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Treatment Effectiveness of Venetoclax‐Based Therapy After Bruton Tyrosine Kinase Inhibitors in Chronic Lymphocytic Leukemia: An International Real‐World Study Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-19 Nilanjan Ghosh, Toby A. Eyre, Jennifer R. Brown, Nicole Lamanna, Beenish S. Manzoor, Catherine C. Coombs, Hande H. Tuncer, Chaitra Ujjani, Lori A. Leslie, Lindsey E. Roeker, Matthew S. Davids, Joanna M. Rhodes, Alan P. Skarbnik, Wendy Sinai, Isabelle Fleury, Brian T. Hill, Nicolas Martinez‐Calle, Paul M. Barr, Dureshahwar Jawaid, Nnadozie Emechebe, Laurie Pearson, Frederick Lansigan, Yun Choi, Christopher
The treatment landscape of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) has evolved drastically with the introduction of targeted agents, including covalent Bruton tyrosine kinase inhibitors (cBTKis) and B-cell lymphoma 2 inhibitors (BCL2is) [1]. However, the development of cBTKi resistance (leading to disease progression) and intolerance due to adverse events limits durable
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Risk Factors and Prognostic Markers for Posterior Reversible Encephalopathy Syndrome in Patients With Thalassemia Major After Allogeneic Hematopoietic Stem Cell Transplantation Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-19 Meiru Bu, Kemei Deng, Meiqing Wu, Lingling Shi, Yuhong Qin, Muliang Jiang, Bihong T. Chen
Posterior reversible encephalopathy syndrome (PRES) has been known as a neurological complication in patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). PRES is an acute or subacute syndrome with various neurological symptoms such as headache, encephalopathy, visual disturbance and seizures [1]. Neuroimaging may show subcortical white matter vasogenic edema typically in bilateral
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Clinical Use of Eltrombopag and Avatrombopag in Pediatric ITP in China: A Real‐World Multicenter Retrospective Cohort Study Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-18 Jingjing Liu, Zhifa Wang, Nan Wang, Jingyao Ma, Yu Hu, Jie Ma, Lijuan Wang, Yan Liu, Juntao Ouyang, Zhenping Chen, Xiaoling Cheng, Runhui Wu
Immune thrombocytopenia (ITP) is an acquired bleeding disorder characterized by a reduced platelet count of less than 100 × 109/L, with an estimated incidence of 2–5/100 000 children per year [1]. Since 2014, the China National Medical Products Administration (NMPA) has approved five TPO-RAs for treating ITP in China. Among these, eltrombopag (ELT) and avatrombopag (AVA) are the two most evidence-based
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Blastic Plasmocytoid Dendritic Cell Neoplasm With Pseudo‐Lymphoid Morphology Mimicking Lymphoid Malignancy Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-18 Romain Ravel‐Chapuis, Gérard Buchonnet, Catherine Boutet, Francine Garnache Ottou, Anne Roggy, Florian Renosi, Olivier Matray, Victor Bobée
A 60-year-old male presented with spontaneous hematomas and cervical adenopathy. Laboratory workup revealed hemoglobin 12.3 g/dL, platelet 56 × 109/L, and leukocyte 14.7 × 109/L. The automated cell counter (Sysmex XN) reported 9% neutrophils (1.32 × 109/L) and 86% lymphocytes (12.6 × 109/L), suggestive of a lymphoproliferative disorder. Blood smear showed atypical lymphoid cells with normal size, mature
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Pseudothrombocytopenia due to Phagocytosis of Platelets by Polymorphonuclear Leukocytes Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-16 Iliana Stamatiou, Zoe Bezirgiannidou, Evangelia Charitaki, Ioannis Kotsianidis, Konstantinos Liapis
A 75-year-old woman presented to the emergency department with progressive abdominal pain, fever, and diarrhea after taking levofloxacin for a respiratory tract infection. On evaluation, she was in clinical shock, with blood pressure 88/58 mmHg and heart rate 122 beats per minute. A complete blood count provided by the Sysmex XN-1000 analyzer showed leukocytosis (13.8 × 109/L, 95% neutrophils) and
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Efficacy and Safety of Obinutuzumab in Immune-Mediated Thrombotic Thrombocytopenic Purpura Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-12 Julia Weisinger, Raïda Bouzid, Jehane Fadlallah, Christelle Barbet, Francois Provot, Pascale Poullin, Antoine Neel, Manon Marie, Virginie Rieu, Tarik Kanouni, Olivier Moranne, Elie Azoulay, Zora Marjanovic, Elise Corre, Anne-Christine Joly, Minh-Tam Baylatry, Bérangère S. Joly, Agnes Veyradier, Paul Coppo
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare form of thrombotic microangiopathy (TMA) caused by an autoantibody-mediated deficiency of the von Willebrand factor (vWF) cleaving protease ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin-1 motifs, 13th Member) [1]. Without treatment, the disease is almost always fatal. In the last decades, the combination of therapeutic
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The Diversity of Spiculated Erythrocytes Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-13 Barbara J. Bain
Spiculated erythrocytes come in a variety of forms, which are of diagnostic importance. They can be broadly classified as echinocytes, acanthocytes, keratocytes, and schistocytes. However, complexity is added by the superimposition of one abnormality on another. Other spiculated cells can, for example, undergo an echinocytic or acanthocytic change. In addition, other poikilocytes, for example, spherocytes
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Mayo Genetic Risk Models for Newly Diagnosed Acute Myeloid Leukemia Treated With Venetoclax + Hypomethylating Agent Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-13 Naseema Gangat, Azeem Elbeih, Nour Ghosoun, Kristen McCullough, Fnu Aperna, Isla M. Johnson, Maymona Abdelmagid, Aref Al‐Kali, Hassan B. Alkhateeb, Kebede H. Begna, Michelle Elliott, Abhishek Mangaonkar, Aasiya Matin, Antoine N. Saliba, Mehrdad Hefazi Torghabeh, Mark R. Litzow, William Hogan, Mithun Shah, Mrinal M. Patnaik, Animesh Pardanani, Talha Badar, Hemant Murthy, James Foran, Jeanne Palmer,
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Peri‐Transplant Management of JAK Inhibitor Therapy in Myelofibrosis Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-13 Ayalew Tefferi, Vincent T. Ho
Conflicts of Interest The authors declare no conflicts of interest.
