Zelal Guel Dilbaz, Sophy Denker, Carla Ankermann, Joerg-Thomas Bittenbring, Dominic Kaddu-Mulindwa, Ameya S. Kunte, Sascha Hünecke, Viola Poeschel, Stephan Stilgenbauer, Lorenz Thurner, Il-Kang Na, Moritz Bewarder, Konstantinos Christofyllakis
{"title":"Comparison of R-CHOP-14 and R-mini-CHOP in older adults with diffuse large B-cell lymphoma—A retrospective multicenter cohort study","authors":"Zelal Guel Dilbaz, Sophy Denker, Carla Ankermann, Joerg-Thomas Bittenbring, Dominic Kaddu-Mulindwa, Ameya S. Kunte, Sascha Hünecke, Viola Poeschel, Stephan Stilgenbauer, Lorenz Thurner, Il-Kang Na, Moritz Bewarder, Konstantinos Christofyllakis","doi":"10.1111/ejh.14268","DOIUrl":"10.1111/ejh.14268","url":null,"abstract":"<p>Diffuse large B-cell lymphoma (DLBCL) is the most common lymphoma entity, and its incidence increases with age. There is a paucity of data regarding use of biweekly R-CHOP (R-CHOP-14) in patients ≥80 years of age. We performed a retrospective cohort study of patients with DLBCL aged ≥80 years treated with R-CHOP-14 and R-miniCHOP in two academic tertiary centers in Germany between 01/01/2005 and 12/30/2019. Overall, 79 patients were included. Median age was 84 years (range 80–91). Despite higher CR rates with R-CHOP-14 (71.4% vs. 52.4%), no statistically significant difference could be found between patients treated with R-CHOP-14 and R-miniCHOP regarding overall survival (OS) (<i>p</i> = .88, HR 0.94, 95% CI = 0.47–1.90) and progression-free survival (PFS) (<i>p</i> = .26, HR 0.66, 95% CI = 0.32–1.36). At a median follow-up of 40 months, the 2-year OS rates were 56% with R-CHOP-14 and 53% with R-miniCHOP. Two-year PFS rates were 46% for R-CHOP-14 and 50% for R-mini-CHOP. Relative dose intensity of chemotherapy did not correlate with OS (<i>p</i> = .72). With the caveat of a retrospective cohort study, we conclude that lacking a difference in OS, R-miniCHOP should be preferred for most patients with untreated DLBCL aged ≥80 years.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"675-684"},"PeriodicalIF":2.3,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14268","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859424","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Retroperitoneal and lower extremities muscle bleeding in acquired haemophilia A (AHA): Risk factors and implications in disability and survival","authors":"José Pardos-Gea, Olga Benítez","doi":"10.1111/ejh.14279","DOIUrl":"10.1111/ejh.14279","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To assess risk factors of retroperitoneal and lower extremity musculoskeletal bleed in acquired haemophilia (AHA) and perform an objective assessment of disability and influence on survival.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We included 49 patients with AHA from November 2017 to May 2023. The occurrence of any retroperitoneal or/and lower extremities bleeding manifestation was investigated. On clinical follow-up, we search for compressive femoral neuropathy and quadriceps amyotrophy. The lower extremity functional scale (LEFS) was carried out one year after the last bleeding event in all AHA patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A 61.2% of patients in our AHA cohort presented with any retroperitoneal and/or lower extremities musculoskeletal manifestation. Those patients had higher percentage of major bleeding EACH2/ISTH criteria (90% vs. 57%, <i>p</i> = .01), needs of blood transfusions (86% vs. 57% of patients, <i>p</i> = .03), and haemostatic by-pass products (90% vs. 63%, <i>p</i> = .02). Hypertension (HR 2.6, 95% CI 1.1–5.9, <i>p</i> = .02), presence of autoimmune disease (HR 13, 95% CI 1.7–99, <i>p</i> = .01), and inhibitor level > 20 BU (HR 2.6 95% CI 1.0–6.8, <i>p</i> = .04) significantly predicted retroperitoneal/lower extremities clinical manifestations. Most frequent sequelae were quad atrophy (30.6%) and femoral nerve palsy (20.4%). Quad atrophy and LEFS scores under 50 were associated with increased mortality (HR 3, 95% CI 1.1–8.6 and HR 12, 95% CI 3.3–45, respectively).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>AHA with retroperitoneal/lower extremities bleeding involvement is of greater severity and shows high disability and worst survival outcomes. Quadriceps atrophy and LEFS scale scoring under 50 predicted mortality in our AHA patients.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"685-692"},"PeriodicalIF":2.3,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859425","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Antiemetic prophylaxis regimens in haematologic malignancies patients undergoing a hematopoietic stem cell transplantation. Which is the best standard of care? A systematic review","authors":"Nerea Báez-Gutiérrez, Paloma Suárez-Casillas, Maria Antonia Pérez-Moreno, Cristina Blázquez-Goñi, Laila Abdelkader-Martín","doi":"10.1111/ejh.14282","DOIUrl":"10.1111/ejh.14282","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>This systematic review, adhering to PRISMA guidelines, aimed to evaluate the efficacy and safety of antiemetic prophylaxis in haematological patients undergoing high-dose chemotherapy as part of their hematopoietic stem cell transplantation (HSCT) conditioning regimens.