American Journal of Hematology最新文献

{"title":"Ibrutinib-related stellar hematomas of the palms and soles.","authors":"Naia Oillarburu, Loic Ysebaert, Caroline Protin, Ariadna Ortiz-Brugues, Sarah Baali, Estelle Parriel, Vincent Sibaud","doi":"10.1002/ajh.27514","DOIUrl":"https://doi.org/10.1002/ajh.27514","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142556966","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":"Prognostic significance of early acute splenic sequestration in children with severe sickle cell genotypes: A comprehensive longitudinal neonatal cohort study","authors":"Alizée Soulié, Cécile Arnaud, Serge Pissard, Isabelle Hau, Mickaël Shum, Fouad Madhi, Céline Delestrain, Sandra Biscardi, Sabine Blary, Bassem Khazem, Ekaterina Belozertsteva, Eric Guemas, Ralph Epaud, Annie Kamdem, Corinne Pondarré","doi":"10.1002/ajh.27517","DOIUrl":"https://doi.org/10.1002/ajh.27517","url":null,"abstract":"<p>Acute splenic sequestration crisis (ASSC) is one of the earliest acute clinical manifestations of sickle cell anemia (SCA), with a median age at first episode of 1.8 years [range: 0.4–12.9] as reported for our recently published regional longitudinal newborn cohort, beginning with the introduction of newborn screening (1986) and ending just before the introduction of preventive intensification with hydroxyurea (HU) in 2015.<span><sup>1</sup></span> Early predictive biomarkers have been identified for ASSC, but little is known about the impact of early ASSC on disease severity.<span><sup>2</sup></span> Unlike early dactylitis, early ASSC was not found to be associated with an increase in the risk of adverse outcomes, including death, stroke, frequent vaso-occlusive crisis (VOC), and recurrent acute chest syndrome (ACS), in a cohort of newborns with SCA.<span><sup>3</sup></span></p>\u0000<p>Our main objective here was to determine, from our SCA birth cohort, whether children experiencing early ASSC have a higher disease burden. In addition, we aimed to update clinical information on ASSC and confirm the prognostic factors identified in previous studies. Consistent with the French standards of care, for the whole cohort, disease-modifying therapies (DMT) were started only after the occurrence of complications: transfusion program (TP) was mainly implemented for stroke prevention, and HU was prescribed only to children over the age of 3 years for low hemoglobin (Hb) levels and/or recurrence of VOC/ACS. Specifically at our center, TP was offered for frequent VOC/ACS or anemia despite HU, or in children younger than 3 years, and hematopoietic stem cell transplantation (HSCT) to patients with cerebral vasculopathy or frequent VOC/ACS with a human leukocyte antigen-identical sibling. In our cohort-study, the use of DMT was thus considered a surrogate for disease severity.</p>\u0000<p>ASSC was defined as splenic enlargement (increase of at least 2 cm from baseline) measured below the costal margin and associated with acute anemia (decrease in Hb concentration >2 g/dL relative to the previous measurement). Early and late ASSC were defined as a first episode of ASSC occurring before or after the age of 2 years respectively. During ASSC, standard management was prompt transfusion to restore effective circulating volume. After the resolution of a first ASSC, local guidelines recommended watchful waiting, unless children had another reason for receiving TP or HU. After the second or third episode, then either splenectomy or a temporary prophylactic TP were considered, to prevent ASSC recurrence. The age at which splenectomy was considered (usually after 3 years of age) and the indication for splenectomy after TP (only if persistent splenomegaly during TP or systematic) varied over time.