HaematologicaPub Date : 2026-04-16DOI: 10.3324/haematol.2026.300520
Rutuja S Gupte,Sara Rovira Puig,Liliana R Loureiro,Margot Dierickx,Alexandra Von Jutrzenka-Trzebiatowski,Michael Bachmann,Jan Voorberg,Karen Vanhoorelbeke,Claudia Arndt,Anja Feldmann
{"title":"Establishing reverse chimeric antigen receptor T cells for precise targeting of immunemediated thrombotic thrombocytopenic purpura.","authors":"Rutuja S Gupte,Sara Rovira Puig,Liliana R Loureiro,Margot Dierickx,Alexandra Von Jutrzenka-Trzebiatowski,Michael Bachmann,Jan Voorberg,Karen Vanhoorelbeke,Claudia Arndt,Anja Feldmann","doi":"10.3324/haematol.2026.300520","DOIUrl":"https://doi.org/10.3324/haematol.2026.300520","url":null,"abstract":"Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) is a life-threatening autoimmune disorder caused by autoantibodies that target the metalloprotease ADAMTS13. This prevents the cleavage of Ultra-Large von Willebrand Factor (UL-VWF), thereby forming microthrombi in the microvasculature. While current treatments are effective, ADAMTS13 relapses occur in up to 15% of the patients after rituximab administration. In this study, we propose a novel approach addressing the unmet need for treatment of patients with ADAMTS13 refractoriness and for patients requiring repeated treatment. Our strategy involves developing two targeted therapies that aim to achieve highly effective and safe B cell depletion. For the first time, we employ adapter Chimeric Antigen Receptor (CAR) T cells called Reverse CAR (RevCAR) T cells to (i) achieve a broader and deeper B cell depletion by targeting CD19 and (ii) selectively deplete the disease-causing anti-ADAMTS13 autoreactive B cells to increase precision and safety in iTTP treatment. Therefore, we have developed novel Target Modules (RevTMs) against CD19+ and anti-ADAMTS13 autoreactive B cells. We show that the RevCAR T cells successfully and specifically kill target hybridomas and Nalm-6 cells in a RevTM-dependent manner and at low effector-to-target cell ratios. Moreover, the RevCAR T cells are able to eliminate the target cells even in the presence of high concentrations of ADAMTS13 autoantibodies. Overall, our findings demonstrate for the first time, a proof-ofconcept that anti-ADAMTS13 autoreactive B cells can be selectively depleted by the RevCAR T cell platform paving the way for an effective and safe therapy for iTTP.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"9 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147685032","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}
{"title":"Gene expression profile of adult T-cell leukemia/lymphoma with human T-cell lymphotropic virus type 1-infected Hodgkin and Reed-Sternberg-like cells is more similar to classic Hodgkin lymphoma than typical adult T-cell leukemia/lymphoma.","authors":"Shohei Miyagi,Kyohei Mori,Kohta Miyawaki,Masatoshi Shimo,Tomoko Tamaki,Shugo Sakihama,Mitsuyoshi Takatori,Koichi Akashi,Kennosuke Karube,Koji Kato","doi":"10.3324/haematol.2025.289270","DOIUrl":"https://doi.org/10.3324/haematol.2025.289270","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"109 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147685025","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}
{"title":"An unusual case of basophilic meningitis revealing a chronicphase chronic myeloid leukemia: a report after almost three years of follow-up.","authors":"Ramy Rahmé,Matteo Dragani,Carole Fleury,Giulia Tueur,Paule Moussounda Bamba,Héloïse Torres-Villaros,Audrey Giocanti-Aurégan,Roland Bangbotche,Gregory Lazarian,Fanny Baran-Marszak,Claude Gardin,Thorsten Braun","doi":"10.3324/haematol.2025.300120","DOIUrl":"https://doi.org/10.3324/haematol.2025.300120","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"138 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147685037","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}
HaematologicaPub Date : 2026-04-16DOI: 10.3324/haematol.2026.300932
Moshe Mittelman,Liran Shlush
{"title":"TP53 mutation analysis in myelodysplastic syndromes - long or short read sequencing?","authors":"Moshe Mittelman,Liran Shlush","doi":"10.3324/haematol.2026.300932","DOIUrl":"https://doi.org/10.3324/haematol.2026.300932","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"19 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147685024","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}
HaematologicaPub Date : 2026-04-16DOI: 10.3324/haematol.2025.300370
Yang Liu,Pablo Giusti,Hong Qian,Ping Chun Wu,Tengyu Wang,Stan De Haan,Jacek Toporski,Agneta Wikman,Petter Höglund,Emöke Deschmann,Roland Fiskesund
{"title":"Neonatal exchange blood is a rich source of hematopoietic stem and progenitor cells for transplantation.","authors":"Yang Liu,Pablo Giusti,Hong Qian,Ping Chun Wu,Tengyu Wang,Stan De Haan,Jacek Toporski,Agneta Wikman,Petter Höglund,Emöke Deschmann,Roland Fiskesund","doi":"10.3324/haematol.2025.300370","DOIUrl":"https://doi.org/10.3324/haematol.2025.300370","url":null,"abstract":"Not available.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"84 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147685036","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}
HaematologicaPub Date : 2026-04-09DOI: 10.3324/haematol.2025.300201
Antonio Vogelsberg,Julia Salmerón-Villalobos,Anna-Lisa Wilhelmi,Franziska Otto,Aylin Schneider,Barbara Mankel,Eyyub Bag,Ariadna Colmenero,Matthias S Dettmer,Marina Narbaitz,Alexandar Tzankov,Itziar Salaverria,Irina Bonzheim,Falko Fend
{"title":"The genetic landscape of extramedullary plasmacytoma: a comparative analysis with extramedullary disease of multiple myeloma.","authors":"Antonio Vogelsberg,Julia Salmerón-Villalobos,Anna-Lisa Wilhelmi,Franziska Otto,Aylin Schneider,Barbara Mankel,Eyyub Bag,Ariadna Colmenero,Matthias S Dettmer,Marina Narbaitz,Alexandar Tzankov,Itziar Salaverria,Irina Bonzheim,Falko Fend","doi":"10.3324/haematol.2025.300201","DOIUrl":"https://doi.org/10.3324/haematol.2025.300201","url":null,"abstract":"Extramedullary (or extraosseous) plasmacytoma (EMP) is a localized plasma cell neoplasm lacking apparent bone marrow involvement and other features of multiple myeloma (MM). The prognosis is usually favorable and progression to MM is infrequent. The genetic landscape of EMP remains largely unknown. Importantly, EMP must be distinguished from extramedullary disease of MM (EMD), which typically exhibits aggressive behavior. We performed a comprehensive molecular analysis of EMP (n=24) in comparison to EMD (n=24) using fluorescence in situ hybridization (FISH), copy number (CN) profiling, and targeted nextgeneration sequencing. Overall, EMP shared several genetic features with MM, but exhibited markedly lower complexity than EMD. EMP revealed a mean of 4.3 CN changes vs. 19.3 in EMD (p.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"21 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2026-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147635769","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}