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We performed a comprehensive search in PubMed, EMBASE, ClinicalTrials.gov and the Cochrane database to identify randomised controlled trials (RCTs) and systematic reviews of antiemetic prophylaxis. Studies in English, French, Italian or Spanish were included. This review is registered with PROSPERO, ID CRD42023406380.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Eight RCTs were analysed. The antiemetic regimens evaluated ranged from monotherapy with 5-Hydroxytryptamine Receptor 3 antagonists (5-HT3RAs) to complex combinations including olanzapine, neurokinin-1 receptor antagonists, 5-HT3RAs and corticosteroids. Complete response rates for triplet or quadruple regimens varied between 23.5% and 81.9%. Although no significant adverse effects were observed, minor symptoms such as diarrhoea, constipation, sedation and headaches were reported.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Existing evidence on HSCT antiemetic therapy highlights its benefits but fails to provide clear clinical directions. The choice between triplet and quadruplet therapies for different patient scenarios is still uncertain. Until more detailed research is available, healthcare providers must rely on the latest guidelines and their judgement to customise antiemetic care for each patient's specific needs and risks.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"564-575"},"PeriodicalIF":2.3,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14282","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141792270","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Juha Ranti, Hanna Pudas, Martta Ranta, Samuli Tuominen, Kristiina Uusi-Rauva, Johanna Lammela
{"title":"Treatment and clinical outcomes of patients with acute myeloid leukemia in Finland 2010–2020: A retrospective analysis of electronic health records","authors":"Juha Ranti, Hanna Pudas, Martta Ranta, Samuli Tuominen, Kristiina Uusi-Rauva, Johanna Lammela","doi":"10.1111/ejh.14272","DOIUrl":"10.1111/ejh.14272","url":null,"abstract":"<p>Our retrospective study (2010–2020) examined treatment patterns, outcomes, and healthcare resource utilization in Finnish acute myeloid leukemia (AML) patients. Data covered 153 patients diagnosed at Hospital District of Southwest Finland (HDSF) and 107 from other districts who underwent allogeneic stem cell transplantation (aSCT) at HDSF. Of the 153 patients, 56.2% received intensive chemotherapy (IC), while 43.8% deemed ineligible for IC received low-intensity therapies or best supportive care (BSC). Median overall survival for IC patients was 31.2 months, compared to 5.3 months for those under azacytidine and 1.2 months on BSC. Majority (57.5%) of patients over 60 with intermediate/high European leukemia network risk had poor outcomes with IC and couldn't proceed to aSCT. These patients carried the highest costs and hospital resource use per patient month. Most common reasons for transplant ineligibility after IC were refractory disease and infection. Our data provides a comprehensive view on AML treatment landscape from a period when the latest treatment advancements were not yet accessible. The data describes patient groups with poor prognosis and increased healthcare burden, emphasizing the need to improve treatment practices and identify better ways to get more patients to transplant, in a rapidly evolving treatment landscape.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"664-674"},"PeriodicalIF":2.3,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14272","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141787656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Posttransplantation cyclophosphamide mediates effective reconstitution of memory B cells after allogeneic hematopoietic cell transplantation","authors":"Tetsuya Hayashi, Yasuhiro Nakashima, Shinichi Takeda, Mitsutaka Nishimoto, Hiroshi Okamura, Teruhito Takakuwa, Masatomo Kuno, Yosuke Makuuchi, Kentaro Ido, Kazuki Sakatoku, Mirei Horiuchi, Hideo Koh, Mika Nakamae, Masayuki Hino, Hirohisa Nakamae","doi":"10.1111/ejh.14280","DOIUrl":"10.1111/ejh.14280","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>Impaired B-cell reconstitution after allogeneic hematopoietic cell transplantation (allo-HCT) contributes to the pathogenesis of chronic graft-versus-host disease (cGVHD). Therefore, methods to consistently achieve effective B cell lymphogenesis are required. We assessed the long-term effects of posttransplantation cyclophosphamide (PTCy) use