Mortality after deferral of treatment or no treatment for choroidal melanoma

Abstract

Purpose: To report mortality of patients who were eligible for enrollment in the Collaborative Ocular Melanoma Study (COMS) clinical trials of medium-sized choroidal melanoma or large-sized choroidal melanoma but chose to defer treatment or receive no melanoma treatment.

Design: Prospective nonrandomized multicenter cohort study as an adjunct to COMS randomized clinical trials.

Methods: Patient follow-up procedures included examinations, correspondence, telephone contacts, and National Death Index searches. Primary outcome was patient death measured by all-cause mortality. Secondary outcomes were melanoma treatment and melanoma metastasis.

Results: Of 77 patients eligible for COMS clinical trials who chose to defer or receive no melanoma treatment, 61 were appropriate candidates and 45 (74%) enrolled in the natural history study (NHS). In all, 42 patients (42 eyes) had medium melanoma, and the median follow-up was 5.3 years (range, 4-10.7 years). In all, 22 patients (52%) had subsequent melanoma treatment, and 20 (48%) had no melanoma treatment. For the 42 patients, Kaplan-Meier estimate of 5-year mortality was approximately 30% [95% confidence interval (CI), 18%-47%]. For COMS medium melanoma trial, 5-year mortality was 18% (95% CI, 16%-20%), not statistically significantly different from the NHS patients. After adjusting for differences in age and longest basal diameter, the 5-year risk of death for NHS patients versus COMS trial patients was 1.54 (95% CI, 0.93-2.56). Three patients had large melanoma. Melanoma metastasis was confirmed or suspected in 8 (42%) of 19 deaths.

Conclusion: Greater mortality and higher risk of death for NHS patients are probative but not conclusive evidence of a beneficial, life-extending effect of medium melanoma treatment.

Keywords: COMS; Choroidal melanoma; melanoma; mortality; uveal melanoma.

Figure 1

Natural history study. Cumulative proportion of patients with medium choroidal melanoma who died by time after determination of eligibility for Collaborative Ocular Melanoma Study medium melanoma trial; enrolled natural history study patients (n = 42) with 95% confidence interval (CI)

Figure 2

Natural history study and Collaborative Ocular Melanoma Study (COMS) medium melanoma trial. Cumulative proportion of patients with medium choroidal melanoma who died by time after determination of eligibility for COMS medium melanoma trial; enrolled COMS medium tumor trial patients (n = 1,317) and natural history study patients (n = 42) with 95% CI