Mediterranean Journal of Hematology and Infectious Diseases最新文献

{"title":"Chimeric Antigen Receptor T Cells for the Treatment of Multiple Myeloma.","authors":"Ugo Testa, Elvira Pelosi, Germana Castelli","doi":"10.4084/MJHID.2024.077","DOIUrl":"https://doi.org/10.4084/MJHID.2024.077","url":null,"abstract":"<p><p>Multiple myeloma (MM), characterized by abnormal proliferation of clonal plasma cells, is an incurable hematological malignancy. Various immunotherapy strategies have emerged as an efficacious approach for the treatment of MM, including monoclonal antibodies, antibody-drug conjugates, bispecific antibodies, and chimeric antigen receptor T (CAR-T) cells. Anti-B-cell maturation antigen (BCMA) CAR-T cells have revolutionized the treatment of MM patients with relapsed/refractory disease and their clinical use was approved for the treatment of these patients. Despite this progress, the efficacy of CAR-T cells in MM is limited by the responsiveness of only a part of the treated patients, the relapse of other patients, the cost of the treatment and the diminished response in patients with prior exposure to anti-BCMA targeting agents. Ongoing clinical trials are evaluating the use of CAR-T cells at an earlier stage of MM disease and the use of CAR-T cells targeting other membrane antigens expressed on malignant plasma cells.</p>","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024077"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11556426/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142624014","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":"Pediatric Hodgkin Lymphoma in Low- and Middle-Income Countries (LMICs). A Narrative Review.","authors":"Maria Luisa Moleti, Anna Maria Testi, Salma Al-Hadad, Mazin Faisal Al-Jadiry, Robin Foà","doi":"10.4084/MJHID.2024.078","DOIUrl":"https://doi.org/10.4084/MJHID.2024.078","url":null,"abstract":"<p><p>Pediatric Hodgkin lymphoma (HL) is a curable disease for more than 90% of children and adolescents in high-income countries. However, similar results cannot be achieved, particularly for advanced disease, in low- and middle-income countries (LMICs), where challenging socio-economic realities and the consequent scarcity of local resources heavily impact the treatment and patients' outcome. Information regarding the management and outcome of pediatric HL in LMICs is still limited. In this narrative review, we summarize the results reported in the literature so far and discuss the critical key points that have emerged from this overview.</p>","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024078"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11556425/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142635988","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":"The Performance of 2023 American College of Rheumatology (ACR) / European Alliance of Associations for Rheumatology (EULAR) Antiphospholipid Syndrome Classification Criteria in a Real-World Rheumatology Department.","authors":"Ahmet Usta, Müçteba Enes Yayla, Emine Uslu, Serdar Sezer, Ebru Us, Aşkın Ateş, Murat Turgay","doi":"10.4084/MJHID.2024.074","DOIUrl":"https://doi.org/10.4084/MJHID.2024.074","url":null,"abstract":"<p><strong>Background: </strong>Antiphospholipid Syndrome (APS) is one of the most common acquired causes of hypercoagulability. The 2023 American College of Rheumatology (ACR) / European Alliance of Associations for Rheumatology (EULAR) APS Classification Criteria were specified as new APS classification criteria with high specificity for use in observational studies and research. The primary objective of this study was to evaluate the performance of the 2023 ACR/EULAR APS classification criteria in a real-world rheumatology department.</p><p><strong>Methods: </strong>This is a retrospective, single-center study evaluating the sensitivity and specificity of the 2006 revised Sapporo and 2023 ACR/EULAR APS classification criteria in patients diagnosed with APS through clinical evaluation. A total of 184 patients, 103 of whom were diagnosed with APS, were included in the study.</p><p><strong>Results: </strong>The 2023 ACR/EULAR APS classification criteria demonstrate higher specificity 98.8% (95% CI 93.3-99.8) and positive predictive value (PPV) 98.7% (95% CI 93.2-99.8). The revised Sapporo criteria exhibit higher sensitivity 90.3% (95% CI 83-96.6), negative predictive value (NPV) 88.1% (95% CI 79.4-93.4), and accuracy 90.8% (95% CI 85.7-94.1). When the diagnosis of APS was accepted according to the revised Sapporo criteria, the sensitivity of the 2023 ACR/EULAR APS classification criteria was 77% (95% CI 67.8-84.2), specificity 97.6% (95% CI 91.7-99.3), PPV 97.5% (95% CI 69.3-84.9) and NPV 78.1% (95% CI 69.3-84.9).</p><p><strong>Conclusion: </strong>The 2023 ACR/EULAR APS classification criteria have low sensitivity and high specificity compared to the revised Sapporo APS classification criteria. The increase in specificity is due to risk assessment in thromboses and strict obstetric and laboratory criteria.