Lourdes M Mendez, Daniel J DeAngelo, Marlise R Luskin
{"title":"Treatment of Older Adults with Newly Diagnosed Philadelphia Chromosome-Negative Acute Lymphoblastic Leukemia.","authors":"Lourdes M Mendez, Daniel J DeAngelo, Marlise R Luskin","doi":"10.1159/000546381","DOIUrl":"10.1159/000546381","url":null,"abstract":"<p><strong>Background: </strong>Approximately half of newly diagnosed cases of acute lymphoblastic leukemia (ALL) occur in adults, but adults experience significantly higher rates of treatment failure and treatment-related mortality due to frequent presence of adverse disease biology and limited tolerability of conventional chemotherapy.</p><p><strong>Summary: </strong>Here, we discuss recent data from clinical trials investigating new approaches for initial treatment of Philadelphia chromosome-negative ALL in older adults. These trials investigate the incorporation of novel agents including the anti-CD22 antibody-drug conjugate inotuzumab, the CD19-CD3 bi-specific T-cell engager blinatumomab, and the BCL2 inhibitor venetoclax into treatment regimens, with some studies attenuating or omitting chemotherapy. We also discuss the role of allogeneic stem cell transplantation consolidation for this population and highlight the possibility of frontline CD19-directed chimeric antigen receptor T-cell therapy consolidation approaches for B-ALL. Finally, we discuss improved understanding of the genetic diversity of ALL in older adults including occurrence of ALL with TP53 mutation, ALL with myeloid gene mutations, and therapy-related ALL.</p><p><strong>Key message: </strong>Overall, we highlight progress for older adults with Ph-negative ALL with patients more frequently achieving a measurable residual disease-negative complete remission, but significant work remains to improve the safety of treatment as well as the depth and durability of treatment response.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":1.7,"publicationDate":"2025-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144265029","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}
Marco Picardi, Claudia Giordano, Annamaria Vincenzi, Novella Pugliese, Alessia Scarpa, Massimo Mascolo, Elena Vigliar, Giancarlo Troncone, Claudia Salvatore, Fabrizio Pane
{"title":"Ultrasonography-Guided Core-Needle Biopsy for Newly Diagnosed Large B-Cell Lymphomas: Analysis on Diagnostic Efficacy and Safety in an Italian Retrospective Study.","authors":"Marco Picardi, Claudia Giordano, Annamaria Vincenzi, Novella Pugliese, Alessia Scarpa, Massimo Mascolo, Elena Vigliar, Giancarlo Troncone, Claudia Salvatore, Fabrizio Pane","doi":"10.1159/000544794","DOIUrl":"10.1159/000544794","url":null,"abstract":"<p><strong>Introduction: </strong>Excisional biopsy (EB) is the gold standard for large B-cell lymphomas (LBCLs) diagnosis. Based on recent advances in interventional radiology enabling accurate sampling with core needle biopsy (CNB), we evaluated efficacy and safety of CNB under imaging guidance for diagnosing LBCLs.</p><p><strong>Methods: </strong>At the Hematology and Pathology Units of the Federico II University Medical School of Naples (Italy), we retrospectively collected patients with lymphadenopathies suspected of lymphomas (during 2009-2022) of which the ultrasonography (US)-guided CNB lymph node samples were available. Subsequently, we investigated the accuracy and safety of US-guided CNB for LBCLs diagnosis.</p><p><strong>Results: </strong>Over a 12-year period, 800 (superficial target, n = 560; deep-seated target, n = 240) lymph node biopsies performed with 16-gauge diameter modified Menghini needle under power-Doppler ultrasonographic guidance have been evaluated in 800 patients. According to the reference standard, 220 were suffering from LBCLs (diffuse LBCL NOS [n = 196], and high-grade B-cell lymphoma with MYC and BCL2 rearrangements [n = 24]) subtypes, other malignancy subtypes (n = 510) and non-malignant findings (n = 70). For the series of LBCLs, the overall diagnostic accuracy of the micro-histological sampling was 100% (95% confidence interval: 98%-100%). The complications occurred with very low incidence and severity (grade ≤2).</p><p><strong>Conclusion: </strong>US-guided CNB is a less invasive method and can be considered an alternative to EB for LBCL diagnosis.