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Impact of busulfan versus treosulfan dose intensity in myelofibrosis undergoing hematopoietic cell transplantation Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-14 Nico Gagelmann, Claudia Schuh, Sarah Flossdorf, Desiree Kunadt, Matthias Stelljes, Igor W. Blau, Arne Brecht, Wolfgang Bethge, Thomas Schroeder, Gerald Wulf, Elisa Sala, Gesine Bug, Katharina Fleischhauer, Nicolaus Kröger
One key aspect of allogeneic hematopoietic cell transplantation (HCT) is pretransplant conditioning, balancing risk for relapse versus non-relapse mortality. Conditioning regimens with different alkylators at different doses can influence outcome, but data are missing for myelofibrosis, a challenging cohort of patients usually presenting at older age and with comorbidities. We evaluated in a multicenter
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Intravenous immunoglobulin as a rescue therapy for severe adult autoimmune hemolytic anemia: Results from a French multicenter observational study Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-14 M. Michel, M. Saïr, E. Rivière, G. Moulis, T. Comont, N. Costedoat‐Chalumeau, C. Pouchelon, D. Boutboul, A. Benyamine, A. Bert, P. ‐Y. Jeandel, S. Hamrouni, N. Belfeki, H. Lobbes, A. Dossier, D. Gobert, M. Mahevas, B. Godeau, Y. Gallien, M. Ebbo
Adult autoimmune hemolytic anemia (AIHA) is a rare but potentially life-threatening acquired autoimmune disease in which autoantibodies directed toward antigens of autologous red blood cells (RBC) membrane lead to their accelerated destruction. Corticosteroids are the cornerstone first-line therapy for primary warm AIHA (wAIHA) and rituximab is commonly used off-label as a second-line option in most
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Cost‐effectiveness of sutimlimab in cold agglutinin disease Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-11 Satoko Ito, Daniel Wang, Adriana Purcell, Karthik Chetlapalli, Alfred I. Lee, Adam Cuker, George Goshua
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Azathioprine/hydroxyurea preconditioning prior to nonmyeloablative matched sibling donor hematopoietic stem cell transplantation in adults with sickle cell disease: A prospective observational cohort study Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-11 Elisabeth Dovern, Mesire Aydin, Mette D. Hazenberg, Man Wai Tang, Elisabeth M. Suijk, Gerianne M. Hoogendoorn, Charlotte F. J. Van Tuijn, Jean‐Louis Kerkhoffs, Caroline E. Rutten, Sacha S. Zeerleder, Josu de la Fuente, Bart J. Biemond, Erfan Nur
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Clinical and biological characterization of involvement of nasal‐associated lymphoid tissues in chronic lymphocytic leukemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-10 Thimali Ranaweera Arachchige, Catherine Mendiburu, Antoine Martin, Carole Fleury, Elisabetta Dondi, Rémi Letestu, Virginie Eclache, Fanny Baran‐Marszak, Florence Cymbalista, Gregory Lazarian
Patients with chronic lymphocytic leukemia (CLL) are prone to infectious complications, including ear, nose, and throat (ENT) infections, due to humoral immunodepression and/or immunosuppression related to therapy. However, specific CLL infiltration in nonlymphoid regions of the head and neck causing ENT symptoms but unrelated to an infection has not been described. CLL is a disease of lymphoid organs
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Ponatinib‐review of historical development, current status, and future research Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-10 Hagop M. Kantarjian, Helen T. Chifotides, Fadi G. Haddad, Nicholas J. Short, Sanam Loghavi, Elias Jabbour
Ponatinib is a third‐generation BCR::ABL1 tyrosine kinase inhibitor (TKI) with high potency against Philadelphia chromosome (Ph)‐positive leukemias, including T315I‐mutated disease, which is resistant to first‐ and second‐generation TKIs. Ponatinib was approved for T315I‐mutated chronic myeloid leukemia (CML), CML resistant/intolerant to ≥2 prior TKIs, advanced phase CML and Ph‐positive acute lymphoblastic
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Correction to “Benefit of axicabtagene ciloleucel vs chemoimmunotherapy in older patients and/or patients with poor ECOG performance status with relapsed or refractory large B‐cell lymphoma after 2 or more lines of prior therapy” Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-07
Lunning, MA, Wang, H, Hu, Z-H, et al. Benefit of axicabtagene ciloleucel versus chemoimmunotherapy in older patients and/or patients with poor ECOG performance status with relapsed or refractory large B-cell lymphoma after 2 or more lines of prior therapy. Am J Hematol. 2024; 99(5): 880–889. doi:10.1002/ajh.27283 In Table 1, a typographical error was made in which the patient numbers and percentages
