Pub Date : 2024-07-13eCollection Date: 2024-08-01DOI: 10.1016/j.eclinm.2024.102728
Christopher R Cederroth, Brian D Earp, Hernando C Gómez Prada, Carlotta M Jarach, Shlomit A Lir, Colleen M Norris, Louise Pilote, Valeria Raparelli, Paula Rochon, Nina Sahraoui, Cassandra Simmon, Bilkis Vissandjee, Chloé Mour, Mathieu Arbogast, José María Armengol, Robin Mason
Gender equality has been a crosscutting issue in Horizon 2020 with three objectives: gender balance in decision-making, gender balance and equal opportunities in project teams at all levels, and inclusion of the gender dimension in research and innovation content. Between 2017 and 2022, the EU funded, in collaboration with national agencies, 13 transnational projects under "GENDER-NET Plus" that explored how to best integrate both sex and gender into studies ranging from social sciences, humanities, and health research. As the projects neared completion, forty researchers from these interdisciplinary teams met in November 2022 to share experiences, discuss challenges, and consider the best ways forward to incorporate sex and gender in research. Here, we summarize the reflections from this workshop and provide some recommendations for i) how to plan the studies (e.g., how to define sex and/or gender and their dimensions, rationale for the hypotheses, identification of data that can best answer the research question), ii) how to conduct them (e.g., adjust definitions and dimensions, perform pilot studies to ensure proper use of terminology and revise until consensus is achieved), and iii) how to analyze and report the findings being mindful of any real-world impact.
{"title":"Integrating gender analysis into research: reflections from the Gender-Net Plus workshop.","authors":"Christopher R Cederroth, Brian D Earp, Hernando C Gómez Prada, Carlotta M Jarach, Shlomit A Lir, Colleen M Norris, Louise Pilote, Valeria Raparelli, Paula Rochon, Nina Sahraoui, Cassandra Simmon, Bilkis Vissandjee, Chloé Mour, Mathieu Arbogast, José María Armengol, Robin Mason","doi":"10.1016/j.eclinm.2024.102728","DOIUrl":"10.1016/j.eclinm.2024.102728","url":null,"abstract":"<p><p>Gender equality has been a crosscutting issue in Horizon 2020 with three objectives: gender balance in decision-making, gender balance and equal opportunities in project teams at all levels, and inclusion of the gender dimension in research and innovation content. Between 2017 and 2022, the EU funded, in collaboration with national agencies, 13 transnational projects under \"GENDER-NET Plus\" that explored how to best integrate both sex and gender into studies ranging from social sciences, humanities, and health research. As the projects neared completion, forty researchers from these interdisciplinary teams met in November 2022 to share experiences, discuss challenges, and consider the best ways forward to incorporate sex and gender in research. Here, we summarize the reflections from this workshop and provide some recommendations for i) how to plan the studies (e.g., how to define sex and/or gender and their dimensions, rationale for the hypotheses, identification of data that can best answer the research question), ii) how to conduct them (e.g., adjust definitions and dimensions, perform pilot studies to ensure proper use of terminology and revise until consensus is achieved), and iii) how to analyze and report the findings being mindful of any real-world impact.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-07-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11299571/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893170","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-13eCollection Date: 2024-08-01DOI: 10.1016/j.eclinm.2024.102730
Boris Tchakounte Youngui, Albert Mambo, Rhoderick Machekano, Rogacien Kana, Emilienne Epée, Sylvain Zemsi Tenkeu, Philippe Narcisse Tsigaing, Marie Louise Aimée Ndongo, Christelle Mayap Njoukam, Lawane Bichara, Tatiana Djikeussi Katcho, Muhamed Awolu Mbunka, Terence Acheliu Longla, Leonie Simo, Adrienne Vanessa Kouatchouang, Patrice Tchendjou, Appolinaire Tiam, Laura Guay, Khairunisa Suleiman, Olukunle Akinwusi, Rigveda Kadam, Paula Akugizibwe, Mario Songane, Godfrey Woelk, Boris Kevin Tchounga
Background: Contact tracing was described as a key strategy to contribute to controlling the spread of severe acute respiratory syndrome of Coronavirus 2 (SARS-CoV-2) but implementing it can be a challenge. Digitalisation of contact tracing is among the proposed solutions being explored in sub-Saharan African settings. We assessed the effectiveness of a digital tool to expand SARS-CoV-2 testing in exposed individuals in Cameroon.
Methods: We conducted a cluster-randomised (1:1) trial in eight health districts, including 22 facilities and SARS-CoV-2 testing units, randomly assigned to a digital (intervention) or standard (control) contact tracing approach. The intervention consisted of a contact tracing module added to the digital platform "Mamal PRO" used for monitoring and coordination of Coronavirus Disease 2019 pandemic response in Cameroon. The primary outcome was the proportion of contacts declared by SAR-CoV-2 index patients who were successfully traced and tested for SARS-CoV-2 evaluated with a Poisson regression model with cluster adjustment. This study is registered with ClinicalTrials.gov (NCT05684887).
Findings: Between October 18, 2022, and March 31, 2023, we enrolled 164 index patients in the intervention arm and 149 in the control arm, who identified 854 and 849 contacts, respectively. In the intervention arm, 93.8% (801/854) of identified contacts were successfully reached by the tracing unit versus 54.5% (463/849) in the control arm. The intervention significantly increased the likelihood of successfully tracing contacts (adjusted relative risks (RR) 1.72 [95% CI: 1.00-2.95], p = 0.049). The median (interquartile range, IQR) time to successfully tracing contacts was 0 days [IQR: 0, 1] in the intervention and 1 day [IQR: 0, 2] in the control arm. In the intervention arm, 21.3% (182/854) of identified contacts received SARS-CoV-2 testing compared to 14.5% (123/849) in the control arm (adjusted RR 1.47 [95% CI: 0.44-4.90], p = 0.530).
Interpretation: Digitalising the contact tracing process improved exposure notification and facilitated the tracing of a greater number of contacts of individuals infected with SARS-CoV-2 in resource-limited settings.
Funding: The study was funded by FIND, United Kingdom (FCDO 40105983), Switzerland (81066910), Netherlands (SDD 4000004160), Canada (DFATD 7429348), The Kingdom of Saudi Arabia (FIND-ACT-A DX PARTNERSHIP 20.08.2020), The Rockefeller Foundation (2020 HTH 059), Germany (BMZ Covid-19 Diagnostic and Surveillance Response 27.07.2021), Australia (DFAT 76442), Kuwait (M239/2020), The Government of Portugal and Partners (ANF, BCP, CGF, APIFARMA) and The BlackRock Foundation (Grant Agreement as of April 20, 2022).
