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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
β‐thalassemia is a disorder characterized by anemia, ineffective erythropoiesis (IE), and iron overload, whose treatment still requires improvement. The activin receptor‐ligand trap Luspatercept, a novel therapeutic option for β‐thalassemia, stimulates erythroid differentiation inhibiting the transforming growth factor β pathway. However, its exact mechanism of action and the possible connection with
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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
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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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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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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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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.
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Hydroxyurea in pregnancy: Reframing the conversation Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-28 Faiza Yasin, Patrick McGann, Layla Van Doren
CONFLICT OF INTEREST STATEMENT The authors have no conflict of interest to disclose.
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A phase 2 trial of orelabrutinib showing promising efficacy and safety in patients with persistent or chronic primary immune thrombocytopenia Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-28 Shi Yan, Hu Zhou, Ruibin Huang, Fang Wang, Heng Mei, Lie Lin, Jingming Guo, Xin Zhou, Zhenyu Li, Yaorong Liu, Sichen Li, Wei Zhou, Yu Hou, Ming Hou
Immune thrombocytopenia (ITP) is an acquired autoimmune hemorrhagic disease without a clear predisposition. Initial treatments often fail to induce long-term responses and until recent years, novel subsequent therapies such as thrombopoietin receptor agonists or the spleen tyrosine kinase inhibitor fostamatinib, have been limited in the management of ITP.1, 2 Expressed in both B and innate immune cells
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The presence of additional cytogenetic abnormalities (ACAs) or Philadelphia chromosome variants do not adversely affect the achievement of treatment‐free remission in chronic myeloid leukemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-28 Fadi G. Haddad, Koji Sasaki, Ghayas C. Issa, Elias Jabbour, Hagop Kantarjian
The long-term survival of patients with chronic myeloid leukemia in the chronic phase (CML-CP) has markedly improved since the introduction of the BCR::ABL1 tyrosine kinase inhibitors (TKIs). Treatment with imatinib or second-generation TKIs is associated with 10-year CML-specific overall survival (OS) of 90 + %.1 Treatment-free remission (TFR) has emerged as an important therapeutic goal for selected
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Mismatched donor allogeneic hematopoietic cell transplantation with post‐transplant cyclophosphamide achieves comparable outcomes between racially and ethnically diverse patient populations Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-28 Teresa Caprice, Wenyi Fan, Jongphil Kim, Rawan Faramand, Asmita Mishra, Lia Perez, Farhad Khimani, Aleksandr Lazar‐yan, Jose L. Ochoa‐Bayona, Hien Liu, Michael D. Jain, Michael Nieder, Claudio Anasetti, Taiga Nishihori, Joseph A. Pidala, Nelli Bejanyan, Hany Elmariah
Allogeneic hematopoietic stem cell transplant (HCT) remains the only cure for many hematologic malignancies, but this costly and highly specialized elective procedure is prone to health disparities. Historically, underrepresented minorities (African/non-Hispanic Black, Hispanic/Latinx, Asian, Native American, Pacific Islander) have lower utilization rates of HCT.1 Among many barriers to transplant
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Bleeding self‐assessments by patients with immune thrombocytopenia (ITP): An agreement study Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-27 Bianca Clerici, Sahrish Masood, Ishac Nazy, Ngan Tang, Madison Cranstone, Yang Liu, Milena Hadzi‐Tosev, Joanne Nixon, Melanie St John, Maryam Shirinzadeh, Erin Jamula, John G. Kelton, Donald M. Arnold
