Results 191 to 200 of about 283,613 (309)
Real‐World Evaluation of Talquetamab for the Treatment of Relapsed/Refractory Multiple Myeloma (RRMM): An International Myeloma Working Group Immunotherapy Registry Real‐World Analysis
American Journal of Hematology, EarlyView.ABSTRACT
Talquetamab is a GPRC5D × CD3 bispecific antibody approved for relapsed/refractory multiple myeloma (RRMM). This is the first real‐world study to comprehensively report muco‐cutaneous toxicities, infections, and efficacy of talquetamab in 151 patients across five countries with the longest follow‐up reported enabling better efficacy and ...Murali Janakiram, Carlyn R. Tan, Hira Mian, Chiung‐Yu Huang, Rakesh Popat, Joaquín Martínez‐Lopez, Efsthathios Kastritis, Wee‐Joo Chng, Prashant Kapoor, Mrugakshi Dave, Susan Bal, Laurent Garderet, Andre De Menezes Silva Corraes, Christine Riedhammer, Torsten Steinbrunn, Magdalene Corona, Chandramouli Nagarajan, Hermann Einsele, Tom Martin, Amrita Krishnan, Yi Lin, Nadine Abdullah, Niels Abildgaard, Ingerid Abrahamsen, Melissa Alsina, Kenneth Anderson, Hervé Avet‐Loiseau, Ashraf Badros, Nizar Jacques Bahlis, Susan Bal, Rahul Banerjee, Dina Ben‐Yehuda, Jesús G. Berdeja, Michele Cavo, Ajai Chari, Wen‐Ming Chen, Marta Chesi, Wee‐Joo Chng, Yael Cohen, Adam Cohen, Gordon Cook, Luciano Costa, Andrew Cowan, Carlos Fernández de Larrea, Benjamin Derman, Juan Du, Hermann Einsele, Gösta Gahrton, Francesca Gay, Sergio A. Giralt, Simon Harrison, J Jean‐Luc Harousseau, Joy Ho, Doris Hansen, Maung Myo Htut, Andrzej J. Jakubowiak, Murali Janakiram, Prashant Kapoor, Efstathios Kastritis, Jonathan Kaufman, Kihyun Kim, Amrita Krishnan, Shaji Kumar, Chara Kyriakou, Xavier Leleu, Yi Lin, Sagar Lonial, Matthew frigault, Johan L. Lund, Elias Mai, Angelo Maiolino, Elias Mai, Angelo Maiolino, Sireesha Asoori Maringanti, Thomas Martin, Joaquin Martinez‐Lopez, María‐Victoria Mateos, Giampaolo Merlini, Hira Mian, Joseph Mikhael, Monique Minnema, Philippe Moreau, Gareth Morgan, Nikhil Munshi, Omar Nadeem, Chandramouli Nagarajan, Ajay Nooka, Michael O'Dwyer, Rimke Oostvogel, Alberto Orfao, Paula Rodriguez Otero, Darren Pan, Krina Patel, Rashesh Popat, Noemí Puig +94 morewiley +1 more sourceA plasma-based DNA test for quantification of disease burden in acute myeloid leukemia patients undergoing bone marrow transplantation. [PDF]
Proc Natl Acad Sci U S AWang Y, Xie J, Pasca S, Popoli M, Ptak J, Dobbyn L, Silliman N, Paul S, Jones RJ, Levis MJ, Curtis SD, Douville C, Shams C, Guo MZ, Mo S, Gocke CD, Malek SN, Bollard CM, Bettegowda C, Kinzler KW, Vogelstein B, Papadopoulos N, Gondek LP. +22 moreeuropepmc +1 more sourceCorrection of Ineffective Erythropoiesis and Normalization of Iron Homeostasis After Exagamglogene Autotemcel in Transfusion‐Dependent β‐Thalassemia
American Journal of Hematology, EarlyView.ABSTRACT
