Acta Haematologica最新文献

{"title":"AI Applications in Transfusion Medicine: Opportunities, Challenges, and Future Directions.","authors":"Omri Cohen, Merav Barzilai, Omri Cohen","doi":"10.1159/000546303","DOIUrl":"10.1159/000546303","url":null,"abstract":"<p><p><p>Background: Artificial intelligence (AI) is reshaping healthcare, with its applications in transfusion medicine (TM) showing great promise to address longstanding challenges. Summary: This review explores the integration of AI-driven tools, including machine learning, deep learning, natural language processing, and predictive analytics, across various domains of TM. From enhancing donor management and optimizing blood product quality to predicting transfusion needs and assessing bleeding risks, AI has demonstrated its potential to improve operational efficiency, patient safety, and resource allocation. Additionally, AI-powered systems enable more accurate blood antigen phenotyping, automate hemovigilance workflows, and streamline inventory management through advanced forecasting models. While these advancements are largely exploratory, early studies highlight the growing importance of AI in improving patient outcomes and advancing precision medicine. However, challenges such as variability in clinical workflows, algorithmic transparency, equitable access, and ethical concerns around data privacy and bias must be addressed to ensure responsible integration. Key Messages: (i) AI-driven tools are being applied across multiple domains of TM. (ii) Early studies demonstrate the potential for AI to improve efficiency, safety, and personalization. (iii) Key implementation challenges include data privacy, workflow integration, and equitable access. </p>.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"516-526"},"PeriodicalIF":1.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12176357/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143955356","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}

{"title":"\"Real-Life\" Data of Zanubrutinib in Patients with Waldenström Macroglobulinemia: A Multicenter Retrospective Study.","authors":"Gilad Itchaki, Ohad Benjamini, Mor Levi, Anatoly Nemets, Shai Ygna, Mahdi Assaly, Anna Gourevitch, Moshe Gatt, Revital Saban, Pia Raanani, Iuliana Vaxman","doi":"10.1159/000542936","DOIUrl":"10.1159/000542936","url":null,"abstract":"<p><strong>Introduction: </strong>Waldenström macroglobulinemia (WM) is a rare indolent lymphoma. Zanubrutinib (ZAN), a second-generation BTK inhibitor, has been approved for the treatment of WM in any line of therapy in 2021. Between November 2020 and January 2022, an expanded access program of ZAN opened in Israel for the treatment of patients with relapsed/refractory (R/R)-WM or those ineligible for chemotherapy or ibrutinib in first line.</p><p><strong>Methods: </strong>This is a multicenter retrospective study aiming to provide real-world data on ZAN in patients with WM in Israel. Demographic and clinical data were collected and coded from electronic files. Response was evaluated by the investigator's assessment. As the program closed, patients transitioned to commercial ZAN.</p><p><strong>Results: </strong>Thirteen patients (12 R/R; 1 treatment-naive) were enrolled across 8 centers in Israel. The median age at ZAN initiation was 71 years (range, 50-85); 6 were males; 10 had high IPSS-WM. R/R patients had a median of 1 (1-4) prior lines of therapy. Other than progressive disease after chemoimmunotherapy, the most common considerations for choosing ZAN were patients' age and/or comorbidities (n = 5), as well as ibrutinib toxicity. The initial ZAN dose was reduced in 4 patients. The median time on ZAN was 19.5 months (2.9-29.5). Of 12 evaluable patients, the ORR was 83% with 3 minor responses, 6 PRs, and 1 VGPR. With a median follow-up of 19.6 months, 7 patients were still on ZAN, 5 progressed, 4 while on ZAN, and 1 after ZAN was stopped due to AE. Eighteen-month PFS and OS were 60.5% and 77%, respectively. Eight (61%) patients had AEs of any grade, and 3 (23%) of grade 3-4; 2 stopped ZAN due to congestive heart failure and extreme fatigue.</p><p><strong>Conclusion: </strong>The results of this real-world high-risk population are consistent with prospective studies highlighting the efficacy and safety of ZAN.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"462-467"},"PeriodicalIF":1.