Shanshan Suo, Rong Feng Fu, Albert Qin, Zonghong Shao, Jie Bai, Hu Zhou, Na Xu, Suning Chen, Xuelan Zuo, Xin Du, Minghui Duan, Li Wang, Pei Li, Xuhan Zhang, Sujiang Zhang, Daoxiang Wu, Jingjing Zhang, Zhijian Xiao, Lei Zhang, Jie Jin
{"title":"Molecular remission uncoupled with complete haematological response in polycythaemia vera treatment with ropeginterferon alfa-2b.","authors":"Shanshan Suo, Rong Feng Fu, Albert Qin, Zonghong Shao, Jie Bai, Hu Zhou, Na Xu, Suning Chen, Xuelan Zuo, Xin Du, Minghui Duan, Li Wang, Pei Li, Xuhan Zhang, Sujiang Zhang, Daoxiang Wu, Jingjing Zhang, Zhijian Xiao, Lei Zhang, Jie Jin","doi":"10.1111/bjh.19846","DOIUrl":"https://doi.org/10.1111/bjh.19846","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491483","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"<sup>18</sup>F-FDG PET/CT impact in grade 3B follicular lymphoma.","authors":"Domenico Albano","doi":"10.1111/bjh.19852","DOIUrl":"https://doi.org/10.1111/bjh.19852","url":null,"abstract":"<p><p>[<sup>18</sup>F]-fluoro-D-glucose positron emission tomography/computed tomography (<sup>18</sup>F-FDG PET/CT) is a non-invasive imaging tool that has a fundamental role in the management of FDG-avid lymphoma (also FL) in different settings, especially in the evaluation of treatment response and prognostication. The report by Barraclough et al. demonstrated that the treatment response evaluation by <sup>18</sup>F-FDG PET/CT was a strong predictor of prognosis in grade 3B follicular lymphoma (G3BFL). Moreover, among semiquantitative baseline PET features, standardized uptake value (SUV) and TGL showed to be useful in predicting progression-free survival (PFS). Commentary on: Barraclough et al. The value of semiquantitative PET features and end-of-therapy PET in grade 3B follicular lymphoma. Br J Haematol 2024 (Online ahead of print). doi: 10.1111/bjh.19823.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491473","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Eleonora Petito, Loredana Bury, Lilian Antunes Heck, Brooke Sadler, Erica De Candia, Gian Marco Podda, Anna Falanga, Lucia Stefanini, Andrea Boccatonda, Patrizia Sciancalepore, Igor Florio, Egidio Imbalzano, Rossella Marcucci, Patrizia Noris, Marta Panella, Rita Carlotta Santoro, Maria Costanza Turi, Gaetano Vaudo, Jorge Di Paola, Matthew T Rondina, Paolo Gresele
{"title":"Association of human leucocyte antigen loci with vaccine-induced immune thrombotic thrombocytopenia: Potential role of the interaction between platelet factor 4-derived peptides and MHC-II.","authors":"Eleonora Petito, Loredana Bury, Lilian Antunes Heck, Brooke Sadler, Erica De Candia, Gian Marco Podda, Anna Falanga, Lucia Stefanini, Andrea Boccatonda, Patrizia Sciancalepore, Igor Florio, Egidio Imbalzano, Rossella Marcucci, Patrizia Noris, Marta Panella, Rita Carlotta Santoro, Maria Costanza Turi, Gaetano Vaudo, Jorge Di Paola, Matthew T Rondina, Paolo Gresele","doi":"10.1111/bjh.19838","DOIUrl":"https://doi.org/10.1111/bjh.19838","url":null,"abstract":"<p><p>No risk factors have been identified for vaccine-induced immune thrombotic thrombocytopenia (VITT) so far. The aim of this study was to identify human leucocyte antigen (HLA) alleles potentially associated with VITT susceptibility. Specific HLA class II alleles were detected with significantly higher frequency in VITT patients compared with Italian controls: DPB1*17:01, DQA1*05:01, and DRB1*11:04. In silico analysis revealed increased affinity of DRB1*11:04 for a platelet factor 4 (PF4)-derived peptide, ITSLEVIKA, that contains two amino acids present in the specific binding site of anti-PF4 antibodies from VITT patients. Our findings show for the first time a genetic predisposition to developing anti-PF4 antibodies in response to Ad-vector vaccines.