Haematologica最新文献_第9页

Pathogenesis and inflammaging in myelodysplastic syndrome. 骨髓增生异常综合征的发病机制和炎症反应

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-24 DOI: 10.3324/haematol.2023.284944

Matthew T Villaume,Michael R Savona

{"title":"Pathogenesis and inflammaging in myelodysplastic syndrome.","authors":"Matthew T Villaume,Michael R Savona","doi":"10.3324/haematol.2023.284944","DOIUrl":"https://doi.org/10.3324/haematol.2023.284944","url":null,"abstract":"Myelodysplastic syndromes (MDS) are a genetically complex and phenotypically diverse set of clonal hematologic neoplasms that occur with increasing frequency with age. MDS has long been associated with systemic inflammatory conditions and disordered inflammatory signaling is implicated in MDS pathogenesis. A rise in sterile inflammation occurs with ageing and the term \"inflammaging\" has been coined by to describe this phenomenon. This distinct form of sterile inflammation has an unknown role in in the pathogenesis of myeloid malignancies despite shared correlations with age and ageing-related diseases. More recent is a discovery that many cases of MDS arise from clonal hematopoiesis of indeterminate potential (CHIP), an age associated, asymptomatic pre-disease state. The interrelationship between ageing, inflammation and clonal CHIP is complex and likely bidirectional with causality between inflammaging and CHIP potentially instrumental to understanding MDS pathogenesis. Here we review the concept of inflammaging and MDS pathogenesis and explore their causal relationship by introducing a novel framing mechanism of \"pre-clonal inflammaging\" and \"clonal inflammaging\". We aim to harmonize research on ageing, inflammation and MDS pathogenesis by contextualizing the current understanding of inflammaging and the ageing hematopoietic system with what is known about the etiology of MDS via its progression from CHIP.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"10 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia. 骨髓增生性肿瘤向急性髓性白血病转化的预防和治疗。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-24 DOI: 10.3324/haematol.2023.283950

Anand A Patel,Raajit K Rampal

{"title":"Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia.","authors":"Anand A Patel,Raajit K Rampal","doi":"10.3324/haematol.2023.283950","DOIUrl":"https://doi.org/10.3324/haematol.2023.283950","url":null,"abstract":"Philadelphia-chromosome negative (Ph-neg) myeloproliferative neoplasms (MPNs) are hematopoietic stem disorders with a risk of progression to the accelerated-phase (AP) or blastphase (BP) that is influenced by clinical, pathologic, cytogenetic, and molecular variables. Overall survival is limited in MPN-AP/BP with current treatment approaches, particularly in those patients that cannot receive an allogeneic hematopoietic stem cell transplant (allo-HCT). In addition, long-term survival with allo-HCT is predominantly seen in chronic-phase MPNs which suggests that the ideal time for intervention may be before MPNs evolve to AP/BP. Over the course of this review we will focus on the risk factors for progression to MPN-AP/BP, identification of high-risk chronic-phase MPNs, potential early-intervention strategies, and considerations around the timing of allo-HCT. We will also summarize current survival outcomes in MPN-AP/BP, discuss the uncertainty around how to best gauge response to therapy, and outline clinical trial considerations for this patient population. Lastly, we will highlight future directions in the management of high-risk MPNs.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"60 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489657","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Branching NOTCH1 to DNA damage in T-cell acute lymphoblastic leukemia. T 细胞急性淋巴细胞白血病 DNA 损伤中的 NOTCH1 分支。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-24 DOI: 10.3324/haematol.2024.286450

Tanaya Kulkarni,Daniel Herranz

引用次数: 0

New approaches to standard of care in early-phase myeloproliferative neoplasms: can interferon-α alter the natural history of the disease? 早期骨髓增生性肿瘤标准治疗的新方法：干扰素-α能否改变疾病的自然史？

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-24 DOI: 10.3324/haematol.2023.283958

Florence Pasquier,Jean Pegliasco,Jean-Edouard Martin,Severine Marti,Isabelle Plo

{"title":"New approaches to standard of care in early-phase myeloproliferative neoplasms: can interferon-α alter the natural history of the disease?","authors":"Florence Pasquier,Jean Pegliasco,Jean-Edouard Martin,Severine Marti,Isabelle Plo","doi":"10.3324/haematol.2023.283958","DOIUrl":"https://doi.org/10.3324/haematol.2023.283958","url":null,"abstract":"The classical BCR::ABL-negative myeloproliferative neoplasms (MPN) include Polycythemia Vera (PV), Essential Thrombocytemia (ET), and Primary Myelofibrosis (PMF). They are acquired clonal disorders of the hematopoietic stem cells (HSC) leading to hyperplasia of one or several myeloid lineages. MPN are caused by three main recurrent mutations, JAK2V617F and mutations in the calreticulin (CALR) and the thrombopoietin receptor (MPL) genes. Here, we review the general diagnosis, the complications, and the management of MPN. Second, we explain the physiopathology of the natural disease development and its regulation, which contributes to MPN heterogeneity. Thirdly, we describe the new paradigm of the MPN development highlighting the early origin of driver mutations decades before the onset of symptoms and the consequence on early detection of MPN cases in the general population for early diagnosis and better medical management. Finally, we present the interferon alpha (IFNα) therapy as a potential early disease-modifying drug after reporting its good hematological and molecular efficacies in ET, PV and early MF in clinical trials as well as its mechanism of action in pre-clinical studies. As a result, we may expect that, in the future, MPN patients will be diagnosed very early during the course of disease and that new selective therapies under development, such as IFNα, JAK2V617F inhibitors and CALRmut monoclonal antibodies, would be able to intercept the mutated clones.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"25 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Posterior cerebral circulation in children with sickle cell anemia: an uncharted territory. 镰状细胞贫血患儿的大脑后循环：未知领域。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-24 DOI: 10.3324/haematol.2024.285773

