Julien Rossignol, Laurence Bouillet, Cristina Bulai-Livideanu, Stéphane Barete, Julie Bruneau, Clément Gourguechon, Laura Polivka, Angèle Soria, Olivier Lortholary, Trevor Stanbury, Benoit Bouquillon, Cedric Sauvage, Rose Marie Javier, Olivier Hermine, Michel Arock
{"title":"Diagnosis, management and the burden of symptoms of mastocytosis from the physician's perspective: A nationwide study.","authors":"Julien Rossignol, Laurence Bouillet, Cristina Bulai-Livideanu, Stéphane Barete, Julie Bruneau, Clément Gourguechon, Laura Polivka, Angèle Soria, Olivier Lortholary, Trevor Stanbury, Benoit Bouquillon, Cedric Sauvage, Rose Marie Javier, Olivier Hermine, Michel Arock","doi":"10.1111/bjh.70148","DOIUrl":"https://doi.org/10.1111/bjh.70148","url":null,"abstract":"<p><p>Mastocytosis is categorized into cutaneous mastocytosis (CM), mast cell sarcoma and systemic mastocytosis (SM). Within SM, indolent SM (ISM) is the more frequent subtype. Adult patients with CM but without an extracutaneous biopsy are classified as having mastocytosis in the skin (MIS), a provisional diagnosis. Mastocytosis patients may experience a wide range of symptoms that significantly impact their quality of life (QoL). In France, the estimated prevalence of mastocytosis and the burden of symptoms remain unknown. To address this, we conducted a national online survey to estimate the number of mastocytosis diagnoses and assess the burden of symptoms from the physician's perspective. Overall, 1169 of the 6239 physicians solicited completed the survey. These physicians reported managing 4121 patients of whom only 53% had ISM. By contrast, CM and MIS were reported in more patients than expected at 17% and 25% respectively. The estimated prevalence of mastocytosis in France was 8.5 per 100,000, which is lower than recent epidemiological studies and is associated with significant regional variability (p < 0.001). Among patients with ISM or MIS, 53% experienced moderate to severe symptoms-mainly affecting the skin, digestive system and general health-which impacted most QoL domains assessed. These findings highlight the need to improve awareness, access to diagnostic workup for mastocytosis (especially for patients with MIS and CM) and enhance QoL for patients.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032369","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":"Adoptive transfer of third-party donor CMV-specific T cells for refractory cytomegalovirus infection following umbilical cord blood transplantation.","authors":"Guangyu Sun, Jingwei Tu, Baolin Tang, Zhenyi Lu, Xin Fang, Huilan Liu, Yongsheng Han, Zimin Sun, Changcheng Zheng, Xiaoyu Zhu, Juan Tong","doi":"10.1111/bjh.70136","DOIUrl":"https://doi.org/10.1111/bjh.70136","url":null,"abstract":"<p><p>Refractory cytomegalovirus (CMV) infection is a severe complication following umbilical cord blood transplantation (UCBT). Antiviral agents, the standard first-line therapy, are limited by toxicity and resistance without robust T-cell immunity. We evaluated third-party donor (TPD)-derived CMV-specific T cells (CMVSTs) as a treatment option. In a prospective phase I trial, UCBT recipients under 60 years with refractory CMV infection were enrolled. CMV-seropositive TPDs, matched for at least one human leucocyte antigen (HLA) class I allele at high resolution, were selected from family members. CMVSTs were manufactured for two infusions within 1 month. Safety and feasibility were primary end-points while efficacy and immune reconstitution were secondary end-points. Ten patients (median age: 15.5 years, range: 3-46 years) received CMVSTs (median doses: 2.2 × 10<sup>7</sup>/m<sup>2</sup> and 4.2 × 10<sup>7</sup>/m<sup>2</sup>). Products showed high CD3<sup>+</sup> T-cell purity and memory T-cell dominance. Infusions caused mild adverse events, with no severe toxicities. At 6 months post-first infusion, 80% (8/10) achieved durable virological clearance; 60% (6/10) survived at 2 years with sustained CMV control. TPD-derived CMVST infusions are safe and feasible for refractory CMV infection post-UCBT, offering durable virological control while larger studies are needed to confirm efficacy. This trial was registered at www.chictr.org.cn as # ChiCTR2000034951.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032399","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}
