HaematologicaPub Date : 2024-11-01DOI: 10.3324/haematol.2024.285170
A Vera De Jonge, Wassilis S C Bruins, Carolien Duetz, Charlotte L B M Korst, Rosa Rentenaar, Meliha Cosovic, Merve Eken, Marie-Jose Kersten, Yorick Sandberg, Rozemarijn S Van Rijn, Rob Fijnheer, Pim Mutsaers, Vibeke K J Vergote, Djamila Issa, Aart Beeker, Yavuz M Bilgin, Otto Visser, Erik Van Werkhoven, Margaretha G M Roemer, Martine E D Chamuleau, Tuna Mutis
{"title":"Response to DA-EPOCH-R is associated with activation of 'fitter' cytotoxic T cells in patients with newly diagnosed double and triple hit high-grade B-cell lymphoma.","authors":"A Vera De Jonge, Wassilis S C Bruins, Carolien Duetz, Charlotte L B M Korst, Rosa Rentenaar, Meliha Cosovic, Merve Eken, Marie-Jose Kersten, Yorick Sandberg, Rozemarijn S Van Rijn, Rob Fijnheer, Pim Mutsaers, Vibeke K J Vergote, Djamila Issa, Aart Beeker, Yavuz M Bilgin, Otto Visser, Erik Van Werkhoven, Margaretha G M Roemer, Martine E D Chamuleau, Tuna Mutis","doi":"10.3324/haematol.2024.285170","DOIUrl":"10.3324/haematol.2024.285170","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"3760-3765"},"PeriodicalIF":8.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11532709/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141497899","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}
HaematologicaPub Date : 2024-10-31DOI: 10.3324/haematol.2024.285245
Imran A Nizamuddin, Nancy L Bartlett
{"title":"Bispecific antibodies in follicular lymphoma.","authors":"Imran A Nizamuddin, Nancy L Bartlett","doi":"10.3324/haematol.2024.285245","DOIUrl":"https://doi.org/10.3324/haematol.2024.285245","url":null,"abstract":"<p><p>Bispecific antibodies, specifically anti-CD20 T-cell engaging constructs, are poised to alter the treatment paradigm of multiple B-cell malignancies, including follicular lymphoma. Two CD20xCD3 bispecific antibodies, mosunetuzumab and epcoritamab, are now approved in the United States for third-line or later treatment of follicular lymphoma. A third agent, odronextamab, remains under review by regulatory agencies. In pivotal phase II trials, these bispecific antibodies demonstrated overall response rates of approximately 80%, with complete response rates of 60-70%, the majority of which have been durable at two years. Important safety signals included risk of infections, neutropenia, and cytokine release syndrome, which occurred in approximately half of patients, but was rarely high grade. Despite similar efficacy and toxicity profiles, key differences exist among agents, primarily relating to treatment duration, route of administration, and prophylactic corticosteroid use. Several ongoing studies are exploring bispecific antibodies in earlier lines of treatment, either as single agents or in combination with other active therapies. This novel class of agents is likely to play a pivotal role in improving outcomes for patients with follicular lymphoma.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HaematologicaPub Date : 2024-10-31DOI: 10.3324/haematol.2024.285828
Nilanjan Ghosh, Alison Sehgal, Fei Fei Liu, Ana Kostic, Alessandro Crotta, Marc De Benedetti, Jillian Faccone, Lily Peng, Leo I Gordon
{"title":"Comparative efficacy of lisocabtagene maraleucel in the PILOT study <i>versus</i> second-line chemotherapy regimens in the real world.","authors":"Nilanjan Ghosh, Alison Sehgal, Fei Fei Liu, Ana Kostic, Alessandro Crotta, Marc De Benedetti, Jillian Faccone, Lily Peng, Leo I Gordon","doi":"10.3324/haematol.2024.285828","DOIUrl":"https://doi.org/10.3324/haematol.2024.285828","url":null,"abstract":"<p><p>This study assessed the comparative efficacy of lisocabtagene maraleucel (liso-cel) in PILOT (NCT03483103), an open-label, phase II study, versus conventional second-line (2L) chemotherapy regimens in the real world administered to patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) who were not intended for hematopoietic stem cell transplantation (HSCT). The liso-cel-treated cohort (n=61) was based on patients who received liso-cel in the PILOT study. The conventional chemotherapy cohort included patients who met PILOT eligibility criteria and received conventional 2L chemotherapy in the real-world clinical setting (n=273). After using the trimmed stabilized inverse probability of treatment weighting method to balance cohorts according to baseline characteristics, there were statistically significant differences in all tested measures of efficacy. Compared with real-world conventional chemotherapy regimens, liso-cel demonstrated higher overall response rates (79.6% with liso-cel vs. 50.5% with conventional chemotherapy; relative risk [RR], 1.6; P.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HaematologicaPub Date : 2024-10-31DOI: 10.3324/haematol.2024.286343
