ITP. These patients are at higher risk of ongoing disease burden and merit additional investigation as well as consideration for clinical trials or novel therapies. Future efforts to define disease burden and disease response will be completed in collaboration with the ITP International Working Group.
期刊介绍：
Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016.
Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.
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