Christina Poh, Xueyan Chen, Jenna Voutsinas, Kikkeri Naresh, Jerlin J Dizon, Mazyar Shadman, Ryan C Lynch, Brian G Till, Chaitra S Ujjani, Mengyang Di, Vikram Raghunathan, Edus H Warren, Stephen D Smith, Qian V Wu, Sindhu Cherian, Ajay K Gopal
{"title":"Impact of immunophenotype on clinical disease characteristics and outcomes in T-cell prolymphocytic leukaemia.","authors":"Christina Poh, Xueyan Chen, Jenna Voutsinas, Kikkeri Naresh, Jerlin J Dizon, Mazyar Shadman, Ryan C Lynch, Brian G Till, Chaitra S Ujjani, Mengyang Di, Vikram Raghunathan, Edus H Warren, Stephen D Smith, Qian V Wu, Sindhu Cherian, Ajay K Gopal","doi":"10.1111/bjh.70113","DOIUrl":"https://doi.org/10.1111/bjh.70113","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145032334","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":"Evans syndrome in pregnancy and the postpartum period: A retrospective cohort study.","authors":"Debbie Jiang, Annemarie E Fogerty, David J Kuter","doi":"10.1111/bjh.70138","DOIUrl":"https://doi.org/10.1111/bjh.70138","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022501","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":"Taiwan consensus on pulmonary chronic graft-versus-host disease: A joint statement from the Taiwan Society of Blood and Marrow Transplantation (TBMT) and Taiwan Society of Pulmonary and Critical Care Medicine (TSPCCM).","authors":"Xavier Cheng-Hong Tsai, Zong-Han Yao, Shu-Wei Chou, Han-Chung Hu, Yi-Chang Liu, Chia-Hung Chen, Su-Peng Yeh, Wei-Chang Huang, Tung-Liang Lin, Tsung-Ming Yang, Ching-Han Lai, Hsin-Kuo Ko, Yao-Wen Kuo, Hsao-Hsun Hsu, Shih-Lung Cheng, Chi-Cheng Li, Bor-Sheng Ko, Hao-Chien Wang","doi":"10.1111/bjh.70074","DOIUrl":"https://doi.org/10.1111/bjh.70074","url":null,"abstract":"<p><p>Pulmonary chronic graft-versus-host disease (cGVHD), particularly bronchiolitis obliterans syndrome (BOS), is a severe complication of allogeneic haematopoietic stem cell transplantation (allo-HSCT) with significant morbidity and mortality. This report, developed collaboratively by experts from the Taiwan Society of Blood and Marrow Transplantation (TBMT) and the Taiwan Society of Pulmonary and Critical Care Medicine (TSPCCM), provides consensus statements for the diagnosis, surveillance and management of pulmonary cGVHD. Early detection through pulmonary function tests (PFTs) is critical, with serial monitoring recommended after allo-HSCT. High-resolution computed tomography (HRCT) serves as a valuable diagnostic tool, whereas bronchoalveolar lavage along with multiplex polymerase chain reaction (PCR) helps rule out infectious aetiologies. The first-line treatment approach includes inhaled and systemic corticosteroids, bronchodilators and azithromycin. The fluticasone-azithromycin-montelukast (FAM) regimen has demonstrated notable efficacy in stabilizing lung function. Emerging therapies such as ruxolitinib, belumosudil, abatacept and axatilimab offer promising benefits for refractory cases, whereas lung transplantation is a viable last resort for advanced disease. Comprehensive supportive care, encompassing pulmonary rehabilitation and infection prophylaxis, is an essential component of management. This consensus report highlights the importance of standardized PFT surveillance, early intervention and individualized immunosuppressive strategies. Future research should focus on refining diagnostic biomarkers and optimizing therapeutic approaches to improve patient outcomes.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028645","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}
Michael F Murphy, Damien Carson, Anwen Davies, Stephanie Ditcham, Graham Donald, Roger Graham, Lizzie Hutchinson, Paul Kerr, Denise McKeown, John Moppett, Shruthi Narayan, Paolo Polzella, Ella Poppitt, Megan Rowley, Tracey Shackleton, Julie Staves, James Neuberger
