Real-World Patient Experience of Pexidartinib for Tenosynovial Giant-Cell Tumor.

BACKGROUND: Pexidartinib (Turalio) is the only systemic therapy approved by the FDA for the treatment of adult patients with symptomatic tenosynovial giant-cell tumor (TGCT) associated with severe morbidity or functional limitations, and not amenable to improvement with surgery. This study assessed patient-reported treatment experiences and symptom improvement among patients receiving pexidartinib. METHODS: A cross-sectional, web-based survey collected data on demographics, disease history, pexidartinib dosing, and symptoms before and after pexidartinib use. RESULTS: Of 288 patients enrolled in the Turalio REMS program in May 2021, 83 completed the survey: mean age was 44.2 years, 62.7% were female, and most common tumor sites were in knee (61%) and ankle (12%). Mean initial dose was 622 mg/day: 29 patients reported reduction from initial dose and 8 had dose reduction after titrating up to a higher dose. At the time of survey completion, median time on pexidartinib was 6.0 months; 22 (26.5%) patients discontinued pexidartinib due to physician suggestion, abnormal laboratory results, side effect, or symptom improvement. Compared with before pexidartinib initiation, most patients reported improvement in overall TGCT symptom (78.3%) and physical function (77.2%) during pexidartinib treatment. Significant improvement was reported during pexidartinib treatment in worst stiffness numeric rating scale (NRS) (3.0 vs. 6.2, P < .05) and worst pain NRS (2.7 vs. 5.7, P < .05). CONCLUSION: Findings from this cross-sectional survey confirmed the benefit of pexidartinib in improving symptoms and functional outcomes among patients with symptomatic TGCTs from the patients' perspective. Future research is warranted to examine the long-term benefit and risk of pexidartinib.

Cost of early progression: patients with epidermal growth factor receptor mutated metastatic non-small-cell lung cancer.

Aim: Compare healthcare costs for patients with epidermal growth factor receptor mutated (EGFRm) metastatic non-small-cell lung cancer (mNSCLC) with and without progression and estimate costs of progression. Materials & methods: Retrospective claims analysis (2015-2020) from adults with EGFRm mNSCLC initiating EGFR tyrosine kinase inhibitors. Adjusted costs for 12 months were compared (with vs without progression) and cumulative costs for early versus late progression were predicted over 36 months. Results: A total of 228 patients with EGFRm mNSCLC were included. Patients with progression within 12 months incurred significantly higher total costs despite lower treatment costs (vs without progression). Medical costs were significantly higher among early versus late progressors. Conclusion: These data may aid providers aiming to administer quality care in a cost-efficient way.

Lung cancer is the leading cause of cancer death among both men and women in the US. Among US patients with adenocarcinoma histology, approximately 17% have epidermal growth factor activating mutations (EGFRm) that include exon 19 deletions or L858R mutations. These common mutations make up approximately 85% of all EGFR mutations. The aim of this study was to compare healthcare resource utilization and costs for patients with EGFRm metastatic non-small-cell lung cancer with and without disease progression within the first 12 months following first-line treatment initiation using data from insurance claims. The results suggest that patients with EGFRm metastatic non-small-cell lung cancer with disease progression in the first 12 months (after treatment initiation) have significantly higher costs compared with patients without disease progression in the first 12 months (and highest in the first 6 months). These data may help inform oncology providers aiming to administer high quality cancer care in a cost-efficient way.

Treatment patterns and outcomes among hospitalized patients with venous thromboembolism in the United States: an analysis of electronic health records data.

BACKGROUND: With the advent of new treatment options for venous thromboembolism (VTE), it is valuable to gain insights into current clinical practices. OBJECTIVE: Assess treatment patterns and recurrence among patients hospitalized for VTE. METHODS: This retrospective study evaluated patients hospitalized with an incident VTE diagnosis (index) from 2008 to 2012 in a de-identified electronic health record database. Patients were further required to receive anticoagulant treatment and/or a VTE-related procedure for study inclusion. Patients were excluded if they: (1) did not have a medical encounter in the 6 months before index (baseline); (2) had a prior VTE diagnosis or used an anticoagulant during the baseline period; or (3) had a diagnosis of atrial fibrillation/flutter, cardiomyopathy, or a coagulation disorder during baseline or the year after index (follow-up). Hospitalization for recurrent VTE and bleeding were evaluated. RESULTS: A total of 2060 patients were identified (mean age, 60.9 years; 53.0% women), with a mean length of stay of 8.1 days. Of the VTE types, acute DVT was the most common (41.9%), followed by PE (33.3%), and DVT + PE (24.7%). Almost all patients (96.9%) received anticoagulants, of which 94.3% received heparin and 76.5% received warfarin. Although 77.4% of warfarin users were prescribed it at discharge, only (40.2%) had a warfarin prescription within 30 days of discharge. Overall 30 day, 90 day and 1-year VTE recurrence rates were 2.0%, 4.2%, and 7.5%, respectively, and the major bleeding rate was 6.8%. CONCLUSION: In a real-world population of hospitalized VTE patients, heparin treatment in combination with warfarin was common. However, continuation of warfarin post-discharge was challenging. Initiatives to improve continuation of therapy may be important to reduce VTE recurrence.