福斯塔替尼在原发性慢性免疫血小板减少症治疗中的应用和定位:意大利专家意见。

IF 3.4 3区 医学 Q2 HEMATOLOGY Therapeutic Advances in Hematology Pub Date : 2023-02-28 eCollection Date: 2023-01-01 DOI:10.1177/20406207221147777
Alessandro Lucchesi, Bruno Fattizzo, Valerio De Stefano, Marco Ruggeri, Sergio Siragusa, Nicola Vianelli, Francesco Zaja, Francesco Rodeghiero
{"title":"福斯塔替尼在原发性慢性免疫血小板减少症治疗中的应用和定位:意大利专家意见。","authors":"Alessandro Lucchesi, Bruno Fattizzo, Valerio De Stefano, Marco Ruggeri, Sergio Siragusa, Nicola Vianelli, Francesco Zaja, Francesco Rodeghiero","doi":"10.1177/20406207221147777","DOIUrl":null,"url":null,"abstract":"<p><p>Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, represents a new therapeutic opportunity for patients with immune thrombocytopenia (ITP) in Europe and Italy. However, the positioning of this drug in patient's therapeutic sequence is undefined within the most recent international guidelines. The conclusions from a consensus meeting between Italian experts, whose task was to outline the profile of the ideal candidate to receive fostamatinib, are reported here. A modified Delphi methodology was used to achieve shared statements, which were reported in a narrative form. In particular, the panelists examined the strengths and weaknesses of the registration studies in terms of clinical outcomes, the safety profile of fostamatinib, the drug's impact on the quality of life of patients with chronic ITP, and the potential benefits of its use in the pandemic era. Although the experience with thrombopoietin receptor agonists (TPO-RAs) and the amount of data from real-world studies suggest the preferential use of these drugs as a second-line treatment in most patients, the absence of an increased thrombotic risk in the clinical trials could make fostamatinib a reasonable choice in patients with an increased risk of vascular events. An unstable platelet count during TPO-RAs might also justify a switch to the Syk inhibitor, which is more likely to stabilize the platelet count in responders. Fostamatinib may be preferred to immunosuppressors during the SARS-CoV-2 pandemic, in patients at infectious risk, or in case of contraindication to splenectomy. Finally, the novel mechanism of action makes it an attractive drug in multi-refractory patient.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4000,"publicationDate":"2023-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/17/63/10.1177_20406207221147777.PMC10326469.pdf","citationCount":"0","resultStr":"{\"title\":\"Use and positioning of fostamatinib in the management of primary chronic immune thrombocytopenia: an Italian expert opinion.\",\"authors\":\"Alessandro Lucchesi, Bruno Fattizzo, Valerio De Stefano, Marco Ruggeri, Sergio Siragusa, Nicola Vianelli, Francesco Zaja, Francesco Rodeghiero\",\"doi\":\"10.1177/20406207221147777\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, represents a new therapeutic opportunity for patients with immune thrombocytopenia (ITP) in Europe and Italy. However, the positioning of this drug in patient's therapeutic sequence is undefined within the most recent international guidelines. The conclusions from a consensus meeting between Italian experts, whose task was to outline the profile of the ideal candidate to receive fostamatinib, are reported here. A modified Delphi methodology was used to achieve shared statements, which were reported in a narrative form. In particular, the panelists examined the strengths and weaknesses of the registration studies in terms of clinical outcomes, the safety profile of fostamatinib, the drug's impact on the quality of life of patients with chronic ITP, and the potential benefits of its use in the pandemic era. Although the experience with thrombopoietin receptor agonists (TPO-RAs) and the amount of data from real-world studies suggest the preferential use of these drugs as a second-line treatment in most patients, the absence of an increased thrombotic risk in the clinical trials could make fostamatinib a reasonable choice in patients with an increased risk of vascular events. An unstable platelet count during TPO-RAs might also justify a switch to the Syk inhibitor, which is more likely to stabilize the platelet count in responders. Fostamatinib may be preferred to immunosuppressors during the SARS-CoV-2 pandemic, in patients at infectious risk, or in case of contraindication to splenectomy. Finally, the novel mechanism of action makes it an attractive drug in multi-refractory patient.</p>\",\"PeriodicalId\":23048,\"journal\":{\"name\":\"Therapeutic Advances in Hematology\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":3.4000,\"publicationDate\":\"2023-02-28\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/17/63/10.1177_20406207221147777.PMC10326469.pdf\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic Advances in Hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/20406207221147777\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2023/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic Advances in Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/20406207221147777","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2023/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