</p>\u0000<p>Children were classified into two groups on the basis of the timing of the first ASSC: before 2 years (early ASSC group), or after 2 years or no ASSC (other group). Descript","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"214 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142563324","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 myelodysplasia-related mutations on 2022 European LeukemiaNet genetic risk classification in de novo acute myeloid leukemia with normal karyotype.","authors":"Srija Shanker, Robert P Hasserjian, Yazan Madanat, Olga K Weinberg, Miguel D Cantu","doi":"10.1002/ajh.27518","DOIUrl":"https://doi.org/10.1002/ajh.27518","url":null,"abstract":"<p><p>De novo normal karyotype AML 2017ELN and 2022ELN Genetic Risk Category Changes and Overall Survival in Induction Treated Patients.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142543149","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":"Harnessing the prognostic potential of PHF6 mutations in chronic myelomonocytic leukemia","authors":"Francesco Onida","doi":"10.1002/ajh.27512","DOIUrl":"10.1002/ajh.27512","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 12","pages":"2266"},"PeriodicalIF":10.1,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142520742","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":"Toxicities and outcome after CD19-directed chimeric antigen receptor T-cell therapy for secondary neurolymphomatosis","authors":"Leon D. Kaulen,&nbsp;Philipp Karschnia,&nbsp;Sofia Doubrovinskaia,&nbsp;Jeremy S. Abramson,&nbsp;Maria Martinez-Lage,&nbsp;Ganesh Shankar,&nbsp;Bryan D. Choi,&nbsp;Jeffrey A. Barnes,&nbsp;Areej El-Jawahri,&nbsp;Ephraim P. Hochberg,&nbsp;P. Connor Johnson,&nbsp;Jacob D. Soumerai,&nbsp;Wolfgang Wick,&nbsp;Marcela V. Maus,&nbsp;Yi-Bin Chen,&nbsp;Matthew J. Frigault,&nbsp;Jorg Dietrich","doi":"10.1002/ajh.27505","DOIUrl":"10.1002/ajh.27505","url":null,"abstract":"<p>Lymphomatous infiltration of the peripheral nervous system (PNS), termed neurolymphomatosis, represents a distinct extranodal non-Hodgkin lymphoma variant with dismal outcome. CD19-directed chimeric antigen receptor (CD19-CAR) T-cell therapy has emerged as a safe and effective treatment for B-cell lymphomas. We aimed to assess toxicity and efficacy of CD19-CAR T-cells in neurolymphomatosis. Neurolymphomatosis patients treated with CD19 CAR T-cells were retrospectively identified at Massachusetts General Hospital over a six-year period. Toxicities were graded according to the ASTCT classification, management, and response rates were recorded. Eleven neurolymphomatosis patients were identified with a median of 2 lines of PNS-directed treatments (range: 1-3) prior to receiving CD19-CAR T-cells. Neurolymphomatosis localized to the nerve roots (8/11, 73%), plexus (5/11, 45%), peripheral (4/11, 36%) and cranial nerves (5/11, 45%). Low grade cytokine release syndrome (CRS) was detected in 8/11 (73%; grade 1: N = 7; grade 2: N = 1) cases. Low- and high-grade immune cell-associated neurotoxicity syndrome (ICANS) were recorded in 5/11 (45%; grade 1: N = 4; grade 2: N = 1) and 1/11 (9%; grade 4) patients, respectively. CRP levels at infusion were predictive of ICANS (area under the curve: 0.96, p = 0.01). Seven of eleven neurolymphomatosis patients (64%) responded to CD19-CAR T-cells. Complete remissions (CR) were achieved in three cases (27%), with 2 patients in sustained CR nine and 46 months after CD19-CAR infusion. Median progression-free survival (PFS) was 4 months. Collectively, CD19-CAR T-cell treatment was well tolerated and showed promising efficacy in recurrent neurolymphomatosis, a difficult to treat condition with unmet medical need. Findings suggest that CD19-CAR may sufficiently penetrate the blood-nerve barrier. Toxicity and outcomes were overall similar to CAR-T cell therapy in CNS lymphoma.