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BEAM versus pharmacokinetics‐directed BuCyVP16 conditioning for patients with peripheral T‐cell lymphoma undergoing high‐dose therapy with autologous hematopoietic cell transplantation Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-25 Robert Stuver, Agrima Mian, Samantha Brown, Sean Devlin, Paolo F. Caimi, Stephanie Chinapen, Parastoo Dahi, Robert Dean, Zachary D. Epstein‐Peterson, Brian Hill, Steven M. Horwitz, Oscar Lahoud, Richard Lin, Alison J. Moskowitz, Craig Sauter, Gunjan Shah, Alison Winter, Deepa Jagadeesh, Michael Scordo
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Effect of allogeneic hematopoietic stem cell transplantation on sickle cell disease‐related organ complications: A systematic review and meta‐analysis Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-22 Elisabeth Dovern, Mesire Aydin, Michael R. DeBaun, Komeil Alizade, Bart J. Biemond, Erfan Nur
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Myopathy related to chronic Graft‐Versus‐Host Disease: From clinic to histological & immunological characterization by imaging mass cytometry Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-21 Amélie Bourhis, Marie Robin, Stéphanie Nguyen, Arnaud Uguen, Patrice Hemon, Nathalie Dhedin, Thierry Maisonobe, Adèle de Masson, Olivier Benveniste, Gérard Socié, Régis Peffault de La Tour, Sarah Leonard‐Louis, Baptiste Hervier
Chronic Graft-Versus-Host Disease (cGVHD) is a common complication of allogenic hematopoietic stem-cells transplantation, potentially affecting all organs.1, 2 Even if muscle involvement is not included in the National Institutes of Health (NIH) consensus criteria it may affect 0.5% to 3,4% of patients.3 We report here a complete clinical and histological series with an extensive characterization by
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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. (IF 12.8) Pub Date : 2024-03-19 Matthew A. Lunning, Hai-lin Wang, Zhen-Huan Hu, Frederick L. Locke, Tanya Siddiqi, Caron A. Jacobson, Sairah Ahmed, David B. Miklos, Yi Lin, Brian T. Hill, Armin Ghobadi, Sattva S. Neelapu, Jason Westin, Chrisopher Dieyi, Polly Field, Harry Miao, Shilpa A. Shahani, Anik Patel, Clare Spooner, Christine Fu, David Muramoto, Hairong Xu, Marcelo C. Pasquini
Axicabtagene ciloleucel (axi-cel) in trials has demonstrated favorable efficacy compared with historical controls after ≥2 lines of therapy for the treatment of relapsed or refractory (R/R) large B cell lymphoma (LBCL). Herein, we compared the real-world effectiveness of axi-cel with efficacy and effectiveness of chemoimmunotherapy (CIT) in patients aged ≥65 years and patients with Eastern Cooperative
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HLA‐haploidentical stem cell transplantation in children with inherited bone marrow failure syndromes: A retrospective analysis on behalf of EBMT severe aplastic Anemia and pediatric diseases working parties Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-18 Stefano Giardino, Dirk‐Jan Eikema, Brian Piepenbroek, Mattia Algeri, Mouhab Ayas, Maura Faraci, Abdelghani Tbakhi, Marco Zecca, Mohammed Essa, Bénédicte Neven, Yves Bertrand, Gaurav Kharya, Tatiana Bykova, Sarah Lawson, Mario Petrini, Alexander Mohseny, Fanny Rialland, Beki James, Anca Colita, Mony Fahd, Simone Cesaro, Ansgar Schulz, Katharina Kleinschmidt, Krzysztof Kałwak, Selim Corbacioglu, Carlo
Haploidentical stem cell transplantation (haplo‐SCT) represents the main alternative for children with inherited bone marrow failure syndrome (I‐BMF) lacking a matched donor. This retrospective study, conducted on behalf of the EBMT SAAWP and PDWP, aims to report the current outcomes of haplo‐SCT in I‐BMFs, comparing the different in vivo and ex vivo T‐cell depletion approaches. One hundred and sixty‐two
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Favorable outcome of non‐myeloablative allogeneic transplantation in adult patients with severe sickle cell disease: A single center experience of 200 patients Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-16 Moussab Damlaj, Bader Alahmari, Ahmed Alaskar, Ayman Alhejazi, Husam Alsadi, Mazin Ahmed, Tahani Alanazi, Rasha Ahmed, Amani Alharbi, Inaam Shehabeddine, Afnan Alzaidi, Suha Alkhuraisat, Isam Mahassnah, Hamza Alquraan, Maybelle Ballili, Mohsen Alzahrani
Allogeneic hematopoietic stem cell transplant (HSCT) for adults with severe sickle cell disease (SCD) is potentially curative but not commonly utilized therapy due to complications such as graft failure (GF) and organ toxicity. Herein, we are reporting our long‐term outcome data of non‐myeloablative (NMA) HSCT in adults with severe SCD with emphasis on factors predicting event free survival (EFS).