on immune reconstitution in clinical settings, an emerging strategy to suppress allogeneic immunological inflammation early after allo-HCT and prevent subsequent GVHD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We comprehensively analyzed peripheral immune cell subsets and measured serum immunoglobulin G (IgG) or cytokine levels in 39 patients who survived for >1 year after allo-HCT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The absolute counts of B1 and IgM memory B cells were significantly lower in patients with severe cGVHD than in those without. The absolute count and percentage (among total CD19+ B cells) of switched memory B cells and serum IgG levels were significantly higher in patients transplanted with PTCy than in those transplanted with conventional GVHD prophylaxis. Interestingly, increased percentages of switched memory B cells and serum IgG levels were observed only in patients transplanted with PTCy and not in those transplanted with umbilical cord blood.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>PTCy administration can mediate favorable memory B-cell reconstitution long after allo-HCT and may therefore suppress cGVHD.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"651-663"},"PeriodicalIF":2.3,"publicationDate":"2024-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141787655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rasmus Rask Kragh Jørgensen, Lasse Hjort Jakobsen, Sandra Eloranta, Karin E. Smedby, Robert Schou Pedersen, Judit M. Jørgensen, Michael Roost Clausen, Peter Brown, Anne Ortved Gang, Inger-Lise Gade, Thomas Stauffer Larsen, Mats Jerkeman, Tarec Christoffer El-Galaly
{"title":"Effectiveness of R-CHOP versus R-CHOEP for treatment of young patients with high-risk diffuse large B-cell lymphoma: A Danish observational population-based study","authors":"Rasmus Rask Kragh Jørgensen, Lasse Hjort Jakobsen, Sandra Eloranta, Karin E. Smedby, Robert Schou Pedersen, Judit M. Jørgensen, Michael Roost Clausen, Peter Brown, Anne Ortved Gang, Inger-Lise Gade, Thomas Stauffer Larsen, Mats Jerkeman, Tarec Christoffer El-Galaly","doi":"10.1111/ejh.14275","DOIUrl":"10.1111/ejh.14275","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>Etoposide to standard R-CHOP is used for high-risk diffuse large B-cell lymphoma (DLBCL) in some countries. Due to the lack of randomized trials, a real-world data study using matching methods was used to test the potential effectiveness of R-CHOEP over R-CHOP.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and Methods</h3>\u0000 \u0000 <p>This study included patients from the Danish Lymphoma Register diagnosed between 2006 and 2020 at the age of 18–60 years with de novo DLBCL and age-adjusted IPI ≥2. R-CHOEP treated patients were matched 1:1 without replacement to R-CHOP treated patients using a hybrid exact and genetic matching technique. Primary endpoints were progression-free survival (PFS) and overall survival (OS).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In total, 396 patients were included; 213 received R-CHOEP and 183 received R-CHOP. Unadjusted 5-year PFS and OS for R-CHOEP were 69% (95% Confidence intervals [CI]; 63%–76%) and 79% (CI;73%–85%) versus 62% (CI;55%–70%) and 76% (CI;69%–82%) for R-CHOP (log-rank test, PFS <i>p</i> = .25 and OS <i>p</i> = .31). A total of 127 patients treated with R-CHOEP were matched to 127 patients treated with R-CHOP. Matching-adjusted 5-year PFS and OS were 65% (CI; 57%–74%) and 79% (CI; 72%–84%) for R-CHOEP versus 63% (CI; 55%–73%) and 79% (CI;72%–87%) for R-CHOP (log-rank test, PFS <i>p</i> = .90 and OS <i>p</i> = .63).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The present study did not confirm superiority of R-CHOEP over R-CHOP for young patients with high-risk DLBCL.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"641-650"},"PeriodicalIF":2.3,"publicationDate":"2024-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14275","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141733839","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Mariuxi Alexandra Viteri Malone, David Alfonso Alejos Castillo, Heitor Tavares Santos, Anahat Kaur, Tarek Elrafei, Lewis Steinberg, Abhishek Kumar
