Acta Haematologica最新文献

{"title":"Toxicity of CAR T-Cell Therapy for Multiple Myeloma.","authors":"Aimaz Afrough, Pearl Rajan Abraham, Laura Turer, Gurbakhash Kaur, Aishwarya Sannareddy, Doris K Hansen, Larry D Anderson","doi":"10.1159/000539134","DOIUrl":"10.1159/000539134","url":null,"abstract":"<p><strong>Background: </strong>Idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel) are novel chimeric antigen receptor (CAR)-T cell therapies targeting B-cell maturation antigen (BCMA), and both have recently gained approval by the US Food Drug Administration (FDA) for the treatment of relapsed and refractory multiple myeloma (RRMM).</p><p><strong>Summary: </strong>These therapies offer unprecedented responses in RRMM but present new challenges including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), non-ICANS neurotoxicity, cytopenias, infections, and hypogammaglobulinemia.</p><p><strong>Key messages: </strong>In the evolving CAR-T landscape, a primary objective is to develop innovative strategies for managing associated toxicities. Through meticulous exploration of underlying mechanisms and tailored interventions, we aim to enhance safety and enable broader outpatient utilization. Refinement of protocols, biomarker identification, and robust monitoring are imperative for sustained efficacy. This comprehensive approach guarantees the continuous advancement and optimization of CAR-T therapy.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":2.4,"publicationDate":"2024-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140891042","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}

{"title":"Historical Perspective of High-Dose Therapy Followed by Autologous Stem Cell Transplantation in Multiple Myeloma.","authors":"Inbar Cohen, Iuliana Vaxman, Morie A Gertz","doi":"10.1159/000539225","DOIUrl":"10.1159/000539225","url":null,"abstract":"<p><strong>Background: </strong>High-dose therapy (HDT) followed by autologous stem cell transplantation (ASCT) has become part of standard of care (SOC) in newly diagnosed multiple myeloma. In this review, we provide a historical perspective on ASCT since its introduction in the 1990s.</p><p><strong>Summary: </strong>Overall survival (OS) benefit for HDT followed by ASCT was demonstrated in studies comparing HDT with ASCT to standard-dose therapy (SDT) before the era of novel agents. Conditioning is done with melphalan 200 mg/m2. Lower doses (MEL140, MEL150) for older patients with comorbidities are safe and have comparable results. The addition of busulfan to melphalan improves progression-free survival (PFS) but not OS. HDT with ASCT after induction with novel agents prolongs PFS but not OS compared to SDT alone. The benefit is more evident in patients with high-risk cytogenetics. Mobilization can be achieved with granulocyte colony-stimulating factor alone, but is improved with the addition of chemotherapy. Plerixafor reduces mobilization failure and enables sufficient stem cell collection after induction with novel agents. ASCT is safe with a low rate of mortality (1%), and selected patients can be managed as outpatients.</p><p><strong>Key messages: </strong>HDT followed by ASCT remains part of SOC due to its PFS benefit and relatively low toxicity.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":2.4,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140846838","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}

{"title":"Persistent COVID-19: A Case Report of an Immunocompromised Patient and a Literature Review.","authors":"Sirine Bekkaoui, Geoffroy Venton, Fannie Bretelle, Victoria Garrido, Victor Chabbert, Stéphane Gayet, Paul Dalmas, Antoine Tichadou, Pierre-André Jarrot, Patrick Villani, Aurélie Daumas, Robin Arcani","doi":"10.1159/000537793","DOIUrl":"10.1159/000537793","url":null,"abstract":"<p><strong>Introduction: </strong>Immunocompromised patients can show prolonged shedding of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and persistent symptoms, which is called persistent COVID-19.</p><p><strong>Case presentation: </strong>We report a case of an immunocompromised patient who was treated for mantle cell lymphoma and was suffering from B-cell depletion. The patient developed persistent COVID-19, which was confirmed by real-time polymerase chain reaction (RT-PCR) tests in only sputum and bronchoalveolar fluid which remained positive for at least 112 days. The patient was successfully treated with SARS-CoV-2 convalescent plasma.</p><p><strong>Conclusion: </strong>It could be of interest to investigate the RT-PCR results of SARS-CoV-2 in sputum/bronchoalveolar lavage samples from immunocompromised patients with unexplained pneumonia.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"571-575"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139740145","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}

{"title":"Pathophysiology of Acute Myeloid Leukemia.","authors":"Franziska Wachter, Yana Pikman","doi":"10.1159/000536152","DOIUrl":"10.1159/000536152","url":null,"abstract":"<p><strong>Background: </strong>Acute myeloid leukemia (AML) is a biologically heterogenous disease arising in clonally proliferating hematopoietic stem cells. Sequential acquisition of mutations leads to expanded proliferation of clonal myeloid progenitors and failure of differentiation, leading to fulminant AML.</p><p><strong>Summary: </strong>Here, we review the pathophysiology of AML with a focus on factors predisposing to AML development, including prior chemo- and radiation therapy, environmental factors, and germline predisposition.</p><p><strong>Key message: </strong>Increasing genomic characterization of AML and insight into mechanisms of its development will be critical to improvement in AML prognostication and therapy.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"229-246"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139477819","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}