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.7,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12173430/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144148792","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":"AI Applications in Transfusion Medicine: Opportunities, Challenges, and Future Directions.","authors":"Merav Barzilai, Omri Cohen","doi":"10.1159/000546303","DOIUrl":"10.1159/000546303","url":null,"abstract":"<p><strong>Background: </strong>Artificial intelligence (AI) is reshaping healthcare, with its applications in transfusion medicine (TM) showing great promise to address longstanding challenges.</p><p><strong>Summary: </strong>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.</p><p><strong>Key messages: </strong>(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>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.7,"publicationDate":"2025-05-09","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}
David McCall, Samanta Catueno, Ramya Ramakrishnan, Priti Tewari, Irtiza Sheikh, Amber Gibson, Cesar A Nuñez, Miriam B Garcia, Branko Cuglievan
{"title":"Beyond Cellular Therapies: The Expanding Role of Antibody-Driven Immunotherapy in Pediatric Acute Lymphoblastic Leukemia.","authors":"David McCall, Samanta Catueno, Ramya Ramakrishnan, Priti Tewari, Irtiza Sheikh, Amber Gibson, Cesar A Nuñez, Miriam B Garcia, Branko Cuglievan","doi":"10.1159/000546249","DOIUrl":"10.1159/000546249","url":null,"abstract":"<p><strong>Background: </strong>The integration of novel antibody-mediated targeted therapies into both relapsed/refractory (R/R) and frontline pediatric acute lymphoblastic leukemia (ALL) treatment protocols has led to critical advancements in the field. Current research efforts focus on optimizing targeted therapies to enhance precision and efficacy while minimizing toxicity by reducing chemotherapy. A notable example is the addition of blinatumomab, demonstrating superiority over conventional chemotherapy, with an 8% increase in disease-free survival at an interim analysis, reaching 96%. Inotuzumab ozogamicin (InO) has also shown promise, achieving nearly a 70% complete response rate in pediatric R/R B-cell ALL (B-ALL) trials. Additionally, daratumumab in T-cell ALL (T-ALL) and chimeric antigen receptor T-cell therapies, particularly CD19-directed (B-ALL) and CD7-directed (T-ALL) strategies, are under active investigation.</p><p><strong>Summary: </strong>This review will provide an overview of targeted antibody-mediated immunotherapies in both B-ALL and T-ALL, with a focus on their pediatric applications, supporting data, and future prospects.</p><p><strong>Key messages: </strong>The next cycle of frontline trials in pediatric ALL will incorporate more immunotherapy with reduction of chemotherapy. Subsequent trials will utilize more concurrent chemoimmunotherapy blocks as precision testing and risk-adapted therapy will continue to develop. These advancements reflect a paradigm shift toward more precise, less toxic treatment strategies in pediatric ALL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143956843","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}
Philipp le Coutre, Andreas Burchert, Susanne Saußele, Thekla Schwarzer, Sebastian Stintzing, Uwe Pelzer, Lars Bullinger, Ahmet Elmaagacli, Christian Jehn, Frank Stegelmann, Andreas Hochhaus, Thomas Ernst, Joachim R Göthert
{"title":"Favorable Rates of Cardiovascular Events with Stringent Cardiovascular Monitoring and a Lowered Dose of Ponatinib as Second-Line Treatment in Chronic Phase Chronic Myeloid Leukemia Patients Failing or Intolerant to First-Line Second-Generation Tyrosine Kinase Inhibitor Treatment: Results of the Prospective PONS Trial.","authors":"Philipp le Coutre, Andreas Burchert, Susanne Saußele, Thekla Schwarzer, Sebastian Stintzing, Uwe Pelzer, Lars Bullinger, Ahmet Elmaagacli, Christian Jehn, Frank Stegelmann, Andreas Hochhaus, Thomas Ernst, Joachim R Göthert","doi":"10.1159/000545826","DOIUrl":"10.1159/000545826","url":null,"abstract":"<p><strong>Introduction: </strong>In chronic myeloid leukemia patients, second-line treatment requires careful consideration of response and tolerability. As most patients need a more efficient tyrosine kinase inhibitor, ponatinib at a lowered dose should be evaluated in this setting.