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142779024","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":"Single-Center Experience of Patients with Plasma Cell Leukemia in the Era of New Therapeutics.","authors":"Maria Dampmann, Sarah Flossdorf, Julius Keyl, Hans Christian Reinhardt, Christine Hanoun","doi":"10.1159/000539223","DOIUrl":"10.1159/000539223","url":null,"abstract":"<p><strong>Introduction: </strong>Plasma cell leukemia (PCL) can occur de novo as primary PCL (pPCL), or in patients with prior diagnosis of multiple myeloma (MM) as secondary PCL (sPCL). In 2021, the diagnostic criteria have been revised, establishing a new cut-off of ≥5% plasma cells in the peripheral blood. Lacking specific clinical trials, PCL is treated similarly to MM; however, outcome for patients with PCL remains poor. Here, we report outcomes for patients with pPCL and sPCL in the era of novel agents.</p><p><strong>Methods: </strong>We performed a retrospective analysis and identified 19 patients (11 pPCL, 8 sPCL) who have been treated for PCL between 2010 and 2022 at University Hospital Essen.</p><p><strong>Results: </strong>Patients with pPCL had a median overall survival (OS) of 37.8 months (95% CI: [15.4; 52.3] months) from diagnosis, with a median time to next treatment (TTNT) of 18.4 (2.0; 22.9) months. All patients were treated with a proteasome-inhibitor (PI)-based induction therapy, and the majority was consolidated with an autologous stem cell transplantation (SCT). Five of these patients received a tandem transplantation. Patients with sPCL had a median OS of only 1.5 months after diagnosis of PCL. Only 1 patient achieved a remission with daratumumab and subsequent allogenic SCT.</p><p><strong>Conclusion: </strong>With our study, we add evidence for a PI-based induction therapy followed by a consolidating autologous SCT for patients with pPCL and give further evidence that a tandem transplant concept might be beneficial. The diagnosis of sPCL remains devastating and needs new therapeutic approaches.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"77-84"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141064633","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":"Eosinophilic Pleocytosis in the Cerebrospinal Fluid following CAR-T Cell Therapy for Central Nervous System Lymphoma: A Case for Warning?","authors":"Mayasa Abu Ata, Israel Henig, Dana Yehudai-Ofir, Inna Tzoran, Shimrit Ringelstein-Harlev, Tsofia Inbar, Ilana Slouzkey, Michal Karmona Fintuch, Anat Stern, Olesya Stanevsky, Michal Weiler-Sagie, Yaniv Zohar, Ido Livneh, Goni Merhav, Ayelet Eran, Tsila Zuckerman, Ofrat Beyar Katz","doi":"10.1159/000539354","DOIUrl":"10.1159/000539354","url":null,"abstract":"<p><strong>Introduction: </strong>Chimeric antigen receptor T (CAR-T) cell therapy, emerging as an efficient treatment option for patients with secondary central nervous system (CNS) lymphoma, is frequently complicated with immune effector cell-associated neurotoxicity syndrome (ICANS).</p><p><strong>Case presentation: </strong>We report a case of a 64-year-old woman with transformed follicular lymphoma, developing high-grade ICANS with eosinophilic pleocytosis following third-line therapy with CAR-T cells (tisagenlecleucel). During bridging therapy, she declined neurologically and was diagnosed with secondary CNS lymphoma. She received methotrexate-cytarabine-thiotepa-rituximab regimen with clinical and radiological improvement. Post-CAR-T cell infusion she developed cytokine release syndrome grade II and ICANS grade III. Given the lack of response to steroids, anakinra was initiated with complete ICANS resolution. Cerebrospinal fluid (CSF) analysis, performed only on day +10 due to thrombocytopenia, revealed eosinophils, while infections were excluded.</p><p><strong>Conclusion: </strong>This report emphasizes the importance of CSF analysis in individuals with CAR-T-related neurotoxicity for elucidating the role of specific immune cells in such complications.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"119-126"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141086471","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":"A Case of Recurrent Localized Pulmonary Nodular Light Chain Amyloidosis Treated with Daratumumab plus CyBorD.","authors":"Ted Raddell, Farah Ashraf, Xiaofeng Zhao, Osheen Abramian, Tulin Budak-Alpdogan","doi":"10.1159/000540272","DOIUrl":"10.1159/000540272","url":null,"abstract":"<p><strong>Introduction: </strong>Nodular pulmonary amyloidosis (NPA) is a localized form of light chain (AL) amyloidosis often found incidentally and typically has an indolent and benign disease course treated with resection or local excision. We present a patient with recurrent localized AL amyloidosis who required further treatment.