Alvise Fattorello Salimbeni,Ludovica De Rosa,Alessia Volpato,Federica Viaro,Alessio Pieroni,Stefano Mozzetta,Francesco Favruzzo,Alessandra Pes,Matteo Zaccagnino,Giulia Reggiani,Maria Paola Boaro,Raffaella Colombatti,Renzo Manara,Claudio Baracchini

引用次数: 0

Treatment of lower-risk myelodysplastic syndromes. 低风险骨髓增生异常综合征的治疗。

IF 8.2 1区医学

Haematologica Pub Date : 2024-10-24 DOI: 10.3324/haematol.2023.284945

Almuth Maria Anni Merz, Uwe Platzbecker

{"title":"Treatment of lower-risk myelodysplastic syndromes.","authors":"Almuth Maria Anni Merz, Uwe Platzbecker","doi":"10.3324/haematol.2023.284945","DOIUrl":"https://doi.org/10.3324/haematol.2023.284945","url":null,"abstract":"<p><p>Myelodysplastic neoplasms (MDS) involve clonal hematopoiesis and cellular dysplasia, driven by genetic and epigenetic alterations. Spliceosome mutations and epigenetic dysregulation underscore the intricate pathogenesis of MDS. The bone marrow microenvironment, stromal dysfunction, chronic inflammation, and immune dysregulation contribute to disease progression. This complex pathogenesis underscores the necessity for targeted therapies, offering a personalized medicine approach, particularly in lower-risk patients. The development of risk scores like the International Prognostic Scoring System (PISS), its revision IPSS-R, and the incorporation of molecular genetics in IPSS-M have refined the diagnostic and prognostic framework of MDS. These scoring systems facilitate tailored treatment strategies and better prognostication, especially for lower-risk MDS patients. The progression from IPSS to IPSS-R and now to IPSS-M epitomizes the shift towards personalized medicine in MDS management. In this review we discuss recent developments and positive phase III studies in lower-risk MDS. The review concludes by proposing a treatment algorithm for LR-MDS and highlighting ongoing trials in this heterogeneous patient population.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499211","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Eleven cases of laryngeal edema after tisagenlecleucel infusion: a 3-year single center retrospective study of CD19-directed chimeric antigen receptor T-cell therapy for relapsed and refractory B-cell lymphomas. 输注替沙根白细胞介素后出现喉水肿的11例病例：CD19定向嵌合抗原受体T细胞疗法治疗复发和难治性B细胞淋巴瘤的3年期单中心回顾性研究。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-17 DOI: 10.3324/haematol.2024.286169

Erina Hosoya,Jun Ando,Shintaro Kinoshita,Yoshiki Furukawa,Yuko Toyoshima,Yoko Azusawa,Toru Mitsumori,Eriko Sato,Hina Takano,Yutaka Tsukune,Naoki Watanabe,Tomoiku Takaku,Hajime Yasuda,Yasuharu Hamano,Makoto Sasaki,Shuko Nojiri,Midori Ishii,Miki Ando

引用次数: 0

Pirtobrutinib: the 'brute' with a softer side. Pirtobrutinib：有柔软一面的 "野蛮人"。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-17 DOI: 10.3324/haematol.2024.286532

Justin Desroches,Diego Villa

引用次数: 0

Belantamab mafodotin monotherapy for relapsed or refractory multiple myeloma: a real-world observational study in the United States. 贝仑单抗马福多汀单药治疗复发或难治性多发性骨髓瘤：美国的一项真实世界观察研究。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-17 DOI: 10.3324/haematol.2024.285893

Malin Hultcrantz,David Kleinman,Ravi Vij,Fernando Escalante,Michel Delforge,Nirali Kotowsky,Jacopo Bitetti,Natalie Boytsov,Leena Camadoo-O'Byrne,Lindsey Powers Happ,Guillaume Germain,Ana Urosevic,Malena Mahendran,Mei Sheng Duh,Francois Laliberte,Michele Cavo,Hans C Lee

引用次数: 0

Frequency of secondary T-cell lymphoma in chimeric antigen receptor Tcell naïve B-cell lymphoid-lineage cancers is higher than that reported on chimeric antigen receptor T-cell therapy. 在嵌合抗原受体 T 细胞幼稚 B 细胞淋巴系癌症中，继发性 T 细胞淋巴瘤的发生率高于嵌合抗原受体 T 细胞疗法。

IF 10.1 1区医学

Haematologica Pub Date : 2024-10-17 DOI: 10.3324/haematol.2024.286002

Christian Brieghel,Søren L Petersen,Peter de N Brown,Carsten U Niemann

引用次数: 0