American Journal of Hematology最新文献

{"title":"Cerebral Small Vessel Disease in Immune‐Mediated Thrombotic Thrombocytopenic Purpura Patients During the Acute Phase and Disease Remission","authors":"Addolorata Truma, Francesco Maria Lo Russo, Giorgio Conte, Ilaria Mancini, Andrea Artoni, Juri Alessandro Giannotta, Barbara Ferrari, Pasquale Agosti, Maria Abbattista, Matteo Gagliardi, Eleonora Piccin, Marco Stroppi, Fabio Maria Triulzi, Flora Peyvandi","doi":"10.1002/ajh.70506","DOIUrl":"https://doi.org/10.1002/ajh.70506","url":null,"abstract":"Immune‐mediated thrombotic thrombocytopenic purpura (iTTP) is a life‐threatening thrombotic microangiopathy characterized by acute neurological manifestations and long‐term sequelae related to microvascular brain injury. Cerebral small vessel disease (cSVD), detectable on MRI through lacunes, microbleeds, white matter hyperintensities, and enlarged perivascular spaces, is a risk factor for stroke and cognitive decline, but its prevalence and evolution in iTTP are unknown. We conducted a prospective study of consecutive patients with acute iTTP and age‐matched controls to assess the prevalence and one‐year progression of cSVD. Brain MRI was performed at enrollment and repeated after 12–18 months in clinical remission. cSVD burden was quantified using a standardized 0–4 score based on four MRI markers. Twenty‐five patients (median age 56 years; 60% women) were enrolled. Acute focal ischemic lesions were detected in 13 (52%). cSVD was present in 48% of patients versus 27% of controls, while moderate burden (score ≥ 2) occurred in 24% versus none. Ordinal logistic regression showed an association between iTTP and higher cSVD scores (OR 3.19, 95% CI 1.00–10.14), independent of traditional cardiovascular risk factors. Follow‐up MRI was available in 22 patients (88%). In paired analyses, cSVD prevalence increased from 50% to 64%, moderate‐to‐high burden from 27% to 41%, and six patients (27%) showed score progression. At 1 year, patients had higher odds of greater cSVD burden than controls (age‐ and sex‐adjusted ordinal OR 7.00, 95% CI 1.65–29.74). These findings indicate that cSVD is common during acute iTTP and progresses during the first year despite hematological remission.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"396 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149019980","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":"Immune Effector Cell Therapies for Immune Thrombocytopenia Concurrent With Hematologic Malignancy","authors":"Saarang R. Deshpande, Debbie Jiang, Rebecca K. Leaf, Andrew Song, P. Connor Johnson, Caron Jacobson, Jeremy Abramson, Hanny Al‐Samkari","doi":"10.1002/ajh.70505","DOIUrl":"https://doi.org/10.1002/ajh.70505","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"13 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149019772","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":"Superficial Vein Thrombosis During Pregnancy and Postpartum—Findings From the French Multicenter Cohort “ HEMOTHEPP ” Study","authors":"Eloïse Laouenan, Brigitte Pan‐Petesch, Karine Morcel, Claire de Moreuil, Emmanuelle Courtois‐Communier, Marie‐Antoinette Sevestre, Sara Robin, François Anouilh, Cécile Tromeur, Christophe Leroyer, Francis Couturaud, Emmanuelle Le Moigne","doi":"10.1002/ajh.70503","DOIUrl":"https://doi.org/10.1002/ajh.70503","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"29 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149009891","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":"Real-World Experience of Mitapivat in Pyruvate Kinase Deficiency.","authors":"Giacinto Luca Pedone, Paolo Ricchi, Michela Ribersani, Francesco Arcioni, Teresa Ceglie, Filippo Mazzi, Antonella Sau, Maria Pia Solfrizzi, Caterina Giovanna Valentini, Anna Marcello, Paola Bianchi, Marta Bortolotti, Francesco Passamonti, Wilma Barcellini, Bruno Fattizzo","doi":"10.1002/ajh.70504","DOIUrl":"https://doi.org/10.1002/ajh.70504","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":9.4,"publicationDate":"2026-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148987603","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":"Venetoclax Plus Hypomethylating Agent for 7- Versus 14- Versus 21- Versus 28-Day Cycles in Newly-Diagnosed Acute Myeloid Leukemia: ELN and Mayo Genetic Risk-Stratified Analysis in 540 Patients.","authors":"Sudhesh Kumar, Momna Warraich, Mahnoor Fatima, Kristen McCullough, Aref Al-Kali, Hassan B Alkhateeb, Kebede H Begna, Abhishek A Mangaonkar, Antoine N Saliba, Mark R Litzow, William Hogan, Mithun Shah, Mrinal M Patnaik, Animesh Pardanani, Talha Badar, James Foran, Jeanne Palmer, Cecilia Arana Yi, Ayalew Tefferi, Naseema Gangat","doi":"10.1002/ajh.70500","DOIUrl":"https://doi.org/10.1002/ajh.70500","url":null,"abstract":"<p><p>Patients with newly-diagnosed acute myeloid leukemia (ND-AML) derive variable survival benefit from venetoclax (Ven) plus hypomethylating agent (HMA) therapy, and the optimal Ven duration across