Bryan Valcarcel, Sara J Schonfeld, Meredith S Shiels, Jorge J Castillo, Lindsay M Morton
{"title":"Survival outcomes in diffuse large B-cell lymphoma patients with and without HIV in the United States from 2001 to 2016: a population-based analysis.","authors":"Bryan Valcarcel, Sara J Schonfeld, Meredith S Shiels, Jorge J Castillo, Lindsay M Morton","doi":"10.3324/haematol.2024.286343","DOIUrl":"https://doi.org/10.3324/haematol.2024.286343","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HaematologicaPub Date : 2024-10-31DOI: 10.3324/haematol.2024.286350
Mattia Algeri, Michele Massa, Daria Pagliara, Valentina Bertaina, Federica Galaverna, Ilaria Pili, Giuseppina Li Pira, Roberto Carta, Francesco Quagliarella, Rita M Pinto, Chiara Rosignoli, Barbarella Lucarelli, Maria G Cefalo, Emilia Boccieri, Francesca Benini, Francesca Del Bufalo, Marco Becilli, Pietro Merli, Gerhard Zugmaier, Franco Locatelli
{"title":"Outcomes of children and young adults with B-cell acute lymphoblastic leukemia given blinatumomab as last consolidation treatment before allogeneic hematopoietic stem cell transplantation.","authors":"Mattia Algeri, Michele Massa, Daria Pagliara, Valentina Bertaina, Federica Galaverna, Ilaria Pili, Giuseppina Li Pira, Roberto Carta, Francesco Quagliarella, Rita M Pinto, Chiara Rosignoli, Barbarella Lucarelli, Maria G Cefalo, Emilia Boccieri, Francesca Benini, Francesca Del Bufalo, Marco Becilli, Pietro Merli, Gerhard Zugmaier, Franco Locatelli","doi":"10.3324/haematol.2024.286350","DOIUrl":"https://doi.org/10.3324/haematol.2024.286350","url":null,"abstract":"<p><p>Blinatumomab has remarkable efficacy in patients with relapsed/refractory (r/r) or measurable residual disease (MRD)-positive B-cell acute lymphoblastic leukemia (B-ALL). In many patients, blinatumomab treatment is followed by allogeneic hematopoietic stem cell transplantation (HSCT). However, the influence of blinatumomab on HSCT outcomes in children and young adults (YA) remains to be fully elucidated. We conducted a single-center, retrospective analysis on patients given blinatumomab as last treatment before HSCT. Seventy-eight pediatric and YA patients were evaluated. With a median follow-up of 23.23 months, the 2-year disease-free (DFS) and overall survival (OS) probability were 72.2% and 89.2%, respectively, with a 2-year cumulative incidence (CI) of non-relapse mortality (NRM) of 2.6%. A trend toward improved 2-year DFS, but not OS, was noted in patients transplanted in first complete remission (CR1) (92.9%) compared to those in second or greater remission (CR2/3) (68.5%, p=0.18) due to a lower CI of relapse (0% vs. 29.9%, p=0.05). Among CR2/3 patients, those receiving the sequential combination of inotuzumab and blinatumomab had a significantly lower CI of relapse as compared to those who did not receive inotuzumab (9.5% vs. 40.4%, p=0.023). Relapse after HSCT occurred in 16 patients, all exhibiting CD19-positive blasts; 10 of them received anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy and 2 inotuzumab as salvage therapy, leading to a 2-year post-relapse OS of 52.7%. Our results indicate that HSCT following blinatumomab in children and YA with B-ALL is highly effective, being associated with low NRM and not affecting the efficacy of subsequent salvage immunotherapies, including CAR-T cells.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545072","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HaematologicaPub Date : 2024-10-31DOI: 10.3324/haematol.2024.286338
Wellington F Silva, Haesook T Kim, Maria S Undurraga, Juan R Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A M Melo, Ana Beatriz F Gloria, Katia B B Pagnano, Elenaide C Nunes, Rosane Isabel Bittencourt, Ninoska Rojas, Shirley M Q Truyenque, Ana Ilda Ayala-Lugo, Ana Carolina Oliver, Lorena L Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M Fagundes, Bruno K L Duarte, Analí Pamela Mora-Alferez, Percy Ortiz, Jose Luis Untama, Martin S Tallman, Raul C Ribeiro, Arnold Ganser, Richard James Dillon, Peter J M Valk, Miguel A Sanz, Bob Löwenberg, Nancy Berliner, Eduardo M Rego
