Clinical hematology international最新文献

{"title":"Genetic and Clinical Progression of MYSM1 Related Bone Marrow Failure into Myeloid Malignancies: Case Series and Review of Literature.","authors":"Alfadil Haroon, Syed Osman Ahmed, Chokri Ben Lamine, Mahmoud Aljurf, Hazzaa Alzahrani","doi":"10.46989/001c.138314","DOIUrl":"10.46989/001c.138314","url":null,"abstract":"<p><p>MYSM1, located on chromosome 1p32.1, encodes histone H2A deubiquitinase, a transcription regulator involved in DNA damage response. Biallelic MYSM1 variants are linked to rare bone marrow failure syndromes, presenting with cytopenia, B-cell deficiency, hypogammaglobulinemia, and developmental abnormalities. We report four cases of MYSM1 mutations progressing from marrow failure to MDS or AML within 9-10 years. Genetic abnormalities, including TP53 mutation and chromosomal anomalies, suggest clonal evolution. Hematopoietic stem cell transplantation achieved remission in two patients with adverse cytogenetics. Further research is needed to refine management strategies and assess long-term outcomes in MYSM1-associated marrow failure and MDS.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 2","pages":"46-54"},"PeriodicalIF":0.0,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12176283/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144328128","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Anemia Management on Bleeding Outcomes in Anticoagulated Patients: A Retrospective Cohort Analysis.","authors":"Shea-Lee Godin, Christopher Hanna, Edgar Naut, Sudhanshu Mulay","doi":"10.46989/001c.138102","DOIUrl":"10.46989/001c.138102","url":null,"abstract":"<p><p>Anticoagulation therapy is essential to manage thromboembolic conditions such as atrial fibrillation and venous thromboembolism. While effective, it carries significant bleeding risks, with annual rates ranging from 10-17% for all events and 2-5% for major bleeding. Anemia is an independent risk factor for anticoagulation-associated bleeding; however, guidelines lack recommendations for anemia screening and management before initiation. In a retrospective analysis of 170 anticoagulated patients (mean age 63.7; 96 males, 74 females), 51.2% had baseline anemia. Anemia severity was significantly associated with bleeding events (χ²=15.7, p=0.003). Multivariate analysis confirmed that moderate (aOR=0.26, 95% CI:0.08-0.82, p=0.021) and no anemia (aOR=0.42, 95% CI:0.22-0.82, p=0.011) were associated with lower bleeding risk than mild anemia, while severe anemia remained uninterpretable due to small sample size. Patients aged ≥65 had higher bleeding risk (OR=2.8, 95% CI:1.5-5.1, p<0.01), though this did not reach significance in multivariate analysis (aOR=1.80, 95% CI:0.95-3.41, p=0.073). Multivariate analysis confirmed higher bleeding risks for warfarin (aOR=4.13, 95% CI:1.91-8.96, p<0.001) and rivaroxaban (aOR=3.67, 95% CI:1.69-7.97, p=0.001) compared to apixaban. Our study found an association between anemia and bleeding events, though severe anemia did not correlate with bleeding, possibly due to small sample size. Direct oral anticoagulants like apixaban and rivaroxaban present lower bleeding risks than warfarin. Given anemia's role in bleeding risk, we recommend routine screening before initiating anticoagulation to improve patient safety. Early assessment may help reduce bleeding complications, particularly in high-risk populations. Future studies should focus on multi-center trials to validate these findings and explore anemia subtypes.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 2","pages":"34-45"},"PeriodicalIF":0.0,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12121077/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144183164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Relapsed/refractory multiple myeloma: standard of care management of patients in the Gulf region.","authors":"Ahmad Alhuraiji, Khalil Al Farsi, Kayane Mheidly, Hesham Elsabah, Honar Cherif, Anas Hamad, Mahmoud Marashi, Hussni Al Hateeti, Hani Osman, Mohamad Mohty","doi":"10.46989/001c.137860","DOIUrl":"https://doi.org/10.46989/001c.137860","url":null,"abstract":"<p><p>Clinical management of patients with relapsed/refractory multiple myeloma (RRMM) can be challenging, whereby each relapse is associated with progressively poorer outcomes. In addition, changes in disease biology and patient characteristics hamper treatment strategies in this setting, as do toxicities accumulated across previous lines of therapy. The availability of several new treatment classes has brought about improvements in outcomes, but with median survival in the RRMM setting at only ~32 months, a