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Efficacy and Prognostic Indicators of Isatuximab, Pomalidomide, and Dexamethasone (IsaPd) in Daratumumab-Refractory Multiple Myeloma Patients: A Multicenter Real-World Study
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-02-03 DOI: 10.1002/hon.70042
Enrica Antonia Martino, Daniele Derudas, Elena Rossi, Paola Stefanoni, Silvia Mangiacavalli, Elena Zamagni, Massimo Offidani, Anna Furlan, Angela Maria Quinto, Roberta Della Pepa, Giuseppe Bertuglia, Emiliano Barbieri, Concetta Conticello, Claudio De Magistris, Velia Bongarzoni, Anna Maria Cafro, Anna Mele, Cirino Botta, Nicola Sgherza, Giuseppe Mele, Ombretta Annibali, Angela Rago, Raffaele Fontana, Ernesto Vigna, Antonella Bruzzese, Katia Mancuso, Angela Amendola, Annalisa Citro, Emilia Cotzia, Sonia Morè, Elena Rivolti, Loredana Pettine, Monica Galli, Valerio De Stefano, Maria Teresa Petrucci, Alessandro Corso, Antonino Neri, Francesco Di Raimondo, Niccolò Bolli, Pellegrino Musto, Fortunato Morabito, Massimo Gentile
{"title":"Efficacy and Prognostic Indicators of Isatuximab, Pomalidomide, and Dexamethasone (IsaPd) in Daratumumab-Refractory Multiple Myeloma Patients: A Multicenter Real-World Study","authors":"Enrica Antonia Martino,&nbsp;Daniele Derudas,&nbsp;Elena Rossi,&nbsp;Paola Stefanoni,&nbsp;Silvia Mangiacavalli,&nbsp;Elena Zamagni,&nbsp;Massimo Offidani,&nbsp;Anna Furlan,&nbsp;Angela Maria Quinto,&nbsp;Roberta Della Pepa,&nbsp;Giuseppe Bertuglia,&nbsp;Emiliano Barbieri,&nbsp;Concetta Conticello,&nbsp;Claudio De Magistris,&nbsp;Velia Bongarzoni,&nbsp;Anna Maria Cafro,&nbsp;Anna Mele,&nbsp;Cirino Botta,&nbsp;Nicola Sgherza,&nbsp;Giuseppe Mele,&nbsp;Ombretta Annibali,&nbsp;Angela Rago,&nbsp;Raffaele Fontana,&nbsp;Ernesto Vigna,&nbsp;Antonella Bruzzese,&nbsp;Katia Mancuso,&nbsp;Angela Amendola,&nbsp;Annalisa Citro,&nbsp;Emilia Cotzia,&nbsp;Sonia Morè,&nbsp;Elena Rivolti,&nbsp;Loredana Pettine,&nbsp;Monica Galli,&nbsp;Valerio De Stefano,&nbsp;Maria Teresa Petrucci,&nbsp;Alessandro Corso,&nbsp;Antonino Neri,&nbsp;Francesco Di Raimondo,&nbsp;Niccolò Bolli,&nbsp;Pellegrino Musto,&nbsp;Fortunato Morabito,&nbsp;Massimo Gentile","doi":"10.1002/hon.70042","DOIUrl":"10.1002/hon.70042","url":null,"abstract":"<p>This multicenter real-world analysis evaluated the efficacy of isatuximab, pomalidomide, and dexamethasone (IsaPd) in 51 patients with multiple myeloma (MM) who were refractory to daratumumab (Dara-R). The majority were under 70 years old (60.8%), predominantly female (56.9%), and heavily pretreated, with 74.5% being triple-class refractory (TCR); 32.1% of the 28 patients with cytogenetic data had high-risk abnormalities. The overall response rate (ORR) was 56.9%, including 3 patients with stringent complete response (sCR), 4 with CR, and 7 with very good partial response (VGPR). Neither age, number of prior therapies, TCR status, nor time from Dara refractoriness to IsaPd initiation significantly affected response rates.</p><p>Median progression-free survival (PFS) was 5.8 months, with a 12-month PFS probability of 30.6%. Baseline hemoglobin (Hb) levels were a key predictor of PFS: patients with Hb &lt; 11.8 g/L had a 3.5-fold increased risk of progression, with a median PFS of 4.6 months compared to 22 months in those with higher Hb.</p><p>Median overall survival (OS) was 21.0 months, with a 12-month OS probability of 63.4%. Lower Hb levels (&lt; 11 g/L) were associated with a tenfold increased risk of mortality.