背景:接触者追踪被认为是有助于控制严重急性呼吸系统综合症冠状病毒 2(SARS-CoV-2)传播的关键策略,但实施起来却很困难。接触者追踪数字化是撒哈拉以南非洲地区正在探索的解决方案之一。我们评估了数字化工具在喀麦隆扩大接触者 SARS-CoV-2 检测范围的有效性:我们在八个卫生区进行了分组随机(1:1)试验,包括 22 个设施和 SARS-CoV-2 检测单位,随机分配到数字(干预)或标准(对照)接触者追踪方法。干预措施包括在数字平台 "Mamal PRO "中添加接触者追踪模块,该平台用于监测和协调喀麦隆 2019 年冠状病毒疾病大流行应对工作。主要结果是,SARS-CoV-2指数患者申报的接触者中,成功追踪到并接受SARS-CoV-2检测的接触者所占比例。本研究已在 ClinicalTrials.gov (NCT05684887) 注册:2022年10月18日至2023年3月31日期间,我们在干预组和对照组分别招募了164名和149名指数患者,他们分别确定了854名和849名接触者。在干预组中,93.8%(801/854)的已确认联系人成功联系到追踪单位,而在对照组中,这一比例为 54.5%(463/849)。干预措施极大地提高了成功追踪接触者的可能性(调整后相对风险 (RR) 1.72 [95% CI: 1.00-2.95],p = 0.049)。成功追踪接触者的中位时间(四分位数间距,IQR)在干预组为 0 天 [IQR: 0, 1],在对照组为 1 天 [IQR: 0, 2]。在干预组中,21.3%(182/854)被确认的接触者接受了 SARS-CoV-2 检测,而在对照组中,这一比例为 14.5%(123/849)(调整后 RR 为 1.47 [95% CI:0.44-4.90],p = 0.530):解释:接触者追踪过程的数字化改进了暴露通知,有助于在资源有限的环境中追踪更多感染 SARS-CoV-2 的接触者:本研究由 FIND、英国(FCDO 40105983)、瑞士(81066910)、荷兰(SDD 4000004160)、加拿大(DFATD 7429348)、沙特阿拉伯王国(FIND-ACT-A DX PARTNERSHIP 20.08.2020), The Rockefeller Foundation (2020 HTH 059), Germany (BMZ Covid-19 Diagnostic and Surveillance Response 27.07.2021), Australia (DFAT 76442), Kuwait (M239/2020), The Government of Portugal and Partners (ANF, BCP, CGF, APIFARMA) and The BlackRock Foundation (Grant Agreement as of April 20, 2022).
{"title":"Improving COVID-19 contact tracing and testing of exposed individuals in Cameroon using digital health technology: a cluster randomised trial.","authors":"Boris Tchakounte Youngui, Albert Mambo, Rhoderick Machekano, Rogacien Kana, Emilienne Epée, Sylvain Zemsi Tenkeu, Philippe Narcisse Tsigaing, Marie Louise Aimée Ndongo, Christelle Mayap Njoukam, Lawane Bichara, Tatiana Djikeussi Katcho, Muhamed Awolu Mbunka, Terence Acheliu Longla, Leonie Simo, Adrienne Vanessa Kouatchouang, Patrice Tchendjou, Appolinaire Tiam, Laura Guay, Khairunisa Suleiman, Olukunle Akinwusi, Rigveda Kadam, Paula Akugizibwe, Mario Songane, Godfrey Woelk, Boris Kevin Tchounga","doi":"10.1016/j.eclinm.2024.102730","DOIUrl":"10.1016/j.eclinm.2024.102730","url":null,"abstract":"<p><strong>Background: </strong>Contact tracing was described as a key strategy to contribute to controlling the spread of severe acute respiratory syndrome of Coronavirus 2 (SARS-CoV-2) but implementing it can be a challenge. Digitalisation of contact tracing is among the proposed solutions being explored in sub-Saharan African settings. We assessed the effectiveness of a digital tool to expand SARS-CoV-2 testing in exposed individuals in Cameroon.</p><p><strong>Methods: </strong>We conducted a cluster-randomised (1:1) trial in eight health districts, including 22 facilities and SARS-CoV-2 testing units, randomly assigned to a digital (intervention) or standard (control) contact tracing approach. The intervention consisted of a contact tracing module added to the digital platform \"Mamal PRO\" used for monitoring and coordination of Coronavirus Disease 2019 pandemic response in Cameroon. The primary outcome was the proportion of contacts declared by SAR-CoV-2 index patients who were successfully traced and tested for SARS-CoV-2 evaluated with a Poisson regression model with cluster adjustment. This study is registered with ClinicalTrials.gov (NCT05684887).</p><p><strong>Findings: </strong>Between October 18, 2022, and March 31, 2023, we enrolled 164 index patients in the intervention arm and 149 in the control arm, who identified 854 and 849 contacts, respectively. In the intervention arm, 93.8% (801/854) of identified contacts were successfully reached by the tracing unit versus 54.5% (463/849) in the control arm. The intervention significantly increased the likelihood of successfully tracing contacts (adjusted relative risks (RR) 1.72 [95% CI: 1.00-2.95], p = 0.049). The median (interquartile range, IQR) time to successfully tracing contacts was 0 days [IQR: 0, 1] in the intervention and 1 day [IQR: 0, 2] in the control arm. In the intervention arm, 21.3% (182/854) of identified contacts received SARS-CoV-2 testing compared to 14.5% (123/849) in the control arm (adjusted RR 1.47 [95% CI: 0.44-4.90], p = 0.530).</p><p><strong>Interpretation: </strong>Digitalising the contact tracing process improved exposure notification and facilitated the tracing of a greater number of contacts of individuals infected with SARS-CoV-2 in resource-limited settings.</p><p><strong>Funding: </strong>The study was funded by FIND, United Kingdom (FCDO 40105983), Switzerland (81066910), Netherlands (SDD 4000004160), Canada (DFATD 7429348), The Kingdom of Saudi Arabia (FIND-ACT-A DX PARTNERSHIP 20.08.2020), The Rockefeller Foundation (2020 HTH 059), Germany (BMZ Covid-19 Diagnostic and Surveillance Response 27.07.2021), Australia (DFAT 76442), Kuwait (M239/2020), The Government of Portugal and Partners (ANF, BCP, CGF, APIFARMA) and The BlackRock Foundation (Grant Agreement as of April 20, 2022).</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-07-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11300906/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897103","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-12eCollection Date: 2024-08-01DOI: 10.1016/j.eclinm.2024.102707
Zhenyi Niu, Yuqin Cao, Mingyuan Du, Siying Sun, Yan Yan, Yuyan Zheng, Yichao Han, Xianfei Zhang, Zhengyuan Zhang, Ye Yuan, Jian Li, Yajie Zhang, Chengqiang Li, Dingpei Han, Hailei Du, Wei Guo, Kai Chen, Jie Xiang, Lianggang Zhu, Jiaming Che, Junbiao Hang, Jian Ren, Toni Lerut, Abbas E Abbas, Jules Lin, Runsen Jin, Hecheng Li
Background: The long-term survival and perioperative outcomes of robotic-assisted lobectomy (RAL) and video-assisted lobectomy (VAL) in resectable non-small-cell lung cancer (NSCLC) were found to be comparable in retrospective studies, but they have not been investigated in a randomized trial setting. We conducted the RVlob trial to investigate if RAL was non-inferior to VAL in patients with resectable NSCLC.
