Results 101 to 110 of about 239,349 (307)
Determinants of Long‐Term Benefit From High Dose Melphalan With Autologous Stem Cell Transplant in AL Amyloidosis
American Journal of Hematology, EarlyView.ABSTRACT
High dose melphalan (HDM) with autologous stem cell transplant is an established treatment for systemic light chain amyloidosis, but its incremental benefit in the era of effective standard intensity therapy is unknown. We retrospectively analyzed 475 transplant‐eligible patients who completed standard intensity treatment with or without HDM ...Maximilian J. Steinhardt, Ute Hegenbart, Tamer Hellou, Sara Oubari, Murielle Roussel, Rahel Schwotzer, Francis Buadi, David Dingli, Moritz Binder, Taxiarchis Kourelis, Saurabh S. Zanwar, Max J. Rieger, Caroline Morbach, Vladimir Cejka, Bernhard Gerber, Stefan Störk, Hermann Einsele, Shaji Kumar, Morie Gertz, Alexander Carpinteiro, K. Martin Kortum, Eli Muchtar, Angela Dispenzieri, Stefan Schönland +23 morewiley +1 more sourceReal‐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 sourceAllogeneic Bone Marrow Transplantation for Primary Immunodeficiencies: Pilot Trial of a Novel, Reduced Intensity Platform [PDF]
, 2017 Dimana Dimitrova, Juan Gea‐Banacloche, Alexandra F. Freeman, Gülbu Uzel, Christa S. Zerbe, Jeffrey I. Cohen, V. Koneti Rao, Steven M. Holland, Stephanie Cotton, Jennifer Mann, Lauren R. Skeffington, Lauren M. Curtis, Steven Z. Pavletic, Daniel H. Fowler, Ronald E. Gress, Christopher G. Kanakry, Jennifer A. Kanakry +16 moreopenalex +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 sourceTherapeutic Outcomes in VEXAS Syndrome: A Multicenter Comparative Cohort of Allogeneic Hematopoietic Stem Cell Transplantation and Hypomethylating Agents
American Journal of Hematology, EarlyView.ABSTRACT
Hypomethylating agents (HMA) and allogeneic hematopoietic stem cell transplantation (alloHSCT) have both demonstrated remissions in VEXAS; however, comparative data is lacking. We conducted a multicenter, retrospective analysis of 66 patients diagnosed with VEXAS syndrome treated with HMA (n = 35) or alloHSCT (n = 31). Baseline characteristics 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 sourceAtypical Clinical Course of Griscelli Syndrome Type 2 With Primarily Neurologic Presentation and Adult‐Onset in a 46‐Year‐Old Male
American Journal of Medical Genetics Part A, EarlyView.ABSTRACT
Griscelli Syndrome Type 2 (GS2) is a rare autosomal recessive disorder caused by pathogenic mutations in the RAB27A gene. Typically, it is characterized by cutaneous hypopigmentation, immunodeficiency, with or without neurological abnormalities secondary to hemophagocytic lymphohistiocytosis (HLH). Without treatment, GS2 often results in fatal Dzhoy Papingi, Michael Kutsche, Helena Lichtenfeld, Fanny Kortüm, Angela Abicht, Laura Herrmann, Theresia Herget +6 morewiley +1 more source
