Bridging the gap between clinical trials and real-world for advanced melanoma: Results of the Dutch Melanoma Treatment Registry

Zeijl, M.C.T. van (2023)

Doctoral Thesis

In this dissertation, new immune and targeted therapies for advanced melanoma were investigated in daily practice in the Netherlands.

From 2013 to 2017, new treatments were implemented fast and save with an increase of median survival. Increased LDH, distance metastases in ≥3 organsites, brain and liver metastases and ECOG performance score were associated with death. Survival of advanced mucosal melanoma lagged behind. Forty percent of patients receiving systemic treatment did not meet ≥1 selection criteria of clinical trials. In these patients, LDH was the most important prognostic factor, followed by the ECOG performance score and brain metastases. For anti-PD-1 antibody monotherapy, a median survival of 24 months was observed. A complete response or a shorter time to the first response were associated with disease controle when anti-PD-1 montherapy was discontinued in the absence of progression. Half of the patients treated with anti-CTLA-4 plus anti-PD-1 antibodies had a grade 3-4 adverse event, requiering hospital admission in 50%. On the other hand, 37% of patients reached disease control after 2 years, with a median survival of 28.7 months. This dissertation emphasizes that with new treatments there is an "knowledge-gap" between the clinical trials and real-world that should be bridged with real-world data.

• Advanced melanoma
• Real-world outcomes
• Targeted therapy
• Immune therapy
• Population-based
• Daily clinical practise

• All authors: Zeijl, M.C.T. van
• Supervisor: Haanen, J.B.A.G.; Eertwegh, A.J.M. van
• Co-supervisor: Wreede, L.C. de
• Committee: Wouters, M.W.J.M.; Uyl-de Groot, C.A.; Hage, J.A. van der; Blokx, W.A.M.
• Qualification: Doctor (dr.)
• Awarding Institution: Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
• Date: 2023-09-12
• ISBN (print): 9789464694581