P-169: Outcome of non-transplant eligible patients with newly diagnosed multiple myeloma depending on the eligibility for clinical trials: a single institution experience

Abstract

<h3>Background</h3> Patient participation in clinical trials forms the backbone of cancer clinical research as they represent a key step in the development of new treatment strategies to improve the outcome of incurable diseases such as multiple myeloma (MM). Although the results obtained from them will determine the future therapeutic approaches due to several reasons, the vast majority of patients with MM do not participate in clinical trials. <h3>Methods</h3> To determine the reasons they were or weren't recruited into prospective clinical trials and evaluate their outcome in one group or the other, we retrospectively reviewed from our database all consecutive patients with newly diagnosed MM, non-candidate to autologous stem cell transplantation, who received first line treatment at our institution from 2003 to 2017. We thoroughly reviewed their characteristics and their outcome after first line treatment only. We analyzed the reasons why they were or not included in a trial and compared the outcome of those included in a clinical trial versus those who did not meet the eligibility criteria and who received standard therapy. Patients who didn't have a clinical trial available at the time of diagnosis were excluded from the analysis. <h3>Results</h3> Between January 2003 and December 2017 we analyzed 211 patients diagnosed and treated at our institution; 105 entered a clinical trial and 106 did not meet the eligibility criteria. The causes for not entering in a clinical trial were as: 1) Didn't fulfill inclusion criteria due to comorbidities (26.7%), other previous malignancies (16.2%) or renal insufficiency (13.3%); 2) Didn't have measurable disease (1.9%); 3) The urgency to start treatment didn't allow the delay of a screening period (8.6%); 4) Very advanced age (10.5%), cognitive impairment (1.9%) or performance status (4.85%); 5) Patient refusal (10.5%). Finally 1.9% were screen failures and 3.8% did not participate for unknown reasons. Patients included in clinical trials were significantly younger (median 71 vs. 78 p<0.001) and had better ECOG (0/1 78.4% vs. 35.6 p<0.001). No differences regarding immunological subtype or bone marrow plasma cells infiltration were found. ISS stage was slightly lower in the clinical trial group although not statistically significant. The ORR was 79 vs.46 p<0.001 and the CR rate was 17% vs. 3% p<0.001 both significantly higher in the clinical trial group. Patients included in clinical trial had a significantly longer OS than those who were not (median 62.5 vs.31.8 p<0.001). <h3>Conclusion</h3> Patients included in clinical trials have a significantly higher response rate and OS than those who do not meet the eligibility criteria. Only half of the elderly patients at our institution fulfill the inclusion criteria to enter a clinical trial. This questions the extrapolation of the results of clinical studies to broader populations.