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Clinically meaningful improvements in patient‐reported outcomes in mitapivat‐treated patients with pyruvate kinase deficiency Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-07 Kevin H. M. Kuo, Rachael F. Grace, Eduard J. van Beers, Wilma Barcellini, Andreas Glenthøj, Susanne Holzhauer, Vanessa Beynon, Susan Morris, Junlong Li, Erin Zagadailov, Parija Patel, Hanny Al‐Samkari
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Dendritic cell vaccines extend CAR T‐cell persistence and improve the efficacy of CD19 CAR T‐cell therapy in refractory or relapsed adult B‐ALL patients Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-07 Sanfang Tu, Lijuan Zhou, Rui Huang, Xuan Zhou, Jilong Yang, Yanjie He, Yuxing Hu, Honghao Zhang, Xiaoling Xie, Yuhua Li
Although CD19 chimeric antigen receptor (CAR) T-cell therapy has a favorable complete response (CR) rate ranging from 71% to 93% for adult patients with refractory or relapsed (r/r) B-cell acute lymphoblastic leukemia (B-ALL),1 its long-term efficacy remains poor, with a 31%–100% relapse rate, a median leukemia-free survival (LFS) of 6.1–11.6 months, and a median overall survival (OS) of 12.9–18.2 months
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Unswitched memory B cell deficiency in children with sickle cell disease and response to pneumococcal polysaccharide vaccine Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-06 Venée N. Tubman, Daniel Maysonet, Norma Estrada, Tripti Halder, Lindsey Ramos, Sameera Bhamidipati, Alexandre F. Carisey, Charles G. Minard, Carl E. Allen
Early mortality in sickle cell disease (SCD) is attributed to increased infections due to loss of splenic function. Marginal zone B cells are important for initial opsonization of pathogens and can be absent in spleen histopathology in SCD. The frequency of unswitched memory B cells (UMBC), the circulating correlate of marginal zone B cells, reflects the immunologic function of the spleen. We hypothesized
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Polatuzumab vedotin, venetoclax, and an anti‐CD20 monoclonal antibody in relapsed/refractory B‐cell non‐Hodgkin lymphoma Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-03 Sam Yuen, Tycel J. Phillips, Rajat Bannerji, Paula Marlton, Giuseppe Gritti, John F. Seymour, Anna Johnston, Christopher Arthur, Anna Dodero, Sunil Sharma, Jamie Hirata, Lisa Musick, Christopher R. Flowers
The Phase 2 portion of this study evaluated safety and efficacy of polatuzumab vedotin 1.8 mg/kg and venetoclax 800 mg, plus fixed‐dose obinutuzumab 1000 mg or rituximab 375 mg/m2 in patients with relapsed/refractory (R/R) follicular lymphoma (FL) or diffuse large B‐cell lymphoma (DLBCL), respectively. Patients with complete response (CR) or partial response (PR)/stable disease (FL) or CR/PR (DLBCL)
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High prevalence of iron deficiency and socioeconomic disparities in laboratory screening of non‐pregnant females of reproductive age: A retrospective cohort study Am. J. Hematol. (IF 12.8) Pub Date : 2024-05-02 Sophia Wen, Rosane Nisenbaum, Angela C. Weyand, Grace H. Tang, Michael Auerbach, Michelle Sholzberg
Iron deficiency anemia (IDA) and non‐anemic iron deficiency (NAID) are highly prevalent among non‐pregnant females of reproductive age. Canada has no national screening guidelines for this population. Screening, when performed, is often with a complete blood count alone without ferritin or iron indices. The primary objective was to determine the prevalence of screening for NAID and IDA over a 3‐year
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Long‐term effectiveness of eliglustat treatment: A real‐world analysis from the International Collaborative Gaucher Group Gaucher Registry Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-30 Pramod K. Mistry, Manisha Balwani, Joel Charrow, Jeremy Lorber, Claus Niederau, Jenny L. Carwile, Antonio Oliveira‐dos‐Santos, Maria Gabriela Perichon, Sefika Uslu Cil, Priya S. Kishnani
Gaucher disease type 1 (GD1) is known for phenotypic heterogeneity and varied natural history. Registrational clinical trials enrolled narrowly defined phenotypes, but greater diversity is encountered in clinical practice. We report real‐world outcomes with long‐term eliglustat treatment in adults with GD1 in the International Collaborative Gaucher Group Gaucher Registry. Among 5985 GD1 patients in
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High risk of progression for chronic major organ complications of sickle cell disease in adolescents and young adults: A long‐term neonatal cohort study Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-30 Marion Borgey, Isabelle Genty, Anoosha Habibi, Jean‐Benoit Arlet, Nathalie Dhedin, François Lionnet, Emmanuelle Bernit, Maryse Etienne Julan, Gylna Loko, Cécile Arnaud, Annie Kamdem, Serge Pissard, Eric Guémas, Clara Noizat, Corinne Pondarré
