Purplish granules as a cytological signature of cortical developmental disorders caused by pathogenic variants in WDR81
Wiley - American Journal of Hematology
by Baptiste Le Calvez, Thomas Besnard, Benjamin Cogne, Stéphane Bézieau, Marie C. Béné, Claire Beneteau, Marion Eveillard
1d ago
American Journal of Hematology, EarlyView ..read more
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Bone marrow Tfr2 deletion improves the therapeutic efficacy of the activin‐receptor ligand trap RAP‐536 in β‐thalassemic mice
Wiley - American Journal of Hematology
by Emanuele Tanzi, Simona Maria Di Modica, Jessica Bordini, Violante Olivari, Alessia Pagani, Valeria Furiosi, Laura Silvestri, Alessandro Campanella, Antonella Nai
1d ago
Hematopoietic Tfr2 deletion promotes erythroid differentiation increasing erythropoietin sensitivity of erythroid cells. The activin receptor ligand trap RAP-536 enhances erythroid maturation of late stage erythroblasts. The two approaches additively ameliorate β-thalassemia. Abstract 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 growt ..read more
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Novel germline JAK2R715T mutation causing PV‐like erythrocytosis in 3 generations. Amelioration by Ropeg‐Interferon
Wiley - American Journal of Hematology
by Jihyun Song, Lucie Lanikova, Soo Jin Kim, Nicolas Papadopoulos, Jessica Meznarich, Stefan N. Constantinescu, Brynn Parsegov, Jaroslav F. Prchal, Josef T. Prchal
1d ago
Abstract Polycythemia vera (PV) is a clonal disorder arising from the acquired somatic mutations of the JAK2 gene, including JAK2 V617F 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 JAK2 V617F and exon 12 mutations. Next generation sequencing revealed a novel mutation: JAK2 R715T in the pseudokinase domain (JH2) at 47.5%. Its presence in her nail DNA confirmed a germline origin. Her mother and her son similarly had erythrocytosis and a JAK2 R715T mutation. Computer mo ..read more
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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
Wiley - American Journal of Hematology
by 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
3d ago
American Journal of Hematology, EarlyView ..read more
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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
Wiley - American Journal of Hematology
by Rebecca Silbermann, Jacob Laubach, Jonathan L. Kaufman, Douglas W. Sborov, Brandi Reeves, Cesar Rodriguez, Ajai Chari, Luciano J. Costa, Larry D. Anderson Jr, 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, Saad Z. Usmani, Paul G. Richardson
3d ago
Abstract 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) collected using the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30-item (QLQ-C30), EORTC Quality of Life Questionnaire Multipl ..read more
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Making a virtue out of an evil: Are red blood cells from chronic mountain sickness patients eligible for transfusions?
Wiley - American Journal of Hematology
by 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
3d ago
We investigated highlanders, permanently living at an altitude of 5100 m and compared Chronic Mountain Sickness (CMS) patients with control volunteers. While we found differences in systemic parameters such as blood oxygen content, hematocrit, hemoglobin concentration, and blood viscosity, the mechanical and rheological properties of single red blood cells did not differ between the two investigated groups ..read more
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Exploring the landscape of somatic ASXL2 mutations in myeloid neoplasms: Frequency and clinical implications
Wiley - American Journal of Hematology
by 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
3d ago
American Journal of Hematology, EarlyView ..read more
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High‐risk multiple myeloma: Redefining genetic, clinical, and functional high‐risk disease in the era of molecular medicine and immunotherapy
Wiley - American Journal of Hematology
by Matthew J. Rees, Shaji Kumar
3d ago
Abstract 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 to identify many patients destined for ER. Treatment strategies are increasingly focused on disease biology and trials enriched for high-risk (HR)MM, but consensus on the minimum requ ..read more
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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
Wiley - American Journal of Hematology
by 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
3d ago
American Journal of Hematology, EarlyView ..read more
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Patients with acquired pure red cell aplasia respond to PI3Kδ inhibitor rapidly
Wiley - American Journal of Hematology
by Zhenzhen Wang, Bo Jiang, Lin Song, Mingyuan Sun, Chunhong Li, Xiaoxia Li, Weiwei Zheng, Yuan Tao, Qi Sun, Junyuan Qi
6d ago
American Journal of Hematology, EarlyView ..read more
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