Results 171 to 180 of about 316,174 (240)

Correction 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 more
wiley   +1 more source

Monofocal intraocular lens based on the Bessel principle for improved intermediate vision: a comparative assessment. [PDF]

BMC Ophthalmol
Kremser F, Yan W, Khoramnia R, Auffarth GU, Łabuz G.   +4 more
europepmc   +1 more source

First-line ET plus palbociclib versus standard mono-chemotherapy in high-risk HR-positive/HER2-negative metastatic breast cancer and indication for chemotherapy: primary results from the randomized phase IV PADMA study. [PDF]

ESMO Open
Schmidt M, Thill M, Rey J, Rautenberg B, Bjelic-Radisic V, Decker T, Rom J, Kögel M, Lübbe K, Nacke A, Seiler S, Just M, Müller V, Buss-Steidle R, Terhaag J, Mundhenke C, Denkert C, Holtschmidt J, Loibl S.   +18 more
europepmc   +1 more source

The Changing Landscape of Maintenance Therapy in Newly Diagnosed Multiple Myeloma: A Systematic Review With Network Meta‐Analysis of the European Myeloma Network (EMN)

American Journal of Hematology, EarlyView.
Our meta‐analysis showed significant improvement of PFS with lenalidomide, proteasome inhibitors, and CD38‐based therapies. A significant OS benefit was noted only with lenalidomide in transplant‐eligible (TE) patients, while CD38‐directed therapy showed a trend toward improved OS.
Heinz Ludwig, Evangelos Terpos, Francesca Gay, Niels W. C. J. van de Donk, Sarah Bernhard, Monika Engelhardt, Gordon Cook, Fredrik Schjesvold, Hermann Einsele, Graham Jackson, Charlotte Pawlyn, María‐Victoria Mateos, Sonja Zweegman, Martin Schreder, Meral Beksaç, Christoph Driessen, Enrique M. Ocio, Rakesh Popat, Leo Rasche, Cyrille Touzeau, Pellegrino Musto, Annemiek Broijl, Meletios Dimopoulos, Roman Hajek, Mario Boccadoro, Pieter Sonneveld   +25 more
wiley   +1 more source

Therapeutic 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 more
wiley   +1 more source

Quizartinib in Combination With FLAG‐IDA for Relapsed or Refractory Acute Myeloid Leukemia (FLAG‐QUIDA): A PETHEMA Phase I‐II Trial

American Journal of Hematology, EarlyView.
ABSTRACT Quizartinib is a tyrosine kinase inhibitor with single agent activity in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) and has demonstrated efficacy in first‐line therapy when combined with intensive chemotherapy in both FLT3 ITD‐negative and positive AML.
Teresa Bernal, Javier Zambrano Márquez, Rebeca Rodríguez‐Veiga, Eduardo Rodríguez‐Arbolí, Juan Manuel Bergua‐Burgués, Susana Vives, Montserrat Arnán, Pilar Martínez Sánchez, Pilar Herrera‐Puente, Josefina Serrano‐López, Vicente Rubio, Inmaculada Marchante‐Cepillo, Marta Cervera‐Calvo, Andrés Novo García, Rosa Fernández Martín, Cristina Gil Cortés, Carlos Rodríguez‐Medina, Victor Noriega‐Concepción, Ana Alfonso‐Piérola, Carmen Loredo, María Antuña‐Casal, Blanca Boluda, Isabel Cano‐Ferri, Evelyn Acuña‐Cruz, Laura Torres‐Miñana, Joaquín Sánchez‐García, Cristina Bilbao, Maria Teresa Gómez‐Casares, David Martínez Cuadrón, Pau Montesinos   +29 more
wiley   +1 more source

Analysis of influencing factors and structural changes in hospitalization expenses for heart failure patients under the DRG payment system reform. [PDF]

Front Public Health
Huang D, Gao H, Chen H, Huang J, Zhao J.
europepmc   +1 more source

Dordaviprone Maintenance After Allogeneic HCT for High‐Risk Acute Myeloid Leukemia and Myelodysplastic Neoplasm

American Journal of Hematology, EarlyView.
ABSTRACT Dordaviprone is a first‐in‐class small‐molecule imipridone. In preclinical studies, it is active against leukemia cells harboring a TP53 mutation or complex karyotype and leukemia stem cells while sparing normal bone marrow cells. This study aimed to determine the safety of dordaviprone maintenance after allogeneic hematopoietic cell ...
Vijaya Raj Bhatt, Christopher S. Wichman, Alyssa Bouska, Moataz Ellithi, Michael Haddadin, Javeed Iqbal, James E. Talmadge, Lori J. Maness, Krishna Gundabolu   +8 more
wiley   +1 more source

Sebetralstat for on-demand treatment of hereditary angioedema: A pooled analysis of placebo-controlled clinical trials. [PDF]

World Allergy Organ J
Aygören-Pürsün E, Cohn DM, Agmon-Levin N, Banerji A, Bernstein JA, Busse P, Cancian M, de Castro ED, Farkas H, Grivcheva-Panovska V, Hagin D, Hakl R, Honda D, Kessel A, Kinaciyan T, Ifaad, Kurowski M, Li HH, Lleonart R, Longhurst HJ, Lumry WR, Magerl M, Martinez-Saguer I, Melamed I, Psarros F, Savic S, Soteres DF, Staevska M, Stobiecki M, Wedner HJ, Zanichelli A, Hao J, Patil K, Iverson M, Owiredu-Yeboa S, Smith MD, Yea CM, Audhya PK, Riedl MA, Kiani-Alikhan S, Maurer M.   +40 more
europepmc   +1 more source

Lymphatic Abnormalities in Noonan Syndrome Extend Beyond Clinically Apparent Disease

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Lymphatic disease represents a well‐described manifestation of Noonan syndrome (NS), yet the full phenotypic spectrum remains incompletely characterized, especially in asymptomatic individuals. We conducted a cross‐sectional study including 10 individuals with NS (four with peripheral lymphedema and six without) and 10 age‐ and sex‐matched ...
Inger Norlyk Sheyanth, Benjamin Kelly, Sheyanth Mohanakumar, Steffen Ringgaard, Yoav Dori, Irene Kibæk Nielsen, Uffe Birk Jensen, Vibeke Elisabeth Hjortdal   +7 more
wiley   +1 more source