</p><p><strong>Methods: </strong>We studied a lowered dose of 30 mg ponatinib in second line in patients selected toward a low cardiovascular risk. In 22 screened patients, ponatinib was started in 18 patients previously treated with imatinib (n = 3), dasatinib (n = 9), or nilotinib (n = 6). Patients were frequently monitored for cardiovascular toxicities by testing of blood pressure, vital signs, ankle brachial index or duplex, oral glucose tolerance test, echocardiography, ECG, and fundoscopy. The study protocol allowed dose reductions in patients achieving MMR. Both previously resistant or intolerant patients were recruited.</p><p><strong>Results: </strong>No serious cardiovascular events were observed, and low-grade cardiovascular toxicity was negligible. By 12 months, 13 patients (92.9%) were in complete hematologic remission, 10 patients (55.6%) were in MMR, and 5 patients (27.8%) were in MR4. Most importantly, we demonstrated that thorough monitoring of cardiovascular risk is feasible.</p><p><strong>Conclusions: </strong>We demonstrated that a lowered dose of 30 mg ponatinib in selected patients can be maintained without serious cardiovascular complications, provided cardiovascular risk monitoring is performed. In our patient cohort, this approach resulted in favorable response rates.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":1.7,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143958017","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}
Karin Mayer, Leonie Birk, Katjana Schwab, Ingo G H Schmidt-Wolf, Marie von Lilienfeld-Toal, Peter Brossart, Axel Glasmacher, Corinna Hahn-Ast
{"title":"Dose Reduced Induction Therapy (A)-AIDA in Patients with Acute Myeloid Leukemia, Being Unfit for Standard 7+3 Chemotherapy and Literature Review of Acute Myeloid Leukemia Treatment in Unfit/Older AML Patients.","authors":"Karin Mayer, Leonie Birk, Katjana Schwab, Ingo G H Schmidt-Wolf, Marie von Lilienfeld-Toal, Peter Brossart, Axel Glasmacher, Corinna Hahn-Ast","doi":"10.1159/000545792","DOIUrl":"10.1159/000545792","url":null,"abstract":"<p><strong>Introduction: </strong>The standard induction chemotherapy in newly diagnosed acute myeloid leukemia (AML) is 7 days cytarabine and 3 days daunorubicin. However, older and frail patients cannot be treated intensively. Before hypomethylating agents (HMA) plus venetoclax (VEN) was established we formulated a more tolerable induction therapy for these patients using continuous infusions of reduced doses of cytarabine (A) and idarubicin (IDA) in combination with ATRA (A), the (A)-AIDA regimen and trying to reach noteworthy and relevant remission rates with acceptable toxicities in this special population.</p><p><strong>Methods: </strong>Between 1998 and 2015 older/frail patients with newly diagnosed or relapsed AML received (A)-AIDA. Treatment consisted of cytarabine 100 mg/m2/d d1-5 and IDA 5 mg/m2/d d1-5 as continuous infusion. Since 2003 ATRA (45 mg/m2/d d4-6, 15 mg/m2/d d7-28) was added in some patients.</p><p><strong>Results: </strong>154 patients received (A)-AIDA, median age was 71 years. In 40% the AML was secondary, 15.6% had relapsed AML. The complete remission rate was 41%, 7% reached a partial remission, the median overall survival was 7 months. No significant differences of remission rates regarding various known risk factors for outcome with (A)-AIDA were detected.</p><p><strong>Conlusion: </strong>Recent studies established HMA/VEN as first-line treatment for older/frail AML patients. Because (A)-AIDA is equally effective in patients with secondary or relapsed AML or ECOG >1, this regimen is an interesting treatment option for patients uneligible to HMA/VEN regimens.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-13"},"PeriodicalIF":1.7,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143954978","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":"Emicizumab and Acquired Hemophilia A Secondary to Multiple Myeloma.","authors":"Florence Cuschera, Marie-Christiane Vekemans, Cedric Hermans","doi":"10.1159/000545442","DOIUrl":"10.1159/000545442","url":null,"abstract":"<p><strong>Introduction: </strong>Acquired hemophilia A (AHA) is a severe bleeding disorder, sometimes linked to plasma cell dyscrasias. In this context, emicizumab, a bispecific antibody, provides stable hemostasis by mimicking factor VIII (FVIII), offering convenience and flexibility compared to bypassing agents. Moreover, targeted anti-myeloma therapy directly addresses the underlying plasma cell disorder, potentially achieving better and more durable control of AHA than conventional immunosuppressive therapy, while reducing its associated adverse effects.