Acta HaematologicaPub Date : 2024-01-01Epub Date: 2024-03-11DOI: 10.1159/000538289
{"title":"[Momelotinib bei Myelofibrose und Anämie].","authors":"","doi":"10.1159/000538289","DOIUrl":"10.1159/000538289","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"147 2","pages":"251-253"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140100712","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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2023-09-06DOI: 10.1159/000533990
Evan C Chen, Jacqueline S Garcia
{"title":"Immunotherapy for Acute Myeloid Leukemia: Current Trends, Challenges, and Strategies.","authors":"Evan C Chen, Jacqueline S Garcia","doi":"10.1159/000533990","DOIUrl":"10.1159/000533990","url":null,"abstract":"<p><strong>Background: </strong>In the past decade, there have been significant breakthroughs in immunotherapies for B-cell lymphoid malignancies and multiple myeloma, but progress has been much less for acute myeloid leukemia (AML). Nevertheless, challenge begets innovation and several therapeutic strategies are under investigation.</p><p><strong>Summary: </strong>In this review, we review the state of the art in AML immunotherapy including CD33- and CD123-targeted agents, immune checkpoint inhibition, and adoptive cell therapy strategies. We also share conceptual frameworks for approaching the growing catalog of investigational AML immunotherapies and propose future directions for the field.</p><p><strong>Key messages: </strong>Immunotherapies for AML face significant challenges but novel strategies are in development.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"198-218"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10226340","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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2023-10-09DOI: 10.1159/000534341
Ofrat Beyar Katz, Dana Yehudai-Ofir, Tsila Zuckerman
{"title":"Cellular Therapy in Chronic Lymphocytic Leukemia: Have We Advanced in the Last Decade?","authors":"Ofrat Beyar Katz, Dana Yehudai-Ofir, Tsila Zuckerman","doi":"10.1159/000534341","DOIUrl":"10.1159/000534341","url":null,"abstract":"<p><strong>Background: </strong>Chronic lymphocytic leukemia (CLL) is a heterogeneous B-cell malignancy, affecting mainly older adults. Despite the recent introduction of multiple targeted agents, CLL remains an incurable disease. Cellular therapy is a promptly evolving area that has developed over the last decades from such standard of care as hematopoietic cell transplantation (HCT) to the novel treatment modalities employing genetically engineered immune cells.</p><p><strong>Summary: </strong>Tailoring the proper treatment for each patient is warranted and should take into account the disease biology, patient characteristics, and the available treatment modalities. Nowadays, the most broadly applied cellular therapies for CLL management are HCT and chimeric antigen receptor-T (CAR-T) cells. However, CAR-T cell therapy is currently not yet approved in CLL, and the appropriate sequencing for the administration of these agents remains to be clarified.</p><p><strong>Key messages: </strong>The current review will discuss various available cellular treatment options, their advances and limitations, as well as the optimal timing for the employment of such therapies in CLL patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"99-112"},"PeriodicalIF":1.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41181753","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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2023-10-17DOI: 10.1159/000533666
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
{"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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2024-02-15DOI: 10.1159/000537719
Qing Zhang, Yi Dong, Zhimin Zhai, Lili Tao, Qianshan Tao
{"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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2024-02-24DOI: 10.1159/000537791
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
{"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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2023-11-24DOI: 10.1159/000535013
Pia Raanani
{"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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2023-11-24DOI: 10.1159/000535228
Gi-June Min, Chin Kook Rhee, Tong Yoon Kim, Young-Woo Jeon, Joo Hyun O, Byung-Ock Choi, Gyeongsin Park, Seok-Goo Cho
{"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}
Acta HaematologicaPub Date : 2024-01-01Epub Date: 2023-11-23DOI: 10.1159/000535449
Rutvij A Khanolkar, Nathan Kuehne, Jan Storek
{"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}