Methods: In this single-center, open-label, and parallel-arm randomized controlled trial conducted in Ruijin Hospital (Shanghai, China) between May 2017 and May 2020, we randomly assigned patients with resectable NSCLC in a 1:1 ratio to receive either RAL or VAL. One of the primary endpoints was 3-year overall survival. Secondary endpoints included 3-year disease-free survival. The Kaplan-Meier approach was used to calculate overall survival and disease-free survival at 3 years. This study was registered with ClinicalTrials.gov, NCT03134534.
Findings: A total of 320 patients were randomized to receive RAL (n = 157) or VAL (n = 163). The baseline characteristics of patients were well balanced between the two groups. After a median follow-up of 58.0 months, the 3-year overall survival was 94.6% (95% confidence interval [CI], 91.0-98.3) in the RAL group and 91.5% (95% CI, 87.2-96.0) in the VAL group (hazard ratio [HR] for death, 0.65; 95% CI, 0.33-1.28; P = 0.21); noninferiority of RAL was confirmed according to the predefined margin of -5% (absolute difference, 2.96%; a one-sided 90% CI, -1.39% to ∞; P = 0.0029 for noninferiority). The 3-year disease-free survival was 88.7% (95% CI, 83.6-94.1) in the RAL group and 85.4% (95% CI, 80.0-91.2) in the VAL group (HR for disease recurrence or death, 0.87; 95% CI, 0.50-1.52; P = 0.62).
Interpretation: This study is the first randomized trial to show that RAL resulted in non-inferior overall survival compared with VAL in patients with resectable NSCLC. Based on our results, RAL is an equally oncologically effective treatment and can be considered as an alternative to VAL for resectable NSCLC.
Funding: National Natural Science Foundation of China (82072557), National Key Research and Development Program of China (2021YFC2500900), Shanghai Municipal Education Commission-Gaofeng Clinical Medicine Grant (20172005, the 2nd round of disbursement), program of Shanghai Academic Research Leader from Science and Technology Commission of Shanghai Municipality (20XD1402300), Novel Interdisciplinary Research Project from Shanghai Municipal Health Commission (2022JC023), and Interdisciplinary Program of Shanghai Jiao Tong University (YG2023ZD04).
{"title":"Robotic-assisted versus video-assisted lobectomy for resectable non-small-cell lung cancer: the RVlob randomized controlled trial.","authors":"Zhenyi Niu, Yuqin Cao, Mingyuan Du, Siying Sun, Yan Yan, Yuyan Zheng, Yichao Han, Xianfei Zhang, Zhengyuan Zhang, Ye Yuan, Jian Li, Yajie Zhang, Chengqiang Li, Dingpei Han, Hailei Du, Wei Guo, Kai Chen, Jie Xiang, Lianggang Zhu, Jiaming Che, Junbiao Hang, Jian Ren, Toni Lerut, Abbas E Abbas, Jules Lin, Runsen Jin, Hecheng Li","doi":"10.1016/j.eclinm.2024.102707","DOIUrl":"10.1016/j.eclinm.2024.102707","url":null,"abstract":"<p><strong>Background: </strong>The long-term survival and perioperative outcomes of robotic-assisted lobectomy (RAL) and video-assisted lobectomy (VAL) in resectable non-small-cell lung cancer (NSCLC) were found to be comparable in retrospective studies, but they have not been investigated in a randomized trial setting. We conducted the RVlob trial to investigate if RAL was non-inferior to VAL in patients with resectable NSCLC.</p><p><strong>Methods: </strong>In this single-center, open-label, and parallel-arm randomized controlled trial conducted in Ruijin Hospital (Shanghai, China) between May 2017 and May 2020, we randomly assigned patients with resectable NSCLC in a 1:1 ratio to receive either RAL or VAL. One of the primary endpoints was 3-year overall survival. Secondary endpoints included 3-year disease-free survival. The Kaplan-Meier approach was used to calculate overall survival and disease-free survival at 3 years. This study was registered with ClinicalTrials.gov, NCT03134534.</p><p><strong>Findings: </strong>A total of 320 patients were randomized to receive RAL (n = 157) or VAL (n = 163). The baseline characteristics of patients were well balanced between the two groups. After a median follow-up of 58.0 months, the 3-year overall survival was 94.6% (95% confidence interval [CI], 91.0-98.3) in the RAL group and 91.5% (95% CI, 87.2-96.0) in the VAL group (hazard ratio [HR] for death, 0.65; 95% CI, 0.33-1.28; <i>P</i> = 0.21); noninferiority of RAL was confirmed according to the predefined margin of -5% (absolute difference, 2.96%; a one-sided 90% CI, -1.39% to ∞; <i>P</i> = 0.0029 for noninferiority). The 3-year disease-free survival was 88.7% (95% CI, 83.6-94.1) in the RAL group and 85.4% (95% CI, 80.0-91.2) in the VAL group (HR for disease recurrence or death, 0.87; 95% CI, 0.50-1.52; <i>P</i> = 0.62).</p><p><strong>Interpretation: </strong>This study is the first randomized trial to show that RAL resulted in non-inferior overall survival compared with VAL in patients with resectable NSCLC. Based on our results, RAL is an equally oncologically effective treatment and can be considered as an alternative to VAL for resectable NSCLC.</p><p><strong>Funding: </strong>National Natural Science Foundation of China (82072557), National Key Research and Development Program of China (2021YFC2500900), Shanghai Municipal Education Commission-Gaofeng Clinical Medicine Grant (20172005, the 2nd round of disbursement), program of Shanghai Academic Research Leader from Science and Technology Commission of Shanghai Municipality (20XD1402300), Novel Interdisciplinary Research Project from Shanghai Municipal Health Commission (2022JC023), and Interdisciplinary Program of Shanghai Jiao Tong University (YG2023ZD04).</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11299594/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141893171","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-07-05eCollection Date: 2024-08-01DOI: 10.1016/j.eclinm.2024.102704
Jean-Francois Trani, Yiqi Zhu, Soobin Park, Dauod Khuram, Rahim Azami, Monib Rahim Fazal, Ganesh M Babulal
[This corrects the article DOI: 10.1016/j.eclinm.2023.101906.].