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Correction to “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 10.1) Pub Date : 2024-12-12
Lunning MA, Wang HL, Hu ZH, 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,” American Journal of Hematology 99, no. 5 (2024): 880–889. In Figure 1C, a typographical error was made reporting incorrect median overall survival
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Effect of Ferric Derisomaltose on Fatigue in Iron Deficiency Anemia Associated With Abnormal Uterine Bleeding Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-12 Petra Stute, Imo J. Akpan, Christian Breymann, Ally Murji, Sarah H. O'Brien, Jacquelyn M. Powers, Malcolm G. Munro
Anemia is prevalent among women of reproductive age, with iron deficiency (ID) being the primary etiology. ID can lead to fatigue and reduced quality of life, particularly in the context of abnormal menstrual bleeding [1]. Abnormal menstrual bleeding refers to a set of symptoms collectively known as abnormal uterine bleeding (AUB), and includes heavy menstrual bleeding (HMB), experienced by up to around
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Efficacy of Midostaurin Combined With Intensive Chemotherapy in Core Binding Factor Leukemia: A Phase II Clinical Trial Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-11 Roberto Cairoli, Arianna Gatti, Giovanni Grillo, Marta Rachele Stefanucci, Barbara Di Camillo, Monica Fumagalli, Mauro Krampera, Gianpaolo Nadali, Patrizia Zappasodi, Erika Borlenghi, Elisabetta Todisco, Marta Ubezio, Massimo Bernardi, Alfredo Molteni, Claudia Basilico, Mauro Turrini, Rosa Greco, Valentina Mancini, Marta Riva, Davide Paolo Bernasconi, Bruno Brando, Silvio Marco Veronese, Alessandro
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Mutation of Epigenetic Regulators at Diagnosis Is an Independent Predictor of Tyrosine Kinase Inhibitor Treatment Failure in Chronic Myeloid Leukemia: A Report From the RESIDIAG Study Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-10 Hippolyte Guerineau, Jean‐Michel Cayuela, Stéphanie Dulucq, Violaine Tran Quang, Sihem Tarfi, Guillaume Gricourt, Quentin Barathon, Corine Joy, Orianne Wagner‐Ballon, Stéphane Morisset, Frank‐Emmanuel Nicolini, Erika Brunet, Sébastien Maury, Lydia Roy, Gabriel Etienne, Delphine Réa, Ivan Sloma
Additional mutations at chronic myeloid leukemia (CML) diagnosis have been shown to variably affect tyrosine kinase inhibitor (TKI) response [1], and were inconstantly detected at loss of response [2]. Contradictory observations may have resulted from difficulties in reliably inferring CML clonal architecture from mutations quantified by NGS, BCR::ABL1 by qRT-PCR, and ABL1-tyrosine kinase domain (ABL1-TKD)
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Evaluating the Role of Red Blood Cell Lifespan in Transfusion‐Dependent β‐Thalassemia and Impact of Thalidomide Treatment Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-09 Kun Yang, Yuping Gong, Jian Xiao
β-Thalassemia is characterized by ineffective erythropoiesis (IE), anemia, and iron overload. It involves both intramedullary apoptosis and the destruction of red blood cells (RBCs) owing to membranes developing abnormalities as a result of an excess of unpaired globin chains [1]. RBC destruction caused by IE or hemolysis shortens the lifespan of these cells. Although laboratory indicators can detect
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10‐Year Risk of Gallstones in Congenital Red Blood Cell Disorder Patients: A Nationwide Cohort Study Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-09 Anders Blach Naamansen, Dennis Lund Hansen, Jesper Petersen, Andreas Glenthøj, Henrik Toft Sørensen, Henrik Frederiksen
Chronic hemolysis potentially elevates the risk of gallstones in several types of congenital red blood cell (RBC) disorders. However, the magnitude of the risk is unknown. We investigate the risk of gallstone disease in congenital RBC disorder patients, compared with general population comparators. Patients were identified from the Danish National Patient Registry covering all Danish hospitals and
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Hematopoietic Stem Cell Transplantation in Patients With Myelofibrosis and Splanchnic Vein Thrombosis: A Case Series Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-09 Portia Smallbone, Mallika Sekhar, Samer A. Srour, Jeremy L. Ramdial, Crystal L. Carmicheal Kusy, Elizabeth J. Shpall, Uday R. Popat
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The Importance of Controlling Terminal Complement Activity and Intravascular Hemolysis in Paroxysmal Nocturnal Hemoglobinuria (PNH) Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-09 Anita Hill, Christophe Hotermans, Gianluca Pirozzi
