American Journal of Hematology最新文献

筛选
英文 中文
Sickle Cell Diastolic Cardiomyopathy and Mortality Risk: A Novel Echocardiographic Framework for Prognostic Stratification 镰状细胞舒张性心肌病和死亡风险:一种新的预后分层超声心动图框架
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-22 DOI: 10.1002/ajh.27768
Théo Simon, Laurent Savale, Kristoffer Grundtvig Skaarup, Paul Breillat, Luu‐Ly Pham, Seyed‐Mehdi Nouraie, Niklas Dyrby Johansen, Jocelyn Inamo, Francois Lionnet, Gylna Loko, Christelle Chantalat, Anne Laure Pham Hung d’Alexandry d’Orengiani, Anoosha Habibi, Gonzalo de Luna, Sihem Iles, Frédéric Galactéros, Etienne Audureau, Tor Biering‐Sørensen, Pablo Bartolucci, Geneviève Derumeaux, Thomas d’Humières
{"title":"Sickle Cell Diastolic Cardiomyopathy and Mortality Risk: A Novel Echocardiographic Framework for Prognostic Stratification","authors":"Théo Simon, Laurent Savale, Kristoffer Grundtvig Skaarup, Paul Breillat, Luu‐Ly Pham, Seyed‐Mehdi Nouraie, Niklas Dyrby Johansen, Jocelyn Inamo, Francois Lionnet, Gylna Loko, Christelle Chantalat, Anne Laure Pham Hung d’Alexandry d’Orengiani, Anoosha Habibi, Gonzalo de Luna, Sihem Iles, Frédéric Galactéros, Etienne Audureau, Tor Biering‐Sørensen, Pablo Bartolucci, Geneviève Derumeaux, Thomas d’Humières","doi":"10.1002/ajh.27768","DOIUrl":"https://doi.org/10.1002/ajh.27768","url":null,"abstract":"Cardiovascular complications are the leading cause of mortality in sickle cell anemia (SCA) patients. While extensive data have identified diastolic dysfunction (DD) to increase morbidity and mortality, the unique hemodynamic conditions inherent to SCA challenge the current recommendations to assess diastolic function. Thus, there is an urgent need to refine the echocardiographic definition of DD to improve risk stratification and therapeutic strategies in SCA patients. We analyzed data from the French multicentric Etendard cohort and compared them with an age‐ and sex‐matched control group from the Copenhagen City Heart Study (CCHS). We focused on left ventricular diastolic parameters, specifically lateral e′ velocity (e′ lat), E/e′ ratio, and indexed left atrial volume (LAVi), assessing their association with clinical outcomes over a 12‐year follow‐up. Etendard SCA patients (<jats:italic>n</jats:italic> = 379) had an early impaired diastolic function compared to the CCHS controls (<jats:italic>n</jats:italic> = 672). This was particularly obvious in young SCA patients (<jats:italic>n</jats:italic> = 252, age ≤ 38 years) in whom e′ lat was associated with prognosis (<jats:italic>p</jats:italic> = 0.01), with an optimal cut‐off value below 11 cm/s. Indeed, young SCA patients with DD had a fourfold increased 12‐year mortality rate as compared with SCA patients without DD (16 vs. 4%, <jats:italic>p</jats:italic> &lt; 0.001). Additionally, e′ lat correlated with 6‐min walk test, NT pro‐BNP levels, diastolic blood pressure, and lactate dehydrogenase levels. In young SCA patients, our data contribute to refine the diagnosis of diastolic dysfunction evaluation. We highlight the prognostic value below 11 cm/s of lateral e′ velocity and its association with key contributors of cardiac impairment such as hemolysis and systemic vasculopathy.Trial Registration: <jats:ext-link xmlns:xlink=\"http://www.w3.org/1999/xlink\" xlink:href=\"https://ClinicalTrials.gov\">ClinicalTrials.gov</jats:ext-link> identifier: NCT00434902","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"20 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144677536","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