[此处更正文章 DOI:10.1016/j.eclinm.2023.101906.]。
{"title":"Corrigendum to \"Multidimensional poverty is associated with dementia among adults in Afghanistan\" [eClinicalMedicine 58(2023) 101906].","authors":"Jean-Francois Trani, Yiqi Zhu, Soobin Park, Dauod Khuram, Rahim Azami, Monib Rahim Fazal, Ganesh M Babulal","doi":"10.1016/j.eclinm.2024.102704","DOIUrl":"10.1016/j.eclinm.2024.102704","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1016/j.eclinm.2023.101906.].</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11276918/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141787582","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-21eCollection Date: 2024-07-01DOI: 10.1016/j.eclinm.2024.102685
Long Chang, Min Lang, Ting Liu, He Lin, Zheng-Zheng Liu, Hao Cai, Dao-Bin Zhou, Xin-Xin Cao
Background: Rosai-Dorfman disease (RDD) is a rare heterogeneous histiocytic disorder lacking standardized first-line treatment.
Methods: This single-center, phase 2 prospective study enrolled 13 newly diagnosed and 10 recurrent RDD patients from June 2021 to March 2023 at Peking Union Medical College Hospital (Beijing, China). Lenalidomide 25 mg days 1-21 plus dexamethasone 40 mg days 1, 8, 15, 22 was administered in 28-day cycles, totaling 12 cycles. The primary endpoint was progression-free survival (PFS). Secondary endpoints were overall response rate (ORR) to lenalidomide and dexamethasone (RD) regimen, toxicity, and overall survival (OS) measured from RD start to death or last follow-up. OS and PFS were estimated according to Kaplan-Meier survival analysis and compared with the log-rank test. For OS and OR rate, 95% confidence limits were obtained using the Clopper-Pearson method, with standard methods used for PFS. p < 0.05 was considered statistically significant. The trial was registered with ClinicalTrials.gov (NCT04924647).
Findings: The median age was 44 years (IQR 35-54). All patients had extranodal RDD. MAPK pathway alterations occurred in 6/18 (33%). Elevated IL-6 and TNF-α were found in 39% (n = 9) and 70% (n = 16), respectively. All patients received ≥6 cycles (median 12, range 6-12, IQR 10-12). The ORR was 87% (20/23, 95% CI 66%-97%), 30% (n = 7) complete remission, 57% (n = 13) partial remission). Treatment with RD significantly decreased median serum levels of both IL-6 (from 5.9 (IQR 4.2-8.7) to 2.9 (IQR 2.1-5.9) pg/mL, p = 0.031) and TNF-α (from 12.2 (IQR 8.6-17.9) to 8.3 (IQR 6.1-10.5) pg/mL, p = 0.0012). With a median 26 months follow-up (range 6-28, IQR 16-28), 4 patients relapsed and none died. Two-year OS and PFS were 100.0% (95% CI 85%-100%) and 69.0% (95% CI 51%-94%), respectively. No grade 3-4 adverse events or discontinuations due to adverse events occurred. Twelve patients (n = 12, 52%) had grade 1-2 hematological toxicity. Other toxicities included constipation (n = 2, 9%), glucose intolerance (n = 2, 9%), edema (n = 2, 9%), insomnia (n = 1, 4%), and tremor (n = 1, 4%).
Interpretation: Lenalidomide and dexamethasone regimen is an effective and safe regimen for newly diagnosed and recurrent RDD.
Funding: National Natural Science Foundation of China, Beijing Natural Science Haidian frontier Foundation Funding, and the National High Level Hospital Clinical Research Funding.
背景:罗赛-多夫曼病(RDD)是一种罕见的异质性组织细胞疾病,缺乏标准化的一线治疗方法:罗赛-多夫曼病(RDD)是一种罕见的异质性组织细胞疾病,缺乏标准化的一线治疗方法:这项单中心2期前瞻性研究于2021年6月至2023年3月在北京协和医院(中国北京)招募了13名新诊断的RDD患者和10名复发的RDD患者。来那度胺25毫克,第1-21天加地塞米松40毫克,第1、8、15、22天,28天为一个周期,共12个周期。主要终点是无进展生存期(PFS)。次要终点是来那度胺和地塞米松(RD)方案的总反应率(ORR)、毒性以及从RD开始到死亡或最后一次随访的总生存期(OS)。OS和PFS根据Kaplan-Meier生存分析进行估算,并用log-rank检验进行比较。对于OS和OR率,采用Clopper-Pearson方法得出95%置信区间,PFS采用标准方法:中位年龄为 44 岁(IQR 35-54)。所有患者均患有结节外 RDD。6/18(33%)的患者发生了 MAPK 通路改变。IL-6和TNF-α升高的比例分别为39%(9人)和70%(16人)。所有患者均接受了≥6个周期的治疗(中位数为12个周期,范围为6-12个周期,IQR为10-12个周期)。ORR为87%(20/23,95% CI 66%-97%),30%(7例)完全缓解,57%(13例)部分缓解。)RD治疗可明显降低IL-6(从5.9(IQR 4.2-8.7)降至2.9(IQR 2.1-5.9)pg/mL,p = 0.031)和TNF-α(从12.2(IQR 8.6-17.9)降至8.3(IQR 6.1-10.5)pg/mL,p = 0.0012)的中位血清水平。中位随访 26 个月(6-28 个月,IQR 16-28),4 名患者复发,无死亡病例。两年的OS和PFS分别为100.0%(95% CI 85%-100%)和69.0%(95% CI 51%-94%)。未发生 3-4 级不良事件或因不良事件而停药。12名患者(n = 12,52%)出现了1-2级血液学毒性。其他毒性包括便秘(2例,9%)、葡萄糖不耐受(2例,9%)、水肿(2例,9%)、失眠(1例,4%)和震颤(1例,4%):来那度胺和地塞米松方案是治疗新诊断和复发性RDD的有效而安全的方案:国家自然科学基金、北京市自然科学海淀前沿基金、国家高级医院临床研究基金。