</p><p><strong>Case presentation: </strong>We describe the successful management of AHA secondary to multiple myeloma (MM) using emicizumab and targeted anti-myeloma therapy. The patient initially responded to bortezomib-dexamethasone but required teclistamab due to disease progression. Emicizumab maintained hemostatic stability, allowing time for effective MM management.</p><p><strong>Conclusion: </strong>Emicizumab, in conjunction with targeted myeloma treatment, represents a promising strategy to improve outcomes for AHA patients with MM. This approach is particularly advantageous for older patients with multiple comorbidities, who face elevated risks of thrombotic, bleeding, and infectious complications.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-7"},"PeriodicalIF":1.7,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143959199","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}
Florian Bouclet, Juliette Pénichoux, Sorina-Dana Mihailescu, Stéphanie Seris, Lucie Burel, Doriane Richard, Sabine Collard, Emmanuelle Jennet, Elisabeth Bertrand, Mustafa Alani, Sydney Dubois, Anne-Lise Ménard, Vincent Camus, Aspasia Stamatoullas, Pascal Lenain, Nathalie Contentin, Stéphane Leprêtre, Hervé Tilly, Emilie Lemasle, Hélène Lanic, Mikael Daouphars, Louis-Ferdinand Pépin, Sébastien Moussay, Fabrice Jardin
{"title":"Concordance between Tympanic and Enteral Temperature for Early Fever Detection in Hematologic Intensive Care Unit Patients: A Prospective Study.","authors":"Florian Bouclet, Juliette Pénichoux, Sorina-Dana Mihailescu, Stéphanie Seris, Lucie Burel, Doriane Richard, Sabine Collard, Emmanuelle Jennet, Elisabeth Bertrand, Mustafa Alani, Sydney Dubois, Anne-Lise Ménard, Vincent Camus, Aspasia Stamatoullas, Pascal Lenain, Nathalie Contentin, Stéphane Leprêtre, Hervé Tilly, Emilie Lemasle, Hélène Lanic, Mikael Daouphars, Louis-Ferdinand Pépin, Sébastien Moussay, Fabrice Jardin","doi":"10.1159/000544693","DOIUrl":"10.1159/000544693","url":null,"abstract":"<p><strong>Introduction: </strong>Early detection of fever is crucial in neutropenic patients. Lack of precision in body temperature measurement can occur with peripheral site measurements. Furthermore, intermittent temperature monitoring may result in a delay in fever detection.</p><p><strong>Methods: </strong>We conducted a prospective study in hematologic intensive care unit (HICU) patients receiving autologous stem cell transplant or intensive chemotherapy in order to compare tympanic and enteral temperature measured by an ingested electronic pill (BodyCap®, France).</p><p><strong>Results: </strong>Twenty-six patients ingested at least one capsule with a total of 218 days of tympanic and enteral simultaneous measurement. In 12% of these measurements, we identified a difference over 0.5°C between the left ear versus right ear measurements. Enteral temperature was usually higher than the tympanic one (p < 0.0001) with 14% of the measurements that were discordant with higher enteral temperature and 1.9% that were discordant with higher tympanic temperature. Febrile episodes were detected with the ingestible pill on average almost 24 h earlier than with the tympanic measurement. Early capsule evacuation occurred in 20.4% of the cases, mainly because of diarrhea, a frequent adverse event in these patients. Patients were satisfied with capsule ingestion according to a questionnaire.</p><p><strong>Conclusion: </strong>In this prospective trial (TEMPET), we demonstrated that enteral temperature measured by an ingested pill is feasible in HICU and detects fever earlier than tympanic routine measurements. Digestive symptoms related to hematological disease and/or treatments are limiting factors for its generalized usage.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":1.7,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143957940","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}