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Thromboembolic risk of carfilzomib or bortezomib in combination with lenalidomide and dexamethasone for newly diagnosed multiple myeloma: A comparative systematic review and meta‐analysis Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-16 Bruno Almeida Costa, Thomaz Alexandre Costa, Sara Diaz Saravia, Nicole Felix, Carlyn Rose Tan, Neha Korde, Joshua Richter
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Detection of signature double-negative T cells is a predictive marker to identify autoimmune lymphoproliferative syndrome associated with FAS loss of function Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-14 Nina Eisenhauer, Maurizio Miano, Nora Naumann-Bartsch, Jörg Leyh, Gianluca Dell'Orso, Michael Aigner, Gisela Fecker, Claas Hinze, Helmut Wittkowski, Heiko Bruns, Jakob Zierk, Markus Metzler, Peter D. Arkwright, Frederik Graw, Andreas Mackensen, Simon Völkl
The autoimmune lymphoproliferative syndrome (ALPS) is caused by defects in the FAS-dependent apoptosis leading to chronic lymphoproliferation, autoimmunity, chronic multilineage cytopenia, an increased risk for developing B cell lymphomas, and a characteristic accumulation of CD4/CD8-negative, T-cell receptor (TCR), αβ-positive T-cells (double-negative, DNT-cells).1, 2 The heterogeneous clinical phenotype
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JAK inhibitor treatment-resistant splenomegaly before transplantation in myelofibrosis: Splenectomy or radiotherapy? Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-14 Maria Chiara Finazzi, Ayalew Tefferi, Alessandro Rambaldi
CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.
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A new chemotherapy‐free regimen of olverembatinib in combination with venetoclax and dexamethasone for newly diagnosed Ph+ acute lymphoblastic leukemia: Preliminary outcomes of a prospective study Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-14 Hailong Tang, Weijing Jia, Shuangshuang Jia, Runan Dong, Shan Gao, Juan Feng, Hongjuan Dong, Hongtao Gu, Tao Zhang, Ruifeng Yuan, Xiangxiang Liu, Lu Cheng, Shuya Zhou, Guangxun Gao
In the era following the development of tyrosine kinase inhibitors (TKIs), the current standard of treatment for clinically fit patients with previously untreated Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) is first- or second-generation TKIs combined with chemotherapy or corticosteroids, followed by allogeneic stem cell transplantation (allo-HCT). However, relapse still
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A randomized double‐blind pilot study to evaluate the efficacy, safety, and tolerability of intravenous iron versus oral iron for the treatment of restless legs syndrome in patients with iron deficiency anemia Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-13 Vanessa Short, Richard Allen, Christopher J. Earley, Huzefa Bahrain, Stella Rineer, Kiumarce Kashi, Jesse Gerb, Michael Auerbach
Restless legs syndrome (RLS) is a neurological disorder that can have a profound effect on sleep and quality of life. Idiopathic RLS is associated with brain iron insufficiency despite normal peripheral iron stores. There is, however, a five‐ to six‐fold increase in prevalence of RLS in patients with iron deficiency anemia (IDA). Several open‐label trials have demonstrated symptomatic improvement in
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The latest insights into rare blood disorders: Diagnosis and treatment strategies Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-09 David J. Kuter, Spero R. Cataland, Catherine M. Broome, Cindy Neunert
Because immune‐mediated rare blood disorders are uncommon, healthcare providers often lack the knowledge and experience necessary to identify, diagnose, and treat them in accordance with best practices. As a result, there are significant gaps in care, including delays in diagnosis and suboptimal treatment. To ensure that more patients with these rare disorders are offered quality, evidence‐based care
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Chronic myelomonocytic leukemia: 2024 update on diagnosis, risk stratification and management Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-07 Mrinal M. Patnaik, Ayalew Tefferi
Disease OverviewChronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell disorder with overlapping features of myelodysplastic syndromes and myeloproliferative neoplasms, characterized by prominent monocytosis and an inherent risk for leukemic transformation (~15%–20% over 3–5 years).DiagnosisNewly revised diagnostic criteria include sustained (>3 months) peripheral blood (PB) monocytosis
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2024 update on allogeneic hematopoietic stem cell transplant for myelofibrosis: A review of current data and applications on risk stratification and management Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-07 Haris Ali, Andrea Bacigalupo
Allogeneic hemopoietic stem cell transplantation (HSCT) currently remains the only curative treatment for patients with myelofibrosis (MF). Transplant related mortality (TRM) and relapse, remain two significant complications which need to be addressed.