review of current standard of care treatments and considerations for optimizing care in this setting is warranted. Here, we discuss our preferred approach to treating patients with RRMM, based on our collective experience across the Gulf region. We present position statements for the treatment of lenalidomide-sensitive and -refractory patients, as well as for those patients experiencing late relapse. We discuss the major impact that anti-CD38 agents daratumumab and isatuximab have had on the management of RRMM, which is reflected in our preferred use of daratumumab-based regimens across the lenalidomide-sensitive and -refractory settings. For late-relapse settings, we discuss how bispecific antibodies and chimeric antigen receptor [CAR]-T cells are among the biggest breakthroughs in recent years, achieving excellent responses in triple-class exposed patients. While the use of these agents is not yet widespread in the Gulf region, we advocate their use where available and discuss strategies to manage and minimize common toxicities and adverse events associated with their use.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 2","pages":"20-33"},"PeriodicalIF":0.0,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12065471/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144044697","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Consensus Guidelines and Recommendations for The CD38 Monoclonal Antibody-based Quadruplet Therapy and Management in Clinical Practice for Newly Diagnosed Multiple Myeloma: From the Pan-Pacific Multiple Myeloma Working Group.","authors":"Wenming Chen, Zhen Cai, James Cs Chim, Wee Joo Chng, Juan Du, Chengcheng Fu, Ichiro Hanamura, Jian Hou, Jeffrey Shang-Yi Huang, Tadao Ishida, Aijun Liu, Vadim Ptushkin, Anastasiya Semenova, Naoki Takezako, Raymond Siu Ming Wong","doi":"10.46989/001c.133682","DOIUrl":"https://doi.org/10.46989/001c.133682","url":null,"abstract":"<p><p>The therapeutic outcomes of clinical trials for incorporating anti-CD38 monoclonal antibodies (including isatuximab and daratumumab) into the bortezomib/lenalidomide/dexamethasone (VRd) triplet therapy backbone as the first-line treatment for newly diagnosed multiple myeloma (NDMM) have demonstrated significant improved efficacies. From a safety perspective, the addition of anti-CD38 monoclonal antibodies into the triplet therapies did not raise additional safety concerns. Based on the promising results, the National Comprehensive Cancer Network (NCCN) Guidelines Version 1.2025 had updated the quadruplet therapy incorporating anti-CD38 monoclonal antibodies with VRd-based therapies as the primary therapy for both transplantation-eligible and transplantation-ineligible NDMM patients. Thus, a panel of experts in hematology and oncology with extensive experience in the treatment of NDMM was convened in 2024 to develop consensus recommendations based on recent evidence from pivotal clinical trials and real-world practices, providing clear guidance for optimizing treatment strategies in both transplantation-eligible and transplantation-ineligible patients. The main topics identified for discussion and recommendation were: (i) the benefits and indications for quadruplet therapy for NDMM; (ii) the optimization of quadruplet therapy strategies; (iii) the management and monitoring of potential adverse events for quadruplet therapy, and (iv) the impact of quadruplet regimens on tandem stem cell transplantation and maintenance treatment. Recommendations were then presented to the entire panel for further discussion and amendment before voting. This manuscript presents the recommendations developed, including findings from the expert panel discussions, consensus recommendations and a summary of evidence supporting each recommendation.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 2","pages":"1-19"},"PeriodicalIF":0.0,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12013828/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144000136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"It is time to consider the climate crisis in haematology.","authors":"Robin Noel, Aude Charbonnier, Bérénice Schell, Arthur Dony, Charles Toulemonde, François Eisinger, Olivier Decaux, Joanna Lotocka, Edith Julia, Alya Perthus, Mathilde Seguin, Aurélie Cabannes-Hamy, Pierre Sujobert, Laurie Marrauld, Caroline Besson","doi":"10.46989/001c.133524","DOIUrl":"10.46989/001c.133524","url":null,"abstract":"","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 1","pages":"55-59"},"PeriodicalIF":0.0,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11954563/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143756469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prolonged