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JOURNAL ARTICLE
Robert A Wood, R Sharon Chinthrajah, Amanda K Rudman Spergel, Denise C Babineau, Scott H Sicherer, Edwin H Kim, Wayne G Shreffler, Stacie M Jones, Donald Y M Leung, Brian P Vickery, J Andrew Bird, Jonathan M Spergel, Michael Kulis, Ahmar Iqbal, Derrick Kaufman, Dale T Umetsu, Monica Ligueros-Saylan, Alkaz Uddin, Robert B Fogel, Stephanie Lussier, Kim Mudd, Julian Poyser, Martin MacPhee, Maria Veri, Wendy Davidson, Sanaz Hamrah, Andrew Long, Alkis Togias
BACKGROUND: Food allergy is common and causes substantial morbidity and even mortality. Safe and effective treatments for food allergy would therefore be highly desirable, especially for individuals with multiple food allergies. OBJECTIVES: Our aim was to describe a phase 3 study on treatment of patients with multiple food allergies with omalizumab. METHODS: The study was developed as a collaboration between the Consortium for Food Allergy Research, the National Institute of Allergy and Infectious Diseases, and 2 industry sponsors (Genentech and Novartis)...
November 2022: J Allergy Clin Immunol Glob
#62
JOURNAL ARTICLE
Larysa Sanchez, Ajai Chari, Mu Cheng, Dasha Cherepanov, Maral DerSarkissian, Fei Huang, Dawn Marie Stull, Jonathan Dabora, Melanie Young, Stephen J Noga, Selina Pi, Melody Zhang, Azeem Banatwala, Mei Sheng Duh, Sikander Ailawadhi
BACKGROUND: Economic differences among currently available proteasome inhibitors (PI)-based lenalidomide-dexamethasone (Rd)-backbone triplet regimens-ixazomib (I), bortezomib (V), and carfilzomib (K) plus Rd-remain poorly understood. OBJECTIVE: To assess health care resource utilization (HCRU) and health care costs of patients with relapsed/refractory multiple myeloma (RRMM) in the United States treated with IRd, VRd, and KRd. METHODS: This retrospective longitudinal cohort study using IQVIA PharMetrics Plus adjudicated claims US data (January 1, 2015, to September 30, 2020) included adult patients with all available data who initiated IRd, VRd, or KRd in second line of therapy or later (LOT2+) on or after September 1, 2015...
September 30, 2023: Journal of Managed Care & Specialty Pharmacy
#63
JOURNAL ARTICLE
Todd J Schwedt, Jae Lee, Kerry Knievel, Jennifer McVige, Weiying Wang, Zheng Wu, Patrick Gillard, Darshini Shah, Andrew M Blumenfeld
BACKGROUND: Chronic migraine (CM) is a common neurologic disorder that imposes substantial burden on payers, patients, and society. Low rates of persistence to oral migraine preventive medications have been previously documented; however, less is known about persistence and costs associated with innovative nonoral migraine preventive medications. OBJECTIVE: To evaluate real-world persistence and costs among adults with CM treated with onabotulinumtoxinA (onabotA) or calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs)...
October 2023: Journal of Managed Care & Specialty Pharmacy
#64
JOURNAL ARTICLE
Chun-Jui Huang, Chieh-Hua Lu, Kuang-Chung Shih
BACKGROUND: In patients with kidney or hepatic diseases, an increment of circulating pasireotide is also expected. Therefore, this open-label, phase I study aimed to evaluate the pharmacokinetic profiles and safety of subcutaneous (SC) and long-acting release (LAR) intramuscular injections of pasireotide in male Taiwanese volunteers who are hyperendemic hepatitis B/C and chronic kidney disease (CKD). METHODS: A total of 45 male volunteers were randomized to receive one of nine treatment sequences, involving a single subcutaneous injection of 300, 600, or 900 μg pasireotide, a multiple SC injection of the same dosage of pasireotide [300, 600, or 900 μg, twice daily (b...
