Yttrium-90 Transarterial Radioembolization for Chemotherapy-Refractory Intrahepatic Cholangiocarcinoma: A Prospective, Observational Study.

PURPOSE: To evaluate the safety and efficacy of yttrium-90 transarterial radioembolization (TARE) for the treatment of unresectable, chemotherapy-refractory intrahepatic cholangiocarcinoma (ICC). METHODS: A prospective, observational study was carried out in 10 centers between 2013 and 2017. TARE plus standard care was delivered to patients with unresectable, chemotherapy-refractory or chemotherapy-intolerant ICC. Primary outcome was overall survival. Secondary outcomes included safety, progression-free survival (PFS), and liver-specific progression-free survival (LPFS). RESULTS: Sixty-one patients were treated with TARE. Patients were 53% male; median age was 64 years; 91% had performance status 0/1; 92% had received prior chemotherapy; and 59% had no extrahepatic disease. Median follow-up was 13.9 months (95% confidence interval [CI], 9.6-18.1). Overall survival was 8.7 months (95% CI, 5.3-12.1), and 37% of patients survived to 12 months. PFS was 2.8 months (95% CI, 2.6-3.1), and LPFS was 3.1 months (95% CI, 1.3-4.8). One severe complication (abdominal pain) occurred at the time of the TARE procedure. Thirty patients experienced a total of 49 adverse events, of which 8% were grade ≥3; most common were grade 1-2 fatigue and abdominal pain. A total of 77 abnormal laboratory value events were recorded, of which 4% were grade ≥3. CONCLUSIONS: Patients with advanced ICC have limited therapeutic options and a poor prognosis. This prospective study examined the survival of patients with unresectable, chemotherapy-refractory primary ICC treated with TARE in real-world practice. The results demonstrate that this treatment merits further investigation in this patient cohort in a larger study, including collection of patient-reported outcomes.

QUALITY ASSURANCE OF REGISTRIES FOR HEALTH TECHNOLOGY ASSESSMENT.

OBJECTIVES: The aim of this study was to identify guidelines and assessment tools used by health technology agencies for quality assurance of registries and investigate the current use of registry data by HTA organizations worldwide. METHODS: As part of a European Network for Health Technology Assessment Joint Action work package, we undertook a literature search and sent a questionnaire to all partner organizations on the work package and all organizations listed in the International Society for Pharmaco-economics and Outcomes Research directory. RESULTS: We identified thirteen relevant documents relating to quality assurance of registries. We received fifty-five responses from organizations representing twenty-one different countries, a response rate of 40.5 percent (43/110). Many agencies, particularly in Europe, are already drawing on a range of registries to provide data for their HTA. Less than half, however, use criteria or standards to assess the quality of registry data. Nearly all criteria or standards in use have been internally defined by organizations rather than referring to those produced by an external body. A comparison of internal and external standards identified consistency in several quality dimensions, which can be used as a starting point for the development of a standardized tool. CONCLUSION: The use of registry data is more prevalent than expected, strengthening the need for a standardized registry quality assessment tool. A user-friendly tool developed in conjunction with stakeholders will support the consistent application of approved quality standards, and reassure critics who have traditionally considered registry data to be unreliable.