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491475","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Different phenotypes with different endings-Telomere biology disorders and cancer predisposition with long telomeres.","authors":"Sharon A Savage, Alison A Bertuch","doi":"10.1111/bjh.19851","DOIUrl":"https://doi.org/10.1111/bjh.19851","url":null,"abstract":"<p><p>Rare germline pathogenic variants (GPVs) in genes essential in telomere length maintenance and function have been implicated in two broad classes of human disease. The telomere biology disorders (TBDs) are a spectrum of life-threatening conditions, including bone marrow failure, liver and lung disease, cancer and other complications caused by GPVs in telomere maintenance genes that result in short and/or dysfunctional telomeres and reduced cellular replicative capacity. In contrast, cancer predisposition with long telomeres (CPLT) is a disorder associated with elevated risk of a variety of cancers, primarily melanoma, thyroid cancer, sarcoma, glioma and lymphoproliferative neoplasms caused by GPVs in shelterin complex genes that lead to excessive telomere elongation and increased cellular replicative capacity. While telomeres are at the root of both disorders, the term TBD is used to convey the clinical phenotypes driven by critically short or otherwise dysfunctional telomeres and their biological consequences.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491479","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Gisele Loth, Cilmara C K Dumke, Rafaella R Muratori, Polliany D Pelegrina, Carolina M A Peixoto, Juliana L M Bach, Samantha Nichele, Joanna P Trennepohl, Adriana Koliski, Rebeca T G Mousquer, Adriana M Rodrigues, Rafael Marchesini, Margareth K Feitosa, Daniela V Pilonetto, Lara Gouvea, Fernanda M L Benini, Emanuele S Pirolli, Rebeca A F Tutumi, Alberto C M Lima, Ricardo Pasquini, Carmem Bonfim
{"title":"Haematopoietic cell transplantation for 106 infants and preschoolers with acquired and inherited bone marrow failures.","authors":"Gisele Loth, Cilmara C K Dumke, Rafaella R Muratori, Polliany D Pelegrina, Carolina M A Peixoto, Juliana L M Bach, Samantha Nichele, Joanna P Trennepohl, Adriana Koliski, Rebeca T G Mousquer, Adriana M Rodrigues, Rafael Marchesini, Margareth K Feitosa, Daniela V Pilonetto, Lara Gouvea, Fernanda M L Benini, Emanuele S Pirolli, Rebeca A F Tutumi, Alberto C M Lima, Ricardo Pasquini, Carmem Bonfim","doi":"10.1111/bjh.19831","DOIUrl":"https://doi.org/10.1111/bjh.19831","url":null,"abstract":"<p><p>Aplastic anaemia in infants and young children presents unique challenges due to high prevalence of inherited bone marrow failure syndromes (IBMFS) in this age group. The objective of this study is assessing clinical characteristics and outcomes of haematopoietic cell transplantation in children ≤5 years with bone marrow failure syndromes. We analysied 106 patients (66% males), median age 4.6 years, including 40 with Fanconi anaemia (FA), 32 with Acquired Severe Aplastic anaemia (aSAA), 15 with Diamond-Blackfan Anaemia, 11 with Amegakaryocytic Purpura and 8 with other IBMFS. Molecular testing was limited (39%), with 25.4% confirmed genetically. Retrospective longitudinal study across three paediatric transplantation centres (1982-2020). Overall survival (OS) was 76.4% over a median 10-year follow-up. OS rates were similar between aSAA and IBMFS (FA 77.5%, other IBMFS 76.5%). Transplant-related mortality (TRM) was lower in aSAA (9.4%) compared with IBMFS (16.2%). Recent years showed improved outcomes, with TRM declining post-2010. Choice of stem cell source impacted OS, favouring bone marrow over umbilical cord, but showing encouraging results with haploidentical. Late complications were common, including endocrine-metabolic issues and delayed neuropsychomotor development. Diagnosing and managing bone marrow failures in young children pose significant challenges. Despite advancements in transplant practices, ongoing vigilance and comprehensive care are necessary to improve long-term survival rates.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491481","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Gemma K Reynolds, Emily Klimevski, Natalie R Saunders, Gayani S Teenakoon, Simon J Harrison, Mark Dowling, Mary Ann Anderson, Karin Thursky, Monica A Slavin, Benjamin W Teh