\u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure></p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 12","pages":"2411-2415"},"PeriodicalIF":10.1,"publicationDate":"2024-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27505","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142488235","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Emergency department intravenous fluid resuscitation and renal outcomes among adults with sickle cell disease.","authors":"Marcus A Carden,Jeffrey Lebensburger,Wayne Rosamond,Paula Tanabe,Vimal K Derebail","doi":"10.1002/ajh.27509","DOIUrl":"https://doi.org/10.1002/ajh.27509","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"14 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142488236","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":"Parsaclisib for the treatment of primary autoimmune hemolytic anemia: Results from a phase 2, open-label study","authors":"Wilma Barcellini,&nbsp;Fabrizio Pane,&nbsp;Andrea Patriarca,&nbsp;Irina Murakhovskaya,&nbsp;Louis Terriou,&nbsp;Maria T. DeSancho,&nbsp;Wahid T. Hanna,&nbsp;Lance Leopold,&nbsp;Erica Rappold,&nbsp;Ke Szeto,&nbsp;Shaoceng Wei,&nbsp;Ulrich Jäger","doi":"10.1002/ajh.27493","DOIUrl":"10.1002/ajh.27493","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <p>Autoimmune hemolytic anemia (AIHA) is a group of acquired autoimmune disorders characterized by red blood cell hemolysis. In a phase 2, open-label, multicenter study, adults with warm AIHA, cold agglutinin disease, or mixed-type AIHA were administered once-daily 1.0 or 2.5 mg parsaclisib (selective phosphoinositide 3-kinase δ inhibitor) orally for 12 weeks, followed by an extension period. Dose increases (for AIHA worsening) or decreases (for tolerability) were permitted. Primary efficacy endpoint was the proportion of patients with complete (≥12 g/dL hemoglobin [Hgb]) or partial (10–12 g/dL Hgb or ≥2 g/dL increase from baseline) response at any visit during weeks 6–12 not attributable to transfusion. Among 25 enrolled patients (median age, 63 y), 16 (64%) achieved a partial or complete Hgb response during weeks 6–12. Responses were observed by week 1 in 52.0% of patients with incremental improvements during weeks 6–12 and sustained responses during the extension period. Responses were higher among patients with warm AIHA versus other types (75.0% vs. 44.4%). Clinically meaningful improvements in Functional Assessment of Chronic Illness Therapy-Fatigue scores were observed at weeks 6 and 12. All patients had treatment-emergent adverse events (TEAEs), most commonly diarrhea (32.0%) and pyrexia (28.0%). Grade ≥3 TEAEs occurred in 13 patients (52.0%). TEAEs considered possibly related to treatment occurred in 11 patients (44.0%). No dose reductions were required; six patients (24%) discontinued for a TEAE. In summary, parsaclisib was well tolerated and resulted in substantial improvements in Hgb response at week 1, with durable responses through the extension period.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Clinical trial registration</h3>\u0000 \u0000 <p>This trial was registered at ClinicalTrials.gov (NCT03538041).</p>\u0000 </section>\u0000 </div>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 12","pages":"2313-2320"},"PeriodicalIF":10.1,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27493","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142456088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Fulminant intravascular hemolysis resulting from Clostridium perfringens infection.","authors":"Kyo J P H Renshof, Yorick Sandberg, Floor Weerkamp, Barbara J Bain","doi":"10.1002/ajh.27511","DOIUrl":"10.1002/ajh.27511","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142456087","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":"Clonal hematopoiesis and myeloid skewing in older population-based individuals","authors":"Maaike G. J. M. van Bergen,&nbsp;Priscilla Kamphuis,&nbsp;Aniek O. de Graaf,&nbsp;Jonas B. Salzbrunn,&nbsp;Theresia N. Koorenhof-Scheele,&nbsp;Isabelle A. van Zeventer,&nbsp;Avinash G. Dinmohamed,&nbsp;Jan Jacob Schuringa,&nbsp;Bert A. van der Reijden,&nbsp;Gerwin Huls,&nbsp;Joop H. Jansen","doi":"10.1002/ajh.27495","DOIUrl":"10.1002/ajh.27495","url":null,"abstract":"&lt;p&gt;Hematopoietic stem cells (HSCs) continuously produce blood cells while maintaining their self-renewal, proliferation, and differentiation potential. Normal blood cell production is balanced between myeloid and lymphoid progeny. With aging, the number of HSCs increases but their differentiation potential declines.