</p>","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024074"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11556423/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142624022","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":"Older Adults with Ph Negative Acute Lymphoblastic Leukemia: A Monocentric Experience on 57 Patients Focusing on Treatment Intensity and Age-Related Prognosis.","authors":"Erika Borlenghi, Tatiana Zollner, Giuseppe Rossi, Chiara Pagani, Chiara Cattaneo, Michele Malagola, Alessandra Sottini, Diego Bertoli, Mariella Tonelli, Marco Chiarini, Rossella Leopaldo, Angela Passi, Lorenzo Masina, Francesca Federico, Carlotta Giupponi, Alessandra Tucci","doi":"10.4084/MJHID.2024.080","DOIUrl":"https://doi.org/10.4084/MJHID.2024.080","url":null,"abstract":"","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024080"},"PeriodicalIF":2.0,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11556428/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142624018","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":"Hb H Disease Caused by Uniparental Disomy: First Report of the αT-Saudiα Mutation in the Chinese Population.","authors":"Ge Wang,Hongting Xie,Jun Zhang,Peng Huang,Min Liang,Dina Zhu,Qianqian Zhang,Yuqiu Zhou,Xuan Shang","doi":"10.4084/mjhid.2024.069","DOIUrl":"https://doi.org/10.4084/mjhid.2024.069","url":null,"abstract":"","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"27 1","pages":"e2024069"},"PeriodicalIF":3.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142202572","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":"Anorectal Pathologies in the Course of Acute Leukaemias; Predictive Parameters.","authors":"Fatma Yilmaz,Bugra Saglam,Ufuk Gorduk,Ugur Kalan,Hacer Berna Afacan Ozturk,Ahmet Kursad Gunes,Murat Albayrak","doi":"10.4084/mjhid.2024.071","DOIUrl":"https://doi.org/10.4084/mjhid.2024.071","url":null,"abstract":"IntroductionPatients with leukaemia are exposed to infections as long as they are neutropenic. During this period, anorectal pathologies are among the common foci of infection with high mortality. In this study, we aim to investigate the factors that may have a predictive effect on early diagnosis and rapid intervention in perianal complications occurring in neutropenic patients diagnosed with leukaemia.Materials and MethodsA total of 90 patients with acute leukaemia, including 45 patients with anorectal pathology and 45 patients without anorectal pathology, were analysed. Demographics, blood group, BMI, haemogram and biochemical parameters at the time of diagnosis, and types of perianal pathology were recorded.ResultsIn the group of patients with anorectal pathology, WBC, lymphocytes, monocytes, and LDH were significantly (p<0.05) higher, and platelets, MPV, and PCT were significantly (p<0.05) lower. The multivariate model showed significant-independent (p<0.05) efficacy of WBC and MPV values in differentiating patients with and without anorectal pathology. A significant efficacy was observed at the WBC cut-off of 17000 [area under the curve 0.656 (0.542-0.770)] and the MPV cut-off of 10 [area under the curve 0.667 (0.554-0.780)] in differentiating patients with and without anorectal pathology.DiscussionAnorectal pathologies are common foci of infection in patients with acute leukaemia. Having predictive parameters that may help for early intervention will help the clinician. This is the first study in the literature to compare a control group with a group with anorectal pathologies in leukaemia patients providing a cut-off for WBC.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"14 1","pages":"e2024071"},"PeriodicalIF":3.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142202574","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":"Therapeutic Gene Editing for Hemoglobinopathies.","authors":"Ugo Testa, Giuseppe Leone, Maria Domenica Cappellini","doi":"10.4084/MJHID.2024.068","DOIUrl":"https://doi.org/10.4084/MJHID.2024.068","url":null,"abstract":"<p><p>In the last ten years, a consistent number of clinical studies have evaluated different gene approaches for the treatment of patients with sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Initial studies of gene therapy for hemoglobinopathies involved the use of lentiviral vectors to add functional copies of the gene encoding β-globin in defective CD34 cells; more recently, gene editing techniques have been used involving either CRISPR-Cas9, transcription activation-like effector protein nuclease, zinc finger nuclease, and base editing to either induce fetal hemoglobin production at therapeutic levels or to genetically repair the underlying molecular defect causing the disease. Here, we review recent gene editing studies that have started the development of a new era in the treatment of hemoglobinopathies and, in general, monoallelic hereditary diseases.