{"title":"A systematic review of the literature on localized gastrointestinal tract amyloidosis: Presentation, management and outcomes","authors":"Mariuxi Alexandra Viteri Malone, David Alfonso Alejos Castillo, Heitor Tavares Santos, Anahat Kaur, Tarek Elrafei, Lewis Steinberg, Abhishek Kumar","doi":"10.1111/ejh.14269","DOIUrl":"10.1111/ejh.14269","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>Localized gastrointestinal tract amyloidosis is uncommon and little is known regarding this entity. There is no current standard of care for the management of localized amyloidosis. The objective of this study was to evaluate the characteristics, available treatments, outcomes and surveillance of these patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a systematic review of cases reported in the literature from 1962 to 2021. Patients with gastrointestinal amyloidosis reported in English literature were included in the analysis. We described and summarized the patient's characteristics, treatments, clinical presentations, outcomes and surveillance.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The systematic review of reported clinical cases included 62 patients. In these patients, the most common site of amyloid deposition was the stomach (42%). The median age of diagnosis is 64.4 years old; there is a 2:1 prevalence among males (63%) to females (37%); abdominal pain is the most common type of presentation (41%), although patients could also be asymptomatic. There is a high curative rate (100%) with resection alone. Among patients treated with a type of systemic therapy, 80% achieved a complete response. The minority of cases reported a type of surveillance post treatment, and among those 62% pursued serial clinical evaluations alone.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>To our knowledge, this is the first and largest systematic review of the literature in gastrointestinal tract amyloidosis. This is more common among males and seems to have an excellent curative rate (100%) with surgery alone. Systemic therapy is an option for those with non-resectable amyloidomas. Serial clinical evaluations should be part of the standard surveillance care in these patients.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 4","pages":"400-415"},"PeriodicalIF":2.3,"publicationDate":"2024-07-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14269","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141731084","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jerzy Windyga, Shashikant Apte, Melissa Frei-Jones, Teruhisa Fujii, Chuhl Joo Lyu, Laura Villarreal Martinez, Jameela Sathar, Oleksandra Stasyshyn, Huyen Tran, Nadezhda Zozulya, Renée Brown Frandsen, Jesper Skov Neergaard, Jay Jay Thaung Zaw, Johnny Mahlangu
{"title":"Disease and treatment burden of patients with haemophilia entering the explorer6 non-interventional study","authors":"Jerzy Windyga, Shashikant Apte, Melissa Frei-Jones, Teruhisa Fujii, Chuhl Joo Lyu, Laura Villarreal Martinez, Jameela Sathar, Oleksandra Stasyshyn, Huyen Tran, Nadezhda Zozulya, Renée Brown Frandsen, Jesper Skov Neergaard, Jay Jay Thaung Zaw, Johnny Mahlangu","doi":"10.1111/ejh.14277","DOIUrl":"10.1111/ejh.14277","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>We aimed to characterise baseline disease and treatment burden in a large population with haemophilia A/B, both with (HAwI/HBwI) and without (HA/HB) inhibitors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The prospective, non-interventional explorer6 study included patients ≥12 years old with severe HA, severe/moderate HB or HAwI/HBwI of any severity, treated according to local standard of care (excluding previous/current exposure to concizumab or emicizumab). Baseline characteristics and historical clinical data were collected and patient-reported outcomes, including treatment burden, were assessed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The explorer6 study enrolled 231 patients with haemophilia (84 HAwI/HBwI) from 33 countries. At baseline, patients with HA/HB treated with prophylaxis had the lowest median annualised bleeding rates (ABRs; 2.0), irrespective of haemophilia type; of these patients, 27.5% (HA) and 31.4% (HB) had target joints. Patients with HAwI/HBwI treated episodically reported the highest treatment burden. Of these patients, 28.5% (HAwI) and 25.1% (HBwI) performed sports activities in the month before screening.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Despite receiving routine clinical care, historical and baseline information from patients enrolled in explorer6 showed that patients with HA/HB treated episodically and patients with HAwI/HBwI had higher ABRs, higher treatment burden and participated in sports less than those with HA/HB treated with prophylaxis. Emerging treatments could be beneficial in addressing these unmet medical needs.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"631-640"},"PeriodicalIF":2.3,"publicationDate":"2024-07-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14277","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141731085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Fabian Acker, Jörg Chromik, Emily Tiedjen, Sebastian Wolf, Jonas B. Vischedyk, Philipp Makowka, Julius C. Enßle, Khouloud Kouidri, Martin Sebastian, Björn Steffen, Thomas Oellerich, Hubert Serve, Andreas Neubauer, Jonas A. Schäfer, Jörg T. Bittenbring