Adi Sherban, Avraham Frisch, Alon Rozenthal, Shira Buchrits, Baha Atamna, Daniel Ben-Ner, Sivan Eden, Ala Atamna, Yishai Ofran, Pia Raanani, Ofir Wolach
{"title":"Epiglottitis in Patients Treated for Acute Leukemia: Case Series and Systematic Review of the Literature.","authors":"Adi Sherban, Avraham Frisch, Alon Rozenthal, Shira Buchrits, Baha Atamna, Daniel Ben-Ner, Sivan Eden, Ala Atamna, Yishai Ofran, Pia Raanani, Ofir Wolach","doi":"10.1159/000545927","DOIUrl":"10.1159/000545927","url":null,"abstract":"<p><strong>Introduction: </strong>Acute epiglottitis is a medical emergency characterized by inflammation of supraglottic structures of the larynx and epiglottis. The clinical characteristics and course of this complication in acute leukemia (AL) are largely unknown. We present a case series and a systematic review of the literature of adult patients with AL and acute epiglottitis.</p><p><strong>Case presentation: </strong>Four patients with acute myeloid leukemia (age 38-61 years) were diagnosed with acute epiglottitis during their intensive treatment course. One patient presented with epiglottitis at the end of induction, two at the end of consolidation therapy, and 1 patient presented with a later event. Three patients were in grade 4 neutropenia during infection onset (median aplasia duration to event 9 [range 4-14] days). All patients presented with neck pain and dysphagia and 2 patients also had dyspnea at presentation. Urgent invasive airway protection was required in 3 patients. Two patients died due to this complication, one suffered anoxic brain injury. A systematic literature review identified 8 additional cases of epiglottitis.</p><p><strong>Conclusion: </strong>Acute epiglottis is a rare but significant medical emergency with unique challenges in the setting of AL that is caused by atypical infectious pathogens. Early detection and a multidisciplinary therapeutic effort are crucial to improve patient outcome.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12240564/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144054035","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}
Jingyi Bi, Xuelin Dou, Ruqi Liang, Lei Wen, Yang Liu, Xiaoguang Lei, Liru Wang, Huixia Guo, Xiaojun Huang, Mingdi Wang, Jin Lu
{"title":"Tucidinostat Combined with Bortezomib, Liposomal Doxorubicin, and Dexamethasone in Multiple Myeloma Treatment.","authors":"Jingyi Bi, Xuelin Dou, Ruqi Liang, Lei Wen, Yang Liu, Xiaoguang Lei, Liru Wang, Huixia Guo, Xiaojun Huang, Mingdi Wang, Jin Lu","doi":"10.1159/000545709","DOIUrl":"10.1159/000545709","url":null,"abstract":"<p><strong>Introduction: </strong>We conducted a single-arm, open-label dose-exploration study to evaluate the safety and efficacy of the histone deacetylase inhibitor tucidinostat combined with bortezomib, liposomal doxorubicin, and dexamethasone (C-PDD) in treating relapsed and refractory multiple myeloma (RRMM) patients.</p><p><strong>Methods: </strong>Eighteen patients were enrolled from August 2020 to May 2021, receiving 21-day cycles of C-PDD.</p><p><strong>Results: </strong>Eighteen cases were analysed, with a median prior treatment line of 2 (range: 1-4). The median number of completed treatment cycles was 4 (range: 1-8). The overall response rate was 57%, including 14% complete response, 14% very good partial response, and 29% partial response. Both bortezomib-sensitive and refractory groups had a response rate of 57%. The response rate was 100% in patients with extramedullary extraosseous involvement. The median follow-up was 42 months (range: 3-44), with median progression-free survival of 7 months and median overall survival of 24.5 months. Grade 3-4 haematologic adverse events included thrombocytopaenia (50%), neutropenia (33%), and anaemia (33%). Non-haematologic adverse events were mostly grade 1-2, with 1 case of grade 3 peripheral sensory neuropathy.</p><p><strong>Conclusion: </strong>The C-PDD regimen showed efficacy in RRMM, including bortezomib-refractory disease and EME patients. The optimal dose and combination need to be explored in the future.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143964079","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}