November 2023: European Journal of Drug Metabolism and Pharmacokinetics
#65
JOURNAL ARTICLE
Georgios E Papadakis, Benedicte de Kalbermatten, Alexandre Dormoy, Sylvie Salenave, Severine Trabado, Oceana Vieira-Pinto, Carine Richa, Peter Kamenicky, Philippe Chanson, Luigi Maione, Nelly Pitteloud, Jacques Young
STUDY QUESTION: Does Cushing's syndrome (CS) differently affect the gonadotrope axis and testicular functions (GA/TF) according to the hypercortisolism intensity and underlying etiology? SUMMARY ANSWER: Endogenous cortisol excess caused by CS leads to varying degrees of hypogonadotropic hypogonadism (HH) with more severe GA/TF impairment and altered spermatogenesis in men with intense hypercortisolism associated with paraneoplastic/ectopic adrenocorticotrophic hormone (ACTH) secretion (EAS)...
September 23, 2023: Human Reproduction
#66
RANDOMIZED CONTROLLED TRIAL
Eric Bouffet, Jordan R Hansford, Maria Luisa Garrè, Junichi Hara, Ashley Plant-Fox, Isabelle Aerts, Franco Locatelli, Jasper van der Lugt, Ludmila Papusha, Felix Sahm, Uri Tabori, Kenneth J Cohen, Roger J Packer, Olaf Witt, Larissa Sandalic, Ana Bento Pereira da Silva, Mark Russo, Darren R Hargrave
BACKGROUND: Detection of the BRAF V600E mutation in pediatric low-grade glioma has been associated with a lower response to standard chemotherapy. In previous trials, dabrafenib (both as monotherapy and in combination with trametinib) has shown efficacy in recurrent pediatric low-grade glioma with BRAF V600 mutations, findings that warrant further evaluation of this combination as first-line therapy. METHODS: In this phase 2 trial, patients with pediatric low-grade glioma with BRAF V600 mutations who were scheduled to receive first-line therapy were randomly assigned in a 2:1 ratio to receive dabrafenib plus trametinib or standard chemotherapy (carboplatin plus vincristine)...
September 21, 2023: New England Journal of Medicine
#67
REVIEW
Marty Chaplin, Rebecca Bresnahan, Nigel Fleeman, James Mahon, Rachel Houten, Sophie Beale, Angela Boland, Yenal Dundar, Ashley Marsden, Pinki Munot
As part of the National Institute for Health and Care Excellence (NICE) highly specialised technology (HST) evaluation programme, Novartis submitted evidence to support the use of onasemnogene abeparvovec as a treatment option for patients with pre-symptomatic 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival of motor neuron (SMN) 1 gene and up to three copies of the SMN2 gene. The Liverpool Reviews and Implementation Group at the University of Liverpool was commissioned to act as the External Assessment Group (EAG)...
September 20, 2023: PharmacoEconomics Open
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JOURNAL ARTICLE
Jan Heilinger, Jasmin Weindler, Katrin Sabine Roth, Philipp Krapf, Klaus Schomäcker, Markus Dietlein, Alexander Drzezga, Carsten Kobe
BACKGROUND: In 2022, the American Food and Drug Administration and the European Medicines Agency approved [177 Lu]Lu-PSMA-617 (PLUVICTO™, Novartis AG, Basel, Switzerland) for radionuclide therapy with prostate-specific membrane antigen (PSMA) ligands in metastatic prostate cancer. Theranostics require appropriate patients to be identified by positron emission tomography (PET) prior to radionuclide therapy, usually employing [68 Ga]Ga-PSMA-11. Alternatively, several 18 F-labelled PSMA-PET tracers are available and may increasingly replace 68 Ga-labelled compounds, with respect to their image quality, availability and other practical advantages...