Patient safety is paramount and ingrained in our mission at Alexion, and with our leadership in complement biology for over 30 years, we strive to provide the most efficacious and safe therapeutic products for patients. It must be remembered that the most significant risks to patients with paroxysmal nocturnal hemoglobinuria (PNH) come from intravascular hemolysis (IVH) and thrombosis, and these are
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Issue Information Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-09
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Prevalence of BTK and PLCG2 Mutations in CLL Patients With Disease Progression on BTK Inhibitor Therapy: A Meta‐Analysis Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-07 Stefano Molica, David Allsup, Diana Giannarelli
Bruton's tyrosine kinase inhibitors (BTKis) have revolutionized the treatment of chronic lymphocytic leukemia (CLL), with significantly improved outcomes for both treatment-naïve (TN) and relapsed/refractory (R/R) patients. BTKis bind irreversibly to the cysteine 481 (C481) residue of the BTK molecule inhibiting B-cell receptor (BCR)-mediated intracellular signals crucial for CLL-cell survival [1]
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Iron Deficiency in Familial Mediterranean Fever: A Study on 211 Adult Patients From the JIR Cohort Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-07 Ilenia Di Cola, Léa Savey, Marion Delplanque, Rim Bourguiba, Alessandra Bartoli, Zohra Aknouche, Fatima Bensalek, Isabelle Kone‐Paut, Linda Rossi‐Semerano, Isabelle Melki, Brigitte Bader‐Meunier, Piero Ruscitti, Bénédicte Neven, Pierre Quartier, Guilaine Boursier, Irina Giurgea, Laurence Cuisset, Gilles Grateau, Véronique Hentgen, Sophie Georgin‐Lavialle
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Antihuman T Lymphocyte Globulin Fresenius in Graft‐Versus‐Host Disease Prophylaxis for Unrelated Hematopoietic Stem Cell Transplantation After Myeloablative Conditioning: A Long‐Term Real‐Life Retrospective Study Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-04 Marion Divoux, Matthieu Resche‐Rigon, David Michonneau, Aurélien Sutra Del Galy, Nathalie Dhedin, Alienor Xhaard, Flore Sicre de Fontbrune, Marie Robin, Gérard Socié, Régis Peffault de Latour
Graft-versus-host disease (GvHD) is a frequent complication of hematopoietic stem cell transplantation (HSCT) and remains among the leading causes of post-transplant morbidity and mortality. Acute GvHD (aGvHD) affects 30%–50% of HSCT patients, while chronic GvHD (cGvHD) affects 30%–70%. In vivo T depletion using rabbit antithymocyte globulins (ATG) during conditioning has been shown to reduce the occurrence
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Feasibility of a Biomarker‐Based Screening for Pre‐Symptomatic AL Amyloidosis in Patients With Intermediate/High‐Risk MGUS Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-04 Silvia Mangiacavalli, Paolo Milani, Claudio Salvatore Cartia, Marzia Varettoni, Michele Palumbo, Marco Basset, Valeria Masoni, Mario Nuvolone, Andrea Foli, Virginia Valeria Ferretti, Giampaolo Merlini, Luca Arcaini, Giovanni Palladini
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Overall Survival in Male Patients With Advanced Hematological Disease (Mostly Acute Leukemia) Is Influenced by CYP1B1 C432G Polymorphism and Donor Sex in Allogeneic Stem Cell Transplantation Am. J. Hematol. (IF 10.1) Pub Date : 2024-12-03 Norbert Stute, Michael Koldehoff
Human cytochrome P450 1B1 (CYP1B1) is an extrahepatic key enzyme involved in estrogen metabolism, steroid synthesis, and pro-carcinogen activation. CYP1B1 is strongly overexpressed in multiple human malignancies and has been used as a target for cancer chemotherapy and immunotherapy. CYP1B1 is also overexpressed in acute lymphocytic leukemia, acute myeloid leukemia, lymphoma, and myeloma [1]. In allogeneic
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Methotrexate Versus Mycophenolate Mofetil Prophylaxis in Allogeneic Hematopoietic Cell Transplantation for Chronic Myeloid Malignancies: A Retrospective Analysis on Behalf of the Chronic Malignancies Working Party of the EBMT Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-28 Thomas Luft, Luuk Gras, Linda Koster, Nicolaus Kröger, Thomas Schröder, Uwe Platzbecker, Katja Sockel, Régis Peffault de Latour, Matthias Stelljes, Henrik Sengeloev, Matthias Eder, Igor Wolfgang Blau, Peter Dreger, Ibrahim Yakoub-Agha, Johan Maertens, Urpu Salmenniemi, Wolfgang Bethge, Stephan Mielke, Guido Kobbe, Anastasia Pouli, Liesbeth C. de Wreede, Kavita Raj, Joanna Drozd-Sokolowska, Donal P
Prophylaxis strategies for Graft versus host disease (GVHD) in allogeneic hematopoietic cell transplantation (allo-HCT) frequently encompass a combination of a calcineurin inhibitor (CNI) with either methotrexate (MTX) or mycophenolate mofetil (MMF). The aim of this retrospective, EBMT registry-based study was to determine outcome differences for chronic myeloid malignancies and secondary acute myeloid
