Publications by authors named "P Schafhausen"
Background: Although complement component 5 inhibitors (C5is) eculizumab and ravulizumab improve paroxysmal nocturnal hemoglobinuria (PNH) outcomes, patients may experience persistent anemia. This post hoc analysis investigated whether the complement component 3-targeted therapy pegcetacoplan also improved hematologic outcomes and reduced fatigue in patients with PNH and mild/moderate anemia.
Methods: Patients with PNH and hemoglobin ≥10.
Article Synopsis
- Iptacopan, an oral factor B inhibitor, shows promise in treating paroxysmal nocturnal hemoglobinuria patients suffering from persistent hemolytic anemia, especially those not responding to anti-C5 therapy.
- In two phase 3 trials, iptacopan significantly improved hemoglobin levels in patients with low baseline hemoglobin (under 10 g/dL), with many experiencing increases of at least 2 g/dL without needing blood transfusions.
- The results revealed that 85% of patients in the first trial and nearly all in the second trial experienced a notable increase in hemoglobin levels, leading to reduced fatigue and dependency on transfusions.
Article Synopsis
- Head and neck squamous cell carcinoma (HNSCC) is a common cancer, and over 50% of patients experience recurrent or metastatic disease despite aggressive treatments, highlighting the need for better therapies.
- The FOCUS trial seeks to compare the effectiveness of the UV1 cancer vaccine combined with pembrolizumab to pembrolizumab alone in treating patients with recurrent or metastatic HNSCC and PD-L1 expression.
- The study adheres to ethical guidelines, aiming to publish findings in scientific journals and at conferences, as it explores new treatment strategies for patients who cannot tolerate traditional chemotherapy.
BACKGROUND: Indolent systemic mastocytosis (ISM) is a clonal mast-cell disease driven by the KIT D816V mutation. We assessed the efficacy and safety of avapritinib versus placebo, both with best supportive care, in patients with ISM. METHODS: We randomized patients with moderate to severe ISM (total symptom score [TSS] of ≥28; scores range from 0 to 110, with higher numbers indicating more severe symptoms) two to one to avapritinib 25 mg once daily (n=141) or placebo (n=71).
View Article and Find Full Text PDFInternational Consensus Statement on Allergy and Rhinology: Sinonasal Tumors.
Int Forum Allergy Rhinol
February 2024
Article Synopsis
- Sinonasal neoplasms (both benign and malignant) are complex issues for clinicians, highlighting the need for collaboration to improve patient care, as presented in the International Consensus Statement on Allergy and Rhinology: Sinonasal Tumors (ICSNT).
- The ICSNT document organizes findings into four main sections: general principles, benign neoplasms, malignant neoplasms, and quality of life, covering 48 specific topics and providing evidence-based recommendations and summaries based on their rigor.
- This comprehensive document reflects a collective effort from an international team to advance understanding and intervention methods for sinonasal neoplasms, while also identifying future research opportunities.