Cold Agglutinin Syndrome Secondary to Mycoplasma pneumoniae Infection in Adults: Results From a Large French Observational Study (MyCOLD Study). 成人肺炎支原体感染继发的感冒凝集素综合征:来自法国一项大型观察性研究(MyCOLD研究)的结果。
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-19 DOI: 10.1002/ajh.70010
Kevin Chevalier,Matthieu Holub,Romain Palich,Karine Blanckaert,Laurent Gilardin,Louis Terriou,Bérangère Arnould,Solal Bellaiche,Samuel Deshayes,Julie Mérindol,Simon Rolland,Simon Valentin,Rishma Amarsy,Sylvain Audia,Virginie Baltes,Amaury Barret,Pierre-Louis Cariou,Jessy Cattelan,Hortense Chassepot,Dorothée Chopin,Mélissa Clément,Marine Coeffier,Thibault Comont,Soline De Monteynard,Charles Declerck,Pauline Durand,Mikael Ebbo,Gaël Galli,Amélie Godot,Sarra Hamrouni,Charlotte Kaeuffer,Jean-Emmanuel Kahn,Ludovic Lassel,Sophie Leautez,Anne-Lise Lecapitaine,Gwenael Le Moal,David Luque Paz,Martin Martinot,Natacha Mrozek,Valentine Pagis,Thomas Perpoint,Valéry Salle,Jean-François Viallard,Clément Viguier,Matthieu Mahevas,Bertrand Godeau,Etienne Crickx,Marc Michel
{"title":"Cold Agglutinin Syndrome Secondary to Mycoplasma pneumoniae Infection in Adults: Results From a Large French Observational Study (MyCOLD Study).","authors":"Kevin Chevalier,Matthieu Holub,Romain Palich,Karine Blanckaert,Laurent Gilardin,Louis Terriou,Bérangère Arnould,Solal Bellaiche,Samuel Deshayes,Julie Mérindol,Simon Rolland,Simon Valentin,Rishma Amarsy,Sylvain Audia,Virginie Baltes,Amaury Barret,Pierre-Louis Cariou,Jessy Cattelan,Hortense Chassepot,Dorothée Chopin,Mélissa Clément,Marine Coeffier,Thibault Comont,Soline De Monteynard,Charles Declerck,Pauline Durand,Mikael Ebbo,Gaël Galli,Amélie Godot,Sarra Hamrouni,Charlotte Kaeuffer,Jean-Emmanuel Kahn,Ludovic Lassel,Sophie Leautez,Anne-Lise Lecapitaine,Gwenael Le Moal,David Luque Paz,Martin Martinot,Natacha Mrozek,Valentine Pagis,Thomas Perpoint,Valéry Salle,Jean-François Viallard,Clément Viguier,Matthieu Mahevas,Bertrand Godeau,Etienne Crickx,Marc Michel","doi":"10.1002/ajh.70010","DOIUrl":"https://doi.org/10.1002/ajh.70010","url":null,"abstract":"Mycoplasma pneumoniae (MP), primarily a respiratory pathogen, can cause extra-pulmonary manifestations including cold agglutinin syndrome (CAS). We conducted a national, multicenter, observational, ambispective study to describe the characteristics, risk factors, and outcomes of MP-associated CAS. Adult patients hospitalized for a MP-infection with CAS (hemolytic anemia with hemoglobin < 10 g/dL and C3 positive direct anti-globulin test) were included. Recovery was defined as hemoglobin > 10 g/dL off therapy. We also compared MP-infected patients with or without CAS. Sixty patients (51.7% of females; median age of 48.5 years) were included. CAS was diagnosed a median of 10 days after MP-infection symptoms onset. At diagnosis, the median hemoglobin level was 6.9 g/dL, and 71.7% of patients received red blood cell transfusions. Intensive care unit (ICU) admission was required in 45% of patients, and 16.7% experienced a venous thromboembolic event (VTE). Seventeen patients (28.3%) received glucocorticoids alone, while 40 (66.7%) did not receive any specific treatment for CAS. After a median follow-up of 56 (30-83) days, 90% of patients achieved recovery, while 2 patients (3.3%) died from sepsis and pulmonary embolism. Glucocorticoid use did not significantly impact the rate or timing of recovery. Compared with MP-infected patients from the MYCADO cohort study (n = 1267), CAS patients had significantly more VTE (p < 0.0001) and ICU admissions (p = 0.03). MP-associated CAS typically occurs 10 days after the first symptoms of MP infection and is associated with ICU admissions and VTE. Overall, the prognosis of CAS is good, and glucocorticoids do not appear to influence outcomes.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"267 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144664225","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
