PB2190 THE IMPACT OF ENROLLMENT IN CLINICAL TRIALS OF PATIENTS WITH MULTIPLE MYELOMA
Authors: A. Shacham Abulafia, S. Shemesh, O. Pasvolsky, J. Vaxman, P. Raanani, U. Rozovski
Journal: HemaSphere (2019)

Background: Clinical trials provide access to drugs that are not available in routine practice. Doctors often see the bright side of clinical trials and advocate patients to participate, often based on a dim promise for a closer and tighter monitoring. This ’imaginary’ advantage is often referred to as a “trial effect”. Others point to less relevant interests that drive doctors to promote participation of patients in clinical trials. Whether “trial effect” is present in the oncological setting is debatable. For example, one study found a survival benefit for patients with glioblastoma that participated in clinical trials and randomized to the control arm, compared with patients who received the same treatment outside clinical trials. On the other hand, patients with advanced stage non-small cell lung carcinoma had better overall quality of care but this was not translated into a longer survival. Likewise, the outcome of patients treated for ovarian cancer in a large tertiary center was similar regardless of whether they received treatment within our outside clinical trials. Whether “trial effect” benefits patients with multiple myeloma (MM) has not been yet tested. Aims: We sought to study whether participation in clinical trials benefits our patients. Methods In this single center retrospective study, the medical records of all patients with MM diagnosed between the years 2007 and 2012 were reviewed. Patients above the age of 18 years who participated in randomized double-blind clinical trials and were assigned to the standard care arm were included in this analysis. For all participant patients, we searched our multiple myeloma database and matched non-participant patients based on age (+ 3 years) and date of diagnosis (+ 3 years) whom we followed for at least 12 months. Results: Between 2008 and 2012 we identified 10 patients with MM that participated in 3 different clinical trials and were randomized to the control arm. The median age at diagnosis was 68 years (range 51 to 85) and 16 (41%) were males. For each of these patients we matched between 1 to 8 patients based on age and date of diagnosis. The median follow-up period for the entire cohort was 61 months (range 12 to 115). It was 80 months (range: 35 to 114) for study participants but only 52 months for non-participants (range: 12 to 115) (P = 0.037). Most patients (9/10, 90%) were recruited at relapsed/refractory disease. Patients in both arms typically received bortezomib-based treatments in first line. In second line, more patients that participated in clinical trials received lenalidomide based-treatment (P = 0.03). The number of previous treatments was similar in the two groups. The estimated median OS for patients who participated in clinical trials was not reached and was 52 months (CI: 38 to 66 months) in non-participants (P =0.037, Figure 1). Summary/Conclusion: During the follow up visits only 10 patients with MM who participated in clinical trials were randomized to the “standard of care” arm. Yet, this small sample demonstrated a survival advantage over patients who received similar treatments in “real world”. While selection bias can not be completely excluded our data suggest that patients with MM benefit from trial participation even if they receive standard treatment.