{"title":"Real-world effectiveness of first-line azacitidine or decitabine with or without venetoclax in acute myeloid leukemia patients unfit for intensive therapy","authors":"Fabian Acker, Jörg Chromik, Emily Tiedjen, Sebastian Wolf, Jonas B. Vischedyk, Philipp Makowka, Julius C. Enßle, Khouloud Kouidri, Martin Sebastian, Björn Steffen, Thomas Oellerich, Hubert Serve, Andreas Neubauer, Jonas A. Schäfer, Jörg T. Bittenbring","doi":"10.1111/ejh.14278","DOIUrl":"10.1111/ejh.14278","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>First-line treatment in patients with acute myeloid leukemia (AML) unfit for intensive therapy is the combination of a hypomethylating agent (HMA) with venetoclax (VEN). However, retrospective data confirming the benefits of this regimen outside of clinical trials have shown conflicting results.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We performed a multicenter retrospective analysis of outcomes with first-line HMA–VEN versus HMA in AML patients unfit for intensive chemotherapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 213 patients were included from three German hospitals (125 HMA–VEN, 88 HMA). Median overall survival in the HMA–VEN cohort was 7.9 months (95% confidence interval [CI], 5.1–14.7) versus 4.9 months (3.1–7.1) with HMA. After 1 year, 42% (95% CI, 33–54) and 19% (12–30) of patients were alive, respectively (hazard ratio [HR] for death, 0.64; 95% CI, 0.46–0.88). After adjusting for clinical and molecular baseline characteristics, treatment with HMA–VEN remained significantly associated with both prolonged survival (HR, 0.48; 95% CI, 0.29–0.77) and time to next treatment (HR, 0.63; 95% CI, 0.47–0.85). Patients who achieved recovery of peripheral blood counts had a favorable prognosis (HR for death, 0.52; 95% CI, 0.33–0.84).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion</h3>\u0000 \u0000 <p>These data align with findings from the pivotal VIALE-A trial and support the use of HMA–VEN in patients unfit for intensive therapy.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"623-630"},"PeriodicalIF":2.3,"publicationDate":"2024-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14278","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141633026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Camille Kockerols, Peter J. M. Valk, Jeroen J. W. M. Janssen, Pauline Hogenbirk, Jan J. Cornelissen, Susanne Saussele, Birgit Spiess, Maria Agustina Perusini, Dennis Kim, Peter E. Westerweel
{"title":"Prediction of sustained remission after tyrosine kinase inhibitor discontinuation with BCR::ABL1 digital PCR in chronic myeloid leukemia patients","authors":"Camille Kockerols, Peter J. M. Valk, Jeroen J. W. M. Janssen, Pauline Hogenbirk, Jan J. Cornelissen, Susanne Saussele, Birgit Spiess, Maria Agustina Perusini, Dennis Kim, Peter E. Westerweel","doi":"10.1111/ejh.14271","DOIUrl":"10.1111/ejh.14271","url":null,"abstract":"<p>Precise and reliable predictive parameters to accurately identify chronic myeloid leukemia (CML) patients who can successfully discontinue their tyrosine kinase inhibitor (TKI) treatment are lacking. One promising parameter is depth of molecular response measured by <i>BCR::ABL1</i> digital PCR (dPCR). The aim of this study was to validate a previously described prediction cutoff of 0.0023%<sup>IS</sup> and to assess the value of dPCR for treatment-free remission (TFR) prediction in relation to other clinical parameters. A droplet-based dPCR assay assessed <i>BCR::ABL1</i> %<sup>IS</sup> prior to TKI discontinuation. The primary endpoint was molecular recurrence (MolR) by 36 months. A total of 186 patients from Canada, Germany, and the Netherlands were included. In patients with a first TKI discontinuation attempt (<i>n</i> = 163), a <i>BCR::ABL1</i> dPCR < and ≥0.0023%<sup>IS</sup> had a MolR probability of 33% and 70%, respectively. Patients treated less than 6 years with a <i>BCR::ABL1</i> dPCR <0.0023%<sup>IS</sup> had a MolR probability of 31%. After correction for treatment duration, both high dPCR value and the use of imatinib (vs. second-generation TKI) were significantly associated with a higher risk of MolR (HR of 3.66, 95%CI 2.06–6.51, <i>p</i> < .001; and 2.85, 95%CI 1.25–6.46, <i>p</i> = .013, respectively). <i>BCR::ABL1</i> dPCR was not associated with TFR outcome after second TKI discontinuation, however, with the limitation of a small number of patients analyzed (<i>n</i> = 23). In conclusion, <i>BCR::ABL1</i> digital PCR based on the cutoff of 0.0023%<sup>IS</sup> is a valuable predictive tool to identify CML patients with a high probability of TFR success after first TKI discontinuation, including patients treated for less than 6 years.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 5","pages":"606-613"},"PeriodicalIF":2.3,"publicationDate":"2024-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14271","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141590004","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}