The implementation of newborn screening associated with comprehensive care has led to the transformation of sickle cell disease (SCD) from a fatal illness in children to a chronic disease with multiple organ dysfunctions and premature death in adults.1, 2 Organ injury begins during childhood, as a result of chronic hemolysis-related endothelial dysfunction and hypoxia triggered by repeated vaso-occlusive
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Long‐term follow‐up of children with sickle cell disease diagnosed by newborn screening in the Netherlands: Overview of morbidity and mortality Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-29 Caroline Vuong, Corien L. Eckhardt, Harriët Heijboer, Monique H. Suijker, Lydian A. de Ligt, Aimee L.A. Voigt, Mariska M. G. Leeflang, Marije Bartels, Paul Brons, Louise Hooimeijer, Eva Rettenbacher, Frans J. Smiers, Marjet A. Stein‐Wit, Arian van der Veer, Annemieke Verbaan, Marjon H. Cnossen, Karin Fijnvandraat
Sickle cell disease (SCD) is the most prevalent and severe autosomal recessively inherited hemoglobinopathy. It is associated with chronic hemolytic anemia, recurrent vaso-occlusive events, organ, and tissue ischemia, resulting in a broad range of acute and chronic complications.1 In the Netherlands, approximately 2000 patients live with this disease, half of which are children. To diagnose patients
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RETRACTION: A predictive model of herpes zoster after allogeneic hematopoietic stem cell transplantation: VZV reactivation following antiviral prophylaxis discontinuation Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-26
Retraction: Feng, C‐J. , Zhao, P. , Fu, H‐X. , Yan, C‐H. , Wang, C‐C. , Zhu, X‐L. , He, Y. , Wang, F‐R. , Zhang, Y‐Y. , Mo, X‐D. , Kong, Y. , Han, W. , Wang, J‐Z. , Wang, Y. , Chen, H. , Chen, Y‐H. , Zhao, X‐Y. , Chang, Y‐J. , Xu, L‐P. , Liu, K‐Y. , Huang, X‐J. , Zhang, X‐H. (2023) . American Journal of Hematology. https://doi.org/10.1002/ajh.27090.The above article, published online on 29 September
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Unveiling the genetic landscape of suspected congenital dyserythropoietic anemia type I: A retrospective cohort study of 36 patients Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-26 Roberta Marra, Antonella Nostroso, Barbara Eleni Rosato, Federica Maria Esposito, Vanessa D'Onofrio, Anthony Iscaro, Antonella Gambale, Barbara Bruschi, Paola Coccia, Antonella Poloni, Sule Unal, Alberto Romano, Achille Iolascon, Immacolata Andolfo, Roberta Russo
Congenital Dyserythropoietic Anemia type I (CDA I) is a rare hereditary condition characterized by macrocytic/normocytic anemia, splenomegaly, iron overload, and distinct abnormalities during late erythropoiesis, particularly internuclear bridges between erythroblasts. Diagnosis of CDA I remains challenging due to its rarity, clinical heterogeneity, and overlapping phenotype with other rare hereditary
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Management of post‐autologous transplant relapse in patients with T‐cell lymphomas Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-25 Olivier Veilleux, Francisco Socola, Sally Arai, Matthew J. Frank, Laura Johnston, Robert Lowsky, Judith Shizuru, Everett Meyer, Lori Muffly, Andrew R. Rezvani, Parveen Shiraz, Surbhi Sidana, Saurabh Dahiya, David B. Miklos, Robert S. Negrin, Wen‐Kai Weng
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Combination of a TGF‐β ligand trap (RAP‐GRL) and TMPRSS6‐ASO is superior for correcting β‐thalassemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-25 Amaliris Guerra, Nolan Hamilton, Ariel Rivera, Perry Demsko, Shuling Guo, Stefano Rivella
A recently approved drug that induces erythroid cell maturation (luspatercept) has been shown to improve anemia and reduce the need for blood transfusion in non‐transfusion‐dependent as well as transfusion‐dependent β‐thalassemia (BT) patients. Although these results were predominantly positive, not all the patients showed the expected increase in hemoglobin (Hb) levels or transfusion burden reduction
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GLP‐1 agonists and SGLT‐2 inhibitors in adults with sickle cell disease Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-24 Ryan Sun, Anand Srivastava, Vimal K. Derebail, Jin Han, Robert E. Molokie, Victor Gordeuk, Santosh L. Saraf
Acute and chronic organ damage are key drivers of early mortality in adults with sickle cell disease (SCD). Chronic kidney disease (CKD) occurs in approximately half of adults with SCD and a rapid decline in estimated glomerular filtration rate (eGFR) portends an increased risk of progression to end-stage kidney disease (ESKD) and mortality.1 Although renin-angiotensin-aldosterone system (RAAS) blocking
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Thrombosis risk prediction in lymphoma patients: A multi‐institutional, retrospective model development and validation study Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-24 Shengling Ma, Jennifer La, Kaitlin N. Swinnerton, Danielle Guffey, Raka Bandyo, Giordana De Las Pozas, Katy Hanzelka, Xiangjun Xiao, Cristhiam M. Rojas‐Hernandez, Christopher I. Amos, Vipul Chitalia, Katya Ravid, Kelly W. Merriman, Christopher R. Flowers, Nathanael Fillmore, Ang Li