{"title":"Guidelines from the expert advisory committee on the Safety of Blood, Tissues and Organs (SaBTO) on patient consent and shared decision-making for blood transfusion.","authors":"Michael F Murphy, Damien Carson, Anwen Davies, Stephanie Ditcham, Graham Donald, Roger Graham, Lizzie Hutchinson, Paul Kerr, Denise McKeown, John Moppett, Shruthi Narayan, Paolo Polzella, Ella Poppitt, Megan Rowley, Tracey Shackleton, Julie Staves, James Neuberger","doi":"10.1111/bjh.70075","DOIUrl":"https://doi.org/10.1111/bjh.70075","url":null,"abstract":"<p><p>Evidence from national audits of practice indicates that the provision of information to patients about transfusion and the taking of consent to transfusion have not improved in recent years. Although the final report of the Infected Blood Inquiry did not make a specific recommendation about consent to transfusion, it emphasised the need for cultural change, the importance of openness and giving patients a voice. The purpose of these updated Safety of Blood, Tissues and Organs (SaBTO) guidelines is to enhance the provision of information to patients about blood transfusion, ensure an effective process for obtaining patients' consent and support shared decision-making. The guidelines emphasise that there is a duty on staff administering a transfusion to check that the documentation for consent is present and valid before commencing the transfusion. Hospitals and other healthcare facilities must facilitate this step by ensuring that documentation for consent can be easily found in a standard format and location in the patient's paper or preferably their electronic record. They should employ mechanisms through their arrangements for clinical governance to support the training of all staff who may take consent to transfusion and monitor the implementation and compliance with these SaBTO recommendations with subsequent improvement plans developed and implemented if necessary.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028629","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}
Andrew Clark, Hazel Clouston, Kanchan Rao, Najeem Folarin, Josu De la Fuente, Angela Hamblin, Eduardo Olavarria, Debbie Richardson, Polly Talley, Victoria Potter, Justin Loke, Terri McVeigh, John Snowden
{"title":"UK recommendations for chimerism testing and monitoring following allogeneic haematopoietic stem cell transplantation (HSCT): Best practice consensus guidelines from the British Society for Blood and Marrow Transplant and Cellular Therapies (BSBMTCT), NHS England Genomic Laboratory Hub (GLH) Haematological Malignancies Working Group, UK Cancer Genetics Group (UKCGG) and the UK National External Quality Assessment Service for Leucocyte Immunophenotyping (UK NEQAS LI).","authors":"Andrew Clark, Hazel Clouston, Kanchan Rao, Najeem Folarin, Josu De la Fuente, Angela Hamblin, Eduardo Olavarria, Debbie Richardson, Polly Talley, Victoria Potter, Justin Loke, Terri McVeigh, John Snowden","doi":"10.1111/bjh.70061","DOIUrl":"https://doi.org/10.1111/bjh.70061","url":null,"abstract":"<p><p>In allogeneic haematopoietic stem cell transplantation (HSCT), important clinical decisions depend upon assessment of chimerism, including immunosuppressant dosing and donor lymphocyte infusions (DLI), which in turn can have major impacts on disease control, graft-versus-host disease (GVHD), immunity and ultimately patient survival. There is a complex range of clinical and laboratory procedural considerations including methodology of testing, types of cell subset selection, frequency of testing, urgency of turnaround times (TATs), interplay with measurable residual disease (MRD) monitoring and duration of testing post-transplant. These aspects are routinely adapted according to disease indication, patient characteristics, donor source and intensity of transplant technique. To encourage the harmonisation of clinical and laboratory practice in the United Kingdom, we held a national workshop meeting to bring together key stakeholders to review the current literature with a view to producing a state-of-the-art position paper. Here, we present best practice consensus recommendations and identify key areas for future audit and research from the UK Cancer Genetics Group (UKCGG), NHS England Genomic Laboratory Hub (GLH) Haematological Oncology Malignancies Working Group, UK National External Quality Assessment Service for Leucocyte Immunophenotyping (UK NEQAS LI) and the British Society of Blood and Marrow Transplantation and Cellular Therapy (BSBMTCT).