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Autologous and Allogeneic Stem‐Cell Transplantation for Transformed Waldenström Macroglobulinemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-26 Eric Durot, Lukshe Kanagaratnam, Saurabh Zanwar, Adrienne Kaufman, Shirley D'Sa, Elise Toussaint, Damien Roos‐Weil, Miguel Alcoceba, Josephine M. I. Vos, Anne‐Sophie Michallet, Dipti Talaulikar, Efstathios Kastritis, Jahanzaib Khwaja, Steven P. Treon, Ramon Garcia‐Sanz, Pierre Morel, Javier Munoz, Jorge J. Castillo, Prashant Kapoor, Alain Delmer
Waldenström macroglobulinemia (WM) is characterized by a clonal proliferation of plasmacytoid B-cells in the bone marrow and monoclonal IgM gammopathy, that ultimately require treatment in symptomatic patients. Moreover, a small subset of patients undergoes histological transformation (HT) [1], mainly in the form of diffuse large B-cell lymphoma (DLBCL). They usually present with high-risk features
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Acute Leukemias of Ambiguous Lineage With MDS‐Associated Mutations Show Similar Prognosis Compared to Acute Myeloid Leukemia With MDS‐Associated Mutations: A Study From the Bone Marrow Pathology Group Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-23 Timothy J. Kirtek, Weina Chen, Jaryse C. Harris, Adam Bagg, Kathryn Foucar, Wayne Tam, Attilio Orazi, Eric D. Hsi, Robert P. Hasserjian, Sa A. Wang, David P. Ng, Tracy I. George, Min Shi, Kaaren K. Reichard, Emily Symes, Xinmin Zhang, Daniel A. Arber, Olga K. Weinberg
1 Introduction To the Editor: Acute myeloid leukemia (AML) and acute leukemia of ambiguous lineage (ALAL) comprise a diverse variety of acute leukemias that are defined by their morphologic, immunophenotypic, and genetic features. AML is characterized by clonal expansion of immature hematopoietic precursors of myeloid lineage in the peripheral blood and bone marrow. AML may arise de novo, evolve from
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Basophilia Predicts Poorer Outcomes in Essential Thrombocythemia, Polycythemia Vera, Primary Myelofibrosis, and Myeloproliferative Neoplasm, Unclassifiable Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-23 Lisa Yuen, Tasos Gogakos, Leonardo Boiocchi, Gabriela Hobbs, Robert Hasserjian
Basophils are hematopoietic cells derived from myeloid progenitor cells and are found in increased numbers in some myeloid neoplasms, particularly chronic myeloid leukemia (CML). Marked basophilia is a poor prognostic indicator in CML and defines accelerated phase according to the revised 4th edition World Health Organization (WHO4R) classification and the International Consensus Classification (ICC)
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Venetoclax and Hypomethylating Agent in Previously Untreated Higher‐Risk Myelodysplastic Syndromes and Genotype Signatures for Response and Prognosis: A Real‐World Study Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-22 Jing Wang, Zhijian Fang, Siyi Yang, Kexin Yan, Jingjing Zhang, Yanfang Yu, Yaoliang Ren, Hao Jiang, Jinsong Jia, Jianlin Chen, Botao Li, Yingjun Chang, Xiaosu Zhao, Xiaojun Huang
Higher-risk myelodysplastic syndrome (MDS), as defined by the revised international prognostic scoring system (IPSS-R), requires more aggressive treatment. Despite the use of hypomethylating agents (HMAs), response rates remain low, underscoring the need for more effective therapies. Venetoclax is a potent, orally bioavailable inhibitor of the B-cell lymphoma 2 (BCL-2) protein, which acts synergistically
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Treatment‐Free Remissions in Children With Chronic Myeloid Leukemia (CML): A Prospective Study From the Tata Memorial Hospital (TMH) Pediatric CML (pCML) Cohort Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-21 Nirmalya Roy Moulik, Swaminathan Keerthivasagam, Gaurav Chatterjee, Jayesh Agiwale, Pallavi Rane, Chetan Dhamne, Akanksha Chichra, Shyam Srinivasan, Purvi Mohanty, Hemani Jain, Dhanlaxmi Shetty, Sweta Rajpal, Prashant Tembhare, Nikhil Patkar, Gaurav Narula, Papagudi G. Subramanian, Shripad Banavali
Pediatric chronic myeloid leukemia (pCML) is a rare childhood malignancy, representing 2%–3% of all childhood leukemia. Tyrosine kinase inhibitors (TKIs) have greatly improved survival but pose challenges due to their long‐term effects on growth and bone health in children. We prospectively studied treatment‐free remission (TFR) in 45 children with pCML in chronic phase on imatinib. Eligibility criteria
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Blood Plasma Methylated DNA Markers in the Detection of Lymphoma: Discovery, Validation, and Clinical Pilot Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-20 Thomas E. Witzig, William R. Taylor, Douglas W. Mahoney, William R. Bamlet, Patrick H. Foote, Kelli N. Burger, Karen A. Doering, Mary E. Devens, Jacquelyn R. Arndt, Maria C. O'Connell, Calise K. Berger, Anne J. Novak, James R. Cerhan, Jacquelyn Hennek, Slava Katerov, Hatim T. Allawi, Dragan Jevremovic, Linda N. Dao, Rondell P. Graham, John B. Kisiel