genetic risk groups remains undefined. Among 540 ND-AML patients receiving Ven-HMA at Mayo Clinic, outcomes were compared across Ven 7- (n = 33), 14- (n = 117), 21- (n = 96), and 28-day (n = 294) schedules during Cycle 1 and stratified by ELN 2024 and Mayo genetic risk groups. At a median follow-up of 37.7 months, allogeneic stem cell transplant (ASCT) rates were similar across Ven duration groups (15%, 18%, 18%, and 20% for 7-, 14-, 21-, and 28-day; p = 0.86). Median transplant-censored survival was comparable across Ven durations (13.3, 11.9, 16.8, and 13.2 months for 7, 14, 21, 28 days, respectively; p = 0.65), with outcomes driven by ELN risk (6.3, 11.5, 18.3 months for high, intermediate, low; p < 0.01) and Mayo genetic risk (6.9, 17.8 months, not reached; p < 0.01). Survival was comparable across Ven durations within ELN intermediate/low-risk and all Mayo risk groups. Among ELN high-risk patients, 14-day Ven was associated with inferior transplant-censored survival compared to 21- and 28-day schedules (p < 0.01). Notably, 30- and 60-day mortality were higher with shorter Ven schedules (7-day: 9%/18%; 14-day: 7%/15%) versus 28-day (2%/6%), which likely reflects treatment selection bias. In ND-AML, no significant difference in transplant-censored survival was observed across the 7-, 14-, 21-, and 28-day Ven schedules; prognosis was determined primarily by Mayo and ELN 2024 genetic risk rather than by Ven duration. Prospective trials are needed to establish risk-adapted Ven dosing strategies.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":9.4,"publicationDate":"2026-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148987611","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":"Hairy Cell Leukemia: 2026 Update on Diagnosis, Risk-Stratification, and Treatment.","authors":"Xavier Troussard, Elsa Maître, Jérôme Paillassa","doi":"10.1002/ajh.70441","DOIUrl":"10.1002/ajh.70441","url":null,"abstract":"<p><strong>Disease overview: </strong>Hairy cell leukemia (HCL) and HCL-like disorders, including HCL variant (HCL-V) and splenic diffuse red pulp lymphoma (SDRPL), are a very heterogeneous group of mature lymphoid B-cell disorders characterized by the identification of hairy cells, a specific immunophenotypic and genetic profile, a different clinical course and the need for appropriate treatment.</p><p><strong>Diagnosis: </strong>Diagnosis of HCL is based on morphological evidence of hairy cells, characteristic flow cytometric immunophenotype (CD11c, CD103, CD123, CD25) bone marrow trephine biopsy which makes it possible to specify the degree of tumoral bone marrow infiltration and presence of the Annexin A1 marker in immunohistochemistry, and presence of the BRAF^V600E mutation.</p><p><strong>Risk stratification: </strong>Progression of patients with HCL is based on a large splenomegaly, leukocytosis, a high number of hairy cells in the peripheral blood and the unmutated ImmunoGlobulin HeaVy (IGHV) chain variable region gene mutational status. VH4-34 positive HCL cases are associated with a poor prognosis, as well as the rare cases of HCL with TP53 mutations and HCL-V.</p><p><strong>Treatment: </strong>Patients should be treated only if HCL is symptomatic. Chemotherapy with purine analogs (PNAs) are indicated in first-line HCL patients. The use of chemo-immunotherapy combining cladribine (CDA) and rituximab (R) represents an increasingly used therapeutic approach. Management of relapsed/refractory disease is based on the use of BRAF inhibitors (BRAFi) plus R, MEK inhibitors (MEKi), Bruton Tyrosine Kinase inhibitors (BTKi) and/or Bcl-2 inhibitors (Bcl-2i). The optimal sequence of the different treatments remains to be determined.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":9.4,"publicationDate":"2026-09-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148966180","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":"Could Mixed CAR T-Cell Products and Overlapping Patients Overestimate the Risk of HLH With Tisagenlecleucel?","authors":"Wenqiang Chen, Qiuhong Ma, Zhenzhen Chen","doi":"10.1002/ajh.70493","DOIUrl":"https://doi.org/10.1002/ajh.70493","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":9.4,"publicationDate":"2026-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148962769","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":"Splenic Tropism and Spleen‐Modulated Systemic Inflammation in Acute\u0000 \u0000 Plasmodium vivax\u0000 \u0000 Malaria","authors":"Steven Kho,Hasrini Rini,Noy N. Kambuaya,Syawal Satria,Freis Candrawati,Putu A. I. Shanti,King Alexander,Benediktus Andries,Aisah R. Amelia,Anjana Rai,Kim A. Piera,Agatha M. Puspitasari,Ristya Amalia,Pak Prayoga,Leo Leonardo,Maulina Hafidzah,Theo Situmorang,Dewi S. Margayani,Desandra A. Rahmayenti,Pengxing Cao,Enny Kenangalem,Leily Trianty,Damian Oyong,Julie A. Simpson,Rintis Noviyanti,Pierre A. Buffet,Jeanne R. Poespoprodjo,Nicholas M. Anstey","doi":"10.1002/ajh.70498","DOIUrl":"https://doi.org/10.1002/ajh.70498","url":null,"abstract":"ABSTRACT