</p><p>Among the 28 patients who underwent FISH analysis, while no significant difference in mortality risk was observed, those with high-risk cytogenetic abnormalities exhibited a nearly tenfold increased risk of disease progression.</p><p>These results suggest that IsaPd offers a meaningful option for Dara-R patients, with Hb levels serving as a critical predictor of both PFS and OS. However, PFS remains modest, underscoring the need for novel combination therapies.</p>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 2","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/hon.70042","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079460","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparative Safety of Ibrutinib Versus Zanubrutinib in Patients With Chronic Lymphocytic Leukemia: A Prospective Cohort Study
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-29 DOI: 10.1002/hon.70041
Fuli Fan, Xiaodan Liu, Zhan Su, Saisai Li, Chuanlei Wang, Shibo Wang, Shuxia Cui, Yuting Yan
{"title":"Comparative Safety of Ibrutinib Versus Zanubrutinib in Patients With Chronic Lymphocytic Leukemia: A Prospective Cohort Study","authors":"Fuli Fan,&nbsp;Xiaodan Liu,&nbsp;Zhan Su,&nbsp;Saisai Li,&nbsp;Chuanlei Wang,&nbsp;Shibo Wang,&nbsp;Shuxia Cui,&nbsp;Yuting Yan","doi":"10.1002/hon.70041","DOIUrl":"10.1002/hon.70041","url":null,"abstract":"<div>\u0000 \u0000 <p>This study compares the safety profiles of two Bruton's tyrosine kinase (BTK) inhibitors, Ibrutinib and Zanubrutinib, in patients with chronic lymphocytic leukemia (CLL). While Ibrutinib has transformed CLL treatment, it is associated with significant adverse events (AEs). Zanubrutinib, a second-generation BTK inhibitor, offers potential for improved safety. In this prospective study, 200 CLL patients were enrolled, with 100 receiving Ibrutinib and 100 receiving Zanubrutinib. Baseline characteristics such as age, sex, body mass index (BMI), Eastern Cooperative Oncology Group (ECOG) performance status, and genetic factors were evaluated. AEs and serious AEs (SAEs) were tracked and graded using the Common Terminology Criteria for Adverse Events (CTCAE). Multivariate logistic regression models were conducted to determine predictors of SAE and AEs grade ≥ 3. Adjusted odds ratio (aOR) and 95% confidence interval (CI) were reported. The mean ages of the Ibrutinib and Zanubrutinib groups were 49.65 and 49.16 years, respectively (<i>p</i> = 0.285). The Zanubrutinib group had a higher percentage of patients with worse ECOG status (71% vs. 57%, <i>p</i> = 0.039). Fewer Zanubrutinib patients experienced severe AEs (4% vs. 9%, <i>p</i> = 0.152) or SAEs (8% vs. 17%, <i>p</i> = 0.054). Neutropenia occurred only in the Ibrutinib group (3%). Subgroup analysis showed a higher complication rate with Zanubrutinib in non-refractory patients (11.40% vs. 5.26%, <i>p</i> = 0.065). Stage III CLL was a protective factor of grade ≥ 3 AEs (aOR = 0.007; 95% CI: 0.0003–0.1829) and SAE (aOR = 0.015; 95% CI: 0.001–0.177). While ECOS status (2 vs. 3) resulted in reduced risk of SAE, chromosome 17p deletion emerged as the main risk factor of SAE (aOR = 6.40; 95% CI: 1.33–30.79). Zanubrutinib demonstrated a more favorable safety profile than Ibrutinib, with fewer severe adverse events. It may be a safer alternative for CLL patients, particularly those at higher risk for complications from BTK inhibitors. However, these differences stemmed from variability in baseline clinical characteristics rather than the interventions themselves.</p>\u0000 </div>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 2","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143065188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comprehensive Characterization of Overt Myelofibrosis in an Asian Cohort: Phenotype, Mutation Landscape and Discordance Among Scoring Systems