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Changing hemostatic management in post‐partum hemorrhage Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-07 Agnès Rigouzzo, Pierre‐Antoine Froissant, Nicolas Louvet
Early and fast assessment of hemostasis during postpartum hemorrhage (PPH) is essential to allow early characterization of coagulopathy, estimate bleeding severity and improve outcome. During PPH, fibrinogen decrease occurs earlier than other coagulation factors deficiency and hypofibrinogenemia is an early marker of PPH severity of progression. With good evidence in the context of PPH, point‐of‐care
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Mortality in adults with sickle cell disease: Results from the sickle cell disease implementation consortium (SCDIC) registry Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-07 Franklin Njoku, Norma Pugh, Donald Brambilla, Barbara Kroner, Nirmish Shah, Marsha Treadwell, Robert Gibson, Lewis L. Hsu, Victor R. Gordeuk, Jeffrey Glassberg, Jane S. Hankins, Abdullah Kutlar, Allison A. King, Julie Kanter
The cause of death in people affected by sickle cell disease (SCD) is often challenging to define as prior studies have used retrospective or administrative data for analysis. We used a prospective longitudinal registry to assess mortality and clinical co-morbidities among subjects enrolled in the Sickle Cell Disease Implementation Consortium (SCDIC) registry. At enrollment, we collected the following
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Results of a phase I trial with Haploidentical mbIL-21 ex vivo expanded NK cells for patients with multiply relapsed and refractory AML Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-05 Stefan O. Ciurea, Piyanuch Kongtim, Samer Srour, Julianne Chen, Doris Soebbing, Elizabeth Shpall, Katayoun Rezvani, Robin Nakkula, Aarohi Thakkar, Ella C. Troy, Alex A. Cash, Gregory Behbehani, Kai Cao, Jolie Schafer, Richard E. Champlin, Dean A. Lee
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SHP2 inhibition displays efficacy as a monotherapy and in combination with JAK2 inhibition in preclinical models of myeloproliferative neoplasms Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-05 Garima Pandey, Lucia Mazzacurati, Tegan M. Rowsell, Nathan P. Horvat, Narmin E. Amin, Guolin Zhang, Afua A. Akuffo, Christelle M. Colin‐Leitzinger, Eric B. Haura, Andrew T. Kuykendall, Ling Zhang, Pearlie K. Epling‐Burnette, Gary W. Reuther
Myeloproliferative neoplasms (MPNs), including polycythemia vera, essential thrombocytosis, and primary myelofibrosis, are clonal hematopoietic neoplasms driven by mutationally activated signaling by the JAK2 tyrosine kinase. Although JAK2 inhibitors can improve MPN patients' quality of life, they do not induce complete remission as disease‐driving cells persistently survive therapy. ERK activation
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Myeloid neoplasm with <10% blasts and t(3;5)(q25.1;q34)/NPM::MLF1: A classification dilemma Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-05 Fnu Sameeta, Hong Fang, Wei Wang, Zhenya Tang, Sa A. Wang, Gokce A. Toruner, Xenia Parisi, Joseph D. Khoury, Ghayas Issa, Guillermo Garcia‐Manero, L. Jeffrey Medeiros, Guilin Tang, Sanam Loghavi
CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.
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Hairy cell leukemia 2024: Update on diagnosis, risk-stratification, and treatment—Annual updates in hematological malignancies Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-05 Xavier Troussard, Elsa Maître, Jérôme Paillassa
Hairy cell leukemia (HCL) and HCL-like disorders, including HCL variant (HCL-V) and splenic diffuse red pulp lymphoma (SDRPL), are a very heterogenous group of mature lymphoid B-cell disorders characterized by the identification of hairy cells, a specific genetic profile, a different clinical course and the need for appropriate treatment.
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Treatment‐free remission in CML patients with additional chromosome abnormalities in the Philadelphia‐positive clone or variant Philadelphia translocations Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-04 Simone Claudiani, Lynette Chee, Fiona Fernando, Loretta Brown, Udayakumar M. Achandira, Afzal Khan, Kate Rothwell, Chloe Hayden, Ioannis Koutsavlis, Guy Hannah, Andrew Innes, Jane F. Apperley, Dragana Milojkovic
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Leg ulcers are indicators of systemic dysfunction in individuals with sickle cell disease Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-03 Tiffany A. Jackson, Ashley J. Buscetta, Hasmin C. Ramirez, Vence L. Bonham, Caterina P. Minniti
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Real‐world evidence: Long‐term safety of deferiprone in a large cohort of patients with sickle cell disease enrolled in a registry for up to 10 years Am. J. Hematol. (IF 12.8) Pub Date : 2024-03-02 Janet L. Kwiatkowski, Alexis A. Thompson, Fernando Tricta, Noemi Toiber Temin, Anna Rozova, Caroline Fradette, Sherif M. Badawy
Patients with sickle cell disease (SCD) and other anemias who receive blood transfusions are at risk of organ damage due to transfusional iron overload. Deferiprone is an iron chelator with a well‐established safety and efficacy profile that is indicated for the treatment of transfusional iron overload. Here, we report safety data from the large‐scale, retrospective Ferriprox® Total Care Registry,