Cytopenia with CAR-T Cell Therapy and Management Recommendations.","authors":"Debolanle Dahunsi, Cynthia Eleanya, Akintomiwa Akintunde, Olalekan Oluwole","doi":"10.46989/001c.126463","DOIUrl":"10.46989/001c.126463","url":null,"abstract":"<p><p>Chimeric antigen receptor T-cell (CAR T-cell) therapy has revolutionized the treatment of lymphoid malignancies. Prolonged cytopenias, though poorly understood, have emerged as important considerations in the treatment process. In this review, we classified cytopenias into early (< 30 days post CAR T infusion), and late-occurring (after day 30 post infusion). We identified previous chemotherapy and lymphodepletion chemotherapy as the major risk factors contributing to early cytopenia. Product characteristics, such as costimulatory domains, and side effects of therapy such as cytokine release syndrome (CRS) and immune effector cell associated neurotoxicity syndrome (ICANS) were identified as contributing factors to prolonged cytopenias occurring more than 30 days post CAR-T infusion. We recommend close monitoring with frequent checks, enhanced care with granulocyte colony stimulating factor (GCSF) support for grade 3-4 neutropenia, blood transfusion for severe anemia (Hb < 7g/dL), platelets for severe thrombocytopenia (< 10,000/µL) and thrombopoietin (TPO) mimetics such as eltrombopag or romiplostim for prolonged severe thrombocytopenia in patients at high-risk of hemorrhagic complications.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 1","pages":"47-54"},"PeriodicalIF":0.0,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11952652/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143756471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of primary letermovir prophylaxis for cytomegalovirus infection in paediatric patients undergoing allogeneic transplantation: a single-centre, retrospective, real-world analysis.","authors":"Xin Wang, Chaoqian Jiang, Lipeng Liu, Xia Chen, Yuanyuan Ren, Yang Wan, Aoli Zhang, Xiaoyan Zhang, Yue Shang, Yao Zou, Xiaojuan Chen, Fang Liu, Wenyu Yang, Xiaofan Zhu, Ye Guo","doi":"10.46989/001c.131683","DOIUrl":"https://doi.org/10.46989/001c.131683","url":null,"abstract":"<p><strong>Background: </strong>Cytomegalovirus (CMV) infection is a common and life-threatening complication following allogeneic haematopoietic stem cell transplantation (allo-HSCT). Letermovir (LET) has been the standard prophylaxis for adult recipients, but studies in children remain limited.</p><p><strong>Methods: </strong>We retrospectively analyzed children with or without LET prophylaxis after haploidentical donor (HID) for the Beijing protocol or unrelated cord blood (UCB) transplantation.</p><p><strong>Results: </strong>Of the 151 patients, 67 received LET, including 35 HID recipients and 32 UCB recipients. During the 180 days after transplantation, we found that the LET group had a lower incidence of clinically significant CMV infection (csCMVi) than the non-LET group (13.4% vs. 56.0%, P＜0.001). In the LET group, later LET administration was identified as a risk factor for the occurrence of csCMVi (HR: 1.07, 95% CI: 1.01 - 1.14, P=0.029). Further, the HID subgroup had a lower incidence of csCMVi during follow-up than the UCB subgroup (2.9% vs. 25.0%, P=0.009). In terms of safety, the incidence and severity of adverse events, overall survival, cumulative incidence of relapse, relapse free survival, nonrelapse mortality and graft versus host disease-free, relapse-free survival were similar between the two groups.</p><p><strong>Conclusion: </strong>LET is effective and safe in preventing csCMVi among Chinese children undergoing allo-HSCT. Compared to UCB recipients, children undergoing HID transplantation for the Beijing protocol develop less scCMVi up to 180 days post-HSCT.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 1","pages":"36-46"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11910970/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143652426","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Therapeutics of acute myeloid leukemia with central nervous system involvement.","authors":"Edwin U Suárez, Tamara Castaño-Bonilla, Rocio Salgado, Laura Solán, Alberto Lázaro-García, Juan M Alonso-Domínguez","doi":"10.46989/001c.131722","DOIUrl":"10.46989/001c.131722","url":null,"abstract":"<p><p><i>FLT3</i>-mutated acute myeloid leukemia (AML) with central nervous system (CNS) involvement poses therapeutic challenges. We describe two cases and performed a systematic review evaluating the efficacy of therapeutic strategies in CNS involvement