Acta Haematologica最新文献

{"title":"Addendum to the German Consensus Recommendations on Ponatinib in the Treatment of Chronic Myeloid Leukemia.","authors":"Susanne Saussele, Paul La Rosée, Alexander Kiani, Wilhelm Haverkamp, Kathleen Jentsch-Ullrich, Frank Stegelmann, Christina Rieger, Cornelius F Waller, Georg-Nikolaus Franke, Christian Junghanss, Rudolf Kirchmair, Markus Theurl, Philipp le Coutre","doi":"10.1159/000533666","DOIUrl":"10.1159/000533666","url":null,"abstract":"<p><strong>Background: </strong>Based on the new data from the primary analysis of the OPTIC (Optimizing Ponatinib Treatment in CP-CML) trial on dose optimization of ponatinib in patients with chronic phase (CP)-CML, the German consensus paper on ponatinib published in 2020 (Saussele S et al., Acta Haematol. 2020) has been updated in this addendum.</p><p><strong>Summary: </strong>Focus is on the update of efficacy and safety of ponatinib, reflecting the new data set, as well as the update of the benefit-risk assessment and recommendations for ponatinib starting dose in CP-CML - provided that the decision to use ponatinib has already been made. Furthermore, based on OPTIC and additional empirical data, the expert panel collaborated to develop a decision tree for ponatinib dosing, specifically for intolerant and resistant patients. The recommendations on cardiovascular management have also been updated based on the most recent 2021 guidelines of the European Society of Cardiology (ESC) on cardiovascular disease prevention in clinical practice.</p><p><strong>Key messages: </strong>The OPTIC data confirm the high efficacy of ponatinib in patients with CP-CML and provide the basis for individualized dose adjustment during the course of treatment.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"344-351"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11152025/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41231535","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":"Long Non-Coding RNA RP11-252C15.1 Is a Potential Biomarker of Prognosis and Hallmark for Leukemogenesis in Children with B-Cell Precursor Acute Lymphoblastic Leukemia.","authors":"Lili Pan, Yongzhi Zheng, Hao Zheng","doi":"10.1159/000535461","DOIUrl":"10.1159/000535461","url":null,"abstract":"<p><strong>Introduction: </strong>Improved understanding of the prognostic biomarkers associated with childhood acute lymphoblastic leukemia (ALL) is needed for accurate risk group stratification. This study aimed to identify potential long non-coding RNA (lncRNA) markers and evaluate their prognostic value in children with ALL.</p><p><strong>Methods: </strong>We selected 50 children with newly diagnosed ALL and 20 age-matched patients with idiopathic immune thrombocytopenia (controls). RNA sequencing was performed to identify differentially expressed lncRNAs between the ALL and control groups. Correlation analysis was performed to determine the relationships between candidate lncRNAs, clinical features, and the risk of leukemogenesis.</p><p><strong>Results: </strong>A total of 1,019 differentially expressed lncRNAs were identified between the ALL and control groups. Reverse transcriptase (RT-qPCR) revealed that lncRNA RP11-252C15.1 and lncRNA RP11-701P16.2 were significantly upregulated in patients with ALL. Furthermore, correlation analysis showed that lncRNA RP11-252C15.1 and lncRNA RP11-701P16.2 represent potential predictors of leukemogenesis; however, only lncRNA RP11-252C15.1 was associated with clinical features and outcome in children with B-cell precursor ALL (BCP-ALL). In vitro experiments confirmed that lncRNA RP11-252C15.1 was significantly overexpressed in BCP-ALL cell lines and promoted proliferation and repressed apoptosis in MHH-CALL-3 cells.</p><p><strong>Conclusion: </strong>lncRNA RP11-252C15.1 is a potential oncogene in BCP-ALL pathogenesis and a prognostic biomarker in children with BCP-ALL.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"646-660"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140287962","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":"Lenalidomide Treatment of Isolated Central Nervous System Relapse in Acute Lymphoblastic Leukemia after Hematopoietic Stem Cell Transplantation and Chimeric Antigen Receptor T-Cell Therapy.","authors":"Qing Zhang, Yi Dong, Zhimin Zhai, Lili Tao, Qianshan Tao","doi":"10.1159/000537719","DOIUrl":"10.1159/000537719","url":null,"abstract":"<p><strong>Introduction: </strong>Hematopoietic stem cell transplantation (HSCT) and chimeric antigen receptor T (CAR-T) cell are effective treatments for acute lymphoblastic leukemia (ALL). Various forms of intra- and extramedullary relapses have been reported after HSCT and CAR-T-cell therapy for ALL; however, no reports have investigated isolated central nervous system (CNS) relapse after HSCT and CAR-T-cell therapy. Hence, no clinical treatment has been established for such rare patients.