Pomalidomide is a third generation immunomodulatory drug in the treatment of refractory
and relapsed multiple myeloma patients. Our aim was to investigate the efficacy and
safety of pomalidomide therapy in a real world setting. Eighty-six Hungarian patients
were included, 45 of whom received pomalidomide ± an alkylating agent, while in 38
of them pomalidomide was combined with a proteasome inhibitor. 56 patients (65%) showed
any response to the treatment with 18 complete or very good partial remissions and
38 partial remissions. At a median duration of follow-up of 18.6 months, the median
progression-free survival (PFS) was 9.03 months, while the median overall survival
(OS) was 16.53 months in the whole cohort. Patients with early stage disease (R-ISS
1 and 2) had better survival results than those with stage 3 myeloma ( p = 0.002).
Neither the number of prior treatment lines, nor lenalidomide refractoriness had a
significant impact on PFS . PFS was found similar between the cohort of patients with
impaired renal function and the cohort without kidney involvement. During the study,
eight mortal infections and two fatal bleeding complications occurred, however, mild
hematologic and gastrointestinal toxicities were identified as the most frequent adverse
events. The results of our investigations confirm that pomalidomide is an effective
treatment option for relapsed/refractory MM, besides, the safety profile is satisfactory
in subjects with both normal and impaired renal function.