September 20, 2023: EJNMMI Research
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JOURNAL ARTICLE
Elizabeth Berry-Kravis, Leonard Abbeduto, Randi Hagerman, Christopher S Coffey, Merit Cudkowicz, Craig A Erickson, Andrea McDuffie, David Hessl, Lauren E Ethridge, Flora Tassone, Walter E Kaufmann, Katherine Friedmann, Lauren Bullard, Anne Hoffmann, Jeremy Veenstra-VanderWeele, Kevin Staley, David Klements, Michael Moshinsky, Brittney Harkey, Jeffrey D Long, Janel Fedler, Elizabeth Klingner, Dixie J Ecklund, Michele Costigan, Trevis Huff, Brenda Pearson
BACKGROUND: FXLEARN, the first-ever large multi-site trial of effects of disease-targeted pharmacotherapy on learning, was designed to explore a new paradigm for measuring effects of mechanism-targeted treatment in fragile X syndrome (FXS). In FXLEARN, the effects of mGluR5 negative allosteric modulator (NAM) AFQ056 on language learning were evaluated in 3-6 year-old children with FXS, expected to have more learning plasticity than adults, where prior trials of mGluR5 NAMs have failed...
August 31, 2023: Journal of Clinical Investigation
#70
MULTICENTER STUDY
Akshay Sharma, Jaap-Jan Boelens, Maria Cancio, Jane S Hankins, Prafulla Bhad, Marjohn Azizy, Andrew Lewandowski, Xiaojun Zhao, Shripad Chitnis, Radhika Peddinti, Yan Zheng, Neena Kapoor, Fabio Ciceri, Timothy Maclachlan, Yi Yang, Yi Liu, Jianping Yuan, Ulrike Naumann, Vionnie W C Yu, Susan C Stevenson, Serena De Vita, James L LaBelle
BACKGROUND: Sickle cell disease is caused by a defect in the β-globin subunit of adult hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed red cells that hemolyze and cause vaso-occlusion that results in progressive organ damage and early death. Elevated fetal hemoglobin levels in red cells protect against complications of sickle cell disease. OTQ923, a clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9-edited CD34+ hematopoietic stem- and progenitor-cell (HSPC) product, has a targeted disruption of the HBG1 and HBG2 (γ-globin) gene promoters that increases fetal hemoglobin expression in red-cell progeny...
August 31, 2023: New England Journal of Medicine
#71
RANDOMIZED CONTROLLED TRIAL
Michael I Barbato, Jeannette Nashed, Diana Bradford, Yi Ren, Sachia Khasar, Claudia P Miller, Banu S Zolnik, Hong Zhao, Yangbing Li, Youwei Bi, Stacy S Shord, Anup K Amatya, Pallavi S Mishra-Kalyani, Barbara Scepura, Raniya A Al-Matari, Richard Pazdur, Paul G Kluetz, Martha Donoghue, Harpreet Singh, Nicole Drezner
On March 16, 2023, the FDA approved dabrafenib in combination with trametinib (Tafinlar, Mekinist; Novartis Pharmaceuticals Corporation) for the treatment of pediatric patients with low-grade glioma (LGG) with a BRAFV600E mutation who require systemic therapy. FDA also approved oral formulations of both drugs suitable for patients who cannot swallow pills. This approval was based on the LGG cohort from study CDRB436G2201 (NCT02684058), a multicenter, open-label trial in which pediatric patients with LGG with a BRAFV600E mutation were randomly assigned 2:1 to dabrafenib plus trametinib (D+T) or carboplatin plus vincristine (C+V)...
January 17, 2024: Clinical Cancer Research
#72
JOURNAL ARTICLE
Ryan A Herold, Christopher J Schofield, Fraser A Armstrong
The ability to control enzyme cascades entrapped in a nanoporous electrode material (the "Electrochemical Leaf", e-Leaf) has been exploited to gain detailed kinetic insight into the mechanism of an anti-cancer drug. Ivosidenib, used to treat acute myeloid leukemia, acts on a common cancer-linked variant of isocitrate dehydrogenase 1 (IDH1 R132H) inhibiting its "gain-of-function" activity-the undesired reduction of 2-oxoglutarate (2OG) to the oncometabolite 2-hydroxyglutarate (2HG). The e-Leaf quantifies the kinetics of IDH1 R132H inhibition across a wide and continuous range of conditions, efficiently revealing factors underlying the inhibitor residence time...