</p><p><strong>Case presentation: </strong>A 63-year-old female with monoclonal gammopathy of undetermined significance (MGUS) was found to have pulmonary AL amyloid on wedge resection and later had recurrence. The patient did not have signs of clonal plasma cell proliferation or systemic AL amyloid. She was treated with daratumumab, cyclophosphamide, bortezomib, and dexamethasone. After initiation of treatment, the patient has had significant hematologic and radiographic response.</p><p><strong>Conclusion: </strong>The patient had NPA recurrence with organ dysfunction without systemic disease. Because the presentation of recurrent pulmonary AL amyloidosis is rare, there is no published evidence on treatment. However, the patient has had hematologic and radiographic improvement after initiating treatment with a systemic protocol.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"346-351"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141791633","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":"Sudden Blast Crisis in a Chronic Myeloid Leukemia Patient in Treatment-Free Remission: A Case Report and Literature Review.","authors":"Qiushi Liang, Zhigang Liu, Yu Wu, Huanling Zhu, Yunfan Yang","doi":"10.1159/000542153","DOIUrl":"10.1159/000542153","url":null,"abstract":"<p><strong>Introduction: </strong>Treatment-free remission (TFR) has emerged as a new goal in the treatment of chronic myeloid leukemia (CML). TFR is considered a safe intervention because patients who experienced molecular relapse usually responded well to tyrosine kinase inhibitors resumption and regained molecular response quite efficiently. Nevertheless, there have been reports of occurrence of blast crisis during TFR.</p><p><strong>Case presentation: </strong>We report a case of sudden lymphoid blast crisis in a CML patient who had been in TFR for 21 months without any prior molecular loss. Whole-exon sequencing identified a frameshift mutation of SETD2. In addition, we reviewed the current literature on cases of blast crisis in TFR. Only eleven cases of blast crisis have been reported among thousands of patients who discontinued tyrosine kinase inhibitor (TKI) therapy, including our patient. Of these cases, nine presented with lymphoid blast crisis. Additional gene mutations are frequently observed.</p><p><strong>Conclusion: </strong>This case, along with others, emphasizes the necessity of implementing a long-term monitoring strategy following TKI discontinuation due to the potential for late onset of blast crisis. Systematic genetic studies in patients failing TFR should be properly carried out to further understand the mechanism and, eventually, to predict or prevent such adverse event in patients in TFR.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"371-379"},"PeriodicalIF":1.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142455472","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":"Results of Long-Term Therapy with a Biosimilar of Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria.","authors":"Alexander D Kulagin, Vadim V Ptushkin, Elena A Lukina, Igor L Davydkin, Alexander V Korobkin, Tatiana S Konstantinova, Elena Yu Komartseva, Natalia V Minaeva, Tatiana A Mitina, Olesya U Klimova, Evgeniya G Arshanskaya, Vitalii D Latyshev, Oksana A Markova, Eugene V Zuev","doi":"10.1159/000542294","DOIUrl":"10.1159/000542294","url":null,"abstract":"<p><strong>Introduction: </strong>The study aimed to assess the safety, immunogenicity, and efficacy of long-term therapy with biosimilar of eculizumab (Elizaria®) in paroxysmal nocturnal hemoglobinuria (PNH) patients.</p><p><strong>Methods: </strong>The study included 30 patients with PNH who had completed previous clinical trials. Of these, 25 patients continued receiving the biosimilar product, and 5 patients switched from the originator product Soliris. The maximum duration of follow-up was 104 weeks, during which the investigational product was administered 52 times at a standard dose.</p><p><strong>Results: </strong>Throughout the study, the levels of lactate dehydrogenase, hemoglobin, reticulocytes, and PNH clone remained stable compared to baseline, regardless of the previous therapy (p > 0.05). There were no significant differences in the number of patients with chronic kidney disease at different visits, as well as in the number of patients who received donor red blood cell and platelet transfusions during the study (p > 0.05). There were 2 cases of adverse reactions reported in 2 patients (6.6%): elevated aspartate aminotransferase (3.3%) and alopecia (3.3%). Immunogenicity analysis showed no significant differences in the frequency of antidrug antibody detection compared to baseline (p > 0.05).</p><p><strong>Conclusion: </strong>The study findings confirm the long-term efficacy and safety of biosimilar in patients with PNH.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"443-451"},"PeriodicalIF":1.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12306948/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142715000","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}