Lymphoma is one of the leading causes of cancer and cancer deaths and yet has not been amenable to population screening. The role of methylated DNA markers (MDMs) in the detection of lymphoma has not been extensively studied. We aimed to discover, validate, and test tissue–derived MDMs of lymphoma in archival plasma specimens. Reduced representation bisulfite sequencing (RRBS) was performed on a discovery
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Effect of voxelotor on cerebral perfusion and cerebral oxygen metabolism and cardiac stress in adult patients with sickle cell disease Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-20 Kadère Konté, Liza Afzali-Hashemi, Koen P. A. Baas, Anouk Schrantee, John C. Wood, Erfan Nur, Aart J. Nederveen, Bart J. Biemond
Sickle cell disease (SCD) is complicated by silent cerebral infarcts (SCIs), for which anemia is an important risk factor. Despite normal oxygen delivery (OD), cerebral vascular reserve (CVR), and cerebral metabolic rate of oxygen (CMRO2) are diminished in SCD, possibly causing the formation of SCIs. Voxelotor inhibits polymerization by increasing the hemoglobin oxygen binding, ameliorating hemolytic
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Different impacts of granulocyte colony-stimulating factor administration on allogeneic hematopoietic cell transplant outcomes for adult acute myeloid leukemia according to graft type Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-20 Takaaki Konuma, Kazuaki Kameda, Kaoru Morita, Tadakazu Kondo, Fumihiko Kimura, Hideki Nakasone, Fumihiko Ouchi, Naoyuki Uchida, Masatsugu Tanaka, Tetsuya Nishida, Takahiro Fukuda, Yuta Hasegawa, Mamiko Sakata-Yanagimoto, Makoto Onizuka, Masashi Sawa, Shuichi Ota, Noboru Asada, Shin-Ichiro Fujiwara, Satoshi Yoshihara, Fumihiko Ishimaru, Makoto Yoshimitsu, Yoshinobu Kanda, Marie Ohbiki, Yoshiko Atsuta
We retrospectively evaluated the impacts of using granulocyte colony-stimulating factor (G-CSF) and its timing on posttransplant outcomes for 9766 adults with acute myeloid leukemia (AML) between 2013 and 2022 using a Japanese database. We separately evaluated three distinct cohorts based on graft type: 3248 received bone marrow transplantation (BMT), 3066 received peripheral blood stem cell transplantation
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Prognostic significance of mutation type and chromosome fragility in Fanconi anemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-20 María José Ramírez, Roser Pujol, Jordi Minguillón, Massimo Bogliolo, Ilaria Persico, Debora Cavero, Aurora de la Cal, Paula Río, Susana Navarro, José Antonio Casado, Almudena Bailador, Antonio Sanchez de la Fuente, Miguel López de Heredia, Francisco Almazán, M. Luisa Antelo, Bienvenida Argilés, Isabel Badell, Marta Baragaño, Cristina Beléndez, Mar Bermúdez, Marta Bernués, María Isabel Buedo, Estela
Fanconi anemia (FA) is a rare genetic disease characterized by high phenotypic and genotypic heterogeneity, and extreme chromosome fragility. To better understand the natural history of FA, identify genetic risk and prognostic factors, and develop novel therapeutic strategies, the Spanish Registry of Patients with FA collects data on clinical features, chromosome fragility, genetic subtypes, and DNA
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First-line treatment of Waldenström's macroglobulinemia in Italy: A multicenter real-life study on 547 patients to evaluate the long-term efficacy and tolerability of different chemoimmunotherapy strategies Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-19 Francesco Autore, A. Tedeschi, G. Benevolo, V. Mattiello, E. Galli, N. Danesin, R. Rizzi, J. Olivieri, E. Cencini, B. Puccini, I. Ferrarini, D. Marino, M. Bullo, B. Rossini, M. Motta, I. Innocenti, A. Fresa, L. Stirparo, D. Petrilli, R. Pasquale, P. Musto, G. Scapinello, A. Noto, V. Peri, G. Zamprogna, S. Hohaus, A. M. Frustaci, F. Piazza, S. Ferrero, L. Laurenti
Treatment is indicated in Waldenström macroglobulinemia (WM) when clinical manifestations arise due to the IgM paraprotein or lymphoplasmacytic infiltrate.1 The main classes of drugs used for WM treatment include monoclonal antibodies, chemotherapeutic agents, proteasome inhibitors, and Bruton Tyrosine Kinase inhibitors (BTKi). The most frequently used chemotherapeutic agent is bendamustine, which
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RAS signaling pathway is essential in regulating PIEZO1-mediated hepatic iron overload in dehydrated hereditary stomatocytosis Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-18 Barbara Eleni Rosato, Vanessa D'Onofrio, Roberta Marra, Antonella Nostroso, Federica Maria Esposito, Anthony Iscaro, Vito Alessandro Lasorsa, Mario Capasso, Achille Iolascon, Roberta Russo, Immacolata Andolfo