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Suppression of Hb Bart's to improve tissue oxygenation and fetal development in homozygous alpha‐thalassemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-24 G. Lugthart, E. J. T. Verweij, C. L. Harteveld, R. N. G. B. Tan, M. F. C. M. Knapen, F. Slaghekke, M. C. Haak, A. B. Mohseny, F. J. Smiers
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Lower relapse incidence with haploidentical versus matched sibling or unrelated donor hematopoietic cell transplantation for core‐binding factor AML patients in CR2: A study from the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-24 Yishan Ye, Myriam Labopin, Socié Gérard, Ibrahim Yakoub‐Agha, Igor Wolfgang Blau, Mahmoud Aljurf, Edouard Forcade, Tobias Gedde‐Dahl, David Burns, Jan Vydra, Khalid Halahleh, Rose‐Marie Hamladji, Ali Bazarbachi, Arnon Nagler, Eolia Brissot, Lin Li, Yi Luo, Yanmin Zhao, Fabio Ciceri, He Huang, Mohamad Mohty, Norbert Claude Gorin
Allogeneic hematopoietic cell transplantation (allo‐HCT) is recommended for core‐binding factor mutated (CBF) AML patients achieving second complete remission (CR2). However, approximately 20% of patients may relapse after transplant and donor preference remains unclear. We compared in this EBMT global multicenter registry‐based analysis the allo‐HCT outcomes using either haploidentical (Haplo), matched
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Identification of the characteristics and prognostic impact of FUS::ERG and RUNX1::CBFA2T3 fusion genes in adult acute myeloid leukemia patients Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-23 Ziyue Zhou, Nanfang Zhuo, Yile Zhou, Caihong Sun, Yiyi Yao, Liping Mao, Yi Zhang, Qing Hong, Peifeng Pan, Hongyan Tong, Jie Jin, Huafeng Wang
FUS::ERG/ t(16;21)(p11;q22) and RUNX1::CBFA2T3/ t(16;21)(q24;q22) are two rare non-random chromosome translocations involving chromosomes 16 and 21 and appear in less than 1% of acute myeloid leukemia (AML) cases.1 Prior research reported that FUS::ERG was characterized as an adverse-risk subtype with a high relapse rate and mortality, while the occurrence of RUNX1::CBFA2T3 was generally believed to
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Correction to “Red cell transfusion thresholds in outpatients with myelodysplastic syndromes: Combined results from two randomized controlled feasibility studies” Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-23
Buckstein R, Callum J, Prica A, Bowen D, Wells RA, Leber B, Heddle N, Chodirker L, Cheung M, Mozessohn L, Yee K, Gallagher J, Parmentier A, Jamula E, McQuilten Z, Wood EM, Weinkov R, Zhang L, Mamedov A, Stanworth SJ, and Lin Y. Red cell transfusion thresholds in outpatients with myelodysplastic syndromes: combined results from two randomized controlled feasibility studies. Am J Hematol. 2024, 99: 473–476
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Pediatric refractory chronic immune thrombocytopenia: Identification, patients' characteristics, and outcome Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-23 Thomas Pincez, Helder Fernandes, Mony Fahd, Marlène Pasquet, Wadih Abou Chahla, Jérome Granel, Stéphane Ducassou, Caroline Thomas, Nathalie Garnier, Eric Jeziorski, Sophie Bayart, Pascal Chastagner, Nathalie Cheikh, Corinne Guitton, Catherine Paillard, Julien Lejeune, Frédéric Millot, Valérie Li‐Thiao Te, Coralie Mallebranche, Isabelle Pellier, Martin Castelle, Corinne Armari‐Alla, Liana Carausu, Christophe
Refractory chronic immune thrombocytopenia (r‐cITP) is one of the most challenging situations in chronic immune thrombocytopenia (cITP). Pediatric r‐cITP is inconsistently defined in literature, contributing to the scarcity of data. Moreover, no evidence is available to guide the choice of treatment. We compared seven definitions of r‐cITP including five pediatric definitions in 886 patients with cITP
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miR‐146a−/− mice model reveals that NF‐κB inhibition reverts inflammation‐driven myelofibrosis‐like phenotype Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-22 Ernesto José Cuenca‐Zamora, Pedro J. Guijarro‐Carrillo, María J. López‐Poveda, María Luz Morales, María Luisa Lozano, Rocío Gonzalez‐Conejero, Constantino Martínez, Raúl Teruel‐Montoya, Francisca Ferrer‐Marín
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Chronic neutrophilic leukemia and atypical chronic myeloid leukemia: 2024 update on diagnosis, genetics, risk stratification, and management Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-22 Natasha Szuber, Attilio Orazi, Ayalew Tefferi