{"title":"Lenalidomide and dexamethasone for Rosai-Dorfman disease: a single arm, single center, prospective phase 2 study.","authors":"Long Chang, Min Lang, Ting Liu, He Lin, Zheng-Zheng Liu, Hao Cai, Dao-Bin Zhou, Xin-Xin Cao","doi":"10.1016/j.eclinm.2024.102685","DOIUrl":"10.1016/j.eclinm.2024.102685","url":null,"abstract":"<p><strong>Background: </strong>Rosai-Dorfman disease (RDD) is a rare heterogeneous histiocytic disorder lacking standardized first-line treatment.</p><p><strong>Methods: </strong>This single-center, phase 2 prospective study enrolled 13 newly diagnosed and 10 recurrent RDD patients from June 2021 to March 2023 at Peking Union Medical College Hospital (Beijing, China). Lenalidomide 25 mg days 1-21 plus dexamethasone 40 mg days 1, 8, 15, 22 was administered in 28-day cycles, totaling 12 cycles. The primary endpoint was progression-free survival (PFS). Secondary endpoints were overall response rate (ORR) to lenalidomide and dexamethasone (RD) regimen, toxicity, and overall survival (OS) measured from RD start to death or last follow-up. OS and PFS were estimated according to Kaplan-Meier survival analysis and compared with the log-rank test. For OS and OR rate, 95% confidence limits were obtained using the Clopper-Pearson method, with standard methods used for PFS. p < 0.05 was considered statistically significant. The trial was registered with ClinicalTrials.gov (NCT04924647).</p><p><strong>Findings: </strong>The median age was 44 years (IQR 35-54). All patients had extranodal RDD. MAPK pathway alterations occurred in 6/18 (33%). Elevated IL-6 and TNF-α were found in 39% (n = 9) and 70% (n = 16), respectively. All patients received ≥6 cycles (median 12, range 6-12, IQR 10-12). The ORR was 87% (20/23, 95% CI 66%-97%), 30% (n = 7) complete remission, 57% (n = 13) partial remission). Treatment with RD significantly decreased median serum levels of both IL-6 (from 5.9 (IQR 4.2-8.7) to 2.9 (IQR 2.1-5.9) pg/mL, p = 0.031) and TNF-α (from 12.2 (IQR 8.6-17.9) to 8.3 (IQR 6.1-10.5) pg/mL, p = 0.0012). With a median 26 months follow-up (range 6-28, IQR 16-28), 4 patients relapsed and none died. Two-year OS and PFS were 100.0% (95% CI 85%-100%) and 69.0% (95% CI 51%-94%), respectively. No grade 3-4 adverse events or discontinuations due to adverse events occurred. Twelve patients (n = 12, 52%) had grade 1-2 hematological toxicity. Other toxicities included constipation (n = 2, 9%), glucose intolerance (n = 2, 9%), edema (n = 2, 9%), insomnia (n = 1, 4%), and tremor (n = 1, 4%).</p><p><strong>Interpretation: </strong>Lenalidomide and dexamethasone regimen is an effective and safe regimen for newly diagnosed and recurrent RDD.</p><p><strong>Funding: </strong>National Natural Science Foundation of China, Beijing Natural Science Haidian frontier Foundation Funding, and the National High Level Hospital Clinical Research Funding.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11253284/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141632967","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-21eCollection Date: 2024-07-01DOI: 10.1016/j.eclinm.2024.102683
Epke A Le Rutte, Andrew J Shattock, Inês Marcelino, Sophie Goldenberg, Melissa A Penny
Background: In 2023 severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was declared endemic, yet hospital admissions have persisted and risen within populations at high and moderate risk of developing severe disease, which include those of older age, and those with co-morbidities. Antiviral treatments, currently only available for high-risk individuals, play an important role in preventing severe disease and hospitalisation within this subpopulation. Here, we further explore the public health and economic benefits of extending target populations for treatment, and assess efficacy thresholds for a treatment strategy to be cost-saving.
Methods: We adapted an individual-based transmission model of SARS-CoV-2, OpenCOVID, which was calibrated and validated to 2020-2023 Swiss, European, and Northern Hemisphere epidemiological data. We used the model to estimate hospitalisations and overall costs for preventatively treating three risk groups for a full range of treatment efficacies and coverages with, besides vaccination and hospital treatments, no other interventions in place. We further calculated efficacy thresholds for strategies to be cost-saving. A global sensitivity analysis was conducted to test the sensitivity of all outcomes for a wide range of treatment properties, emerging variant properties, and vaccination coverages.
Findings: In a high vaccination coverage setting, we found that a high efficacy antiviral treatment given to all those at high-risk could reduce hospitalisations by up to 40%. When expanding treatment coverage to also include all those at moderate-risk, an additional 50% of hospitalisations could be averted. Targeting both high-risk and moderate-risk groups was found to be cost-saving for a treatment efficacy greater than ∼40%. This threshold was found to be robust regardless of vaccination coverage and emerging variant properties, but highly sensitive to treatment costs.
Interpretation: For a sufficiently efficacious antiviral treatment, expanding the target population to include both high-risk and moderate-risk groups should be considered. Equitable treatment costs are found crucial in achieving the best possible public health and health economic outcomes.
Funding: Botnar Research Centre for Child Health (DZX2165 to MAP), the Swiss National Science Foundation Professorship of MAP (P00P3_203450) and Swiss National Science Foundation NFP 78 Covid-19 2020 (4079P0_198428 to MAP).