PIEZO1 encodes a mechanoreceptor, a cation channel activated by mechanical stimuli. Gain-of-function (GoF) variants in PIEZO1 cause dehydrated hereditary stomatocytosis (DHS), or xerocytosis, a pleiotropic syndrome characterized by anemia and iron overload. DHS patients develop hepatic iron overload independent of the degree of anemia and transfusion regimen. PIEZO1-GoF variants suppress hepcidin expression
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The role of allogeneic stem cell transplantation in acute myeloid leukemia with translocation t(8;16)(p11;p13) Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-18 Ann-Kristin Schmälter, Myriam Labopin, Jurjen Versluis, Maria Pilar Gallego Hernanz, Matthias Eder, Peter von dem Borne, Gerard Socié, Patrice Chevallier, Edouard Forcade, Andreas Neubauer, Frédéric Baron, Ali Bazarbachi, Gesine Bug, Arnon Nagler, Christoph Schmid, Jordi Esteve, Mohamad Mohty, Fabio Ciceri
Acute myeloid leukemia (AML) with translocation t(8;16)(p11;p13) represents a rare entity that has been categorized as a disease-defining recurring cytogenetic abnormality with adverse risk in the 2022 European LeukemiaNet classification. This rating was mainly based on a retrospective analysis comprising patients from several large clinical trials, which, however, included only 21 patients treated
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Mini-consolidations or intermediate-dose cytarabine for the post-remission therapy of AML patients over 60. A retrospective study from the DATAML and SAL registries Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-18 Christian Récher, Pierre-Yves Dumas, Emilie Bérard, Suzanne Tavitian, Thibaut Leguay, Jean Galtier, Camille Alric, Audrey Bidet, Eric Delabesse, Jean Baptiste Rieu, Jean-Philippe Vial, François Vergez, Isabelle Luquet, Emilie Klein, Anne-Charlotte de Grande, Audrey Sarry, Sven Zukunft, Uwe Platzbecker, Carsten Müller-Tidow, Claudia D. Baldus, Martin Bornhäuser, Hubert Serve, Sarah Bertoli, Arnaud Pigneux
According to current recommendations, older AML patients in first complete remission (CR) after induction chemotherapy should receive consolidation with intermediate-dose cytarabine (IDAC). However, no study has demonstrated the superiority of IDAC over other regimen. In this retrospective study, we compared the efficacy of mini-consolidations (idarubicin 8 mg/m2 day 1, cytarabine 50 mg/m2/12 h, day
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Clinical significance of complete remission and measurable residual disease in relapsed/refractory multiple myeloma patients treated with T-cell redirecting immunotherapy Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-16 Aintzane Zabaleta, Noemi Puig, Maria-Teresa Cedena, Aina Oliver-Caldes, José J. Perez, Cristina Moreno, Luis-Esteban Tamariz-Amador, Paula Rodriguez-Otero, Felipe Prosper, Veronica Gonzalez-Calle, Lucía López-Corral, Beatriz Rey-Búa, Borja Puertas, Fátima Mirás, José María Sánchez-Pina, Nieves López-Muñoz, Manel Juan, E. Azucena González-Navarro, Álvaro Urbano, Carlos Fernández de Larrea, Joan Blade
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Clinical and Immune Effects of Peri‐Transplantation JAK Inhibition for Myelofibrosis Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-16 Kristin Rathje, Nico Gagelmann, Anita Badbaran, Claudia Langebrake, Adrin Dadkhah, Johanna Richter, Radwan Massoud, Mathias Schäfersküpper, Franziska E. Marquard, Sofia Oechsler, Evgeny Klyuchnikov, Ina Rudolph, Silke Heidenreich, Christian Niederwieser, Catherina Lueck, Dietlinde Janson, Christine Wolschke, Boris Fehse, Francis Ayuk, Nicolaus Kröger
Despite the introduction of JAK inhibitors, allogeneic hematopoietic cell transplant remains the only potentially curative treatment for patients with myelofibrosis but has considerable treatment‐related complications. Whether the incorporation of JAK inhibition into the transplant algorithm leads to improved outcomes is still unclear. Here, we analyzed different transplant platforms in myelofibrosis
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A prophylactic tyrosine kinase inhibitor strategy based on measurable residual disease pre-transplantation for Ph+ acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation: A prospective multicenter cohort study Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-16 Hui Liu, Hui Xu, Peiru Chi, Zinan Feng, Xiaojun Xu, Danian Nie, Xudong Li, Xinquan Liang, Zhiping Fan, Na Xu, Fen Huang, Ren Lin, Zhixiang Wang, Hua Jin, Hongsheng Zhou, Xutao Guo, Dongjun Lin, Jing Sun, Qifa Liu, Li Xuan
Relapse is the major cause of treatment failure in Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). This study aimed to evaluate the effect of a prophylactic tyrosine kinase inhibitor (TKI) strategy on relapse in this population. Patients were assigned to prophylactic or control groups based on measurable