</p>","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024068"},"PeriodicalIF":2.0,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11385271/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142290893","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":"CD146 Molecule Expression in B Cells Acute Lymphoblastic Leukemia (B-ALLs): A Flow-Cytometric Marker for an Accurate Diagnostic Workup.","authors":"Alessandro Laganà,Matteo Totaro,Maria Laura Bisegna,Loredana Elia,Stefania Intoppa,Marco Beldinanzi,Mabel Matarazzo,Mariangela di Trani,Alessandro Costa,Raffaele Maglione,Biancamaria Mandelli,Sabina Chiaretti,Maurizio Martelli,Maria Stefania De Propris","doi":"10.4084/mjhid.2024.064","DOIUrl":"https://doi.org/10.4084/mjhid.2024.064","url":null,"abstract":"BackgroundB-lineage acute lymphoblastic leukemias (B-ALL) harboring the t(9;22)(q34;q11)/BCR::ABL1 rearrangement represent a category with previously dismal prognosis whose management and outcome dramatically changed thanks to the use of tyrosine kinase inhibitors (TKIs) usage and more recently full chemo-free approaches. The prompt identification of these cases represents an important clinical need.ObjectivesWe sought to identify an optimized cytofluorimetric diagnostic panel to predict the presence of Philadelphia chromosome (Ph) in B-ALL cases by the introduction of CD146 in our multiparametric flow cytometry (MFC) panels.MethodsWe prospectively evaluated a total of 245 cases of newly diagnosed B-ALLs with a CD146 positivity threshold >10% referred to the Division of Hematology of 'Sapienza' University of Rome. We compared the results of CD146 expression percentage and its mean fluorescence intensity (MFI) between Ph+ ALLs, Ph-like ALLs, and molecularly negative ALLs.ResultsSeventy-nine of the 245 B-ALL cases (32%) did not present mutations at molecular testing, with 144/245 (59%) resulting in Ph+ ALL and 19/245 (8%) Ph-like ALLs. Comparing the 3 groups, we found that Ph+ B-ALLs were characterized by higher expression percentage of myeloid markers such as CD13, CD33, and CD66c and low expression of CD38; Ph+ B-ALL showed a higher CD146 expression percentage and MFI when compared with both molecular negative B-ALL and Ph-like ALLs; neither the mean percentage of CD146 expression neither CD146 MFI were statically different between molecular negative B-ALL and Ph-like ALLs.ConclusionsOur data demonstrate the association between CD146 expression and Ph+ ALLs. CD146, along with myeloid markers, may help to identify a distinctive immunophenotypic pattern, useful for rapid identification in the diagnostic routine of this subtype of B-ALLs that benefits from a specific therapeutic approach.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"4 1","pages":"e2024064"},"PeriodicalIF":3.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142202580","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":"Blinatumomab in the Therapy of Acute B-Lymphoid Leukemia.","authors":"Ugo Testa,Elvira Pelosi,Germana Castelli,Patrizia Chiusolo","doi":"10.4084/mjhid.2024.070","DOIUrl":"https://doi.org/10.4084/mjhid.2024.070","url":null,"abstract":"Blinatumomab, a CD19-CD3 bispecific T cell engager (BiTE), has two recombinant single-chain variable fragments that temporarily link CD3+ T cells and CD19+ B cells, leading to the T cell-mediated lysis of neoplastic B cells. Improved minimal residual disease (MRD)-negative response rates and long-term overall survival have been observed in B-ALL patients who received this drug. These therapeutic successes have led to FDA approval for refractory/relapsed and MRD-positive B-ALL patients. Furthermore, recent studies in newly diagnosed B-ALL patients have led in Philadelphia chromosome-positive patients to the development of chemotherapy-free regimens based on tyrosine kinase inhibitors plus Blinatumomab and in Philadelphia chromosome-negative patients to improvement in outcomes using chemotherapy regimens that have incorporated Blinatumomab in the consolidation phase of treatment.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"20 1","pages":"e2024070"},"PeriodicalIF":3.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142202571","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":"MPN/MDS Overlap Syndrome Anticipated by a Severe Bleeding Diathesis: Hypothesis of a Preexisting Platelet Disorder.","authors":"Paola Ranalli,Stefano Baldoni,Daniela Bruno,Mauro Di Ianni","doi":"10.4084/mjhid.2024.067","DOIUrl":"https://doi.org/10.4084/mjhid.2024.067","url":null,"abstract":"","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"11 1","pages":"e2024067"},"PeriodicalIF":3.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142202573","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}