Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study.

IF 3 3区医学 Q2 HEMATOLOGY

Annals of Hematology Pub Date : 2025-05-24 DOI:10.1007/s00277-025-06381-3

Luana Fianchi, Alfonso Piciocchi, Fabio Guolo, Francesco Marchesi, Giovanni Marsili, Chiara Cattaneo, Michele Gottardi, Francesco Restuccia, Anna Candoni, Elettra Ortu La Barbera, Rita Fazzi, Crescenza Pasciolla, Olimpia Finizio, Nicola Fracchiolla, Mario Delia, Federica Lessi, Michela Dargenio, Valentina Bonuomo, Maria Ilaria Del Principe, Patrizia Zappasodi, Marco Picardi, Claudia Basilico, Monica Piedimonte, Paola Minetto, Patrizia Chiusolo, Lucia Prezioso, Caterina Buquicchio, Lorella Melillo, Daniele Zama, Francesca Farina, Valentina Mancini, Michela Rondoni, Alessandro Busca, Livio Pagano

{"title":"Matching-adjusted indirect comparison of CPX- 351 in secondary Acute Myeloid Leukemia between the registrative trial and a real-life study.","authors":"Luana Fianchi, Alfonso Piciocchi, Fabio Guolo, Francesco Marchesi, Giovanni Marsili, Chiara Cattaneo, Michele Gottardi, Francesco Restuccia, Anna Candoni, Elettra Ortu La Barbera, Rita Fazzi, Crescenza Pasciolla, Olimpia Finizio, Nicola Fracchiolla, Mario Delia, Federica Lessi, Michela Dargenio, Valentina Bonuomo, Maria Ilaria Del Principe, Patrizia Zappasodi, Marco Picardi, Claudia Basilico, Monica Piedimonte, Paola Minetto, Patrizia Chiusolo, Lucia Prezioso, Caterina Buquicchio, Lorella Melillo, Daniele Zama, Francesca Farina, Valentina Mancini, Michela Rondoni, Alessandro Busca, Livio Pagano","doi":"10.1007/s00277-025-06381-3","DOIUrl":null,"url":null,"abstract":"<p><p>A real-life study on CPX-351 and the standard arm ('7 + 3') of the CPX-351 registrative trial in adults with secondary Acute Myeloid Leukemia were compared by an unanchored Matching-adjusted indirect comparison (MAIC), in order to evaluate the efficacy and toxicity of CPX-351. Results of this study are important to confirm the role of CPX-351 in significantly improving survival and remission rates compared with '7 + 3' with a good safety profile in AML patients with high-risk features, a target group traditionally with a very poor prognosis. Moreover, this pilot analysis underlines the potentiality of the statistical method to compare studies with strong differences.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":3.0000,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Annals of Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s00277-025-06381-3","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

A real-life study on CPX-351 and the standard arm ('7 + 3') of the CPX-351 registrative trial in adults with secondary Acute Myeloid Leukemia were compared by an unanchored Matching-adjusted indirect comparison (MAIC), in order to evaluate the efficacy and toxicity of CPX-351. Results of this study are important to confirm the role of CPX-351 in significantly improving survival and remission rates compared with '7 + 3' with a good safety profile in AML patients with high-risk features, a target group traditionally with a very poor prognosis. Moreover, this pilot analysis underlines the potentiality of the statistical method to compare studies with strong differences.

查看原文本刊更多论文

注册试验和现实研究中CPX- 351治疗继发性急性髓系白血病的匹配调整间接比较

通过非锚定匹配调整间接比较（MAIC），对CPX-351在成人继发性急性髓性白血病患者中的实际研究和CPX-351注册试验的标准组（'7 + 3'）进行比较，以评估CPX-351的疗效和毒性。这项研究的结果对于确认CPX-351在具有高风险特征的AML患者（传统上预后非常差的目标群体）中与具有良好安全性的“7 + 3”相比，在显着提高生存率和缓解率方面的作用非常重要。此外，这项初步分析强调了统计方法在比较具有强烈差异的研究方面的潜力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Annals of Hematology 医学-血液学

CiteScore

5.60

自引率

2.90%

发文量

304

审稿时长

2 months

期刊介绍： Annals of Hematology covers the whole spectrum of clinical and experimental hematology, hemostaseology, blood transfusion, and related aspects of medical oncology, including diagnosis and treatment of leukemias, lymphatic neoplasias and solid tumors, and transplantation of hematopoietic stem cells. Coverage includes general aspects of oncology, molecular biology and immunology as pertinent to problems of human blood disease. The journal is associated with the German Society for Hematology and Medical Oncology, and the Austrian Society for Hematology and Oncology.