</p><p><strong>Case presentation: </strong>An 18-year-old male patient with B-cell ALL suffered from isolated CNS relapse after HSCT and CAR-T-cell therapy. Conventional systemic intravenous and intrathecal chemotherapies were ineffective and intolerable. A unique immunosuppressive microenvironment of decreasing NK cell percentage and increasing IL-8 concentration and CAR-T-cell exhaustion had been illustrated in the cerebrospinal fluid. Finally, the patient received immunomodulatory therapy with lenalidomide and obtained complete remission.</p><p><strong>Conclusion: </strong>Lenalidomide might be a therapeutic strategy for isolated CNS relapse after HSCT and CAR-T-cell therapy.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"592-597"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139740144","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":"Venous Thromboembolism Prophylaxis in Inflammatory Bowel Disease Inpatients: Systematic Review and Meta-Analysis.","authors":"Rotem McNeil, Danielle Fredman, Ofir Eldar, Anat Gafter-Gvili, Tomer Avni","doi":"10.1159/000538086","DOIUrl":"10.1159/000538086","url":null,"abstract":"<p><strong>Introduction: </strong>Inflammatory bowel disease (IBD) patients are three times more likely to develop venous thromboembolism (VTE), and guidelines recommend prophylaxis during all hospitalizations. In this systematic review, we sought to assess for the benefits and risks of VTE prophylaxis in hospitalized IBD patients.</p><p><strong>Methods: </strong>We performed a systematic review and meta-analysis. We searched MEDLINE and others up to 2/2022, for studies on IBD inpatients treated with prophylactic anticoagulation during hospitalization, compared to no prophylaxis. Primary efficacy and safety outcomes were any VTE and major bleeding, respectively. Results were pooled using random-effects models, calculating odds ratios (OR), and 95% confidence intervals (CI). The ROBINS-I tool was used to assess bias.</p><p><strong>Results: </strong>We extracted data from 18 observational studies and 2 randomized-trial subgroups. The studies were highly variable regarding the included populations, interventions, and outcome definitions. Meta-analysis of all studies showed a nonsignificant effect of prophylaxis on VTEs (OR: 0.97 [95% CI: 0.49-1.95]). An analysis of eight lower-risk-of-bias studies showed a significant reduction in VTEs (OR: 0.27 [95% CI: 0.13-0.55], number needed to treat (NNT) 34.8 [95% CI: 26.8-49.8]). A significant protective effect persisted in several subgroups. Major bleeding was reported in three studies and showed a significant increase with prophylaxis (OR: 2.02 [95% CI: 1.11-3.67], number needed to harm (NNH) 113.6 [95% CI: 40.7-very-large-number]).</p><p><strong>Conclusion: </strong>In studies with lower-risk-of-bias, a significant reduction in VTEs was shown in patients treated with VTE prophylaxis (NNT = 35), which should be carefully considered against an increased major-bleeding risk (NNH = 114). However, current data are limited and randomized trials dedicated to IBD inpatients would aid in understating whether universal prophylaxis should be recommended.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"702-715"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11610454/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140020714","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":"Oncogenic MTOR Signaling Axis Compensates BTK Inhibition in a Chronic Lymphocytic Leukemia Patient with Richter Transformation: A Case Report and Review of the Literature.","authors":"Thomas Parigger, Stephan Drothler, Christian Scherhäufl, Franz Josef Gassner, Maria Schubert, Markus Steiner, Jan Philip Höpner, Alexandra Hödlmoser, Lena Schultheis, Aryunni Abu Bakar, Daniel Neureiter, Lisa Pleyer, Alexander Egle, Richard Greil, Roland Geisberger, Nadja Zaborsky","doi":"10.1159/000537791","DOIUrl":"10.1159/000537791","url":null,"abstract":"<p><strong>Introduction: </strong>Targeting the B-cell receptor pathway via ibrutinib, a specific inhibitor of Bruton's tyrosine kinase, has shown marked clinical efficacy in treatment of patients with chronic lymphocytic leukemia (CLL), thus becoming a preferred first line option independent of risk factors. However, acquired resistance to ibrutinib poses a major clinical problem and requires the development of novel treatment combinations to increase efficacy and counteract resistance development and clinical relapse rates.