{"title":"Early death and intracranial hemorrhage prediction in acute promyelocytic leukemia: validation of a risk score in a chemotherapy plus ATRA cohort from an international consortium.","authors":"Wellington F Silva, Haesook T Kim, Maria S Undurraga, Juan R Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A M Melo, Ana Beatriz F Gloria, Katia B B Pagnano, Elenaide C Nunes, Rosane Isabel Bittencourt, Ninoska Rojas, Shirley M Q Truyenque, Ana Ilda Ayala-Lugo, Ana Carolina Oliver, Lorena L Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M Fagundes, Bruno K L Duarte, Analí Pamela Mora-Alferez, Percy Ortiz, Jose Luis Untama, Martin S Tallman, Raul C Ribeiro, Arnold Ganser, Richard James Dillon, Peter J M Valk, Miguel A Sanz, Bob Löwenberg, Nancy Berliner, Eduardo M Rego","doi":"10.3324/haematol.2024.286338","DOIUrl":"https://doi.org/10.3324/haematol.2024.286338","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HaematologicaPub Date : 2024-10-24DOI: 10.3324/haematol.2023.284937
Howard S Oster,Arjan A Van de Loosdrecht,Moshe Mittelman
{"title":"Diagnosis of myelodysplastic syndromes: the classic and the novel.","authors":"Howard S Oster,Arjan A Van de Loosdrecht,Moshe Mittelman","doi":"10.3324/haematol.2023.284937","DOIUrl":"https://doi.org/10.3324/haematol.2023.284937","url":null,"abstract":"The Myelodysplastic syndromes (MDS) are a heterogenous group of clonal bone marrow (BM) stem cell myeloid neoplasms, characterized by bone marrow (BM) dysplasia, macrocytic anemia or cytopenia with a tendency for leukemic transformation. The suspicion of MDS is raised by a typical but not specific clinical picture and routine labs, but the gold standard for MDS diagnosis is still BM examination with the presence of uni-or multi-lineage dysplasia and blast percentage, together with exclusion of other reasons. Cytogenetics is also a part of the diagnostic process. Flow cytometry and genetics are helpful but are not always mandatory for MDS diagnosis. This review summarizes the current steps in the diagnostic approach for a patient suspected of having MDS. We also describe new concepts that use non-invasive diagnostic technologies, especially digital methods as well as peripheral blood genetics. The hope is that one day these will mature, be introduced into clinical practice, and perhaps in many cases even replace the invasive BM biopsy.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"4 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
HaematologicaPub Date : 2024-10-24DOI: 10.3324/haematol.2023.284947
Mario Cazzola,Luca Malcovati
{"title":"Genome sequencing in the management of myelodysplastic syndromes and related disorders.","authors":"Mario Cazzola,Luca Malcovati","doi":"10.3324/haematol.2023.284947","DOIUrl":"https://doi.org/10.3324/haematol.2023.284947","url":null,"abstract":"Myeloid neoplasms originate from the clonal proliferation of hematopoietic stem cells, which is driven by the acquisition of somatic genetic mutations. Within these disorders, myelodysplastic syndromes (MDS) are specifically characterized by morphologic abnormalities (dysplasia) and impaired maturation of myeloid precursors (ineffective hematopoiesis), resulting in peripheral blood cytopenia. Several studies have advanced the field of MDS, with a few landmark papers leading to a paradigm shift, opening new avenues of research and enabling a molecular revolution. These seminal papers include the first description of the 5q- syndrome, the identification of somatic mutations of TET2 in myeloid neoplasms, the detection of common pathway mutations in the splicing machinery, and the discovery of clonal hematopoiesis. The somatic genomic landscape of MDS is now well-defined. Genes that are recurrently mutated include epigenetic regulators, as well as genes of RNA splicing machinery, transcription regulation, DNA repair control, cohesin complex, and signal transduction. Furthermore, several disorders with a germline genetic predisposition to MDS have been identified, collectively accounting for up to 15% of all MDS cases. Genomic profiling can significantly improve the diagnostic approach to MDS, allowing the identification of distinct nosologic entities such as SF3B1-mutant or TP53-mutant MDS. The Molecular International Prognostic Scoring System for MDS (IPSS-M) has already proven to be a valuable tool for individualized risk assessment and treatment decisions. In addition, the recently developed molecular taxonomy of MDS will likely facilitate the implementation of precision medicine approaches for these disorders. This will necessitate the establishment of specialized infrastructures within public health systems, involving close collaboration between healthcare institutions, academia, and the life sciences industry.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"5 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}