{"title":"Efficacy thresholds and target populations for antiviral COVID-19 treatments to save lives and costs: a modelling study.","authors":"Epke A Le Rutte, Andrew J Shattock, Inês Marcelino, Sophie Goldenberg, Melissa A Penny","doi":"10.1016/j.eclinm.2024.102683","DOIUrl":"10.1016/j.eclinm.2024.102683","url":null,"abstract":"<p><strong>Background: </strong>In 2023 severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was declared endemic, yet hospital admissions have persisted and risen within populations at high and moderate risk of developing severe disease, which include those of older age, and those with co-morbidities. Antiviral treatments, currently only available for high-risk individuals, play an important role in preventing severe disease and hospitalisation within this subpopulation. Here, we further explore the public health and economic benefits of extending target populations for treatment, and assess efficacy thresholds for a treatment strategy to be cost-saving.</p><p><strong>Methods: </strong>We adapted an individual-based transmission model of SARS-CoV-2, OpenCOVID, which was calibrated and validated to 2020-2023 Swiss, European, and Northern Hemisphere epidemiological data. We used the model to estimate hospitalisations and overall costs for preventatively treating three risk groups for a full range of treatment efficacies and coverages with, besides vaccination and hospital treatments, no other interventions in place. We further calculated efficacy thresholds for strategies to be cost-saving. A global sensitivity analysis was conducted to test the sensitivity of all outcomes for a wide range of treatment properties, emerging variant properties, and vaccination coverages.</p><p><strong>Findings: </strong>In a high vaccination coverage setting, we found that a high efficacy antiviral treatment given to all those at high-risk could reduce hospitalisations by up to 40%. When expanding treatment coverage to also include all those at moderate-risk, an additional 50% of hospitalisations could be averted. Targeting both high-risk and moderate-risk groups was found to be cost-saving for a treatment efficacy greater than ∼40%. This threshold was found to be robust regardless of vaccination coverage and emerging variant properties, but highly sensitive to treatment costs.</p><p><strong>Interpretation: </strong>For a sufficiently efficacious antiviral treatment, expanding the target population to include both high-risk and moderate-risk groups should be considered. Equitable treatment costs are found crucial in achieving the best possible public health and health economic outcomes.</p><p><strong>Funding: </strong>Botnar Research Centre for Child Health (DZX2165 to MAP), the Swiss National Science Foundation Professorship of MAP (P00P3_203450) and Swiss National Science Foundation NFP 78 Covid-19 2020 (4079P0_198428 to MAP).</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11246010/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141616092","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-21eCollection Date: 2024-07-01DOI: 10.1016/j.eclinm.2024.102691
Muhammad Aaqib Shamim, Jogender Kumar, Amol N Patil, Krishna Tiwari, Sakshi Sharma, Abhishek Anil, Aswini Saravanan, Mokanpally Sandeep, Shoban Babu Varthya, Surjit Singh, Molla Imaduddin Ahmed, Ahmad Najmi, Muhammad Aasim Shamim, Aravind Gandhi, Prakisini Satapathy, Ranjit Sah, Sarvesh Rustagi, Abhay M Gaidhane, Quazi Syed Zahiruddin, Mahalaqua Nazli Khatib, Bijaya Kumar Padhi, Kuldeep Singh, Pradeep Dwivedi
Background: Initial randomised controlled trials (RCTs) showed that prophylactic azithromycin in pregnant women improved maternal and neonatal outcomes; however, the recent evidence did not show any benefit to neonatal survival. There is conflicting evidence over the role of azithromycin prophylaxis in antenatal and intrapartum periods. We explored whether azithromycin prophylaxis in pregnant women improves maternal and neonatal outcomes.
Methods: For this systematic review and meta-analysis registered on PROSPERO [CRD42023411093], we searched seven databases (PubMed, Scopus, Embase, Cochrane Library, EBSCOHost, ProQuest, and Web of Science) and clinical trial registries until 04/23/2024, for RCTs evaluating antenatal/intrapartum azithromycin prophylaxis against placebo/routine care in pregnant women. The primary outcome was neonatal mortality. Intrapartum and antenatal administration were assessed separately. We used random-effects meta-analysis. The risk of bias was assessed using the Cochrane RoB 2 tool. The GRADE approach was used to evaluate the certainty of the evidence.
Findings: Screening 2161 records retrieved 20 RCTs (56,381 participants). Intrapartum azithromycin may make little or no difference to neonatal mortality [5 RCTs, 44,436 participants; Risk Ratio (RR): 1.02, 95% CI 0.86-1.20, I2 = 0%, very low certainty], and maternal mortality [3 RCTs, 44,131 participants, RR: 1.26, 0.65-2.42, I2 = 0%, low certainty]. Similarly, antenatal azithromycin may have little or no effect on neonatal mortality [3 RCTs; 5304 participants; RR: 0.74, 0.35-1.56, I2 = 43%, very-low certainty] and maternal mortality [3 RCTs; 8167 participants RR: 1.62, 0.67-3.91, I2 = 0%, low certainty]. There is no data on long-term adverse outcomes and antimicrobial resistance.
Interpretation: Low to very low certainty evidence suggests that intrapartum or antenatal azithromycin prophylaxis in pregnant women might not reduce maternal or neonatal mortality.
{"title":"PeRinatal, neOnatal, and Maternal OuTcomEs with azithromycin prophylaxis in pregnancy and labour (PROMOTE-PROPHYLAXIS): systematic review and meta-analysis.","authors":"Muhammad Aaqib Shamim, Jogender Kumar, Amol N Patil, Krishna Tiwari, Sakshi Sharma, Abhishek Anil, Aswini Saravanan, Mokanpally Sandeep, Shoban Babu Varthya, Surjit Singh, Molla Imaduddin Ahmed, Ahmad Najmi, Muhammad Aasim Shamim, Aravind Gandhi, Prakisini Satapathy, Ranjit Sah, Sarvesh Rustagi, Abhay M Gaidhane, Quazi Syed Zahiruddin, Mahalaqua Nazli Khatib, Bijaya Kumar Padhi, Kuldeep Singh, Pradeep Dwivedi","doi":"10.1016/j.eclinm.2024.102691","DOIUrl":"10.1016/j.eclinm.2024.102691","url":null,"abstract":"<p><strong>Background: </strong>Initial randomised controlled trials (RCTs) showed that prophylactic azithromycin in pregnant women improved maternal and neonatal outcomes; however, the recent evidence did not show any benefit to neonatal survival. There is conflicting evidence over the role of azithromycin prophylaxis in antenatal and intrapartum periods. We explored whether azithromycin prophylaxis in pregnant women improves maternal and neonatal outcomes.