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High-grade B-cell lymphoma not otherwise specified, with diffuse large B-cell lymphoma gene expression signatures: Genomic analysis and potential therapeutics Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-16 Waseem Lone, Alyssa Bouska, Tyler A. Herek, Catalina Amador, Joo Song, Alexander M. Xu, Dylan Jochum, Issa Ismail Issa, Dennis D. Weisenburger, Xuan Zhang, Sharath Kumar Bhagavathi, Tayla B. Heavican-Foral, Sunandini Sharma, Ab Rauf Shah, Abdul Rouf Mir, Aisha Ahmad Alkhinji, Dalia El-Gamal, Bhavana J. Dave, Keenan Hartert, Jiayu Yu, Mallick Saumyaranjan, Timothy C. Greiner, Julie Vose, Timothy W.
High-grade B-cell lymphoma not otherwise specified (HGBCL, NOS) has overlapping morphological and genetic features with diffuse large B-cell lymphoma (DLBCL) and Burkitt lymphoma (BL), leading to uncertainty in its diagnosis and clinical management. Using functional genomic approaches, we previously characterized HGBCL and NOS, that demonstrate gene expression profiling (GEP), and genetic signatures
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Issue Information Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-12
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Hyperparathyroidism and the Hematologist Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-12 Hajer Oun, Kirsteen Harper, Mike Leach, Barbara J. Bain
A 76-year-old man with a history of chronic obstructive pulmonary disease with lung fibrosis, type 2 diabetes mellitus, and chronic kidney disease underwent computed tomography imaging of the chest due to increasing dyspnea. The bones appeared sclerotic, and a bone scan showed diffuse tracer uptake throughout the axial and appendicular skeleton. The prostate showed no features of malignancy on magnetic
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Globalization in clinical drug development for sickle cell disease Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-12 Enrico Costa, Russell E. Ware, Léon Tshilolo, Julie Makani, Hubert G. M. Leufkens, Lucio Luzzatto
1 BACKGROUND Globalization of clinical trials, defined operationally as conduct in the international arena, has grown over the past few decades. The pharmaceutical industry is expanding its activities not only in High-Income countries but also in Low- and Middle-Income countries (LMICs).1 For pharmaceutical companies, this shift can be associated with several benefits: a larger pool of potential participants
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Efficacy and safety of zanubrutinib monotherapy for chronic lymphocytic leukemia/small lymphocytic lymphoma: A multicenter, real-world study in China Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-04 Jing Luo, Jiaojiao Zhang, Ligen Liu, Rong Wei, Yonghua Yao, Min Xu, Jumei Shi, Jianmin Yang, Jian Hou, Jin Wang, Jian-Qing Mi
Bruton tyrosine kinase (BTK) inhibitor is now the standard of care for both treatment-naïve (TN) and relapsed/refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Zanubrutinib, a next-generation BTK inhibitor with better BTK specificity and less off-target inhibition, has demonstrated superior efficacy and improved safety profile compared with first-generation BTK inhibitor
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The impact of post-remission granulocyte colony-stimulating factor use in the phase 3 studies of venetoclax combination treatments in patients with newly diagnosed acute myeloid leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-01 Courtney D. DiNardo, Keith W. Pratz, Panayiotis Panayiotidis, Xudong Wei, Vladimir Vorobyev, Árpád Illés, Inho Kim, Vladimir Ivanov, Grace Ku, Catherine L. Miller, Meng Zhang, Fernando Tatsch, Jalaja Potluri, Xenia Schmidt, Christian Récher
Based on results from the randomized, placebo-controlled phase 3 VIALE-A (NCT02993523) and VIALE-C (NCT03069352) trials,1-4 venetoclax in combination with hypomethylating agents or low-dose cytarabine (LDAC) has become standard of care in patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. Cytopenias are common adverse events with venetoclax and
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Prognostic significance of early acute splenic sequestration in children with severe sickle cell genotypes: A comprehensive longitudinal neonatal cohort study Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-01 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
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
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Ibrutinib‐related stellar hematomas of the palms and soles Am. J. Hematol. (IF 10.1) Pub Date : 2024-11-01 Naia Oillarburu, Loic Ysebaert, Caroline Protin, Ariadna Ortiz‐Brugues, Sarah Baali, Estelle Parriel, Vincent Sibaud
CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.