{"title":"Bortezomib and Vorinostat Therapy as Maintenance Therapy Post-Autologous Transplant for Non-Hodgkin's Lymphoma Using R-BEAM or BEAM Transplant Conditioning Regimen.","authors":"Leona A Holmberg, David G Maloney, Laura Connelly-Smith","doi":"10.1159/000533944","DOIUrl":"10.1159/000533944","url":null,"abstract":"<p><strong>Introduction: </strong>The success of autologous stem cell transplantation (ASCT) for treating non-Hodgkin's lymphoma (NHL) is limited by its high relapse rates. To reduce the risk of relapse, additional maintenance therapy can be added post-transplant. In a non-transplant setting at the time of initiation of this study, both bortezomib and vorinostat had been studied alone or in combination for some NHL histology and showed some clinical activity. At our center, this combination therapy post-transplant for multiple myeloma showed acceptable toxicity. Therefore, it seemed reasonable to study this combination therapy post-ASCT for NHL.</p><p><strong>Methods: </strong>NHL patients underwent conditioning for ASCT with rituximab, carmustine, etoposide, cytarabine, melphalan/carmustine, etoposide, cytarabine, melphalan. After recovery from the acute transplant-related toxicity, combination therapy with IV bortezomib and oral vorinostat (BV) was started and was given for a total of 12 (28-day) cycles.</p><p><strong>Results: </strong>Nineteen patients received BV post-ASCT. The most common toxicities were hematologic, gastrointestinal, metabolic, fatigue, and peripheral neuropathy. With a median follow-up of 10.3 years, 11 patients (58%) are alive without disease progression and 12 patients (63%) are alive.</p><p><strong>Conclusions: </strong>BV can be given post-ASCT for NHL and produces excellent disease-free and overall survival rates.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"300-309"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10590639","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}

{"title":"Consensus Statements Highlight the Need of Harmonizing Chronic Lymphocytic Leukemia Management Worldwide.","authors":"Stefano Molica, Marco Rossi, David Allsup","doi":"10.1159/000533349","DOIUrl":"10.1159/000533349","url":null,"abstract":"<p><p>In addition to the European Society for Medical Oncology (ESMO) and National Comprehensive Cancer Network (NCCN) guidelines that are reference standards for the treatment of chronic lymphocytic leukemia (CLL) in Europe and the USA, several consensus statements, formulated by independent, multidisciplinary panels of specialists, have been developed to provide region-specific guidance for the management of CLL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"257-259"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9944849","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}

{"title":"Belantamab Mafodotin and Relapsed/Refractory Multiple Myeloma: This Is Not Game Over.","authors":"Annalisa Condorelli, Bruno Garibaldi, Caterina Gagliano, Alessandra Romano, Vittorio Del Fabro, Nunziatina Laura Parrinello, Antonio Longo, Sebastiano Cosentino, Francesco Di Raimondo, Concetta Conticello","doi":"10.1159/000521112","DOIUrl":"10.1159/000521112","url":null,"abstract":"<p><p>Although the therapeutic landscape for multiple myeloma (MM) has expanded, the disease always tends to relapse. In an attempt to obtain deep and durable responses, each relapse requires the use of a new strategy. In recent years, new treatment options have emerged, even for heavily treated patients. Novel, well-tolerated, and highly effective therapies in the relapsed/refractory (RRMM) setting currently represent a real hope. Belantamab mafodotin (BLENREP™) is a first-in-class monoclonal antibody-drug conjugate whose target is B-cell maturation antigen conjugated to the cytotoxic microtubule inhibitor monomethyl auristatin F. Here, we present two cases of heavily pre-treated RRMM patients that were favorably treated with belantamab mafodotin, obtaining at least a partial response. Treatment was well tolerated and is ongoing. This is a rare report on real life clinical use of belantamab mafodotin outside of controlled clinical trials and provides information on efficacy and safety of this anti-myeloma new class of drugs.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"493-498"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39940141","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}

