The kinase inhibitor ruxolitinib (Jakafi) is now approved to treat chronic graft-vs-host disease.
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- Ruxolitinib cream, shown to be safe and effective in a phase 3 study, has demonstrated its anti-inflammatory and itch-relieving properties in children aged 2-11 with mild to moderate atopic dermatitis (AD).
- The study focused on assessing safety, tolerability, pharmacokinetics, and quality of life while using the cream at maximum levels over longer periods, with 29 children participating and applying the cream twice daily for 4 weeks, followed by individualized usage.
- Results indicated that 31% of participants experienced treatment-related adverse events, but no severe health issues arose, and significant improvements in skin condition and quality of life were sustained for up to 52 weeks.
Article Synopsis
- Atopic dermatitis (AD) significantly affects patients' quality of life, causing itching, skin pain, and sleep disturbances; ruxolitinib cream has shown effectiveness in treating these symptoms in adults and adolescents through two phase III clinical trials.
- In the TRuE-AD studies, patients applied different strengths of ruxolitinib cream or a vehicle cream, with results indicating that those using ruxolitinib experienced quick relief from symptoms like skin pain and sleep issues within hours or weeks of application.
- Analysis of patient-reported outcomes revealed notable improvements in overall quality of life and symptom burden after two weeks of using ruxolitinib cream compared to the vehicle, with sustained benefits observed throughout the study periods.
JAK inhibitors to treat STAT3 gain-of-function: a single-center report and literature review.
Front Immunol
September 2024
Department of Rheumatology and Immunology, Hannover Medical School, Hannover, Germany.
Article Synopsis
- STAT3 gain-of-function syndrome is an immune disorder that results in various immune system issues, often requiring immunomodulatory treatments like blocking interleukin-6 receptors or using JAK inhibitors (JAKi).
- A study at Hannover University involved seven patients with STAT3-GOF, where five were treated with JAK inhibitors, leading to positive outcomes for many autoimmune and inflammatory symptoms.
- Overall, JAKi treatment showed significant improvement for the majority of patients, but more long-term studies are necessary to understand the risks associated with extended use.
Analysis of publicly available adverse events reported for pediatric patients treated with Janus kinase inhibitors.
Pediatr Dermatol
November 2024
Department of Dermatology and Cutaneous Biology, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.
Janus kinase inhibitors (JAKi) are drugs that block tyrosine kinases responsible for transducing cytokine signals. The first JAKi was approved by the US Food and Drug Administration (FDA) in 2011 to treat rheumatoid arthritis in adults. A pediatric indication was not approved until 8 years later, for acute graft-versus-host disease.
View Article and Find Full Text PDFThe evolving landscape of polycythemia vera therapies.
Expert Opin Pharmacother
August 2024
Department of Internal Medicine, Division of Hematology & Medical Oncology, Tisch Cancer Institute/Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Introduction: The treatment landscape of polycythemia vera (PV) has seen major advancements within the last decade including approval of ruxolitinib in the second line setting after hydroxyurea, ropegylated interferon-α2b, and advanced clinical development of a novel class of agents called hepcidin mimetics.
Areas Covered: We provide a comprehensive review of the evidence discussing the risk stratification, treatment indications, role and limitations of phlebotomy only approach and pivotal trials covering nuances related to the use of interferon-α (IFN-α), ruxolitinib, hepcidin mimetics, and upcoming investigational agents including HDAC and LSD1 inhibitors.
Expert Opinion: The research paradigm in PV is slowly shifting from the sole focus on hematocrit control and moving toward disease modification.
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