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Drug-induced autoimmune hemolytic anemias related to immune checkpoint inhibitors, therapeutic management, and outcome Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-20 Marion Plaçais, Ariane Laparra, Alexandre Thibault Jacques Maria, Nora Kramkimel, Audrey Perret, Guillaume Manson, Thibault Comont, Laetitia Coutte, Charlee Nardin, Kaissa Ouali, Francois-Xavier Danlos, Nicolas Noël, Sabine Messayke, Marc Michel, Olivier Lambotte, Jean-Marie Michot
Autoimmune hemolytic anemia (AIHA) is defined as augmented destruction of erythrocytes by autoimmune mechanisms, usually mediated by autoantibodies against erythrocyte surface antigens.1 Drug-induced hemolytic anemia is distinct cause of AIHA and is generally mediated by immunological mechanisms.1 Immune checkpoint inhibitors (ICIs) that are used to treat cancer, in particular PD-1 inhibitors, can
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Bone marrow Tfr2 deletion improves the therapeutic efficacy of the activin-receptor ligand trap RAP-536 in β-thalassemic mice Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-17 Emanuele Tanzi, Simona Maria Di Modica, Jessica Bordini, Violante Olivari, Alessia Pagani, Valeria Furiosi, Laura Silvestri, Alessandro Campanella, Antonella Nai
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Purplish granules as a cytological signature of cortical developmental disorders caused by pathogenic variants in WDR81 Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-17 Baptiste Le Calvez, Thomas Besnard, Benjamin Cogne, Stéphane Bézieau, Marie C. Béné, Claire Beneteau, Marion Eveillard
A girl, born to unrelated healthy Caucasian parents, presented since birth with severe epileptic encephalopathy. Her neurological development was extremely deficient. Brain magnetic resonance imaging displayed severe microencephaly with hypoplasia of the corpus callosum. During her childhood, she developed numerous infections (such as aspiration pneumonia) related to her neurological condition. At
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Novel germline JAK2R715T mutation causing PV-like erythrocytosis in 3 generations. Amelioration by Ropeg-Interferon Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-17 Jihyun Song, Lucie Lanikova, Soo Jin Kim, Nicolas Papadopoulos, Jessica Meznarich, Stefan N. Constantinescu, Brynn Parsegov, Jaroslav F. Prchal, Josef T. Prchal
Polycythemia vera (PV) is a clonal disorder arising from the acquired somatic mutations of the JAK2 gene, including JAK2V617F or several others in exon 12. A 38-year-old female had a stroke at age 32 and found to have elevated hemoglobin, normal leukocytes, normal platelets, and tested negative for JAK2V617F and exon 12 mutations. Next generation sequencing revealed a novel mutation: JAK2R715T in the
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Health‐related quality of life in transplant‐eligible patients with newly diagnosed multiple myeloma treated with daratumumab, lenalidomide, bortezomib, and dexamethasone: Patient‐reported outcomes from GRIFFIN Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-16 Rebecca Silbermann, Jacob Laubach, Jonathan L. Kaufman, Douglas W. Sborov, Brandi Reeves, Cesar Rodriguez, Ajai Chari, Luciano J. Costa, Larry D. Anderson, Nitya Nathwani, Nina Shah, Naresh Bumma, Sarah A. Holstein, Caitlin Costello, Andrzej Jakubowiak, Robert Z. Orlowski, Kenneth H. Shain, Andrew J. Cowan, Katharine S. Gries, Huiling Pei, Annelore Cortoos, Sharmila Patel, Thomas S. Lin, Peter M. Voorhees
In the phase 2 GRIFFIN trial (ClinicalTrials.gov identifier: NCT02874742), daratumumab added to lenalidomide, bortezomib, and dexamethasone (D‐RVd) improved depth of response and progression‐free survival (PFS) versus lenalidomide, bortezomib, and dexamethasone (RVd) alone in transplant‐eligible (TE) patients with newly diagnosed multiple myeloma (NDMM). Here, we present patient‐reported outcomes (PROs)
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Making a virtue out of an evil: Are red blood cells from chronic mountain sickness patients eligible for transfusions? Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-16 Emeric Stauffer, Aurélien P. Pichon, Benoit Champigneulle, Michaël Furian, Ivan Hancco, Alexis Darras, Paul Robach, Julien V. Brugniaux, Elie Nader, Philippe Connes, Samuel Verges, Lars Kaestner
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Comparison of outcomes of immunosuppressive therapy with rabbit versus horse antithymocyte globulin and cyclosporine a in children with acquired severe aplastic anemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-16 Ayami Yoshimi, Peter Noellke, Jan Starý, Krisztián Kállay, Owen Smith, Franco Locatelli, Jochen Buechner, Ivana Bodova, Julian Sevilla, Markus Schmugge, Marc Bierings, Tania Masmas, Michael Dworzak, Veerle Labarque, Katarzyna Pawelec, Kirsi Jahnukainen, Sophia Polychronopoulou, Paula Kjollerstrom, Marko Kavcic, Miriam Erlacher, Charlotte M. Niemeyer, Brigitte Strahm