Exagamglogene autotemcel (exa‐cel) is a one‐time, ex vivo, CRISPR‐Cas9 gene edited cell therapy approved for patients with transfusion dependent β‐thalassemia (TDT) aged 12–35 years. In a Phase 3 study (CLIMB THAL‐111), exa‐cel treatment resulted in reactivation of fetal hemoglobin and increases in total hemoglobin, leading to transfusion ...Sujit Sheth, Selim Corbacioglu, Josu de la Fuente, Mattia Algeri, Joachim Rupprecht, Kevin H. M. Kuo, Ami J. Shah, Peter Lang, Hayley Merkeley, Ben Carpenter, Markus Y. Mapara, Robert I. Liem, Stephan Grupp, Yogi Chopra, Amanda M. Li, Janet L. Kwiatkowski, Melanie Kirby‐Allen, Maria Domenica Cappellini, Antonis Kattamis, Sakellarios Zairis, Tina Liu, William Hobbs, Haydar Frangoul, Franco Locatelli, Roland Meisel, on behalf of the CLIMB THAL‐111 and CLIMB‐131 Study Groups +25 morewiley +1 more sourceSequential in vivo CRISPR screens identify the clonal dominance of <i>Nf1</i> loss in long-term hematopoiesis. [PDF]
Blood SciYuan S, Wu Q, Yin T, Chen L, Zhang B, Li P, Xie X, Sun G, Cheng T, Wei J, Cheng H. +10 moreeuropepmc +1 more sourceTherapeutic Outcomes in VEXAS Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents
American Journal of Hematology, EarlyView.Key findings of therapeutic outcomes from a multi‐center cohort of VEXAS patients. ABSTRACT
Hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation (alloHSCT) have both demonstrated remissions in VEXAS; however, comparative data is lacking.Saubia Fathima, Margaret Mei‐Kay Wong, Jesus Gonzalez‐Lugo, Susan M. Geyer, Ali Alsugair, Maria Sirenko, Kimberly J. Langer, Terra L. Lasho, Christy Finke, Jun Choi, Maher Abdul‐Hay, Gary Ho, Mark R. Litzow, Aasiya Matin, Urshila Durani, Mehrdad Hefazi, William J. Hogan, Mithun V. Shah, Aref Al‐Kali, Kebede H. Begna, Naseema Gangat, Antoine N. Saliba, Ronald S. Go, Tariq Kewan, Gabriel Bartoo, Jade Kutzke, Kristen McCullough, Kenneth J. Warrington, Megan Sullivan, Kaaren K. Reichard, Horatiu Olteanu, Hemant Murthy, Talha Badar, Yael Kusne, Jeanne Palmer, Saurabh Chhabra, Nathan Punwani, Mary Riwes, Joseph P. McGuirk, Elizabeth F. Krakow, Amelia Langston, Taxiarchis Kourelis, David Dingli, James Foran, Matthew J. Koster, Mrinal M. Patnaik, David B. Beck, Hassan B. Alkhateeb, Abhishek A. Mangaonkar +48 morewiley +1 more sourceThe 9th International RASopathies Symposium
American Journal of Medical Genetics Part A, EarlyView.ABSTRACT
The RASopathies are a group of congenital disorders with overlapping clinical manifestations that are caused by pathogenic germline or early somatic variants that result in the hyperactivation of the RAS/mitogen‐activated protein kinase (MAPK) signaling pathway.Pau Castel, Lisa Schoyer, Beth Stronach, Raya Bogdanova, Anton M. Bennett, Jaishri Blakeley, Miriam Bornhorst, Tammy Bowers, Saskia M. Brachmann, Emma Burkitt‐Wright, Kathryn Chatfield, Alessandro De Luca, Khalil El‐Chammas, Abdul Elkadri, John E. Fortunato, Bruce D. Gelb, Anne Goriely, Karen Gripp, Kassidy Grover, Lindsay Homan, Kenneth A. Kern, Maija Kiuru, Charles (Chuck) Lawson, Yong‐Seok Lee, Frank McCormick, Gina Ney, Cristina Nuevo‐Tapioles, Sara Pardej, Elizabeth I. Pierpont, Julia Plank, Nancy Ratner, Katherine A. Rauen, J. Elliott Robinson, Les Rogers, Sarah E. Sheppard, Keir Shiels, David Stevenson, Dagmar Tiemens, Matthew Traylor, K. Nicole Weaver, Marielle Yohe, Tamar Green +41 morewiley +1 more source