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Psychometric validation of the Cystic Fibrosis Impact Questionnaire (CF-IQ): A patient-reported outcome assessing impacts of cystic fibrosis.
PLoS One
January 2025
INSERM U1151, Université Paris Cité, Centre de Références Maladies Rares Mucoviscidose et Maladies Apparentées, Hôpital Necker Enfants Malades, Paris, France.
The Cystic Fibrosis (CF) Impact Questionnaire (CF-IQ) was qualitatively developed to assess the impact of CF in the context of treatment advancements and increased longevity. This study reports the CF-IQ validation. In this noninterventional validation study, people with CF completed the 40-item CF-IQ and validating patient-reported outcome measures (PROMs) via electronic diaries at enrollment (baseline) and at the 4-week follow-up.
View Article and Find Full Text PDFPhase 2 Randomised Study of Bulevirtide as Monotherapy or Combined With Peg-IFNα-2a as Treatment for Chronic Hepatitis Delta.
Liver Int
February 2025
Department of Gastroenterology, Hepatology, Endocrinology, and Infectious Diseases, Hannover Medical School, Hannover, Germany.
Background And Aim: Bulevirtide (BLV) leads to beneficial virologic and biochemical responses when given alone to treat hepatitis delta virus (HDV) infection, which causes the most severe form of chronic viral hepatitis. We evaluated 48 weeks of BLV monotherapy, BLV + tenofovir disoproxil fumarate (TDF) and BLV + pegylated interferon alfa-2a (Peg-IFNα-2a), with 24-week follow-up.
Methods: Ninety patients were enrolled into six arms of 15 each (A-F); 60 patients were included in the main randomisation (arms A-D), and 30 patients (arms E-F) were randomised to the extension phase: (A) Peg-IFNα-2a 180 μg once weekly (QW); (B) BLV 2 mg once daily (QD) + Peg-IFNα-2a 180 μg QW; (C) BLV 5 mg QD + Peg-IFNα-2a 180 μg QW; (D) BLV 2 mg QD; (E) BLV 10 mg QD + Peg-IFNα-2a 180 μg QW and (F) BLV 10 mg (5 mg twice daily) + TDF QD.
Effects, Mechanisms of Action and Application of Vitex agnus-castus for Improvement of Health and Female Reproduction.
Phytother Res
January 2025
Constantine the Philosopher University in Nitra, Nitra, Slovak Republic.
This narrative review describes the provenance and chemical composition of Vitex agnus-castus, as well as the currently available knowledge concerning its action. To search the related articles, Cochrane Library, PubMed, Web of Science, SCOPUS databases between the years 1995 and 2024, and the keywords "Vitex," "review," "fertility," "ovarian" and "mechanisms" were used in various combinations. The data listed in this review demonstrate that Vitex agnus-castus and its constituents (isoflavones and essential oils) affect a number of physiological actions via multiple extra- and intracellular mechanisms of action.
View Article and Find Full Text PDFResearch progress on N6-methyladenosine RNA modification in osteosarcoma: functions, mechanisms, and potential clinical applications.
Med Oncol
January 2025
School of Basic Medicine, Gannan Medical University, Ganzhou, 341000, Jiangxi, China.
Osteosarcoma (OS) is the most commonly diagnosed primary malignant bone tumor in children and adolescents. Despite significant advancements in therapeutic strategies against OS over the past few decades, the prognosis for this disease remains poor, largely due to its high invasiveness and challenges associated with its treatment. N6-methyladenosine (m6A) modification is one of the most abundant epigenetic modifications of RNAs, and many studies have highlighted its crucial role in OS.
View Article and Find Full Text PDFSystemic Mastocytosis: Current Status and Challenges in 2024.
Blood Adv
January 2025
University of Michigan, Ann Arbor, Michigan, United States.
Systemic mastocytosis (SM) is a rare disease and has had significant discoveries in its biology, prognostication, and management in the past two decades. The latest update of the WHO classification and the new ICC classification are current standards in the diagnosis and prognostication of SM. In clinical practice, SM can be divided into two main categories: non-advanced SM (nonAdvSM) and advanced SM (AdvSM).
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