Real-World Outcomes of High-Risk Multiple Myeloma: Retrospective Analysis of Cams&pumc
Authors: Sui Weiwei , Shuhui Deng , Gang An , Xu Yan , Yi Shuhua , Dehui Zou , Qiu Lugui 
Published: October 2019

Abstract
OBJECTIVE:
To record and describe the current status of real world treatment and outcome of patients with high risk multiple myeloma by retrospective ,single-center, non-interventional, observational study
METHODS:
The data were collected from the CAMS&PUMC estimated real-world outcomes in high-risk multiple myeloma patients between January 2013 to June 2017.
RESULTS:
A total of 160 patients were enrolled. Of these patients, 1 case discontinued treatment with complications. 52 cases had not received standardized first-line treatment (9 cases of traffic inconvenience, 40 cases were unable to bear treatment costs, and 3 cases were refused consolidation / maintenance therapy after induction). In 107 patients received standardized treatment, the median age 56 years (31-77), 37 and 70 did and did not undergo stem cell transplantation (SCT) at any time during treatment, respectively. In these 37 patients, 4 patients received double auto stem cell transplantation(ASCT), 2 cases of allogeneic hematopoietic stem cell transplantation (allo-SCT), and the other 31 received single ASCT. Immunological subtype IgA was found in 21 cases (19.6%), IgG in 42 cases (39.3%), IgD in 18 cases (16.8%), and light chain in 23 cases (21.4%), non secretory 3 cases(2.8%).The proportion of IgD subtypes was significantly higher than that of MM patients in the same period (5.8%).Non parosteal extramedullary myeloma was found in 24 cases (22.4%), primary plasma cell leukemia in 12 cases (11.2%). 9.3%(10 cases) of the patients had received bortezomib-based therapy without thalidomide/lenalidomide, 8.4%(9 cases) had received thalidomide/lenalidomide-based therapy without bortezomib, and 82.2%(88 cases) had received bortezomib plus thalidomide/ lenalidomide-based therapy as frontline treatment, respectively. After a median follow-up of 22.31 months, the median disease-free survival (PFS) and overall survival (OS) were 28.52 and 48.10 months, respectively. Although there was no difference in OS between the treatment regimens, the SCT group had a better survival benefit than the non SCT group. The median PFS in the two group was 46.78 months vs 24.84 months (p=0.037), the SCT group had not reached the median OS, but the median OS in the non SCT group was 43.30 months, and the SCT group could obtain longer survival time than the non SCT group, p=0.012.However, the PFS of single, double and allo-SCT in the SCT group was 46.78, 19.32 and 54.51 months respectively (P single vs double = 0.770, P single vs allo = 0.547, P double vs allo = 0.317), and the three subgroups had not reached the median OS.
CONCLUSION:
The results of our study suggest that there are significant differences in the treatment regimen used in the real world for the therapy of high risk MM. About 1/3 of high-risk MM patients can not adhere to the standard treatment. Patients who received transplantation were only 34.6% of the patients in the standard treatment. Patients with high-risk MM who receive hematopoietic stem cell transplantation can obtain longer remission time and have significant survival benefits even in the novel drugs era. This diversity provides references for treatment recommendations. Moreover, our results provide real world data for clinical benefits.

Disclosures
No relevant conflicts of interest to declare.