Postapproval trials versus patient registries: comparability of advanced melanoma patients with brain metastases. Issue 1 (February 2021)

• Record Type: Journal Article
• Authors: Ismail, Rawa K.; Sikkes, Nienke O.; Wouters, Michel W.J.M.; Hilarius, Doranne L.; Pasmooij, Anna M.G.; van den Eertwegh, Alfonsus J.M.; Aarts, Maureen J.B.; van den Berkmortel, Franchette W.P.J.; Boers-Sonderen, Marye J.; de Groot, Jan Willem B.; Haanen, John B.A.G.; Hospers, Geke A.P.; Kapiteijn, Ellen; Piersma, Djura; van Rijn, Roos S.; Suijkerbuijk, Karijn P.M.; ten Tije, Bert-Jan; van der Veldt, Astrid A.M.; Vreugdenhil, Art; van Dartel, Maaike; de Boer, Anthonius
• Is Part Of: Melanoma research. Volume 31:Issue 1(2021)
• Publication Date: 2021-02
• Subjects: advanced melanoma -- clinical outcomes -- patient registries -- postapproval trials; Melanoma -- Periodicals; Melanoma -- Periodicals; Melanomen; 616.99477
• DOI: 10.1097/CMR.0000000000000707
• Languages: English
• ISSNs: 0960-8931

Abstract

Abstract : Postapproval trials and patient registries have their pros and cons in the generation of postapproval data. No direct comparison between clinical outcomes of these data sources currently exists for advanced melanoma patients. We aimed to investigate whether a patient registry can complement or even replace postapproval trials. Postapproval single-arm clinical trial data from the Medicines Evaluation Board and real-world data from the Dutch Melanoma Treatment Registry were used. The study population consisted of advanced melanoma patients with brain metastases treated with targeted therapies (BRAF- or BRAF-MEK inhibitors) in the first line. A Cox hazard regression model and a propensity score matching (PSM) model were used to compare the two patient populations. Compared to patients treated in postapproval trials ( n = 467), real-world patients ( n = 602) had significantly higher age, higher ECOG performance status, more often ≥3 organ involvement and more symptomatic brain metastases. Lactate dehydrogenase levels were similar between both groups. The unadjusted median overall survival (mOS) in postapproval clinical trial patients was 8.7 (95% CI, 8.1–10.4) months compared to 7.2 (95% CI, 6.5–7.7) months ( P < 0.01) in real-world patients. With the Cox hazard regression model, survival was adjusted for prognostic factors, which led to a statistically insignificant difference in mOS for trial and real-world patients of 8.7 (95% CI, 7.9–10.4) months compared to 7.3 (95% CI, 6.3–7.9) months, respectively. The PSM model resulted in 310 matched patients with similar survival ( P = 0.9). Clinical outcomes of both data sources were similar. Registries could be a complementary data source to postapproval clinical trials to establish information on clinical outcomes in specific subpopulations.