Determinants of 15-Year Progression-Free Survival in Multiple Myeloma; Real-World Data From a Single Institution. 多发性骨髓瘤患者15年无进展生存期的决定因素来自单一机构的真实世界数据。
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-18 DOI: 10.1002/ajh.70012
Meletios A Dimopoulos,Ioannis Ntanasis-Stathopoulos,Despina Fotiou,Foteini Theodorakakou,Eirini Solia,Vasiliki Spiliopoulou,Panagiotis Malandrakis,Erasmia Psimenou,Stavroula Giannouli,Nikolaos Kanellias,Vassilis Koutoulidis,Maria Roussou,Angeliki Andrikopoulou,Magdalini Migkou,Evangelos Eleutherakis-Papaiakovou,Maria Gavriatopoulou,Evangelos Terpos,Efstathios Kastritis
{"title":"Determinants of 15-Year Progression-Free Survival in Multiple Myeloma; Real-World Data From a Single Institution.","authors":"Meletios A Dimopoulos,Ioannis Ntanasis-Stathopoulos,Despina Fotiou,Foteini Theodorakakou,Eirini Solia,Vasiliki Spiliopoulou,Panagiotis Malandrakis,Erasmia Psimenou,Stavroula Giannouli,Nikolaos Kanellias,Vassilis Koutoulidis,Maria Roussou,Angeliki Andrikopoulou,Magdalini Migkou,Evangelos Eleutherakis-Papaiakovou,Maria Gavriatopoulou,Evangelos Terpos,Efstathios Kastritis","doi":"10.1002/ajh.70012","DOIUrl":"https://doi.org/10.1002/ajh.70012","url":null,"abstract":"The therapeutic advances during the last two decades have rendered multiple myeloma a chronic disease and, thus, it is important to identify patient subgroups which may have extremely favorable outcomes and optimize their treatment. The current study aimed to evaluate the clinical and disease characteristics of patients with very long follow-up (minimum 15 years), to identify those with very long survival (> 15 years) and those with very long disease remissions (> 15 years) after frontline treatment diagnosed at a single center from 1994 to 2009. Among 323 consecutive, unselected patients, the calculated 15-year and 20-year cumulative survival rates were 18% and 14%, respectively. Forty-nine survived for more than 15 years. Furthermore, the calculated 15-year and 20-year cumulative PFS rates for the 323 patients were 9% and 7%, respectively; 25 (8%) patients were identified as long-term progression-free survivors. Younger age (≤ 65 years), good performance status (ECOG PS 0-1), low/intermediate risk stratification (ISS 1 or 2), absence of adverse cytogenetic abnormalities, autologous transplantation and achievement of CR to initial therapy were significantly associated with prolonged PFS. Importantly, all patients who were in complete response with negative minimal residual disease at 15 years remained at the same disease status at last follow-up. In conclusion, among patients treated with either conventional chemotherapy or with first-generation novel agents (thalidomide, bortezomib, or lenalidomide), about 15% were long-term survivors and 8% remained in long-term remission for more than 15 years without maintenance treatment.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"94 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144664226","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
Reversal of Glomerular Hyperfiltration Following Hematopoietic Stem Cell Transplantation in Children With Sickle-Cell Anemia. 镰状细胞性贫血患儿造血干细胞移植后肾小球高滤过的逆转。
