American Journal of Hematology最新文献

{"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}

{"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}

{"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}

{"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}

{"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}

{"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}

{"title":"Impact of Cytogenetic Response to Therapy on Long‐Term Survival in Acute Myeloid Leukemia","authors":"John Hanna, Emily C. Zabor, Moath Albliwi, Jessica El‐Asmar, Daniel P. Nurse, Ameed Bawwab, Hasan Abuamsha, Yomna Abu‐Farsakh, Heya Batah, Asad Rauf, Joy Nakitandwe, David S. Bosler, Akriti G. Jain, John C. Molina, Sophia Balderman, Abhay Singh, Aaron T. Gerds, Sudipto Mukherjee, Ronald M. Sobecks, Anjali S. Advani, Hetty E. Carraway, Caroline Astbury, Moaath K. Mustafa Ali","doi":"10.1002/ajh.70000","DOIUrl":"https://doi.org/10.1002/ajh.70000","url":null,"abstract":"Prognostication in acute myeloid leukemia (AML) relies on clinical, molecular, and cytogenetic factors. In this retrospective study, we examined the impact of different levels of cytogenetic response on overall survival (OS) and event‐free survival (EFS) in AML. Among 973 adult AML patients treated at Cleveland Clinic (5/2017–9/2023), 563 patients had baseline cytogenetic data and post‐treatment response assessment available. Based on baseline and response cytogenetic status, patients were categorized into: normal to normal (NL‐Cy to NL‐Cy, <jats:italic>n</jats:italic> = 221, 39%), normal or abnormal to gain (NL/Abnl‐Cy to Gain‐Cy, <jats:italic>n</jats:italic> = 46, 8.2%), abnormal to persistent (Abnl‐Cy to Persistent‐Cy, <jats:italic>n</jats:italic> = 81, 14%), abnormal to partial response (Abnl‐Cy to Partial‐Cy, <jats:italic>n</jats:italic> = 20, 3.6%), and abnormal to complete response (Abnl‐Cy to NL‐Cy, <jats:italic>n</jats:italic> = 195, 35%). Landmark analysis was used to account for post‐treatment assessments. The cohort had a median age of 62 years (interquartile range: 52–69), 256 females (45%), 90% were White, and median follow‐up of 45.8 months (range: 0.73–191.3). The median OS and hazard ratios (HRs) from multivariable regression analysis were as follows: NL‐Cy to NL‐Cy: 37 months (95% CI: 27–91), HR = reference; NL/Abnl‐Cy to Gain‐Cy: 14 months (95% CI: 8.6–30), HR = 1.5 (95% CI: 0.99–2.39); Abnl‐Cy to Persistent‐Cy: 13 months (95% CI: 12–18), HR = 1.61 (95% CI: 1.13–2.31); Abnl‐Cy to Partial‐Cy: 25 months (95% CI: 14‐NC), HR = 0.76 (95% CI: 0.39–1.49); and Abnl‐Cy to NL‐Cy: 27 months (95% CI: 19–101), HR = 1.25 (95% CI: 0.93–1.68) (<jats:italic>p</jats:italic> = 0.038). Achieving cytogenetic remission, complete or partial, was associated with better survival outcomes. These findings highlight the importance of monitoring cytogenetic responses to inform treatment decisions and support integrating cytogenetic response into risk‐adapted, personalized AML management strategies.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"197 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144602891","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":"Novel ADAMTS13 Mutation in a Patient With Congenital TTP Diagnosed in Pregnancy","authors":"William Frank Mawalla, Clara Chamba, Ahlam Nasser, Irene Jonathan, Emmanuel Josephat, Mbonea Yonazi, Zainab Yusuph, Elena Bresin, Giuseppe Remuzzi, Lucio Luzzatto","doi":"10.1002/ajh.70007","DOIUrl":"https://doi.org/10.1002/ajh.70007","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"697 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144602890","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":"Maintenance Therapy in the Era of Quadruplets for Multiple Myeloma: When, What, and for How Long?","authors":"Hira Mian, Luciano J. Costa","doi":"10.1002/ajh.70002","DOIUrl":"https://doi.org/10.1002/ajh.70002","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"21 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144593991","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":"Not All Hereditary Iron Overload Is Hemochromatosis: A Case of Hereditary Xerocytosis Unmasked by Blood Smear Morphology.","authors":"María-Angustias Molina-Arrebola,Barbara J Bain","doi":"10.1002/ajh.27766","DOIUrl":"https://doi.org/10.1002/ajh.27766","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"33 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144586586","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}