ABSTRACT
BACKGROUND: Several publications reported on the safety and efficacy of bortezomib, a novel first-in-class anti-cancer agent, in the treatment of relapsed/refractory multiple myeloma in controlled clinical trials. Complementary data on the experience with bortezomib in daily oncology practice are needed. We report on the use of bortezomib to treat patients with relapsed/refractory multiple myeloma in routine clinical practice. METHODS: Patients were treated at investigators' discretion within a Compassionate Use Program, allowing third-line or subsequent treatment. Post-hoc safety and efficacy analysis of patient records was performed using predefined data capture forms. Marker response was defined as a decrease in paraprotein level of at least 25% as compared to baseline. RESULTS: Eighty-eight patients entered the program involving 62 oncologists/ haematologists (62% institutional). Median patient age was 66 years [44-86], median time since diagnosis 4 years [0.5-14], median number of previous treatments 3 [2-6]. Marker response was observed in 61% of patients (17% CR/nCR). At the time of data collection, median 4.75 months [0.5-13] after last bortezomib injection, overall median response duration was 4.5 months [1.5-22], and response was ongoing in 45% of patients. The most frequently reported adverse events were thrombocytopenia (34%), peripheral neuropathy (31%), diarrhoea (20%) and fatigue (19%). Among the cases of peripheral neuropathy, 68% were due to aggravation of a pre-existing condition and the remaining cases to onset under bortezomib. No cases of bortezomib-related haemorrhage were reported. CONCLUSIONS: Our results in daily oncology practice confirm findings from clinical trials, demonstrating high response rates and predictable adverse events in patients with relapsed/refractory multiple myeloma treated with bortezomib.