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-16 DOI: 10.1002/ajh.70004
Stella Huet,Annie Kamdem,Valentine Beaufront,Karima Yakouben,Nathalie Dhedin,Catherine Paillard,Isabelle Hau,Claire Falguière,Aoufa Dahmani,Bassem Khazem,Ekaterina Belozertsteva,Céline Delestrain,Stéphane Bechet,Cécile Arnaud,Corinne Pondarré
{"title":"Reversal of Glomerular Hyperfiltration Following Hematopoietic Stem Cell Transplantation in Children With Sickle-Cell Anemia.","authors":"Stella Huet,Annie Kamdem,Valentine Beaufront,Karima Yakouben,Nathalie Dhedin,Catherine Paillard,Isabelle Hau,Claire Falguière,Aoufa Dahmani,Bassem Khazem,Ekaterina Belozertsteva,Céline Delestrain,Stéphane Bechet,Cécile Arnaud,Corinne Pondarré","doi":"10.1002/ajh.70004","DOIUrl":"https://doi.org/10.1002/ajh.70004","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"74 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144640281","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
Lymphocyte Kinetics and Outcomes of Chimeric Antigen Receptor T Cell Therapy in Multiple Myeloma With Out of Specification Products 嵌合抗原受体T细胞治疗不合规格产品多发性骨髓瘤的淋巴细胞动力学和结果
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-15 DOI: 10.1002/ajh.70001
Sarah Dingli, Paul Rothweiler, Moritz Binder, Joselle Cook, Morie A. Gertz, Suzanne Hayman, Prashant Kapoor, Taxiarchis Kourelis, Shaji K. Kumar, Mustaqeem Siddiqui, Rahma Warsame, Yi Lin, Arthur G. Erdman, David Dingli
{"title":"Lymphocyte Kinetics and Outcomes of Chimeric Antigen Receptor T Cell Therapy in Multiple Myeloma With Out of Specification Products","authors":"Sarah Dingli, Paul Rothweiler, Moritz Binder, Joselle Cook, Morie A. Gertz, Suzanne Hayman, Prashant Kapoor, Taxiarchis Kourelis, Shaji K. Kumar, Mustaqeem Siddiqui, Rahma Warsame, Yi Lin, Arthur G. Erdman, David Dingli","doi":"10.1002/ajh.70001","DOIUrl":"https://doi.org/10.1002/ajh.70001","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"39 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629675","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
Validation of the ELN2024 and Mayo Genetic Risk Models in an External Cohort of 352 Patients With Newly Diagnosed AML Receiving Less-Intensive Therapies. ELN2024和Mayo遗传风险模型在352例接受低强度治疗的新诊断AML患者外部队列中的验证
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-15 DOI: 10.1002/ajh.70003
Marco Roncador,Maddalena Marconato,Jeremy Werner Deuel,Stefan Balabanov
{"title":"Validation of the ELN2024 and Mayo Genetic Risk Models in an External Cohort of 352 Patients With Newly Diagnosed AML Receiving Less-Intensive Therapies.","authors":"Marco Roncador,Maddalena Marconato,Jeremy Werner Deuel,Stefan Balabanov","doi":"10.1002/ajh.70003","DOIUrl":"https://doi.org/10.1002/ajh.70003","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"84 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144630347","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
Anti-ADAMTS13 Antibodies Trajectory is Associated With ADAMTS13 Recovery in Immune-Mediated TTP. 抗ADAMTS13抗体轨迹与免疫介导的TTP中ADAMTS13的恢复相关
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-15 DOI: 10.1002/ajh.70005
Marie Robert,Arthur Mageau,Ygal Benhamou,François Provôt,Jehane Fadlallah,Lionel Galicier,Elie Azoulay,Hafid Ait-Oufella,Tomas Urbina,Pascale Poullin,Alain Wynckel,Coralie Poulain,Nihal Martis,Pierre Perez,Virginie Rieu,Yahsou Delmas,Jean-Michel Halimi,Christelle Barbet,Amélie Seguin,Valérie Chatelet,Jean-François Augusto,Mathieu Legendre,Olivier Moranne,Carole Philipponnet,Bérengère Cador,Raïda Bouzid,Bérangère S Joly,Agnès Veyradier,Paul Coppo,