In chronic Plasmodium vivax (Pv) infection, the spleen accounts for over 98% of total‐body parasite biomass. Whether splenic tropism also occurs in acute infection and how the spleen influences pathogenesis have not been systematically explored. In Papua, Indonesia, we compared clinical and hematology data in 24 spleen‐intact and 25 previously splenectomized patients with acute uncomplicated vivax malaria. Plasma levels of Pv lactate dehydrogenase (PvLDH) and markers of intravascular haemolysis, inflammation, endothelial activation and neutrophil activation were measured by ELISA. Circulating parasitaemia was four times higher in splenectomized compared to spleen‐intact patients but total‐body Pv biomass (PvLDH) was three times lower in patients without a spleen. Parasite staging and greater organ‐specific symptoms suggest parasite redistribution in the absence of a spleen. Linear regression modeling adjusting for circulating parasitaemia, age, sex and fever duration demonstrated an 8.1‐fold higher PvLDH concentration in spleen‐intact patients, indicating a splenic biomass accounting for 89% of total‐body parasites. Giemsa‐based splenic histology in an untreated patient splenectomized during acute vivax malaria revealed a spleen‐to‐blood biomass ratio of 10.7. In spleen‐intact patients, splenic biomass correlated strongly with markers of disease intensity, endothelial activation and systemic inflammation, whereas circulating parasitaemia correlated weakly or not at all. Compared to spleen‐intact patients, cell‐free hemoglobin, endothelial activation and systemic inflammation were higher in splenectomized patients while inflammasome‐dependent responses were lower. In conclusion, Pv is predominantly an infection of the spleen in acute clinical vivax malaria. While the size of this hidden population correlates with disease intensity, the spleen likely modulates inflammatory pathways and heme‐associated pathology.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"3 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148922944","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":"The Presence and Percentage of Circulating Nucleated Red Blood Cells Reveal Distinct Characteristics in Adults With Sickle Cell Disease","authors":"Ugo Boccadifuoco, Ivan Lerner, Auria Godard, Luc Darnige, Laure Joseph, Wassim El Nemer, Geoffrey Cheminet, Jean‐Benoit Arlet","doi":"10.1002/ajh.70497","DOIUrl":"https://doi.org/10.1002/ajh.70497","url":null,"abstract":"Although circulating nucleated red blood cells (cNRBCs) have previously been detected in patients with sickle cell disease (SCD), their prevalence and clinical significance during steady‐state disease have not been established. We retrospectively analyzed 270 adults with an SS or Sβ <jats:sup>0</jats:sup> ‐thalassemia genotype. cNRBCs were counted during steady‐state disease. Patients were classified as high cNRBC+ (&gt; 3 per 100 white blood cells (WBCs)), low cNRBC+ (1–3 per 100 WBCs), or cNRBC− (no cNRBCs). cNRBC+ and cNRBC− patients were compared with regard to clinical and laboratory data and in vitro erythroid differentiation from peripheral blood CD34+ cells. cNRBCs were detected in 65.6% of the patients and encompassed all terminal erythroid stages. High cNRBC+ patients were more likely to be receiving hydroxyurea ( <jats:italic>p</jats:italic> = 0.024). cNRBC+ patients exhibited a lower median [IQR] hemoglobin level (8.8 [7.8–10] and 8.4 [7.4–9.4] in the high and low cNRBC+ groups, respectively vs. 9.3 [8.3–9.9] g/dL in the cNRBC− group; <jats:italic>p</jats:italic> = 0.024). The cNRBC+ percentage was correlated negatively with the reticulocyte and WBC counts and positively with the mean corpuscular volume and the ferritin level. Clinically, a trend toward a greater likelihood of acute chest syndrome was observed in cNRBC+ patients. High cNRBC+ patients' cells exhibited accelerated erythroid differentiation in vitro, suggestive of dyserythropoiesis. cNRBCs constitute a common, easily measurable marker of a distinct hematological phenotype in adult patients with SCD: more severe anemia and signs of dyserythropoiesis. Prospective studies are needed to determine the value of the cNRBC percentage for predicting disease severity and guiding the treatment of dyserythropoiesis.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"37 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148915496","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":"Basophilic Stippling Unmasks Pyrimidine 5′‐Nucleotidase Deficiency in a G6PD ‐Deficient Patient","authors":"Victor Bobée, Stéphane Moutereau, Maïssa Souissi, Pierre Lebreton, Loïc Garcon","doi":"10.1002/ajh.70495","DOIUrl":"https://doi.org/10.1002/ajh.70495","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"277 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2026-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148915495","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}