Clinical presentation, pathological features and natural course of metastatic uveal melanoma (MUM) as an orphan and commonly fatal disease

Abstract

Abstract Purpose Uveal melanoma (UM) is a rare disease characterized by an unpredictable course and variable outcome ranging from cure by local treatment to the occurrence of untreatable metastasis. The current project is focused on the characteristics of the metastatic phenotype of the disease Methods We performed data collection from 76 pts with MUM treated in Leuven between 1957‐2008. Statistical analysis involved nonparametric technics, Kaplan Meyer and log rank test Results The median age at diagnosis of UM was 58 yrs (range 30‐94). Common initial treatments were surgery (71%), brachytherapy (20%) and external beam RT (7%). Synchronous metastasis was found in only 9% of cases, all others had metachronous disease after a median interval of 40 mo (range, 7‐420). Metastasis in >1 organ, was seen in 47% of cases. The most frequent metastatic site was the liver (96%), followed by lung, subcutaneous, bone and brain lesions. The median OS from diagnosis of UM was 46 months (range, 2‐182), and only 4,5 months in pts with MUM (range, 1‐128). 65% of MUM pts qualified for further treatment, The most common drugs given were DTIC, cisplatin, tamoxifen or phase I agents. Patient benefit (PR+SD) was seen in 16/45 pts (36%), including 2 PR Conclusion In this orphan disease with female predominance metastasis occurs late, is mainly found to the liver, and is associated with high morbidity, as >1/3 of pts do not qualify for further therapy. Advances in MUM can only be achieved by networking of sites interested in this tumour type with systematic collection of data and tissue to improve our understanding of the molecular biology of the disease