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-24 DOI: 10.1002/hon.70040
Yu-Sung Chang, Yu-Hung Wang, Chao-Hung Wei, Yu-Wen Chen, Hsing-Yu Lin, Chien-Chin Lin, Xavier Cheng-Hong Tsai, Chang-Tsu Yuan, Feng-Ming Tien, Yun-Chu Lin, Sze-Hwei Lee, Yuan-Yeh Kuo, Ming-Kai Chuang, Bor-Sheng Ko, Ming Yao, Hwei-Fang Tien, Wen-Chien Chou, Hsin-An Hou
{"title":"Comprehensive Characterization of Overt Myelofibrosis in an Asian Cohort: Phenotype, Mutation Landscape and Discordance Among Scoring Systems","authors":"Yu-Sung Chang,&nbsp;Yu-Hung Wang,&nbsp;Chao-Hung Wei,&nbsp;Yu-Wen Chen,&nbsp;Hsing-Yu Lin,&nbsp;Chien-Chin Lin,&nbsp;Xavier Cheng-Hong Tsai,&nbsp;Chang-Tsu Yuan,&nbsp;Feng-Ming Tien,&nbsp;Yun-Chu Lin,&nbsp;Sze-Hwei Lee,&nbsp;Yuan-Yeh Kuo,&nbsp;Ming-Kai Chuang,&nbsp;Bor-Sheng Ko,&nbsp;Ming Yao,&nbsp;Hwei-Fang Tien,&nbsp;Wen-Chien Chou,&nbsp;Hsin-An Hou","doi":"10.1002/hon.70040","DOIUrl":"10.1002/hon.70040","url":null,"abstract":"<div>\u0000 \u0000 <p>Various prognostic scoring systems in myelofibrosis (MF) have been developed to guide clinical decision-making in MF. However, discrepancies between different scoring systems for individual patients remain poorly understood, which can result in conflicting treatment recommendations. Moreover, data regarding there applicability in Asian populations remain scarce. We conducted a retrospective analysis of patients with overt MF at National Taiwan University Hospital from November 1, 2006, to November 30, 2023. This study evaluates the distribution, concordance, and prognostic value of various scoring systems (IPSS, DIPSS, DIPSS+, MYSEC-PM, MIPSS70, MIPSS70+v2, GIPSS), focusing on the mutational landscape, particularly <i>TP53</i> mutations, and cytopenic phenotypes in an Asian MF cohort. All prognostic systems effectively stratified patients by risk (<i>p</i> &lt; 0.005); however, concordance between systems was low (Cohen's <i>κ</i> &lt; 0.4, except for IPSS <i>vs</i>. DIPSS+). GIPSS demonstrated superior performance with a comparable C-index but lower Akaike and Bayesian information criterion values. Besides high-molecular risk mutations, <i>TP53</i> mutations (5.2%) were associated with worse overall survival (OS) (5-year: 30% <i>vs</i>. 68%, <i>p</i> &lt; 0.001) and a trend toward higher leukemic transformation (5-year: 22% <i>vs</i>. 10%, <i>p</i> = 0.055). Cytopenic phenotype (59%) was associated with a higher incidence of <i>ASXL1</i> mutations (44% <i>vs</i>. 28%, <i>p</i> = 0.04). In multivariable analysis, elderly patients, higher-risk GIPSS, <i>TP53</i> mutation and cytopenic phenotype were associated with a poorer OS. This study validated multiple prognostic scoring systems in an Asian MF cohort, with GIPSS showing superior risk stratification. Further, cytopenic phenotype and <i>TP53</i> mutations were identified as independent factors linked to poorer survival.</p>\u0000 </div>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033084","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Correction to Elotuzumab Plus Pomalidomide and Dexamethasone in Relapsed/Refractory Multiple Myeloma: Extended Follow-Up of a Multicenter, Retrospective Real-World Experience With 321 Cases Outside of Controlled Clinical Trials