Immunosuppressive therapy (IST) combining antithymocyte globulin (ATG) and cyclosporine (CSA) is a consolidated therapy for aplastic anemia (AA). Currently, there are two animal sources of ATG available, namely horse (h-ATG) and rabbit (r-ATG) ATG. While the h-ATG Atgam® (Pfizer) continues to be the standard ATG for IST in the US, the h-ATG traditionally used in Europe and Asia, Lymphoglobulin® (Genzyme)
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Exploring the landscape of somatic ASXL2 mutations in myeloid neoplasms: Frequency and clinical implications Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-13 Tareq Abuasab, Gautam Borthakur, Rashmi Kanagal‐Shamanna, Lucia Masarova, Keyur Patel, Koichi Takahashi, Prithviraj Bose, John Villarreal, Sherry Pierce, Tapan Kadia, Guillermo Garcia‐Manero, Nicholas J. Short, Courtney DiNardo, Naval Daver, Farhad Ravandi, Hagop Kantarjian, Srdan Verstovsek, Musa Yilmaz
Additional sex combs-like (ASXL) genes consist of three family members which are involved in epigenetic regulation and the three genes are: ASXL1, ASXL2, and ASXL3. Mutations in the family of ASXL have been identified in increased frequencies in myeloid neoplasms (MNs).1 While ASXL1 acts as a haploinsufficient tumor suppressor, mice models have shown that ASXL2 gene plays an essential role in normal
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High‐risk multiple myeloma: Redefining genetic, clinical, and functional high‐risk disease in the era of molecular medicine and immunotherapy Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-13 Matthew J. Rees, Shaji Kumar
Multiple myeloma (MM) exhibits significant heterogeneity in its presentation, genetics, and treatment response. Despite therapeutic advances, some patients continue to relapse early (ER, <18‐months) and rapidly cycle through therapies. Myriad prognostic factors have been identified and incorporated into risk stratification models; however, these produce discordant, often three‐tiered outputs that fail
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The t(X;20)(q13;q13) translocation is a good prognostic factor in myeloid neoplasms: A report of 25 cases from the Groupe Francophone de Cytogénétique Hématologique Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-13 Florence Nguyen‐Khac, Marc Muller, Elise Chapiro, Nassera Abermil, Marie‐Agnes Collonge‐Rame, Agnes Daudignon, Baptiste Gaillard, Doina Guzun, Antoine Ittel, Christine Lefebvre, Jean‐Francois Lesesve, Marie‐Joelle Mozziconacci, Dominique Penther, Julie Quessada, Catherine Settegrana, Luce Smagghe, Christine Terre, Lauren Veronese, Pierre Hirsch, Marie‐Bérengère Troadec
t(X;20)(q13;q13) is a very rare but recurrent translocation observed in myeloid neoplasms such as myelodysplastic neoplasm (MDS), myeloproliferative neoplasm (MPN), and acute myeloid leukemia (AML). While only nine cases have been reported previously,1-5 we have collected data (including gene sequencing data) on a large series of 25 cases of myeloid neoplasms with t(X;20). By retrospectively screening
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Characteristics and outcomes of patients with relapsed Philadelphia chromosome‐positive acute lymphoblastic leukemia after failure of a frontline ponatinib‐containing therapy Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-12 Nicholas J. Short, Elias Jabbour, Lewis F. Nasr, Nitin Jain, Fadi G. Haddad, Ghayas C. Issa, Koji Sasaki, Jayastu Senapati, Partow Kebriaei, Rebecca Garris, Marina Konopleva, Farhad Ravandi, Hagop Kantarjian
Frontline therapy of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) consists of chemotherapy or blinatumomab in combination with a BCR::ABL1 tyrosine kinase inhibitor (TKI). Ponatinib is a potent BCR::ABL1 TKI that is active against T315I ABL1 mutations, which are the dominant mechanism of relapse with earlier-generation TKIs. Promising outcomes have been achieved with frontline
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Patients with acquired pure red cell aplasia respond to PI3Kδ inhibitor rapidly Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-12 Zhenzhen Wang, Bo Jiang, Lin Song, Mingyuan Sun, Chunhong Li, Xiaoxia Li, Weiwei Zheng, Yuan Tao, Qi Sun, Junyuan Qi
Pure red cell aplasia (PRCA) is a rare disease for which large controlled trials are challenging and as such recommendations are mostly based on retrospective studies or case accumulations. Cyclosporine A (CsA) provides the most effective treatment for PRCA with an overall response rate of 65%–87%,1 leaving approximately one-third of patients in need of an alternative agent. Moreover, responders typically
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Non-myeloablative allogeneic HSCT in adult patients with sickle cell disease: Multiple take-home points from the Saudi Arabia experience Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-12 Damiano Rondelli
