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.

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.