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-23 DOI: 10.1002/hon.70039
{"title":"Correction to Elotuzumab Plus Pomalidomide and Dexamethasone in Relapsed/Refractory Multiple Myeloma: Extended Follow-Up of a Multicenter, Retrospective Real-World Experience With 321 Cases Outside of Controlled Clinical Trials","authors":"","doi":"10.1002/hon.70039","DOIUrl":"10.1002/hon.70039","url":null,"abstract":"<p>Martino EA, Palmieri S, Galli M, et al. Elotuzumab plus pomalidomide and dexamethasone in relapsed/refractory multiple myeloma: Extended follow-up of a multicenter, retrospective real-world experience with 321 cases outside of controlled clinical trials. <i>Hematol Oncol</i>. 2024;42(4):e3290. https://doi.org/10.1002/hon.3290</p><p>In the article, affiliation 42 for author Barbara Gamberi is not correct. The correct affiliation for Dr. Barbara Gamberi is “Hematology Unit, Azienda Unità Sanitaria Locale – IRCCS of Reggio Emilia, Reggio Emilia, Italy”.</p><p>We apologize for this error.</p>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/hon.70039","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143023212","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Impact of Recent Translational and Therapeutic Developments on Clinical Course of BCR::ABL1-Positive and -Negative Myeloproliferative Neoplasms 近期翻译和治疗进展对BCR: abl1阳性和阴性骨髓增生性肿瘤临床病程的影响
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-18 DOI: 10.1002/hon.70013
Tariq I. Mughal, John Mascarenhas, Raajit K. Rampal, Prithviraj Bose, Thomas Lion, Helen Ajufo, Abdulraheem Yacoub, Soheil Meshinchi, Lucia Masarova, Ruben Mesa, Catriona Jamieson, Tiziano Barbui, Giuseppe Saglio, Richard A. Van Etten
{"title":"Impact of Recent Translational and Therapeutic Developments on Clinical Course of BCR::ABL1-Positive and -Negative Myeloproliferative Neoplasms","authors":"Tariq I. Mughal,&nbsp;John Mascarenhas,&nbsp;Raajit K. Rampal,&nbsp;Prithviraj Bose,&nbsp;Thomas Lion,&nbsp;Helen Ajufo,&nbsp;Abdulraheem Yacoub,&nbsp;Soheil Meshinchi,&nbsp;Lucia Masarova,&nbsp;Ruben Mesa,&nbsp;Catriona Jamieson,&nbsp;Tiziano Barbui,&nbsp;Giuseppe Saglio,&nbsp;Richard A. Van Etten","doi":"10.1002/hon.70013","DOIUrl":"10.1002/hon.70013","url":null,"abstract":"<div>\u0000 \u0000 <p>Despite the study of <i>BCR::ABL1</i>-positive and -negative myeloproliferative neoplasms (MPNs) providing seminal insights into cancer biology, tumor evolution and precision oncology over the past half century, significant challenges remain. MPNs are clonal hematopoietic stem cell-derived neoplasms with heterogenous clinical phenotypes and a clonal architecture which impacts the often-complex underlying genetics and microenvironment. The major driving molecular abnormalities have been well characterized, but debate on their role as disease-initiating molecular lesions continues. The introduction of the ABL1 tyrosine kinase inhibitors have been extremely successful in the treatment of chronic myeloid leukemia with most patients having a near-normal life expectancy. Similar success has, however, not been achieved for <i>BCR::ABL1</i>-negative MPNs in terms of disease course modification and most patients remain incurable. In both disease categories, genomic instability seems to increase the risk of disease progression to accelerated/blast phase, which is resistant/refractory to conventional treatment and associated with a poor prognosis. To address some of these issues, the late John Goldman and Tariq Mughal founded a scientific and clinical platform in 2006, the