</p><p><strong>Case presentation: </strong>In this study, we performed exome and transcriptome analyses of an ibrutinib resistant CLL patient in order to investigate genes and expression patterns associated with ibrutinib resistance. Here, we provide evidence that ibrutinib resistance can be attributed to aberrant mammalian target of rapamycin (MTOR) signaling.</p><p><strong>Conclusion: </strong>Thus, our study proposes that combined use of MTOR inhibitors with ibrutinib could be a possible option to overcome therapy resistance in ibrutinib treated patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"604-611"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11441378/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139970590","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":"In Memoriam: Isaac Ben-Bassat (1937-2023) A Lifelong Legacy in Hematology.","authors":"Pia Raanani","doi":"10.1159/000535013","DOIUrl":"10.1159/000535013","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"247-248"},"PeriodicalIF":2.4,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138440092","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":"Long-Term Clinical Outcomes of Optimizing Combination Therapy for Primary Pulmonary Mucosa-Associated Lymphoid Tissue Lymphoma: A Retrospective Study.","authors":"Gi-June Min, Chin Kook Rhee, Tong Yoon Kim, Young-Woo Jeon, Joo Hyun O, Byung-Ock Choi, Gyeongsin Park, Seok-Goo Cho","doi":"10.1159/000535228","DOIUrl":"10.1159/000535228","url":null,"abstract":"<p><strong>Introduction: </strong>Pulmonary mucosa-associated lymphoid tissue (MALT) lymphoma progresses with advancing disease stage. However, no standard treatment approach has been established. This single-center retrospective study evaluated clinical and radiological characteristics, treatment modalities, and long-term prognosis of pulmonary MALT lymphoma.</p><p><strong>Methods: </strong>The study included 42 patients diagnosed with pulmonary MALT lymphoma between October 2004 and July 2019. Primary therapeutic modalities were determined using modified Ann Arbor staging. Therapeutic response was evaluated via computed tomography and laboratory analyses every 6 months for 5 years. Radiological findings were categorized based on the Lugano classification as complete response (CR), partial response, stable disease (SD), or progressive disease.</p><p><strong>Results: </strong>Initial treatment included observation (n = 2), surgical resection (n = 6), or systemic chemotherapy (n = 34). Patients treated surgically had localized disease and achieved initial and long-term CR. Of the 34 patients who underwent chemotherapy, 30 achieved CR, 2 achieved SD, and 2 died. Overall and progression-free survival (PFS) rates were 93.9% and 54.3%, respectively. Multivariate analysis indicated that PFS was lower in patients with modified Ann Arbor stage III-IV lymphoma and those who did not achieve CR.</p><p><strong>Conclusions: </strong>Optimized treatment based on anatomical location, pulmonary function, and disease stage can improve long-term survival in patients with pulmonary MALT lymphoma.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"413-426"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11296562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138440093","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":"Complete Response to Donor Lymphocyte Infusion for Primary Hemophagocytic Lymphohistiocytosis Relapse after Allogeneic Hematopoietic Cell Transplantation.","authors":"Rutvij A Khanolkar, Nathan Kuehne, Jan Storek","doi":"10.1159/000535449","DOIUrl":"10.1159/000535449","url":null,"abstract":"<p><strong>Introduction: </strong>Primary hemophagocytic lymphohistiocytosis (HLH) is a hyper-inflammatory disorder characterized by dysregulation of inflammatory cells and cytokine signaling. Although first-line treatment consisting of immunosuppressive therapy and allogeneic hematopoietic cell transplantation (HCT) is often curative, it remains unknown whether any effective therapies exist for disease relapse/progression after HCT.