</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145028657","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":"Efficacy of ciclosporin monotherapy in non-severe aplastic anaemia not requiring transfusions: Results from a multicentre phase II study.","authors":"Ken Ishiyama, Masahide Yamazaki, Hiroyuki Maruyama, Naoko Hosono, Hiroki Yamaguchi, Noboru Asada, Kazuki Tanimoto, Hiroyuki Sugiura, Kensuke Usuki, Kenichi Yoshimura, Seishi Ogawa, Yuzuru Kanakura, Itaru Matsumura, Koichi Akashi, Shinji Nakao","doi":"10.1111/bjh.70144","DOIUrl":"https://doi.org/10.1111/bjh.70144","url":null,"abstract":"<p><p>The efficacy of ciclosporin (CsA) to treat transfusion-independent non-severe aplastic anaemia (TI-NSAA) has not yet been systematically evaluated. We conducted a prospective trial in patients with TI-NSAA treated with CsA monotherapy. CsA (3.5 mg/kg/day) was administered to patients with TI-NSAA aged ≥16. The CsA dose was adjusted to maintain a blood CsA level of ≥600 ng/mL at 2 h post-administration. Blood cell counts were assessed after 8, 16 and 52 weeks of therapy. Thirty-two evaluable patients from 21 institutions were enrolled. The median age was 63.5 (range: 16-83) years. At 8 weeks, haematological improvement, with increases in haemoglobin (Hb) ≥1.5 g/dL (haematological improvement in erythrocytes [HI-E]) and platelet count ≥30 × 10<sup>9</sup>/L (haematological improvement in platelets [HI-P]), was observed in 0/25 (0%) and 6/32 (19%) evaluable cases respectively. HI-E and HI-P occurred in 1/25 (4%) and 10/32 (31%) patients at 16 weeks, respectively, and at 52 weeks in 5/25 (20%) and 16/32 (50%) patients respectively. Nine grade 3 adverse events (AEs) occurred in six patients, but there were no grade ≥4 AEs. Ten of the 32 patients experienced grade 2 renal toxicity. Low-dose CsA is effective in TI-NSAA patients and demonstrates minimal renal toxicity. However, at least 16 weeks are necessary to adequately evaluate its efficacy.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022458","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}
Markus Metzler, Susan Branford, Nobuko Hijiya, Kathleen Sakamoto
{"title":"An old leukaemia in young patients-Genetic characteristics of paediatric chronic myeloid leukaemia.","authors":"Markus Metzler, Susan Branford, Nobuko Hijiya, Kathleen Sakamoto","doi":"10.1111/bjh.70134","DOIUrl":"https://doi.org/10.1111/bjh.70134","url":null,"abstract":"<p><p>Chronic myeloid leukaemia (CML) accounts for 2% of leukaemias in children and 9% in adolescents. While the BCR::ABL1 fusion gene remains a hallmark across all age groups, emerging evidence suggests that paediatric CML exhibits unique biological and clinical characteristics compared to its adult counterpart. Children often present with more aggressive clinical features and show distinct treatment response patterns. Recent genomic and transcriptomic studies have highlighted a higher prevalence of germline variants in haematopoiesis-related genes and unique cytogenetic alterations, as well as age-specific distributions of BCR and ABL1 breakpoints. Transcriptomic profiling of paediatric CML stem cells reveals differential expression signatures that suggest the presence of distinct signalling networks. Somatic mutations-particularly in ASXL1-are observed at diagnosis in some children, though their prognostic relevance remains uncertain. Additionally, while the BCR::ABL1 transcript subtype is known to affect treatment-free remission in adults, its role in paediatric CML is still unclear. Collectively, these findings support the notion that paediatric CML may constitute a biologically distinct disease rather than simply a younger form of adult CML. To advance risk stratification and develop age-specific treatment approaches, larger international studies and comprehensive genomic analyses are urgently needed.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022473","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}
Jason E Love, Vicki R Ratliff, Steven J Kussick, Afshin Shameli
{"title":"Early identification of circulating neuroendocrine tumour cells by flow cytometry prior to surgical diagnosis.","authors":"Jason E Love, Vicki R Ratliff, Steven J Kussick, Afshin Shameli","doi":"10.1111/bjh.70141","DOIUrl":"https://doi.org/10.1111/bjh.70141","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145022432","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}