Post-American Society of Hematology (ASH) MPN workshop, to appraise novel cancer biology, candidate therapeutic targets, treatments and other clinical challenges and pay tribute to all the many scientists and clinicians around the world instrumental to the progress made and continuing advances being made. This paper summarizes some of the recent data discussed at the 18<sup>th</sup> edition of the workshop and includes reference to some data presented or published after the workshop, including the 26<sup>th</sup> John Goldman CML conference.</p>\u0000 </div>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143004409","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Successful Pregnancies During Crizotinib Therapy for Anaplastic Lymphoma Kinase Positive Lymphoma 克唑替尼治疗间变性淋巴瘤激酶阳性淋巴瘤期间妊娠成功。
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-14 DOI: 10.1002/hon.70038
Veronica Guglielmana, Francesco Maria Fusi, Valentina Giardini, Federica Cocito, Carlo Gambacorti-Passerini
{"title":"Successful Pregnancies During Crizotinib Therapy for Anaplastic Lymphoma Kinase Positive Lymphoma","authors":"Veronica Guglielmana,&nbsp;Francesco Maria Fusi,&nbsp;Valentina Giardini,&nbsp;Federica Cocito,&nbsp;Carlo Gambacorti-Passerini","doi":"10.1002/hon.70038","DOIUrl":"10.1002/hon.70038","url":null,"abstract":"<p>Anaplastic lymphoma kinase positive (ALK+) anaplastic large cell lymphoma (ALCL) typically affects young individuals and, despite high responsiveness to cytotoxic drugs, relapses occur in over 50% of patients. Crizotinib has improved outcomes, but its management in patients desiring parenthood remains an issue. This study presents the first description of four successful pregnancies during crizotinib treatment for ALK+ALCL: a female patient achieving two pregnancies through assisted reproductive technologies (ART), temporarily discontinuing crizotinib and maintaining a complete remission (CR), and a male patient conceiving naturally while on continuous therapy. These cases demonstrate crizotinib potential to support conception without compromising disease control, even in the absence of specific guidelines on treatment discontinuation.</p>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11732784/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142983299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Acalabrutinib in High-Risk Chronic Lymphocytic Leukaemia Naïve Patients: An Italian Multicenter Retrospective Observational Real-Life Experience 阿卡拉布替尼在高危慢性淋巴细胞白血病Naïve患者中的应用:一项意大利多中心回顾性观察现实生活经验。
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-14 DOI: 10.1002/hon.70033
Idanna Innocenti, Annamaria Tomasso, Diana Giannarelli, Lydia Scarfò, Roberta Murru, Andrea Visentin, Anna Maria Frustaci, Francesca Morelli, Candida Vitale, Antonio Mosca, Alessandro Sanna, Giuliana Farina, Roberta Laureana, Massimo Gentile, Andrea Galitzia, Raffaella Pasquale, Francesco Autore, Jacopo Olivieri, Azzurra Romeo, Marina Deodato, Luca Stirparo, Andrea Corbingi, Vanessa Innao, Francesca Perutelli, Francesca Martini, Paolo Sportoletti, Alberto Fresa, Maria Ilaria Del Principe, Alessandra Tedeschi, Marta Coscia, Paolo Ghia, Luca Laurenti