Allogeneic hematopoietic stem cell transplantation has been the only curative option for patients affected by sickle cell disease (SCD), and over a thousand patients have undergone this procedure over the last decades. The initial results of innovative clinical trials utilizing autologous transplantation of gene-edited CD34+ hematopoietic stem cells1, 2 suggest that this could become another potentially
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Beware of methylene blue in possible G6PD deficiency Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-12 Olga Tatarinova, Kirstin Lund, Barbara J. Bain
A seven-year-old, previously fit and well, Northern European boy presented with fever, shortness of breath, pallor, jaundice, and left upper quadrant pain. His family reported a two-day history of upper respiratory tract infection and fever. He was found to have tachycardia, tachypnea, and low oxygen saturation of 85% on room air. He was commenced on oxygen supplementation. Despite this, the oxygen
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Comparative efficacy of carfilzomib, lenalidomide, and dexamethasone (KRd) versus bortezomib, lenalidomide, and dexamethasone (VRd) in newly‐diagnosed multiple myeloma: A systematic review and meta‐analysis Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-12 Bruno Almeida Costa, Thomaz Alexandre Costa, Kevin Pak, Aesha Patel, Nicole Felix, Tarek H. Mouhieddine, Joshua Richter
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Evaluating ChatGPT as an educational resource for patients with multiple myeloma: A preliminary investigation Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-11 Ludovic Saba, Chieh-Lin Fu, Jack Khouri, Beth Faiman, Faiz Anwer, Chakra P. Chaulagain
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Let's get “real” in sickle cell disease: Real-world data and long-term patients' registries Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-11 Caterina P. Minniti
CONFLICT OF INTEREST STATEMENT Authors do not have any conflicts of interest.
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Feminist issues in clinic care, research, and healthcare professionals in thrombosis and hemostasis Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-09 Jan Hartmann, Beverley J. Hunt
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CAR T‐cell therapy induces a high rate of prolonged remission in relapsed primary CNS lymphoma: Real‐life results of the LOC network Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-08 Sylvain Choquet, Carole Soussain, Nabih Azar, Véronique Morel, Carole Metz, Renata Ursu, Agathe Waultier‐Rascalou, Roberta di Blasi, Roch Houot, Laetitia Souchet, Damien Roos‐Weil, Madalina Uzunov, Stéphanie Nguyen Quoc, Nathalie Jacque, Inès Boussen, Nicolas Gauthier, Maya Ouzegdouh, Marie Blonski, Arnaud Campidelli, Guido Ahle, Blandine Guffroy, Lise Willems, Emilie Corvilain, Maryline Barrie, Marion
The prognosis of relapsed primary central nervous system lymphoma (PCNSL) remains dismal. CAR T‐cells are a major contributor to systemic lymphomas, but their use in PCNSL is limited. From the LOC network database, we retrospectively selected PCNSL who had leukapheresis for CAR‐T cells from the third line of treatment, and, as controls, PCNSL treated with any treatment, at least in the third line and
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Efficacy and safety of mammalian target of rapamycin inhibitors in systemic mastocytosis: A nationwide French pilot study Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-06 Josquin Moraly, Julien Rossignol, Claire Rouzaud, Thomas Gabas, Hassiba Bouktit, Ludovic Lhermitte, Danielle Canioni, Sylvie Fraitag, Julie Bruneau, Stéphane Barete, Felipe Suarez, Thomas Ballul, Cécile Meni, Laura Polivka, Louis Terriou, David Launay, Laurence Bouillet, Caroline Gaudy-Marqueste, Marie Gousseff, Edwige Le Mouel, Antoine Neel, Dana Ranta, Roland Jaussaud, Philippe Guilpain, Laurent
Systemic mastocytosis (SM) corresponds to a rare and heterogeneous spectrum of diseases characterized by the accumulation of atypical mast cells (MCs). Advanced mastocytosis (Adv-SM) is associated with poor survival; in contrast, patients with non-advanced SM (non-Adv-SM) usually have a normal life expectancy but may experience poor quality of life. Despite recent therapeutic progress including tyrosine
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Distinct bone marrow findings associated with a noncanonical UBA1 variant in VEXAS syndrome Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-06 Devin R. Allison, Bhagirathbhai Dholaria, Ashwin Kishtagari, Sanjay Mohan, Eli Steigelfest, Aaron C. Shaver, Emily F. Mason
VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome is a recently described syndrome associated with adult-onset inflammatory disease, hematologic abnormalities, and somatic mutations in the UBA1 (ubiquitin-activating enzyme 1) gene, which occurs predominantly in male patients.1 Common hematologic manifestations of VEXAS include macrocytic anemia, progressive cytopenias, and an