摘要

在欧洲和意大利,脾脏酪氨酸激酶(Syk)抑制剂福斯塔替尼为免疫性血小板减少症(ITP)患者带来了新的治疗机会。然而,在最新的国际指南中,这种药物在患者治疗序列中的定位尚未明确。本文报告了意大利专家共识会议的结论,该会议的任务是概述接受福斯塔替尼治疗的理想候选者的特征。会议采用了改良的德尔菲方法来达成共识,并以叙述的形式进行了报告。特别是,小组成员审查了注册研究在临床结果、福司他替尼的安全性、该药物对慢性ITP患者生活质量的影响以及在大流行病时代使用该药物的潜在益处等方面的优缺点。尽管使用促血小板生成素受体激动剂(TPO-RA)的经验和实际研究的大量数据表明,大多数患者优先选择这些药物作为二线治疗,但临床试验中没有发现血栓风险增加的情况,因此对于血管事件风险增加的患者来说,福斯他替尼是一个合理的选择。服用TPO-RA期间血小板计数不稳定,也有理由改用Syk抑制剂,因为后者更有可能稳定应答者的血小板计数。在SARS-CoV-2大流行期间,有感染风险的患者或有脾切除术禁忌症的患者可能会首选福斯他替尼,而不是免疫抑制剂。最后,新的作用机制使其成为治疗多重难治性患者的一种有吸引力的药物。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

摘要图片

摘要图片

摘要图片

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
Use and positioning of fostamatinib in the management of primary chronic immune thrombocytopenia: an Italian expert opinion.

Fostamatinib, a spleen tyrosine kinase (Syk) inhibitor, represents a new therapeutic opportunity for patients with immune thrombocytopenia (ITP) in Europe and Italy. However, the positioning of this drug in patient's therapeutic sequence is undefined within the most recent international guidelines. The conclusions from a consensus meeting between Italian experts, whose task was to outline the profile of the ideal candidate to receive fostamatinib, are reported here. A modified Delphi methodology was used to achieve shared statements, which were reported in a narrative form. In particular, the panelists examined the strengths and weaknesses of the registration studies in terms of clinical outcomes, the safety profile of fostamatinib, the drug's impact on the quality of life of patients with chronic ITP, and the potential benefits of its use in the pandemic era. Although the experience with thrombopoietin receptor agonists (TPO-RAs) and the amount of data from real-world studies suggest the preferential use of these drugs as a second-line treatment in most patients, the absence of an increased thrombotic risk in the clinical trials could make fostamatinib a reasonable choice in patients with an increased risk of vascular events. An unstable platelet count during TPO-RAs might also justify a switch to the Syk inhibitor, which is more likely to stabilize the platelet count in responders. Fostamatinib may be preferred to immunosuppressors during the SARS-CoV-2 pandemic, in patients at infectious risk, or in case of contraindication to splenectomy. Finally, the novel mechanism of action makes it an attractive drug in multi-refractory patient.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
CiteScore
4.30
自引率
0.00%
发文量
54
审稿时长
7 weeks
期刊介绍: Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
期刊最新文献
Pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease: final results from the phase II SOLACE-adults study. Long-term risks of cardiovascular-specific mortality among myeloproliferative neoplasms patients. Clinical features and treatment of newly diagnosed multiple myeloma with secondary myelofibrosis: a retrospective study. Real-world efficacy of chidamide plus R-CHOP in newly diagnosed double-expressor diffuse large B-cell lymphoma. Iron deficiency anemia: an early clinical presentation of cytomegalovirus-induced hemorrhagic colitis in chronic myeloid leukemia patients under dasatinib treatment.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1