Treatments And Outcomes For Patients With Myelodysplastic Syndromes (Mds) By Revised International Prognostic Scoring System (Ipss-R) Scores At The Huntsman Cancer Institute (Hci)

Context: IPSS-R is used to classify risk of disease progression and guide treatment decisions for patients with MDS. Recent data shows mutational profiling may improve the prognostic stratification of MDS. Minimal real-world evidence exists for this population. Objective: Assess treatment patterns and clinical outcomes in patients with MDS by IPSS-R risk scores. Design: A retrospective cohort study assessed real-world, patient-level data from adults diagnosed with MDS between 2010–2019 at HCI. All data were obtained from electronic medical records via chart review. Patients with intermediate to very-high IPSS-R scores comprised the higher-risk cohort. Results: Of 259 patients with MDS, 90 had an IPSS-R score at diagnosis, 65 patients who were not clinical trial participants were included. IPSS-R score distribution: 10/65 (15%) very low; 18/65 (28%) low, 14/65 (22%) intermediate, 15/65 (23%) high, and 8/65 (12%) very high. Average patient age was 68 years, 52% (n=34) of patients were female, 91% (n=59) were White, 8% (n=5) had autoimmune disorders, and 5% (n=3) had cerebrovascular disease. In the higher-risk cohort, hypomethylating agents (HMAs) were used to treat 62% (n=23) of patients (median 2 cycles), while 27% (n=10) received no MDS-directed therapy and 11% (n=4) received other MDS-related medications. Stem-cell transplant (SCT) was received more frequently (n=13, 35%) and sooner (median 4.8 months from diagnosis) by higher-risk patients compared with lower-risk patients (18%, n=5; median 15.0 months from diagnosis). Complete (CR) or partial response was achieved by 17% (n=4) of higher-risk patients treated with HMAs versus 7% (n=1) of lower-risk patients. Transfusion independence was achieved by 60% (n=3) of higher-risk and 100% (n=4) of lower-risk patients who received PLT or pRBC prior to treatment initiation. Disease progression or death occurred in 82% (n=53) of patients during the study period, with a median progression-free survival of 8.3 months and 25.3 months for higher-risk and lower-risk patients, respectively. Conclusions: These results show that higher-risk patients have a low likelihood of achieving and maintaining CR. Limited treatment options for patients with higher-risk MDS reveals a large unmet need. Specific genetic profiles may indicate the need for more aggressive treatments and management of relevant comorbidities.