Transfusion independence after lenalidomide discontinuation in patients with del(5q) myelodysplastic neoplasm: a HARMONY Alliance study

IF 12.8 1区 医学 Q1 HEMATOLOGY
Elena Crisà, Elvira Mora, Ulrich Germing, Cecile Bally, Maria Diez Campelo, Mikko Myllymäki, Martin Jädersten, Rami Komrokji, Uwe Platzbecker, Detlef Haase, Wolf-Karsten Hofmann, Najla H. Al Ali, Daniela Barraco, Juan José Bargay, Teresa Bernal, Felix López Cadenas, Anna Calvisi, Isabella Capodanno, Marco Cerrano, Rosanna Ciancia, Monica Crugnola, Andrea Kündgen, Carlo Finelli, Claudio Fozza, Chiara Frairia, Ebeling Freja, Christina Ganster, Anne Sophie Kubasch, Maria Jose Jimenez, Roberto Latagliata, Francisca Hernandez Mohedo, Antonieta Molero, Miriam Vara Pampliega, Clara Aparicio Perez, Giuseppe Pietrantuono, Antonella Poloni, Helena Pomares, Valle Recasens, Axel Rüfer, Alessio Signori, Eva Hellstrom-Lindberg, Pierre Fenaux, Guillermo Sanz, Valeria Santini
{"title":"Transfusion independence after lenalidomide discontinuation in patients with del(5q) myelodysplastic neoplasm: a HARMONY Alliance study","authors":"Elena Crisà, Elvira Mora, Ulrich Germing, Cecile Bally, Maria Diez Campelo, Mikko Myllymäki, Martin Jädersten, Rami Komrokji, Uwe Platzbecker, Detlef Haase, Wolf-Karsten Hofmann, Najla H. Al Ali, Daniela Barraco, Juan José Bargay, Teresa Bernal, Felix López Cadenas, Anna Calvisi, Isabella Capodanno, Marco Cerrano, Rosanna Ciancia, Monica Crugnola, Andrea Kündgen, Carlo Finelli, Claudio Fozza, Chiara Frairia, Ebeling Freja, Christina Ganster, Anne Sophie Kubasch, Maria Jose Jimenez, Roberto Latagliata, Francisca Hernandez Mohedo, Antonieta Molero, Miriam Vara Pampliega, Clara Aparicio Perez, Giuseppe Pietrantuono, Antonella Poloni, Helena Pomares, Valle Recasens, Axel Rüfer, Alessio Signori, Eva Hellstrom-Lindberg, Pierre Fenaux, Guillermo Sanz, Valeria Santini","doi":"10.1038/s41375-024-02360-1","DOIUrl":null,"url":null,"abstract":"Lenalidomide (LEN) can induce red blood cell-transfusion independence (RBC-TI) in 60–70% of del(5q) myelodysplastic neoplasm (MDS) patients. Current recommendation is to continue LEN in responding patients until failure or progression, with likelihood of toxicity and a high cost for healthcare systems. This HARMONY Alliance study investigated the outcome of MDS del(5q) patients who discontinued LEN while RBC-transfusion independent. We enrolled 118 patients with IPSS-R low-intermediate risk. Seventy patients (59%) discontinued LEN for intolerance, 38 (32%) per their physician decision, nine (8%) per their own decision and one (1%) for unknown reasons. After a median follow-up of 49 months from discontinuation, 50/118 patients lost RBC-TI and 22/30 who underwent cytogenetic re-evaluation lost complete cytogenetic response. The median RBC-TI duration was 56 months. In multivariate analysis, RBC-TI duration after LEN discontinuation correlated with low transfusion burden before LEN therapy, treatment ≥ 12 LEN cycles, younger age and higher Hb level at LEN withdrawal. Forty-eight patients were re-treated with LEN for loss of response and 28 achieved again RBC-TI. These data show that stopping LEN therapy in MDS del(5q) patients who reached RBC-TI allows prolonged maintenance of TI in a large subset of patients.","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":null,"pages":null},"PeriodicalIF":12.8000,"publicationDate":"2024-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Leukemia","FirstCategoryId":"3","ListUrlMain":"https://www.nature.com/articles/s41375-024-02360-1","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Lenalidomide (LEN) can induce red blood cell-transfusion independence (RBC-TI) in 60–70% of del(5q) myelodysplastic neoplasm (MDS) patients. Current recommendation is to continue LEN in responding patients until failure or progression, with likelihood of toxicity and a high cost for healthcare systems. This HARMONY Alliance study investigated the outcome of MDS del(5q) patients who discontinued LEN while RBC-transfusion independent. We enrolled 118 patients with IPSS-R low-intermediate risk. Seventy patients (59%) discontinued LEN for intolerance, 38 (32%) per their physician decision, nine (8%) per their own decision and one (1%) for unknown reasons. After a median follow-up of 49 months from discontinuation, 50/118 patients lost RBC-TI and 22/30 who underwent cytogenetic re-evaluation lost complete cytogenetic response. The median RBC-TI duration was 56 months. In multivariate analysis, RBC-TI duration after LEN discontinuation correlated with low transfusion burden before LEN therapy, treatment ≥ 12 LEN cycles, younger age and higher Hb level at LEN withdrawal. Forty-eight patients were re-treated with LEN for loss of response and 28 achieved again RBC-TI. These data show that stopping LEN therapy in MDS del(5q) patients who reached RBC-TI allows prolonged maintenance of TI in a large subset of patients.

Abstract Image

Abstract Image

del(5q)骨髓增生异常肿瘤患者停用来那度胺后的输血独立性:HARMONY联盟研究。
来那度胺(LEN)可使60%-70%的del(5q)骨髓增生异常肿瘤(MDS)患者摆脱红细胞输血(RBC-TI)。目前的建议是继续对有反应的患者进行 LEN 治疗,直到失败或病情恶化,但这样做可能会产生毒性,而且会给医疗系统带来高昂的成本。这项 HARMONY 联盟研究调查了停用 LEN 且不依赖红细胞输注的 MDS del(5q) 患者的预后。我们招募了 118 名 IPSS-R 低中危患者。70名患者(59%)因不耐受而停用LEN,38名患者(32%)根据医生的决定停用,9名患者(8%)根据自己的决定停用,1名患者(1%)原因不明。在停药后中位随访49个月后,50/118名患者失去了RBC-TI,22/30名接受细胞遗传学再评估的患者失去了完全细胞遗传学反应。中位 RBC-TI 持续时间为 56 个月。在多变量分析中,LEN停药后RBC-TI持续时间与LEN治疗前输血负担低、LEN治疗周期≥12个、年龄较小及LEN停药时Hb水平较高相关。48例患者因反应消失而再次接受LEN治疗,其中28例再次实现了RBC-TI。这些数据表明,对达到RBC-TI的MDS del(5q)患者停止LEN治疗可使一大部分患者的TI得到长期维持。
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
Leukemia
Leukemia 医学-血液学
CiteScore
18.10
自引率
3.50%
发文量
270
审稿时长
3-6 weeks
期刊介绍: Title: Leukemia Journal Overview: Publishes high-quality, peer-reviewed research Covers all aspects of research and treatment of leukemia and allied diseases Includes studies of normal hemopoiesis due to comparative relevance Topics of Interest: Oncogenes Growth factors Stem cells Leukemia genomics Cell cycle Signal transduction Molecular targets for therapy And more Content Types: Original research articles Reviews Letters Correspondence Comments elaborating on significant advances and covering topical issues
×
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
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
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 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信