August 21, 2023: Angewandte Chemie
#73
JOURNAL ARTICLE
Fred S Sarfo, Nana K Ayisi-Boateng, Samuel B Nguah, Osei Sarfo-Kantanka, Collins Kokuro, Hanson Ababio, Yaw Adu-Boakye, Bruce Ovbiagele
OBJECTIVE: To identify the determinants of systolic blood pressure variability (SBPV) among Ghanaians. DESIGN: We undertook a secondary analysis of data collected in a prospective study. SETTING: The study involved patients with hypertension and or diabetes receiving care in five hospitals in Ghana. MAIN OUTCOME MEASURES: We assessed determinants of SBPV among 2,785 Ghanaian patients. We calculated the standard deviation (SD) of systolic BP recordings of 3 to 10 visits per patient over 18 months as a measure of SBPV...
January 2023: Ghana Medical Journal
#74
JOURNAL ARTICLE
Philip Hartout, Bojana Počuča, Celia Méndez-García, Christian Schleberger
MOTIVATION: Identifying peptides associated with the major histocompability complex class II (MHCII) is a central task in the evaluation of the immunoregulatory function of therapeutics and drug prototypes. MHCII-peptide presentation prediction has multiple biopharmaceutical applications, including the safety assessment of biologics and engineered derivatives in silico, or the fast progression of antigen-specific immunomodulatory drug discovery programs in immune disease and cancer. This has resulted in the collection of large-scale data sets on adaptive immune receptor antigenic responses and MHC-associated peptide proteomics...
August 1, 2023: Bioinformatics
#75
RANDOMIZED CONTROLLED TRIAL
Christopher W Ryan, Catherine M Tangen, Elisabeth I Heath, Mark N Stein, Maxwell V Meng, Ajjai S Alva, Sumanta K Pal, Igor Puzanov, Joseph I Clark, Toni K Choueiri, Neeraj Agarwal, Robert G Uzzo, Naomi B Haas, Timothy W Synold, Melissa Plets, Ulka N Vaishampayan, Brian M Shuch, Ian M Thompson, Primo N Lara
BACKGROUND: Patients undergoing resection of renal cell carcinoma are at risk of disease relapse. We evaluated the effectiveness of the mammalian target of rapamycin inhibitor everolimus administered after surgery. METHODS: In this randomised, double-blind, phase 3 trial, we enrolled adults with histologically confirmed renal cell carcinoma who had undergone a full surgical resection and were at intermediate-high or very high risk of recurrence at 398 academic and community institution centres in the USA...
September 23, 2023: Lancet
#76
JOURNAL ARTICLE
M Julia Machline-Carrion, Alysson Nathan Girotto, Josué Nieri, Pedro Marton Pereira, Frederico Monfardini, Francisco Forestiero, Priscila Raupp, Fabiana Roveda, Karla Santo, Otávio Berwanger, Raul D Santos
BACKGROUND: The digitization of the primary care system provides an opportunity to evaluate the current use of statins in secondary prevention populations (myocardial infarction or stroke). METHODS: We conducted a cross-sectional study (ClinicalTrials.gov, NCT05285085), analysing anonymised data routinely collected by community health workers (CHW) in Brazil between May 2016 and September 2021 to assess the proportion of self-reported statins use and associated factors...