{"title":"Anti-ADAMTS13 Antibodies Trajectory is Associated With ADAMTS13 Recovery in Immune-Mediated TTP.","authors":"Marie Robert,Arthur Mageau,Ygal Benhamou,François Provôt,Jehane Fadlallah,Lionel Galicier,Elie Azoulay,Hafid Ait-Oufella,Tomas Urbina,Pascale Poullin,Alain Wynckel,Coralie Poulain,Nihal Martis,Pierre Perez,Virginie Rieu,Yahsou Delmas,Jean-Michel Halimi,Christelle Barbet,Amélie Seguin,Valérie Chatelet,Jean-François Augusto,Mathieu Legendre,Olivier Moranne,Carole Philipponnet,Bérengère Cador,Raïda Bouzid,Bérangère S Joly,Agnès Veyradier,Paul Coppo, ","doi":"10.1002/ajh.70005","DOIUrl":"https://doi.org/10.1002/ajh.70005","url":null,"abstract":"Current triplet regimens associating therapeutic plasma exchange (TPE), immunosuppression with corticosteroids and rituximab, and caplacizumab have dramatically improved the outcome of immune-mediated thrombotic thrombocytopenic purpura (iTTP). However, nearly half of the patients require extended caplacizumab treatment (i.e., > 30 days) due to persistent ADAMTS13 deficiency, raising cost and tolerance concerns. Therefore, we investigated whether anti-ADAMTS13 antibodies titer and their trajectory during the acute phase of the disease could predict ADAMTS13 improvement (i.e., activity ≥ 20% before day-30 post-TPE). From a cohort of 286 patients receiving the triplet regimen, we identified on diagnosis a cut-off value for anti-ADAMTS13 IgG antibodies of 90.5 U/mL, with a modest discriminating ability (AUC: 0.57) for predicting long-term response, precluding its use to guide therapeutic strategies. Nonetheless, the analysis of anti-ADAMTS13 IgG antibodies titer trajectory from diagnosis revealed that the proportion of iTTP patients with ADAMTS13 activity improvement was higher in patients who decreased (Dec+) their antibodies titer within the 7-14 days interval post-TPE compared to those without decrease (Dec-) (65% vs. 25% of cases, respectively, p < 0.001), a finding confirmed in a validation cohort (N = 51). These results highlight the possibility of intensifying immunosuppression in an early period post-TPE to shorten time to ADAMTS13 activity recovery. Close monitoring of anti-ADAMTS13 antibodies titer may guide immunomodulation strategies, including additional courses of B-cell depleting agents when appropriate.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"35 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144630346","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
Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report 遗传性出血性毛细血管扩张出血的术语、定义和结果标准的标准化:国际共识报告
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-15 DOI: 10.1002/ajh.70011
Hanny Al‐Samkari, Raj S. Kasthuri, Hans‐Jurgen Mager, Jenny Y. Zhou, Marcelo M. Serra, Bethany T. Samuelson‐Bannow, Layla N. Van Doren, Jay F. Piccirillo, Marianne S. Clancy, Keith R. McCrae, Sonia M. Thomas, Antoni Riera‐Mestre, Allyson M. Pishko, Sarah Sewaralthahab, James R. Gossage, Vivek N. Iyer, Cedric Hermans, Adrienne Hammill, Ingrid Winship, Meir Mei‐Zahav, Annette von Drygalski, Scott Olitsky, Marie E. Faughnan