{"title":"Acalabrutinib in High-Risk Chronic Lymphocytic Leukaemia Naïve Patients: An Italian Multicenter Retrospective Observational Real-Life Experience","authors":"Idanna Innocenti,&nbsp;Annamaria Tomasso,&nbsp;Diana Giannarelli,&nbsp;Lydia Scarfò,&nbsp;Roberta Murru,&nbsp;Andrea Visentin,&nbsp;Anna Maria Frustaci,&nbsp;Francesca Morelli,&nbsp;Candida Vitale,&nbsp;Antonio Mosca,&nbsp;Alessandro Sanna,&nbsp;Giuliana Farina,&nbsp;Roberta Laureana,&nbsp;Massimo Gentile,&nbsp;Andrea Galitzia,&nbsp;Raffaella Pasquale,&nbsp;Francesco Autore,&nbsp;Jacopo Olivieri,&nbsp;Azzurra Romeo,&nbsp;Marina Deodato,&nbsp;Luca Stirparo,&nbsp;Andrea Corbingi,&nbsp;Vanessa Innao,&nbsp;Francesca Perutelli,&nbsp;Francesca Martini,&nbsp;Paolo Sportoletti,&nbsp;Alberto Fresa,&nbsp;Maria Ilaria Del Principe,&nbsp;Alessandra Tedeschi,&nbsp;Marta Coscia,&nbsp;Paolo Ghia,&nbsp;Luca Laurenti","doi":"10.1002/hon.70033","DOIUrl":"10.1002/hon.70033","url":null,"abstract":"&lt;p&gt;In recent years, the treatment of chronic lymphocytic leukaemia (CLL) has changed considerably, in favour of a chmo-free approach with covalent BTK inhibitors (cBTKis), such as ibrutinib, acalabrutinib and zanubrutinib, that have improved therapeutic results even in patients with an unfavourable risk profile [del(17p), &lt;i&gt;TP53&lt;/i&gt;m, unmutated IGHV genes (uIGHV)] [&lt;span&gt;1-4&lt;/span&gt;]. The ‘continuous therapy’ with cBTKis achieves a high response rate, predominantly partial, with an excellent progression free survival (PFS) and overall survival (OS) even in the setting of high-risk (HR) patients [&lt;span&gt;5, 6&lt;/span&gt;]. ESMO Guideline update on new targeted therapies recommends the use of BTKi as first line for patients with a &lt;i&gt;TP53&lt;/i&gt;m and/or del(17p) [&lt;span&gt;7&lt;/span&gt;], because reported PFS rates suggest longer duration of disease control, and for patients with an uIGHV without a &lt;i&gt;TP53&lt;/i&gt;m or del(17p), especially if unfit or elderly. Despite these data, in clinical practice it's still unclear whether there's a difference of efficacy of cBTKis in HR subgroups [del(17p), &lt;i&gt;TP53&lt;/i&gt;m, uIGHV]. Therefore, this nationwide multicentre retrospective study aims to describe efficacy and safety of acalabrutinib in TN patients with HR CLL and to identify which subgroup of HR may benefit most from this treatment. This study was conducted according to the Helsinki Declaration, Good Clinical Practice and the applicable national regulations, and approved by the local Ethical Committee (study number 6390). Its primary endpoint was to evaluate the efficacy of acalabrutinib as first-line treatment in patients with CLL with an unfavourable biological profile [del(17p) and/or &lt;i&gt;TP53&lt;/i&gt;m and/or uIGHV] in terms of overall response rate (ORR). The secondary endpoints were to compare PFS and OS across subgroups of HR CLL treated with frontline acalabrutinib and to assess its tolerability profile. We enrolled 98 TN patients with HR CLL, who started acalabrutinib between June 2021 and June 2023. Response evaluation was done at 12 months since the start of treatment [&lt;span&gt;8&lt;/span&gt;]. Data regarding definitive treatment discontinuation and safety were also collected. We analysed the survival data by stratifying patients into further subgroups according to the HR biological characteristics [del(17p) and/or &lt;i&gt;TP53&lt;/i&gt;m only, with uIGHV only, uIGHV with del(17p) and/or &lt;i&gt;TP53&lt;/i&gt;m]. Two patients were not analyzed due to unavailability of all biological data. Clinical characteristics and biological features at baseline are reported in Table 1. The 10% of patients had del(17p) and/or &lt;i&gt;TP53&lt;/i&gt;m only (group A), 71% uIGHV only (group B) and 19% had uIGHV with del(17p) and/or &lt;i&gt;TP53&lt;/i&gt;m (group C). After a median follow up of treatment of 16 months, the 86% of patients remained on treatment. ORR among all patients at 12 months was 94% as follows: complete remission (CR) and partial remission (PR) rate were 17% and 77%, respectively. ORR at 12 months were 80%, 95% and 93% in","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11732783/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142983333","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