</p><p><strong>Methods: </strong>For this systematic review and meta-analysis registered on PROSPERO [CRD42023411093], we searched seven databases (PubMed, Scopus, Embase, Cochrane Library, EBSCOHost, ProQuest, and Web of Science) and clinical trial registries until 04/23/2024, for RCTs evaluating antenatal/intrapartum azithromycin prophylaxis against placebo/routine care in pregnant women. The primary outcome was neonatal mortality. Intrapartum and antenatal administration were assessed separately. We used random-effects meta-analysis. The risk of bias was assessed using the Cochrane RoB 2 tool. The GRADE approach was used to evaluate the certainty of the evidence.</p><p><strong>Findings: </strong>Screening 2161 records retrieved 20 RCTs (56,381 participants). Intrapartum azithromycin may make little or no difference to neonatal mortality [5 RCTs, 44,436 participants; Risk Ratio (RR): 1.02, 95% CI 0.86-1.20, <i>I</i> <sup><i>2</i></sup> = 0%, very low certainty], and maternal mortality [3 RCTs, 44,131 participants, RR: 1.26, 0.65-2.42, <i>I</i> <sup><i>2</i></sup> = 0%, low certainty]. Similarly, antenatal azithromycin may have little or no effect on neonatal mortality [3 RCTs; 5304 participants; RR: 0.74, 0.35-1.56, <i>I</i> <sup><i>2</i></sup> = 43%, very-low certainty] and maternal mortality [3 RCTs; 8167 participants RR: 1.62, 0.67-3.91, <i>I</i> <sup><i>2</i></sup> = 0%, low certainty]. There is no data on long-term adverse outcomes and antimicrobial resistance.</p><p><strong>Interpretation: </strong>Low to very low certainty evidence suggests that intrapartum or antenatal azithromycin prophylaxis in pregnant women might not reduce maternal or neonatal mortality.</p><p><strong>Funding: </strong>None.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11253273/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141632968","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-21eCollection Date: 2024-07-01DOI: 10.1016/j.eclinm.2024.102679
Alizée Bozonnat, Marie Beylot-Barry, Olivier Dereure, Michel D'Incan, Gaëlle Quereux, Emmanuella Guenova, Marie Perier-Muzet, Stephane Dalle, Florent Grange, Manuelle-Anne Viguier, Caroline Ram-Wolff, Laurence Feldmeyer, Helmut Beltraminelli, Nathalie Bonnet, Florent Amatore, Eve Maubec, Nathalie Franck, Laurent Machet, François Chasset, Florence Brunet-Possenti, Jean-David Bouaziz, Maxime Battistella, Marie Donzel, Anne Pham-Ledard, Claudia Bejar, Hélène Moins-Teisserenc, Samia Mourah, Philippe Saiag, Ewa Hainaut, Catherine Michel, Guido Bens, Henri Adamski, François Aubin, Serge Boulinguez, Pascal Joly, Billal Tedbirt, Isabelle Templier, Laura Troin, Henri Montaudié, Saskia Ingen-Housz-Oro, Sarah Faiz, Laurent Mortier, Gabor Dobos, Martine Bagot, Matthieu Resche-Rigon, Claire Montlahuc, Arnaud Serret-Larmande, Adèle de Masson
Background: Sézary syndrome is an extremely rare and fatal cutaneous T-cell lymphoma (CTCL). Mogamulizumab, an anti-CCR4 monoclonal antibody, has recently been associated with increased progression-free survival in a randomized clinical trial in CTCL. We aimed to evaluate OS and prognostic factors in Sézary syndrome, including treatment with mogamulizumab, in a real-life setting.
Methods: Data from patients with Sézary (ISCL/EORTC stage IV) and pre-Sézary (stage IIIB) syndrome diagnosed from 2000 to 2020 were obtained from 24 centers in Europe. Age, disease stage, plasma lactate dehydrogenases levels, blood eosinophilia at diagnosis, large-cell transformation and treatment received were analyzed in a multivariable Cox proportional hazard ratio model. This study has been registered in ClinicalTrials (SURPASSe01 study: NCT05206045).
Findings: Three hundred and thirty-nine patients were included (58% men, median age at diagnosis of 70 years, Q1-Q3, 61-79): 33 pre-Sézary (9.7% of 339), 296 Sézary syndrome (87.3%), of whom 10 (2.9%) had large-cell transformation. One hundred and ten patients received mogamulizumab. Median follow-up was 58 months (95% confidence interval [CI], 53-68). OS was 46.5% (95% CI, 40.6%-53.3%) at 5 years. Multivariable analysis showed that age ≥ 80 versus <50 (HR: 4.9, 95% CI, 2.1-11.2, p = 0.001), and large-cell transformation (HR: 2.8, 95% CI, 1.6-5.1, p = 0.001) were independent and significant factors associated with reduced OS. Mogamulizumab treatment was significantly associated with decreased mortality (HR: 0.34, 95% CI, 0.15-0.80, p = 0.013).
Interpretation: Treatment with mogamulizumab was significantly and independently associated with decreased mortality in Sézary syndrome.
Funding: French Society of Dermatology, Swiss National Science Foundation (IZLIZ3_200253/1) and SKINTEGRITY.CH collaborative research program.
{"title":"Real-life efficacy of immunotherapy for Sézary syndrome: a multicenter observational cohort study.","authors":"Alizée Bozonnat, Marie Beylot-Barry, Olivier Dereure, Michel D'Incan, Gaëlle Quereux, Emmanuella Guenova, Marie Perier-Muzet, Stephane Dalle, Florent Grange, Manuelle-Anne Viguier, Caroline Ram-Wolff, Laurence Feldmeyer, Helmut Beltraminelli, Nathalie Bonnet, Florent Amatore, Eve Maubec, Nathalie Franck, Laurent Machet, François Chasset, Florence Brunet-Possenti, Jean-David Bouaziz, Maxime Battistella, Marie Donzel, Anne Pham-Ledard, Claudia Bejar, Hélène Moins-Teisserenc, Samia Mourah, Philippe Saiag, Ewa Hainaut, Catherine Michel, Guido Bens, Henri Adamski, François Aubin, Serge Boulinguez, Pascal Joly, Billal Tedbirt, Isabelle Templier, Laura Troin, Henri Montaudié, Saskia Ingen-Housz-Oro, Sarah Faiz, Laurent Mortier, Gabor Dobos, Martine Bagot, Matthieu Resche-Rigon, Claire Montlahuc, Arnaud Serret-Larmande, Adèle de Masson","doi":"10.1016/j.eclinm.2024.102679","DOIUrl":"10.1016/j.eclinm.2024.102679","url":null,"abstract":"<p><strong>Background: </strong>Sézary syndrome is an extremely rare and fatal cutaneous T-cell lymphoma (CTCL). Mogamulizumab, an anti-CCR4 monoclonal antibody, has recently been associated with increased progression-free survival in a randomized clinical trial in CTCL. We aimed to evaluate OS and prognostic factors in Sézary syndrome, including treatment with mogamulizumab, in a real-life setting.</p><p><strong>Methods: </strong>Data from patients with Sézary (ISCL/EORTC stage IV) and pre-Sézary (stage IIIB) syndrome diagnosed from 2000 to 2020 were obtained from 24 centers in Europe. Age, disease stage, plasma lactate dehydrogenases levels, blood eosinophilia at diagnosis, large-cell transformation and treatment received were analyzed in a multivariable Cox proportional hazard ratio model. This study has been registered in ClinicalTrials (SURPASSe01 study: NCT05206045).