{"title":"Seropositivity against vaccine preventable infections in the early post chimeric antigen receptor T-cell period: Preservation of vaccine-associated antibodies between 0 and 6 months.","authors":"Gemma K Reynolds, Emily Klimevski, Natalie R Saunders, Gayani S Teenakoon, Simon J Harrison, Mark Dowling, Mary Ann Anderson, Karin Thursky, Monica A Slavin, Benjamin W Teh","doi":"10.1111/bjh.19807","DOIUrl":"https://doi.org/10.1111/bjh.19807","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491489","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Goodbye etoposide? Taking the leap to ruxolitinib in haemophagocytic lymphohistiocytosis.","authors":"Samuel A Merrill, Caroline Spaner, Luke Y C Chen","doi":"10.1111/bjh.19864","DOIUrl":"https://doi.org/10.1111/bjh.19864","url":null,"abstract":"<p><p>Fang et al. report a retrospective analysis of paediatric patients with haemophagocytic lymphohistiocytosis (HLH) associated with autoimmune/autoinflammatory disorders treated with ruxolitinib. Responses were impressive and rapid, and ruxolitinib was well tolerated. This study demonstrates that a subset of patients with HLH can be treated with JAK inhibition without the need for cytotoxic chemotherapy. Further work will be needed to better define patient selection for therapy, as some patient groups and HLH triggers, such as malignancy-associated HLH, may be better suited for etoposide-based therapy. Commentary on: Fang et al. Ruxolitinib-based regimen in children with autoimmune disease or autoinflammatory disease related hemophagocytic lymphohistiocytosis. Br J Haematol 2024 (Online ahead of print). doi: 10.1111/bjh.19803.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491480","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sophie Cousson, Charlotte Calvo, Lauriane Goldwirt, Mathieu Simonin, Julie Roupret-Serzec, Marie Émilie Dourthe, Marion Strullu, André Baruchel, Jean-Hugues Dalle, Benoît Brethon
{"title":"Monocentric experience of venetoclax-based regimen in paediatric refractory and relapsed AML/MDS.","authors":"Sophie Cousson, Charlotte Calvo, Lauriane Goldwirt, Mathieu Simonin, Julie Roupret-Serzec, Marie Émilie Dourthe, Marion Strullu, André Baruchel, Jean-Hugues Dalle, Benoît Brethon","doi":"10.1111/bjh.19849","DOIUrl":"https://doi.org/10.1111/bjh.19849","url":null,"abstract":"<p><p>BCL-2 inhibitor venetoclax demonstrates promising efficacy in paediatric relapsed/refractory acute myeloid leukaemia (r/r AML). This retrospective analysis evaluated 12 patients treated with venetoclax-based regimens under compassionate use for r/r myeloid malignancies. The overall response rate (ORR) was 41.6%, with complete response (CR) achieved in 33% of patients. Three patients successfully underwent allogeneic haematopoietic scell transplantation (HSCT) after venetoclax bridging therapy. Venetoclax demonstrated a favourable safety profile with manageable side effects. These findings suggest venetoclax's potential as a valuable therapeutic option for paediatric r/r AML, particularly for heavily pretreated patients. Further investigation in larger multicentre trials is warranted to refine treatment strategy.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142491484","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}