{"title":"Evidence-Based Management of Chronic Lymphocytic Leukemia: Consensus Statements from the Gulf Region.","authors":"Salem H Alshemmari, Mustaqeem A Siddiqui, Ramesh Pandita, Hani Y Osman, Honar Cherif, Susan O'Brien, Mahmoud Marashi, Khalil Al Farsi","doi":"10.1159/000531675","DOIUrl":"10.1159/000531675","url":null,"abstract":"<p><strong>Introduction: </strong>Despite recent advances in diagnosis, prognostication, and treatment options, chronic lymphocytic leukemia (CLL) is still a largely incurable disease. New concepts on diagnosis, staging, treatment, and follow-up on CLL have been incorporated throughout recent years. The lack of regional consensus guidelines has led to varying practices in the management of patients with CLL in the region. This manuscript aims to reach a consensus among expert hematologists regarding the definitions, classifications, and related practices of CLL. The experts developed a set of statements utilizing their personal experience together with the current literature on CLL management. This consensus aims to provide guidance for healthcare professionals involved in the management of CLL and serves as a step in developing regional guidelines.</p><p><strong>Methods: </strong>Eight experts responded to 50 statements regarding the diagnosis, staging, treatment, and prognosis of CLL with three potential answering alternatives ranging between agree, disagree, and abstain. This consensus adopted a modified Delphi consensus methodology. A consensus was reached when at least 75% of the agreement to the answer was reached. This manuscript presents the scientific insights of the participating attendees, panel discussions, and the supporting literature review.</p><p><strong>Results: </strong>Of the 50 statements, a consensus was reached on almost all statements. Statements covered CLL-related topics, including diagnostic evaluation, staging, risk assessment, different patient profiles, prognostic evaluation, treatment decisions, therapy sequences, response evaluation, complications, and CLL during the COVID-19 pandemic.</p><p><strong>Conclusion: </strong>In recent years, CLL management has progressed significantly, with many diagnostic tests and several novel treatments becoming available. This consensus gathers decades of consolidated principles, novel research, and promising prospects for the management of this disease.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"260-279"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41094645","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}

{"title":"Utility of Echocardiogram in Neutropenic Patients with Gram-Positive Bacteremia: A Retrospective Study.","authors":"Neta Sternbach, Haim Ben-Zvi, Ofir Wolach, Moshe Yeshurun, Pia Raanani, Dafna Yahav, Liat Shargian","doi":"10.1159/000538449","DOIUrl":"10.1159/000538449","url":null,"abstract":"<p><strong>Introduction: </strong>Hemato-oncology patients are vulnerable to bloodstream infections due to immunocompromised state and use of intravascular catheters. Data regarding risk of infective endocarditis (IE) among those with gram-positive bacteremia are limited. We aimed to evaluate the incidence of IE among neutropenic hemato-oncology patients and explore the yield of echocardiogram in this population.</p><p><strong>Methods: </strong>We conducted a single retrospective study of all hospitalized hemato-oncology neutropenic patients with gram-positive blood cultures between 2007 and 2021. Data regarding patients' characteristics, blood cultures, and echocardiogram were collected.</p><p><strong>Results: </strong>The study included 241 patients, with 283 isolates. Coagulase-negative Staphylococcus (CONS) was the most commonly isolate found, followed by Streptococcus viridans. Transthoracic echocardiography (TTE) was performed in 45% of patients overall, of which 5.8% had additional transesophageal echocardiogram (TEE). Only a single case of IE was identified in a 47-year-old multiple myeloma patient with neutropenic fever, S. viridans bacteremia, and stroke caused by septic emboli. TTE and TEE failed to demonstrate valvular pathology consistent with IE.</p><p><strong>Conclusion: </strong>In our experience, the yield of echocardiogram in hemato-oncological neutropenic patients with bacteremia is extremely low, owing to reduced probability of IE in this population, and thus could be avoided in most cases.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"671-675"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140287964","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}

{"title":"COVID-19 in Patients with Chronic Lymphocytic Leukemia: What Have We Learned?","authors":"Reut Harel, Gilad Itchaki","doi":"10.1159/000534540","DOIUrl":"10.1159/000534540","url":null,"abstract":"<p><strong>Background: </strong>Chronic lymphocytic leukemia (CLL) is a prevalent hematological malignancy (HM) characterized by inherent immunodeficiency, which is further pronounced by disease-directed therapy. The COVID-19 pandemic has had devastating outcomes, and although its impact has diminished over time, it continues to be a cause of significant morbidity and mortality, particularly among immunodeficient patients.</p><p><strong>Summary: </strong>In this review, we describe mechanisms of immune dysfunction in CLL in relation to COVID-19, provide an overview of the clinical outcomes of the disease in this patient population, and identify risk factors associated with severe morbidity and mortality. Additionally, we acknowledge the influence of the rapidly evolving landscape of new disease variants. The review further delineates the humoral and cellular responses to vaccination and their clinical efficacy in preventing COVID-19 in CLL patients. Moreover, we explore potential approaches to enhance these immune responses. Pre- and post-exposure prophylaxis strategies are discussed, along with description of common agents in the treatment of the disease in both outpatient and inpatient setting. Throughout the review, we emphasize the interplay between novel therapies for CLL and COVID-19 outcomes, prevention, and treatment and describe the impact of COVID-19 on the utilization of these novel agents. This information has the potential to guide clinical decision making in the management CLL patients.</p><p><strong>Key messages: </strong>CLL patients are at risk for severe COVID-19 infection. Vaccinations and COVID-19 directed therapy have improved outcomes in patients with CLL, yet clinical challenges persist.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"60-72"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11251671/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41187816","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}