</p><p><strong>Findings: </strong>Three hundred and thirty-nine patients were included (58% men, median age at diagnosis of 70 years, Q1-Q3, 61-79): 33 pre-Sézary (9.7% of 339), 296 Sézary syndrome (87.3%), of whom 10 (2.9%) had large-cell transformation. One hundred and ten patients received mogamulizumab. Median follow-up was 58 months (95% confidence interval [CI], 53-68). OS was 46.5% (95% CI, 40.6%-53.3%) at 5 years. Multivariable analysis showed that age ≥ 80 versus <50 (HR: 4.9, 95% CI, 2.1-11.2, p = 0.001), and large-cell transformation (HR: 2.8, 95% CI, 1.6-5.1, p = 0.001) were independent and significant factors associated with reduced OS. Mogamulizumab treatment was significantly associated with decreased mortality (HR: 0.34, 95% CI, 0.15-0.80, p = 0.013).</p><p><strong>Interpretation: </strong>Treatment with mogamulizumab was significantly and independently associated with decreased mortality in Sézary syndrome.</p><p><strong>Funding: </strong>French Society of Dermatology, Swiss National Science Foundation (IZLIZ3_200253/1) and SKINTEGRITY.CH collaborative research program.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11245986/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141616149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-20eCollection Date: 2024-07-01DOI: 10.1016/j.eclinm.2024.102690
Xianming Zhu, Eshan U Patel, Stephen A Berry, Mary K Grabowski, Alison G Abraham, Thibaut Davy-Mendez, Brenna Hogan, Keri N Althoff, Andrew D Redd, Oliver Laeyendecker, Thomas C Quinn, Kelly A Gebo, Aaron A R Tobian
Background: Thirty-day hospital readmission measures quality of care, but there are limited data among people with HIV (PWH) and people without HIV (PWoH) in the era of universal recommendation for antiretroviral therapy. We descriptively compared 30-day all-cause, unplanned readmission risk between PWH and PWoH.
Methods: A retrospective cohort study was conducted using the 2019 Nationwide Readmissions Database (2019/01/01-2019/12/31), an all-payer database that represents all US hospitalizations. Index (initial) admissions and readmissions were determined using US Centers for Medicare & Medicaid Services definitions. Crude and age-adjusted risk ratios (aRR) comparing the 30-day all-cause, unplanned readmission risk between PWH to PWoH were estimated using random effect logistic regressions and predicted marginal estimates. Survey weights were applied to all analyses.
Findings: We included 24,338,782 index admissions from 18,240,176 individuals. The median age was 52(IQR = 40-60) years for PWH and 61(IQR = 38-74) years for PWoH. The readmission risk was 20.9% for PWH and 12.2% for PWoH (age-adjusted-RR:1.88 [95%CI = 1.84-1.92]). Stratified by age and sex, young female (age 18-29 and 30-39 years) PWH had a higher readmission risk than young female PWoH (aRR = 3.50 [95%CI = 3.11-3.88] and aRR = 4.00 [95%CI = 3.67-4.32], respectively). While the readmission risk increased with age among PWoH, the readmission risk was persistently high across all age groups among PWH. The readmission risk exceeded 30% for PWH admitted for hypertensive heart disease, heart failure, and chronic kidney disease.
Interpretation: PWH have a disproportionately higher risk of readmission than PWoH, which is concerning given the aging profile of PWH. More efforts are needed to address readmissions among PWH.
{"title":"Hospital readmissions among adults living with and without HIV in the US: findings from the Nationwide Readmissions Database.","authors":"Xianming Zhu, Eshan U Patel, Stephen A Berry, Mary K Grabowski, Alison G Abraham, Thibaut Davy-Mendez, Brenna Hogan, Keri N Althoff, Andrew D Redd, Oliver Laeyendecker, Thomas C Quinn, Kelly A Gebo, Aaron A R Tobian","doi":"10.1016/j.eclinm.2024.102690","DOIUrl":"10.1016/j.eclinm.2024.102690","url":null,"abstract":"<p><strong>Background: </strong>Thirty-day hospital readmission measures quality of care, but there are limited data among people with HIV (PWH) and people without HIV (PWoH) in the era of universal recommendation for antiretroviral therapy. We descriptively compared 30-day all-cause, unplanned readmission risk between PWH and PWoH.</p><p><strong>Methods: </strong>A retrospective cohort study was conducted using the 2019 Nationwide Readmissions Database (2019/01/01-2019/12/31), an all-payer database that represents all US hospitalizations. Index (initial) admissions and readmissions were determined using US Centers for Medicare & Medicaid Services definitions. Crude and age-adjusted risk ratios (aRR) comparing the 30-day all-cause, unplanned readmission risk between PWH to PWoH were estimated using random effect logistic regressions and predicted marginal estimates. Survey weights were applied to all analyses.</p><p><strong>Findings: </strong>We included 24,338,782 index admissions from 18,240,176 individuals. The median age was 52(IQR = 40-60) years for PWH and 61(IQR = 38-74) years for PWoH. The readmission risk was 20.9% for PWH and 12.2% for PWoH (age-adjusted-RR:1.88 [95%CI = 1.84-1.92]). Stratified by age and sex, young female (age 18-29 and 30-39 years) PWH had a higher readmission risk than young female PWoH (aRR = 3.50 [95%CI = 3.11-3.88] and aRR = 4.00 [95%CI = 3.67-4.32], respectively). While the readmission risk increased with age among PWoH, the readmission risk was persistently high across all age groups among PWH. The readmission risk exceeded 30% for PWH admitted for hypertensive heart disease, heart failure, and chronic kidney disease.</p><p><strong>Interpretation: </strong>PWH have a disproportionately higher risk of readmission than PWoH, which is concerning given the aging profile of PWH. More efforts are needed to address readmissions among PWH.</p><p><strong>Funding: </strong>US National Institutes of Health.</p>","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11246008/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141616093","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-06-20eCollection Date: 2024-06-01DOI: 10.1016/j.eclinm.2024.102705
eClinicalMedicine
{"title":"The potential role of Artificial Intelligence in transforming childhood cancer care.","authors":"eClinicalMedicine","doi":"10.1016/j.eclinm.2024.102705","DOIUrl":"https://doi.org/10.1016/j.eclinm.2024.102705","url":null,"abstract":"","PeriodicalId":11393,"journal":{"name":"EClinicalMedicine","volume":null,"pages":null},"PeriodicalIF":9.6,"publicationDate":"2024-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11247146/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141619529","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}