{"title":"Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report","authors":"Hanny Al‐Samkari, Raj S. Kasthuri, Hans‐Jurgen Mager, Jenny Y. Zhou, Marcelo M. Serra, Bethany T. Samuelson‐Bannow, Layla N. Van Doren, Jay F. Piccirillo, Marianne S. Clancy, Keith R. McCrae, Sonia M. Thomas, Antoni Riera‐Mestre, Allyson M. Pishko, Sarah Sewaralthahab, James R. Gossage, Vivek N. Iyer, Cedric Hermans, Adrienne Hammill, Ingrid Winship, Meir Mei‐Zahav, Annette von Drygalski, Scott Olitsky, Marie E. Faughnan","doi":"10.1002/ajh.70011","DOIUrl":"https://doi.org/10.1002/ajh.70011","url":null,"abstract":"<jats:label/>Hereditary hemorrhagic telangiectasia (HHT, Osler‐Weber‐Rendu disease) is the second most common inherited bleeding disorder worldwide, affecting approximately 1 in 5000 people. Development of disease‐modifying and efficacious hemostatic agents to treat HHT has finally begun after decades without such medical therapies. However, the lack of consensus on standardized severity definitions, outcome criteria, and terminology remains a major obstacle to clinical investigation and therapeutic development in HHT. Additionally, with the ongoing repurposing of antiangiogenic agents and emerging development of novel HHT‐specific therapies, comparative clinical trials are expected in the future. Therefore, to end the problematic heterogeneity hindering these efforts, the Global Research and Medical Advisory Board (GRMAB) of the Cure HHT Foundation, an international group of recognized HHT experts, convened a conference of expert HHT clinician‐investigators from within GRMAB as well as invited external experts in HHT and bleeding disorders generally, patients with HHT, and patient advocates to define standard terminology and definitions for outcomes and severity classification for bleeding and anemia in HHT. These criteria and definitions should be adopted by regulators, investigators, and the pharmaceutical industry in the development and performance of interventional clinical trials and cohort studies to allow improved comparability between clinical trials, facilitate communication between clinicians and investigators, improve therapeutic guideline development, and provide a standardized framework for regulatory agencies.Search Strategy and Selection CriteriaEvidence for this report was systematically identified and evaluated utilizing two search strategies in Ovid MEDLINE, described in full detail in the Appendix, pp.4–13. The searches were conducted on January 7, 2025. The titles and abstracts of each record were reviewed, and the inclusion criteria were applied to all search results to identify full text articles to be retrieved for further review. All retrieved full texts were then reviewed to reach a final determination if a study met the inclusion criteria. Included references were then compiled into evidence tables, which were then utilized by the International Consensus Report Working Group throughout the development of report recommendations.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"87 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144629674","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
Hemolysis and Acquired Pyruvate Kinase Deficiency in a Child With a Malignant Myeloid Disorder. 儿童恶性髓系疾病的溶血和获得性丙酮酸激酶缺乏。
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-14 DOI: 10.1002/ajh.70009
Wendy B Wong,Michael Jeng,Louise Lo,Bertil Glader
{"title":"Hemolysis and Acquired Pyruvate Kinase Deficiency in a Child With a Malignant Myeloid Disorder.","authors":"Wendy B Wong,Michael Jeng,Louise Lo,Bertil Glader","doi":"10.1002/ajh.70009","DOIUrl":"https://doi.org/10.1002/ajh.70009","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"23 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144622121","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
