Contributions
Abstract: PB1625
Type: Publication Only
Session title: Myelodysplastic syndromes - Clinical
Background
IPSS-R is used to classify risk of disease progression & 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.
Aims
Primary objectives were to assess treatment patterns & clinical outcomes in patients with MDS by IPSS-R risk scores.
Methods
A retrospective cohort study assessed real-world, patient-level data from adults diagnosed with MDS between 2010-2019 at the Huntsman Cancer Institute (HCI), a NCCN, NCI-designated Comprehensive Cancer Center. All data were obtained via chart review from electronic medical records. IPSS-R scores were manually calculated from lab & cytogenetic data derived within 30 days of diagnosis. Patients with an intermediate to very-high IPSS-R score comprised the higher risk (HR) cohort; those with low or very low-risk IPSS-R comprised the lower risk (LR) cohort.
Results
Of the 259 MDS patients at HCI, 90 had an available IPSS-R score at diagnosis (ANC results were missing for 64% of cohort). After excluding clinical trial participants, 65 patients were included. Distribution of IPSS-R scores was: 15% (n=10) very low, 28% (n=18) low, 22% (n=14) intermediate, 23% (n=15) high, & 12% (n=8) very high. The average age of subjects was 68 years. 52% (n=34) of patients were female, 91% (n=59) were white, 8% (n=5) had autoimmune disorders, & 5% (n=3) had cerebrovascular disease. Twelve percent of NGS-tested patients had TP53 alterations (n=7), while the most frequently altered gene for both cohorts was DNMT3A (19%, n=11). In the HR cohort, HMAs were used to treat 62% (n=23) of patients (median 2 cycles), while 27% (n=10) received no MDS-directed therapy, & 11% (n=4) received other MDS-related medications. In the LR cohort HMAs were used to treat 54% (n=15) (median 5.5 cycles), 39% (n=11) received no MDS-related medications, & 7% (n=2) received lenalidomide. Second-line treatment was received by 8% (n=5) of patients across both cohorts. Stem-cell transplant (SCT) was received more frequently (n=13, 35%) & sooner (median 4.8 months from diagnosis) by HR patients compared to LR patients (18%, n=5; median 15.0 months from diagnosis). Complete or partial response was achieved by 17% (n=4) of HR patients treated with a HMA versus 7% (n=1) of LR patients treated with a HMA. Transfusion independence was achieved by 60% (n=3) & 100% (n=4) of HR & LR HMA-treated patients that received PLT or pRBC prior to treatment initiation, respectively. Disease progression or death occurred in 82% (n=53) of patients during the study period with a median progression free survival (PFS) of 8.3 months & 25.3 months for HR & LR patients, respectively. PFS was significantly shorter for patients with TP53 alterations compared to wild-type (HR: 3.07, 95% CI: 1.13-8.28), while controlling for IPSS-R using multivariable cox regression. Median overall survival was 18.8 & 31.0 months for HR & LR patients, respectively.
Conclusion
These results show that patients with MDS have a low likelihood of achieving & maintaining complete remission. In addition, few patients receive second-line therapy, revealing a large unmet need. Real world data are further limited by the reduction of patients with calculable IPSS-R scores at diagnosis. Specific genetic profiles may indicate the need for more aggressive treatments & management of relevant comorbidities. Updated results will be presented, including economic outcomes.
Keyword(s): MDS
Abstract: PB1625
Type: Publication Only
Session title: Myelodysplastic syndromes - Clinical
Background
IPSS-R is used to classify risk of disease progression & 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.
Aims
Primary objectives were to assess treatment patterns & clinical outcomes in patients with MDS by IPSS-R risk scores.
Methods
A retrospective cohort study assessed real-world, patient-level data from adults diagnosed with MDS between 2010-2019 at the Huntsman Cancer Institute (HCI), a NCCN, NCI-designated Comprehensive Cancer Center. All data were obtained via chart review from electronic medical records. IPSS-R scores were manually calculated from lab & cytogenetic data derived within 30 days of diagnosis. Patients with an intermediate to very-high IPSS-R score comprised the higher risk (HR) cohort; those with low or very low-risk IPSS-R comprised the lower risk (LR) cohort.
Results
Of the 259 MDS patients at HCI, 90 had an available IPSS-R score at diagnosis (ANC results were missing for 64% of cohort). After excluding clinical trial participants, 65 patients were included. Distribution of IPSS-R scores was: 15% (n=10) very low, 28% (n=18) low, 22% (n=14) intermediate, 23% (n=15) high, & 12% (n=8) very high. The average age of subjects was 68 years. 52% (n=34) of patients were female, 91% (n=59) were white, 8% (n=5) had autoimmune disorders, & 5% (n=3) had cerebrovascular disease. Twelve percent of NGS-tested patients had TP53 alterations (n=7), while the most frequently altered gene for both cohorts was DNMT3A (19%, n=11). In the HR cohort, HMAs were used to treat 62% (n=23) of patients (median 2 cycles), while 27% (n=10) received no MDS-directed therapy, & 11% (n=4) received other MDS-related medications. In the LR cohort HMAs were used to treat 54% (n=15) (median 5.5 cycles), 39% (n=11) received no MDS-related medications, & 7% (n=2) received lenalidomide. Second-line treatment was received by 8% (n=5) of patients across both cohorts. Stem-cell transplant (SCT) was received more frequently (n=13, 35%) & sooner (median 4.8 months from diagnosis) by HR patients compared to LR patients (18%, n=5; median 15.0 months from diagnosis). Complete or partial response was achieved by 17% (n=4) of HR patients treated with a HMA versus 7% (n=1) of LR patients treated with a HMA. Transfusion independence was achieved by 60% (n=3) & 100% (n=4) of HR & LR HMA-treated patients that received PLT or pRBC prior to treatment initiation, respectively. Disease progression or death occurred in 82% (n=53) of patients during the study period with a median progression free survival (PFS) of 8.3 months & 25.3 months for HR & LR patients, respectively. PFS was significantly shorter for patients with TP53 alterations compared to wild-type (HR: 3.07, 95% CI: 1.13-8.28), while controlling for IPSS-R using multivariable cox regression. Median overall survival was 18.8 & 31.0 months for HR & LR patients, respectively.
Conclusion
These results show that patients with MDS have a low likelihood of achieving & maintaining complete remission. In addition, few patients receive second-line therapy, revealing a large unmet need. Real world data are further limited by the reduction of patients with calculable IPSS-R scores at diagnosis. Specific genetic profiles may indicate the need for more aggressive treatments & management of relevant comorbidities. Updated results will be presented, including economic outcomes.
Keyword(s): MDS