for both <i>FLT3</i>-mutated and wild-type (WT) AML. A MEDLINE, EMBASE, and Cochrane literature search identified relevant studies. Although CNS involvement in AML is associated with poor prognosis, routine CNS prophylaxis is not standard. Due to the uncertainty regarding the effect of intermediate doses of cytarabine on CNS involvement, we support a diagnostic lumbar puncture (LP) after achieving complete remission in patients with risk factors for CNS infiltration. Consolidation management should be modified depending on the result of the LP. The impact of total body irradiation (TBI) as a conditioning regimen in allogeneic stem cell transplantation on CNS AML outcomes remains ambiguous. Routine craniospinal irradiation is not recommended due to its associated higher morbidity rates, while cranial radiotherapy is preferred, particularly when combined with TBI. Fortunately, currently we can employ a FLT3 inhibitor with CNS penetrance in <i>FLT3</i>-mutated (either gilteritinib or sorafenib) or <i>FLT3</i>-WT (sorafenib) AML patients.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 1","pages":"40-46"},"PeriodicalIF":0.0,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906164/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143627064","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Frontline management of multiple myeloma patients: optimizing treatment for patients in the Gulf region.","authors":"Mahmoud Marashi, Khalil Al Farsi, Hussni Al Hateeti, Ahmad Alhuraiji, Hesham Elsabah, Honar Cherif, Anas Hamad, Kayane Mheidly, Hani Osman, Mohamad Mohty","doi":"10.46989/001c.128113","DOIUrl":"10.46989/001c.128113","url":null,"abstract":"<p><p>Treatment options for newly diagnosed multiple myeloma (NDMM) have expanded dramatically over the last two decades, resulting in remarkable improvements in response rates and median survival times. In eligible patients, autologous stem cell transplant plays the central role of an overall treatment strategy comprising induction, transplantation, consolidation, and maintenance. In this article, we draw from our own collective clinical experience of treating patients with NDMM in the Gulf region to discuss treatment strategies in both transplant-eligible and -ineligible patients, as well as in high-risk patients. We present position statements for these distinct patient populations specifically for treatment in the Gulf region, where patients with NDMM have a younger median age than and different comorbidity profile from Western populations. We discuss how the introduction of anti-CD38 agents, including daratumumab and isatuximab, have had a major impact on the frontline treatment landscape in MM, with daratumumab-based quadruplet and triplet regimens emerging as the new standard of care in transplant-eligible and -ineligible patients, respectively. In addition, we advocate aggressive quadruplet treatment of high-risk patients with NDMM, as part of a strategy including single or tandem transplant when eligible. Finally, we discuss the clinical and practical rationale behind our statements, which is intended to serve as a useful reference for hematologists treating physicians within the Gulf region and beyond.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 1","pages":"14-28"},"PeriodicalIF":0.0,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11820852/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143411965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How to perform a high-quality peer review.","authors":"Mohamad Mohty, Junia V Melo","doi":"10.46989/001c.128601","DOIUrl":"https://doi.org/10.46989/001c.128601","url":null,"abstract":"<p><p>High-quality peer review is a cornerstone of credible and impactful scientific and medical publishing. This manuscript provides a comprehensive overview of the best practices, responsibilities, and evaluation criteria for peer reviewers in clinical and translational research. By adhering to high standards of objectivity, rigor and professionalism, peer reviewers support the integrity of scientific research and contribute to the evolution of evidence-based medicine. We elaborate on the principles and structured processes that ensure a thorough, impartial review, aiming to guide reviewers in producing evaluations that enrich the scientific discourse and foster innovation in clinical practice.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"7 1","pages":"10-13"},"PeriodicalIF":0.0,"publicationDate":"2025-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11797007/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143257543","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}