Superior GVHD‐Free, Relapse‐Free Survival for Haploidentical Transplant With PTCy Than Matched Unrelated Donor for AML Patients Transplanted in Second Complete Remission: A Study From the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation 来自欧洲血液和骨髓移植学会急性白血病工作组的一项研究:在第二次完全缓解的AML患者中,PTCy单倍体移植比匹配的无亲属供体移植具有更高的无GVHD、无复发生存率
IF 12.8 1区 医学
American Journal of Hematology Pub Date : 2025-07-12 DOI: 10.1002/ajh.70008
Yishan Ye, Myriam Labopin, Ibrahim Yakoub‐Agha, Gérard Socié, Didier Blaise, Tobias Gedde‐Dahl, Igor Wolfgang Blau, Anna Maria Raiola, Jennifer Byrne, Etienne Daguindau, Hélène Labussière‐Wallet, Anne Huynh, Ali Bazarbachi, Arnon Nagler, Eolia Brissot, Lin Li, Yi Luo, Jimin Shi, Mohamad Mohty, He Huang, Fabio Ciceri
{"title":"Superior GVHD‐Free, Relapse‐Free Survival for Haploidentical Transplant With PTCy Than Matched Unrelated Donor for AML Patients Transplanted in Second Complete Remission: A Study From the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation","authors":"Yishan Ye, Myriam Labopin, Ibrahim Yakoub‐Agha, Gérard Socié, Didier Blaise, Tobias Gedde‐Dahl, Igor Wolfgang Blau, Anna Maria Raiola, Jennifer Byrne, Etienne Daguindau, Hélène Labussière‐Wallet, Anne Huynh, Ali Bazarbachi, Arnon Nagler, Eolia Brissot, Lin Li, Yi Luo, Jimin Shi, Mohamad Mohty, He Huang, Fabio Ciceri","doi":"10.1002/ajh.70008","DOIUrl":"https://doi.org/10.1002/ajh.70008","url":null,"abstract":"Donor preference for acute myeloid leukemia (AML) patients transplanted in second complete remission (CR2) remains unclear, and hematopoietic cell transplantation (HCT) with post‐transplant cyclophosphamide (PTCy) from a haploidentical donor (HAPLO) merits attention. Data of 3878 adult AML patients receiving a first allo‐HCT in CR2 from the European Society of Blood and Marrow Transplantation registry between 2010 and 2022 were analyzed. Univariate analyses and Cox regression models were used. Results of HCTs from 803 HAPLO PTCy, 1271 matched sibling donor (MSD), and 1804 matched unrelated donor (MUD) were analyzed. A higher proportion (80.7%) of patients with European LeukemiaNet (ELN2022) intermediate−/adverse‐risk cytogenetics received an allo‐HCT from HAPLO PTCy than from either MUD (79.6%) or MSD (70.2%). On multivariate analysis, HAPLO PTCy grafts (hazard ratio [HR] = 0.65, 95% confidence interval [CI] 0.51–0.82; <jats:italic>p</jats:italic> &lt; 0.001) were associated with a lower relapse incidence (RI) compared with MSD HCTs, although non‐relapse mortality was higher (HR = 1.77, 95% CI 1.34–2.34; <jats:italic>p</jats:italic> &lt; 0.001). No difference was observed with respect to leukemia‐free survival and graft‐versus‐host disease (GVHD)‐free, relapse‐free survival (GRFS) for HAPLO PTCy compared to MSD grafts. Notably, HAPLO PTCy HCT was associated with significantly lower RI (HR = 0.64, 95% CI 0.48–0.82; <jats:italic>p</jats:italic> &lt; 0.001), chronic GVHD (cGVHD) (HR = 0.64, 95% CI 0.51–0.81; <jats:italic>p</jats:italic> &lt; 0.001) and extensive cGVHD (HR = 0.47, 95% CI 0.34–0.66; <jats:italic>p</jats:italic> &lt; 0.001) incidences compared to MUD HCTs. Collectively, HAPLO PTCy HCT was associated with superior GRFS (HR = 0.81, 95% CI 0.68–0.95; <jats:italic>p</jats:italic> = 0.013) than MUD HCT. For AML patients in CR2, HAPLO PTCy HCT is associated with lower RI and cGVHD, leading to superior GRFS compared with MUD HCTs.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"6 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144611121","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
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信