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Fedratinib for the treatment of myelofibrosis: a critical appraisal of clinical trial and "real-world" data.
Blood Cancer J
January 2025
School of Medicine, Faculty of Medicine and Health Sciences, Tel Aviv University, Tel Aviv, Israel.
Fedratinib is a predominantly JAK2 inhibitor that has shown efficacy in untreated and ruxolitinib-exposed patients with myelofibrosis (MF). Based on randomized clinical trial data, it is approved for use in patients with International Prognostic Scoring System (IPSS) or Dynamic International Prognostic Scoring System (DIPSS) intermediate-2 or high-risk disease and is distinguished from ruxolitinib in that it can be administered without dose reduction in patients with thrombocytopenia, to a platelet count above 50,000/µL. In these trials, fedratinib achieved significant spleen volume reduction in ~30-45% of patients and improvement in total symptom scores in 35-40% with good tolerability.
View Article and Find Full Text PDFPeficitinib suppresses diffuse-type tenosynovial giant cell tumor by targeting TYK2 and JAK/STAT signaling.
Sci China Life Sci
January 2025
Institute of Cardiovascular Sciences, School of Basic Medical Sciences, Peking University Health Science Center; Department of Cardiology and Institute of Vascular Medicine, Peking University Third Hospital; State Key Laboratory of Vascular Homeostasis and Remodeling, Peking University, Beijing, 100191, China.
Diffuse-type tenosynovial giant cell tumor (dTGCT) is a destructive but rare benign proliferative synovial neoplasm. Although surgery is currently the main treatment modality for dTGCT, the recurrence risk is up to 50%. Therefore, there is a great need for effective drugs against dTGCT with minor side effects.
View Article and Find Full Text PDFRisankizumab Efficacy and Safety Based on Prior Inadequate Response or Intolerance to Advanced Therapy: Post Hoc Analysis of the INSPIRE and COMMAND Phase 3 Studies.
J Crohns Colitis
January 2025
Department of Gastroenterology and Hepatology, University Hospitals Leuven, KU Leuven, Leuven, Belgium.
Background And Aims: Treating ulcerative colitis (UC) in patients with prior advanced therapy (AT) exposure may be challenging. We report the efficacy and safety of risankizumab, a monoclonal interleukin 23p19 antibody, in patients with UC and prior inadequate response or intolerance to AT (AT-IR).
Methods: In the 12-week phase 3 INSPIRE induction study, patients were randomized to intravenous risankizumab 1200 mg or placebo.
Evaluation of Real-World Evidence to Assess Effectiveness Outcomes of Janus Kinase Inhibitors for Rheumatoid Arthritis: A Systematic Review of US Studies.
Drug Healthc Patient Saf
January 2025
Department of Pharmacy Administration, University of Mississippi School of Pharmacy, University, MS, 38677 USA.
Objective: This review summarized the real-world effectiveness outcomes of Janus kinase inhibitors (JAKi) for rheumatoid arthritis (RA) based on observational studies.
Methods: A systematic review followed PRISMA guidelines, with searches conducted in PubMed, Embase, and CINAHL from each database's inception to June 2, 2023. Studies were included if they evaluated real-world effectiveness outcomes of JAKi for US RA patients.
Advances in Myelofibrosis Management: New Janus Kinase Inhibitors Beyond Ruxolitinib.
J Adv Pract Oncol
May 2024
Levine Cancer Institute, Atrium Health, Charlotte, North Carolina.
Myelofibrosis is a myeloproliferative neoplasm characterized by the buildup of fibrous scar tissue in the bone marrow occurring secondary to the secretion of inflammatory cytokines, leading to cytopenias, dysfunctional hematopoiesis, and constitutional symptoms. One of the pathologic mechanisms that underlies myelofibrosis is aberrant activation of the Janus kinase (JAK)-STAT pathway. Targeting the JAK-STAT pathway via JAK inhibition can lead to significant improvements in spleen volume reduction and symptom improvement in intermediate- and high-risk myelofibrosis.
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