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Accurate identification of a precursor B‐cell neoplasm Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-06 Abdalla Dikair, Kirsteen Harper, Mike Leach, Barbara J. Bain
A 23-year-old man with a history of Crohn's disease and liver transplantation for sclerosing cholangitis developed pancytopenia during corticosteroid therapy. His blood count showed hemoglobin concentration 81 g/L, white cell count 7.1 × 109/L, neutrophils 0.7 × 109/L, and platelets 34 × 109/L. His blood film showed a population of medium sized lymphoid cells with a high nucleocytoplasmic ratio, some
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Bendamustine, followed by obinutuzumab and idelalisib in chronic lymphocytic leukemia (CLL2-BCG): Final analysis of a multicenter, open-label phase-II-trial Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-05 Paula Cramer, Julia von Tresckow, Anna-Maria Fink, Sandra Robrecht, Adam Giza, Eugen Tausch, Lothar Müller, Wolfgang Knauf, Matthias Zingerle, Othman Al-Sawaf, Petra Langerbeins, Kirsten Fischer, Karl-Anton Kreuzer, Michael Kneba, Clemens-Martin Wendtner, Stephan Stilgenbauer, Barbara Eichhorst, Michael Hallek
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Redefining hyperviscosity in acute leukemia: Potential implications for red cell transfusions in the microvasculature Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-04 Jamie O. Musick, Evelyn K. Williams, Kirby S. Fibben, Dan Y. Zhang, Christina Caruso, Yumiko Sakurai, Reginald Tran, Melissa L. Kemp, Wilbur A. Lam
Hyperleukocytosis is an emergency of acute leukemia leading to blood hyperviscosity, potentially resulting in life-threatening microvascular obstruction, or leukostasis. Due to the high number of red cells in the circulation, hematocrit/hemoglobin levels (Hct/Hgb) are major drivers of blood viscosity, but how Hct/Hgb mediates hyperviscosity in acute leukemia remains unknown. In vivo hemorheological
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Pre‐B acute lymphoblastic leukemia presenting with NPM1 and FLT3 mutations Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-03 Alesia A. Khan, Daniel James, Vibeke Andresen, Julie Atkey, Rachel Bradbury, Catherine Cargo, Richard Dillon, Bjørn Tore Gjertsen, Antony R. Goldstone, Richard Leach, Daniel Lock, Mayanka Narayanan, Nigel Russell, Eleni‐Anna Verigou, Simone Green, Adele K. Fielding, Brunangelo Falini
CONFLICT OF INTEREST STATEMENT B.F. holds a patent on NPM1 mutants (number 102004901256449). The remaining authors declare no competing financial interests.
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Sex dimorphisms in coagulation: Implications in trauma‐induced coagulopathy and trauma resuscitation Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-03 Julia R. Coleman, Richard Gumina, Thomas Hund, Mitchell Cohen, Matthew D. Neal, Kristy Townsend, Bryce A. Kerlin
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Involvement of the JAK-STAT pathway in the molecular landscape of tyrosine kinase fusion-negative hypereosinophilic syndromes: A nationwide CEREO study Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-02 Matthieu Groh, Laurène Fenwarth, Mathilde Labro, Augustin Boudry, Elise Fournier, Mathieu Wemeau, Alice Marceau-Renaut, Rafael Daltro de Oliveira, Julie Abraham, Marly Barry, Philippe Blanche, Quentin Bodard, Thorsten Braun, Safia Chebrek, Matthieu Decamp, Cécile-Audrey Durel, Edouard Forcade, Mathieu Gerfaud-Valentin, Camille Golfier, Clément Gourguechon, Nathalie Grardel, Olivier Kosmider, Nihal
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A 10‐year follow‐up of high‐dose ambroxol treatment combined with enzyme replacement therapy for neuropathic Gaucher disease Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-02 Soojin Hwang, Hyunwoo Bae, Ji‐Hee Yoon, Dohyung Kim, Hyo‐Sang Do, Sun Hee Heo, Soyoung Kim, Han‐Wook Yoo, Majdolen Istaiti, Ari Zimran, Beom Hee Lee
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Glycolytic activity and in vitro effect of the pyruvate kinase activator AG-946 in red blood cells from low-risk myelodysplastic syndromes patients: A proof-of-concept study Am. J. Hematol. (IF 12.8) Pub Date : 2024-04-02 Bruno Fattizzo, Cristina Vercellati, Anna Marcello, Parija Patel, Megan Wind-Rotolo, Loredana Pettine, Marcella Bonanomi, Daniela Gaglio, Marta Bortolotti, Claudia Leoni, Elisa Fermo, Paola Bianchi, Anna Zaninoni, Francesco Passamonti, Wilma Barcellini
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World Health Organization and International Consensus Classification of eosinophilic disorders: 2024 update on diagnosis, risk stratification, and management Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-29 William Shomali, Jason Gotlib
The eosinophilias encompass a broad range of non-hematologic (secondary or reactive) and hematologic (primary or clonal) disorders with the potential for end-organ damage.