{"title":"Integrating Artificial Intelligence into Hematology: New Frontiers and Practical Applications.","authors":"Shelly Soffer, Eyal Klang","doi":"10.1159/000546889","DOIUrl":"10.1159/000546889","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"514-515"},"PeriodicalIF":1.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144300937","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":"Highlights from the 66th ASH Annual Meeting 2024.","authors":"Ine Schmale","doi":"10.1159/000544847","DOIUrl":"10.1159/000544847","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"244-246"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143456582","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":"Safety of Extended Pirtobrutinib Exposure in Relapsed and/or Refractory B-Cell Malignancies.","authors":"Lindsey E Roeker, Catherine C Coombs, Nirav N Shah, Wojciech Jurczak, Jennifer A Woyach, Chan Y Cheah, Krish Patel, Kami Maddocks, Yucai Wang, Pier Luigi Zinzani, Talha Munir, Youngil Koh, Meghan C Thompson, Catherine E Muehlenbein, Chunxiao Wang, Richard Sizelove, Sarang Abhyankar, Safarulla Hasanabba, Donald E Tsai, Toby A Eyre, Michael Wang","doi":"10.1159/000539587","DOIUrl":"10.1159/000539587","url":null,"abstract":"<p><strong>Introduction: </strong>Pirtobrutinib, a highly selective, noncovalent (reversible) Bruton tyrosine kinase inhibitor, has demonstrated promising efficacy in B-cell malignancies and is associated with low rates of discontinuation and dose reduction. Pirtobrutinib is administered until disease progression or toxicity, necessitating an understanding of the safety profile in patients with extended treatment.</p><p><strong>Methods: </strong>Here we report the safety of pirtobrutinib in patients with relapsed/refractory B-cell malignancies with extended (≥12 months) drug exposure from the BRUIN trial. Assessments included median time-to-first-occurrence of adverse events (AEs), dose reductions, and discontinuations due to treatment-emergent AEs (TEAEs) and select AEs of interest (AESIs).</p><p><strong>Results: </strong>Of 773 patients enrolled, 326 (42%) received treatment for ≥12 months. In the extended exposure cohort, the median time-on-treatment was 19 months. The most common all-cause TEAEs were fatigue (32%) and diarrhea (31%). TEAEs leading to dose reduction occurred in 23 (7%) and discontinuations in 11 (3%) extended exposure patients. One patient had a fatal treatment-related AE (COVID-19 pneumonia). Infections (73.0%) were the most common AESI with a median time-to-first-occurrence of 7.4 months. Majority of TEAEs and AESIs occurred during the first year of therapy.</p><p><strong>Conclusions: </strong>Pirtobrutinib therapy continues to demonstrate an excellent safety profile amenable to long-term administration without evidence of new or worsening toxicity signals.</p><p><strong>Introduction: </strong>Pirtobrutinib, a highly selective, noncovalent (reversible) Bruton tyrosine kinase inhibitor, has demonstrated promising efficacy in B-cell malignancies and is associated with low rates of discontinuation and dose reduction. Pirtobrutinib is administered until disease progression or toxicity, necessitating an understanding of the safety profile in patients with extended treatment.</p><p><strong>Methods: </strong>Here we report the safety of pirtobrutinib in patients with relapsed/refractory B-cell malignancies with extended (≥12 months) drug exposure from the BRUIN trial. Assessments included median time-to-first-occurrence of adverse events (AEs), dose reductions, and discontinuations due to treatment-emergent AEs (TEAEs) and select AEs of interest (AESIs).</p><p><strong>Results: </strong>Of 773 patients enrolled, 326 (42%) received treatment for ≥12 months. In the extended exposure cohort, the median time-on-treatment was 19 months. The most common all-cause TEAEs were fatigue (32%) and diarrhea (31%). TEAEs leading to dose reduction occurred in 23 (7%) and discontinuations in 11 (3%) extended exposure patients. One patient had a fatal treatment-related AE (COVID-19 pneumonia). Infections (73.0%) were the most common AESI with a median time-to-first-occurrence of 7.4 months. Majority of TEAEs and AESIs occurred during the first year ","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"180-197"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11617602/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141199232","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}