</p><p><strong>Case presentation: </strong>Here we present a case of a 29-year-old male with primary HLH who failed HLH-94 protocol and subsequently underwent myeloablative HCT. Disease relapse occurred at 9 months following HCT, and donor lymphocyte infusion (DLI) was initiated as salvage therapy. The patient subsequently achieved durable long-term disease-free survival following a DLI, without significant treatment-related complications.</p><p><strong>Conclusion: </strong>To our knowledge, this represents the first case demonstrating the efficacy of DLI for relapsed primary HLH.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"489-492"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138298067","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 Rare Clinical Case of Secondary Central Nervous System Involvement without Transformation in Hairy Cell Leukemia: A Case Report and Literature Review.","authors":"Kenichi Ito, Kunihiko Harada, Yoshihito Uchino, Kazuhiko Hirano, Naohiro Sekiguchi","doi":"10.1159/000535066","DOIUrl":"10.1159/000535066","url":null,"abstract":"<p><strong>Introduction: </strong>Hairy cell leukemia (HCL) is an indolent B-cell lymphoma characterized by a specific genetic mutation, BRAF V600E, which affects the specific morphology and oncogenesis. For HCL, few reports regarding secondary central nervous system involvement (SCNSI) are available. Herein, we present the case of an 80-year-old woman who had a relapse of HCL with SCNSI.</p><p><strong>Case presentation: </strong>The diagnosis of HCL was made in June 2015 after identifying BRAF V600E proteins by immunohistochemical analysis, and the disease was then controlled for 6 years by employing chemoimmunotherapy. In February 2021, the patient was admitted with neurological symptoms such as dizziness. Magnetic resonance imaging of the brain showed abnormal enhancement in the cerebrum, and cerebrospinal fluid analysis revealed neoplastic cells without transformation into large cells. Thus, the patient was diagnosed as having SCNSI in HCL.</p><p><strong>Conclusion: </strong>We report a case of a rare clinical presentation of SCNSI in HCL with literature review.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"482-488"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89716591","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":"Clinical Outcomes, Survival, and Predictors in Lower-Risk Myelodysplastic Syndrome Patients Treated with Cyclosporine A.","authors":"Yingjia Lu, Lina Zhang, Weiying Qu, Zhou Feng, Yuan Deng, Lin Zhao","doi":"10.1159/000537773","DOIUrl":"10.1159/000537773","url":null,"abstract":"<p><strong>Introduction: </strong>Therapeutic options to improve myelodysplastic syndrome (MDS)-related cytopenias in patients with lower-risk MDS are limited, and cyclosporin A (CSA) is an available option.</p><p><strong>Methods: </strong>We retrospectively analysed the clinical data of 153 consecutive patients with lower-risk MDS at our institution from July 1997 to October 2017. The propensity score matching method was used to balance the influence of confounding factors between patients with MDS treated with CSA and other conventional treatments (excluding CSA), and 50 pairs of cases were successfully identified for the final analysis. We assessed response rates, progression-free survival (PFS), overall survival (OS), and factors affecting response and survival.</p><p><strong>Results: </strong>Haematological improvement (HI) was observed in 35 (70%) patients treated with CSA and in 25 (50%) patients treated with conventional therapies (p < 0.05). Treatment with CSA was a favourable prognostic factor for HI in lower-risk MDS patients in the entire population in univariate analysis (odds ratio (OR) 2.333, p < 0.05), but not in multivariate analysis. In the multivariate analysis, hypocellular marrow was the only independent prognostic factor for HI in the CSA group (OR 6.259, p < 0.05) and in the overall cohort (OR 3.102, p < 0.05). CSA treatment did not improve PFS or OS (p > 0.05).</p><p><strong>Conclusion: </strong>CSA is a safe treatment and can significantly improve cytopenias in a substantial proportion of patients with MDS, especially in individuals with hypocellular bone marrow. However, CSA is not associated with improved PFS or OS.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"716-728"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139899181","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}