MicroRNA Profiling of Bone Marrow Plasma Extracellular Vesicles in Multiple Myeloma, Extramedullary Disease, and Plasma Cell Leukemia 多发性骨髓瘤、髓外疾病和浆细胞白血病患者骨髓浆细胞外囊泡的MicroRNA谱分析
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-13 DOI: 10.1002/hon.70036
Jana Gregorova, Monika Vlachova, Petra Vychytilova-Faltejskova, Adela Dostalova, Tereza Ruzickova, Marek Vecera, Lenka Radova, Vendula Pospichalova, Stanislava Sladecek, Martina Hyzdalova, Jana Kotaskova, Marie Jarosova, Josef Masek, Klara Benesova, Jiri Jarkovsky, Lucie Rihova, Renata Bezdekova, Martina Almasi, Ivanna Boichuk, Martin Stork, Ludek Pour, Sabina Sevcikova
{"title":"MicroRNA Profiling of Bone Marrow Plasma Extracellular Vesicles in Multiple Myeloma, Extramedullary Disease, and Plasma Cell Leukemia","authors":"Jana Gregorova,&nbsp;Monika Vlachova,&nbsp;Petra Vychytilova-Faltejskova,&nbsp;Adela Dostalova,&nbsp;Tereza Ruzickova,&nbsp;Marek Vecera,&nbsp;Lenka Radova,&nbsp;Vendula Pospichalova,&nbsp;Stanislava Sladecek,&nbsp;Martina Hyzdalova,&nbsp;Jana Kotaskova,&nbsp;Marie Jarosova,&nbsp;Josef Masek,&nbsp;Klara Benesova,&nbsp;Jiri Jarkovsky,&nbsp;Lucie Rihova,&nbsp;Renata Bezdekova,&nbsp;Martina Almasi,&nbsp;Ivanna Boichuk,&nbsp;Martin Stork,&nbsp;Ludek Pour,&nbsp;Sabina Sevcikova","doi":"10.1002/hon.70036","DOIUrl":"10.1002/hon.70036","url":null,"abstract":"<p>Multiple myeloma is a plasma cell malignancy characterized by an abnormal increase in monoclonal immunoglobulins. Despite significant advances in treatment, some patients progress to more aggressive forms of multiple myeloma, including extramedullary disease or plasma cell leukemia. Although the exact molecular mechanisms are not known, several studies have confirmed the involvement of small extracellular vesicle-enriched microRNAs in multiple myeloma progression. Therefore, we performed expression profiling of these molecules in bone marrow plasma of multiple myeloma, extramedullary disease, and plasma cell leukemia patients using small RNA sequencing to identify novel molecules involved in disease pathogenesis. In total, 42 microRNAs were significantly dysregulated among analyzed subgroups. Independent validation by RT-qPCR confirmed elevated levels of miR-140-3p, miR-584-5p, miR-191-5p, and miR-143-3p in multiple myeloma patients compared to extramedullary disease and plasma cell leukemia patients. Subsequent statistical analysis revealed significant correlations between patient clinical characteristics or flow cytometry parameters and microRNA expression. These results indicate that dysregulation of microRNAs could contribute to multiple myeloma progression.</p>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11727818/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970397","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Clinical Impact of Osteolytic Bone Lesions in POEMS Syndrome: A Single-Center Experience of 114 Patients 溶骨性骨病变对POEMS综合征的临床影响:114例患者的单中心经验。
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-11 DOI: 10.1002/hon.70037