July 2023: Lancet Reg Health Am
#77
JOURNAL ARTICLE
Christine Nyonator, Emefa Amoah, Etta Forson Addo, Maureen Mukanga, Augustine Kwabena Asubonteng, Kwaku Ohene-Frempong, Jonathan Spector Michael, Solomon Fiifi Ofori-Acquah
Novartis, a global medicines company, and the Sickle Cell Foundation of Ghana (SCFG), an advocacy organization, have endeavored to support the implementation of global best practices in the care of people living with sickle cell disease (SCD) in Africa, and to address unmet needs relating to this condition on the continent. Beginning in 2019, a multifaceted SCD program was implemented in Ghana through a public-private partnership involving the government of Ghana, the SCFG, Novartis, and other partners. A key component of the program involved expanding the reach of hydroxyurea (HU), the only approved disease-modifying generic treatment for SCD, in ways that would promote sustainable access...
July 7, 2023: Seminars in Hematology
#78
JOURNAL ARTICLE
Pilar Brito-Zerón, Alejandra Flores-Chávez, Ildiko Fanny Horváth, Astrid Rasmussen, Xiaomei Li, Peter Olsson, Arjan Vissink, Roberta Priori, Berkan Armagan, Gabriela Hernandez-Molina, Sonja Praprotnik, Luca Quartuccio, Nevsun Inanç, Burcugül Özkızıltaş, Elena Bartoloni, Agata Sebastian, Vasco C Romão, Roser Solans, Sandra G Pasoto, Maureen Rischmueller, Carlos Galisteo, Yasunori Suzuki, Virginia Fernandes Moça Trevisani, Cecilia Fugmann, Andrés González-García, Francesco Carubbi, Ciprian Jurcut, Toshimasa Shimizu, Soledad Retamozo, Fabiola Atzeni, Benedikt Hofauer, Sheila Melchor-Díaz, Tamer Gheita, Miguel López-Dupla, Eva Fonseca-Aizpuru, Roberto Giacomelli, Marcos Vázquez, Sandra Consani, Miriam Akasbi, Hideki Nakamura, Antónia Szántó, A Darise Farris, Li Wang, Thomas Mandl, Angelica Gattamelata, Levent Kilic, Katja Perdan Pirkmajer, Kerem Abacar, Abdurrahman Tufan, Salvatore de Vita, Hendrika Bootsma, Manuel Ramos-Casals
BACKGROUND: What baseline predictors would be involved in mortality in people with primary Sjögren syndrome (SjS) remains uncertain. This study aimed to investigate the baseline characteristics collected at the time of diagnosis of SjS associated with mortality and to identify mortality risk factors for all-cause death and deaths related to systemic SjS activity measured by the ESSDAI score. METHODS: In this international, real-world, retrospective, cohort study, we retrospectively collected data from 27 countries on mortality and causes of death from the Big Data Sjögren Registry...
July 2023: EClinicalMedicine
#79
JOURNAL ARTICLE
Reinaldo Guimarães
Rare or orphan diseases have played an important role in the pharmaceutical industry. On the other hand, the impact of new technologies derived from genomic research has been growing in this industry, with new drugs being launched on the market at unsustainable prices for health systems and patients. This double tendency poses important and growing challenges to public policies on Health Technology Assessment, whose hegemonic rationale is based on cost-benefit analysis between therapies. The very high prices of these drugs require revisiting this rationale and the recent negotiations between the Brazilian Ministry of Health and Novartis regarding a possible risk-sharing agreement for the incorporation of the drug Zolgensma is an opportunity for this revisitation...
July 2023: Ciência & Saúde Coletiva
#80
Qi Wei, Prof Philip J Mease, Michael Chiorean, Lulu Iles-Shih, Wanessa F Matos, Andrew Baumgartner, Sevda Molani, Yeon Mi Hwang, Basazin Belhu, Alexandra Ralevski, Jennifer Hadlock
BACKGROUND: COVID-19 outcomes, in the context of immune-mediated inflammatory diseases (IMIDs), are incompletely understood. Reported outcomes vary considerably depending on the patient population studied. It is essential to analyse data for a large population, while considering the effects of the pandemic time period, comorbidities, long term use of immunomodulatory medications (IMMs), and vaccination status. METHODS: In this retrospective case-control study, patients of all ages with IMIDs were identified from a large U...
June 28, 2023: medRxiv
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