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Impact of myelodysplasia-related mutations on 2022 European LeukemiaNet genetic risk classification in de novo acute myeloid leukemia with normal karyotype Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-30 Srija Shanker, Robert P. Hasserjian, Yazan Madanat, Olga K. Weinberg, Miguel D. Cantu
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Harnessing the prognostic potential of PHF6 mutations in chronic myelomonocytic leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-29 Francesco Onida
CONFLICT OF INTEREST STATEMENT Francesco Onida received lecture fees from Menarini StemLine, Takeda, Kyowa Kirin and MEDAC.
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Toxicities and outcome after CD19-directed chimeric antigen receptor T-cell therapy for secondary neurolymphomatosis Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-23 Leon D. Kaulen, Philipp Karschnia, Sofia Doubrovinskaia, Jeremy S. Abramson, Maria Martinez-Lage, Ganesh Shankar, Bryan D. Choi, Jeffrey A. Barnes, Areej El-Jawahri, Ephraim P. Hochberg, P. Connor Johnson, Jacob D. Soumerai, Wolfgang Wick, Marcela V. Maus, Yi-Bin Chen, Matthew J. Frigault, Jorg Dietrich
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Emergency department intravenous fluid resuscitation and renal outcomes among adults with sickle cell disease Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-23 Marcus A. Carden, Jeffrey Lebensburger, Wayne Rosamond, Paula Tanabe, Vimal K. Derebail
Acidosis and increased tonicity in plasma can mediate pathologic changes in the membrane and cytoplasm of sickle red blood cells (sRBCs). These changes contribute to intravascular hemolysis, endothelial damage, and endothelial adhesion with propensity to microvascular occlusion, resulting in vaso-occlusive episodes (VOE) and end organ damage.1 Kidney dysfunction, in particular, is common among adults
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Fulminant intravascular hemolysis resulting from Clostridium perfringens infection Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-22 Kyo J. P. H. Renshof, Yorick Sandberg, Floor Weerkamp, Barbara J. Bain
A 70-year-old woman with a history of successfully resected pancreatic cancer 10 years ago presented with dyspnea and fever. Laboratory tests showed leukocytosis (white cell count 20.8 × 109/L) and elevated C-reactive protein (87 mg/L) with normal hemoglobin concentration (Hb) (137 g/L) and platelet count (242 × 109/L). Computed tomography identified a hepatic abscess. The abscess was drained and
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Parsaclisib for the treatment of primary autoimmune hemolytic anemia: Results from a phase 2, open-label study Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-22 Wilma Barcellini, Fabrizio Pane, Andrea Patriarca, Irina Murakhovskaya, Louis Terriou, Maria T. DeSancho, Wahid T. Hanna, Lance Leopold, Erica Rappold, Ke Szeto, Shaoceng Wei, Ulrich Jäger
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
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Clonal hematopoiesis and myeloid skewing in older population-based individuals Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-21 Maaike G. J. M. van Bergen, Priscilla Kamphuis, Aniek O. de Graaf, Jonas B. Salzbrunn, Theresia N. Koorenhof-Scheele, Isabelle A. van Zeventer, Avinash G. Dinmohamed, Jan Jacob Schuringa, Bert A. van der Reijden, Gerwin Huls, Joop H. Jansen
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.1 One of the hallmarks of aged HSCs is a myeloid differentiation bias, with less capability
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Dominance of mutations in epigenetic regulators and a diversity of signaling alterations in blast-phase BCR::ABL1-negative myeloproliferative neoplasms Am. J. Hematol. (IF 10.1) Pub Date : 2024-10-19 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
BCR::ABL1-negative myeloproliferative neoplasms (MPNs) can evolve to secondary acute myeloid leukemia (sAML) or blast-phase (BP) MPN, a very severe condition with lack of effective therapy.1 Leukemic transformation (LT) of MPNs displays a variable incidence according to MPN phenotype: 9%–13% in primary myelofibrosis (PMF), 3%–7% in polycythemia vera (PV), and 1%–4% in essential thrombocythemia (ET)
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