Tatsuzo Mishina, Chikako Ohwada, Tomoki Suichi, Shinichiro Matsui, Arata Ishii, Koji Takaishi, Nagisa Oshima-Hasegawa, Shokichi Tsukamoto, Yusuke Takeda, Sonoko Misawa, Naoya Mimura, Satoshi Kuwabara, Chiaki Nakaseko, Emiko Sakaida
{"title":"Clinical Impact of Osteolytic Bone Lesions in POEMS Syndrome: A Single-Center Experience of 114 Patients","authors":"Tatsuzo Mishina,&nbsp;Chikako Ohwada,&nbsp;Tomoki Suichi,&nbsp;Shinichiro Matsui,&nbsp;Arata Ishii,&nbsp;Koji Takaishi,&nbsp;Nagisa Oshima-Hasegawa,&nbsp;Shokichi Tsukamoto,&nbsp;Yusuke Takeda,&nbsp;Sonoko Misawa,&nbsp;Naoya Mimura,&nbsp;Satoshi Kuwabara,&nbsp;Chiaki Nakaseko,&nbsp;Emiko Sakaida","doi":"10.1002/hon.70037","DOIUrl":"10.1002/hon.70037","url":null,"abstract":"<div>\u0000 \u0000 <p>POEMS syndrome is a multisystemic disease associated with monoclonal plasma cell disorders. Although the presence of bone lesions is included in the diagnostic criteria, their precise manifestations remain unknown. Here, we retrospectively analyzed the bone lesions in patients with POEMS syndrome and evaluated their clinical features. Clinical data and bone lesion information by computed tomography (CT) imaging were obtained from the 114 patients with POEMS syndrome. Regardless of the presence of sclerotic bone lesions, patients were divided into two groups according to the presence (lytic group: <i>n</i> = 17, 14.9%) or absence (non-lytic group: <i>n</i> = 97, 85.1%) of osteolytic lesions. In the lytic group, several patients were histologically diagnosed with plasmacytoma. In the evaluation by CT imaging, osteolytic lesions had a higher response rate than sclerotic lesions (75.0% vs. 42.2%, <i>p</i> = 0.079). Nevertheless, patients in lytic group showed earlier clinical progression than patients in non-lytic group (2-year progression-free survival, 66.7% vs. 90.2%, <i>p</i> = 0.069). The presence of innumerable bone lesions was an independent poor prognostic factor in multivariate analysis, regardless of the presence of osteolytic lesions (hazard ratio, 3.4; 95% confidence interval 1.1–10.9; <i>p</i> = 0.040). Osteolytic and innumerable bone lesions are potential prognostic factors. Further studies involving histopathological evaluations of bone lesions are warranted.</p>\u0000 </div>","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142970396","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Role of 24-Month Progression-Free Survival (PFS24) in the Long-Term Evaluation of Patients With Diffuse Large B Cell Lymphoma (DLBCL): A Real-World Single Center Study 24个月无进展生存期(PFS24)在弥漫性大B细胞淋巴瘤(DLBCL)患者长期评估中的作用:一项真实世界单中心研究
IF 3.3 4区 医学
Hematological Oncology Pub Date : 2025-01-09 DOI: 10.1002/hon.70034
Pinelopi Vryttia, Anthi Bouchla, Christina Apostolopoulou, Artemis Zorba, Thomas Thomopoulos, Ioulia Markaki, Periklis G. Foukas, Vasiliki Pappa, Sotirios G. Papageorgiou
{"title":"The Role of 24-Month Progression-Free Survival (PFS24) in the Long-Term Evaluation of Patients With Diffuse Large B Cell Lymphoma (DLBCL): A Real-World Single Center Study","authors":"Pinelopi Vryttia,&nbsp;Anthi Bouchla,&nbsp;Christina Apostolopoulou,&nbsp;Artemis Zorba,&nbsp;Thomas Thomopoulos,&nbsp;Ioulia Markaki,&nbsp;Periklis G. Foukas,&nbsp;Vasiliki Pappa,&nbsp;Sotirios G. Papageorgiou","doi":"10.1002/hon.70034","DOIUrl":"10.1002/hon.70034","url":null,"abstract":"","PeriodicalId":12882,"journal":{"name":"Hematological Oncology","volume":"43 1","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142947672","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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