&lt;span&gt;&lt;sup&gt;1&lt;/sup&gt;&lt;/span&gt; One of the hallmarks of aged HSCs is a myeloid differentiation bias, with less capability of differentiation toward the lymphoid lineage, resulting in age-related myeloid skewing. Another common feature of the aging hematopoietic system is the increased prevalence of somatic driver mutations within the HSC compartment. Clonal outgrowth of a subpopulation of cells sharing a mutation in a hematological malignancy-associated driver gene is called clonal hematopoiesis (CH).&lt;span&gt;&lt;sup&gt;2&lt;/sup&gt;&lt;/span&gt; Since the prevalence of both conditions increase with age, we questioned whether there is an association between myeloid skewing and CH.&lt;/p&gt;&lt;p&gt;To gain insight into the changes in myeloid and lymphoid progeny upon aging, we analyzed all individuals from the Dutch population-based Lifelines cohort ≥18 years with available myeloid and lymphoid peripheral blood counts (&lt;i&gt;n&lt;/i&gt; = 144 676). In males, the percentage of myeloid cells from the total leukocytes increased significantly with aging (&lt;i&gt;p&lt;/i&gt; &lt; .001; Figures S1 and S2), while in females, the myeloid cells showed a periodic pattern with an initial increase, followed by a decrease during menopause and finally increased again from the age of 70 (Figures S1 and S2). A clear difference was observed between males and females for the changes in myeloid cell counts. This may be explained by changes in sex hormone levels, as the number of neutrophils decreases significantly during menopause in females. However, we observed a clear shift in the mean percentage of myeloid cells upon aging (Figure S1).&lt;/p&gt;&lt;p&gt;To investigate whether there is an association between the myeloid cell percentage and CH, we evaluated all individuals ≥60 years from the Lifelines cohort (&lt;i&gt;n&lt;/i&gt; = 21 727) with available myeloid and lymphoid blood cell counts from whom we had generated CH data previously (&lt;i&gt;n&lt;/i&gt; = 4607; Figures S3 and S4, Supplemental Methods; Data S1, Table S1). The percentage of myeloid cells was normally distributed in this cohort with a mean of 67.8% myeloid cells (Figure S4). From these individuals, &lt;i&gt;n&lt;/i&gt; = 1899 (41.2%) carried at least one driver gene mutation with a variant allele frequency (VAF) ≥1%. A significant association was observed between the percentage of myeloid cells and mutations in &lt;i&gt;JAK2&lt;/i&gt; (OR 1.06, 95% CI 1.03–1.09; &lt;i&gt;p&lt;/i&gt; &lt; .001), &lt;i&gt;SF3B1&lt;/i&gt; (OR 1.03, 95% CI 1.00–1.07; &lt;i&gt;p&lt;/i&gt; = .034), and &lt;i&gt;TET2&lt;/i&gt; (OR 1.01, 95% CI 1.00–1.02; &lt;i&gt;p&lt;/i&gt; = .020; Figure S4). Overall, no significant correlation was observed between the percentage of myeloid cells and the clone size in the cohort with available myeloid cell counts and CH (&lt;i&gt;n&lt;/i&gt; = 1899; &lt;i&gt;p&lt;/i&gt;","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 12","pages":"2402-2405"},"PeriodicalIF":10.1,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27495","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142451911","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dominance of mutations in epigenetic regulators and a diversity of signaling alterations in blast‐phase BCR::ABL1‐negative myeloproliferative neoplasms","authors":"Petruta Gurban, Cristina Mambet, Anca Botezatu, Laura G. Necula, Lilia Matei, Ana Iulia Neagu, Ioana Pitica, Marius Ataman, Aurelia Tatic, Alexandru Bardas, Mihnea A. Gaman, Camelia Dobrea, Mihaela Dragomir, Cecilia Ghimici, Silvana Angelescu, Doina Barbu, Oana Stanca, Marina Danila, Nicoleta Berbec, Andrei Colita, Ana Maria Vladareanu, Saviana Nedeianu, Mihaela Chivu‐Economescu, Coralia Bleotu, Daniel Coriu, Elise Sepulchre, Gabriela Anton, Carmen C. Diaconu, Stefan N. Constantinescu","doi":"10.1002/ajh.27503","DOIUrl":"https://doi.org/10.1002/ajh.27503","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"44 1","pages":""},"PeriodicalIF":12.8,"publicationDate":"2024-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142449401","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}