Results 261 to 270 of about 591,464 (366)
Pathogenic Variants in Complement Genes and Risk of Atypical Hemolytic Uremic Syndrome Relapse after Eculizumab Discontinuation.
American Society of Nephrology. Clinical Journal, 2017 F. Fakhouri, M. Fila, F. Provôt, Yahsou Delmas, C. Barbet, V. Châtelet, C. Rafat, M. Cailliez, J. Hogan, A. Servais, A. Karras, R. Makdassi, F. Louillet, J. Coindre, E. Rondeau, C. Loirat, V. Frémeaux-Bacchi +16 moresemanticscholar +1 more sourceManagement of Iron Overload in Infants and Toddlers With Diamond–Blackfan Anemia Syndrome: A French–Italian Study
American Journal of Hematology, EarlyView.ABSTRACT
Diamond–Blackfan Anemia Syndrome (DBAS) is a rare congenital anemia often requiring chronic red blood cell transfusions from infancy. Without appropriate chelation, iron overload develops early and may be severe; however, no data are available on chelation in patients under 3 years of age.Francesca Torchio, Baptiste Lecalvez, Emanuela Garelli, Coralie Mallebranche, Adriana Carando, Marie‐Pierre Castex, Nathalie Garnier, Marco Zecca, Nathalie Aladjidi, Elisa Bertoni, Arthur Sterin, Maria Licciardello, Anne Sirvent, Antonella Sau, Isabelle Marie, Benedicte Bruno, Lydie M. Da Costa, Franca Fagioli, Paola Quarello, Thierry Leblanc +19 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 sourceEffect of Quinolone Prophylaxis Discontinuation During Pre-engraftment Neutropenia on Incidence, Mortality, and Etiology of Bloodstream Infections in Hematopoietic Stem-cell Transplant Recipients: A Systematic Review and Meta-analysis. [PDF]
Open Forum Infect DisRandi BA, Higashino HR, de Godoy Macedo PL, Guimarães T, Pires BG, Rocha V, Howard A, Levin AS, Costa SF. +8 moreeuropepmc +1 more sourceExploring the Burden on Patients Living With and Receiving Treatment for Immune Thrombocytopenia (ITP): Patient and Physician Perceptions From the ITP World Impact Survey (I‐WISh) 2.0
American Journal of Hematology, EarlyView.ABSTRACT
Limited data exist on how patients and physicians perceive immune thrombocytopenia (ITP) symptoms and treatment‐related burden. I‐WISh (ITP World Impact Survey) 2.0 surveyed 1018 patients and 431 physicians in 15 countries to characterize the impact of ITP and its treatments on patients.Nichola Cooper, James Bussel, Waleed Ghanima, Drew Provan, Yoshiaki Tomiyama, Ming Hou, Donald M. Arnold, Cristina Santoro, Francesco Zaja, Barbara Lovrencic, Mervyn Morgan, Michal Winograd, Jennifer DiRaimo, Danielle Boyle, Olivera Rajkovic‐Hooley, Meritxell Vendranas, Susan Frade, Caroline Kruse +17 morewiley +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 source