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Information about ongoing health services research and public health projects
| The myelodysplastic syndromes: impact of modern treatments by disease risk | |
|---|---|
| Investigator (PI): | Abel, Gregory A |
| Performing Organization (PO): |
(Current): Dana-Farber Cancer Institute / (617) 632-3000 |
| Supporting Agency (SA): | American Cancer Society |
| Initial Year: | 2015 |
| Final Year: | 2017 |
| Record Source/Award ID: | ACS/RSG-14-163-01 |
| OTHER ID: |
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| Funding: | Total Award Amount: $594,000 |
| Award Type: | Grant |
| Abstract: | Up to 50,000 people are diagnosed with myelodysplastic syndromes (MDS) each year in the United States, making MDS one of the most common hematologic malignancies. MDS can also develop as a result of treatment for prior cancers, such that all patients who have undergone chemotherapy or radiation are at risk. The disease, which causes progressively lower blood counts, can be clinically devastating, as patients who do not succumb to infections or bleeding often develop acute myeloid leukemia (AML). MDS has traditionally been managed with supportive care and blood transfusions, but in the mid 2000s, four new potentially disease-modifying medications were approved by the FDA: azacitidine, lenalidomide, deferasirox, and decitabine. Despite these revolutionary advances for a hitherto largely untreatable condition, little is known about the effectiveness of these agents in the "real world" of clinical practice, and physicians must act in conditions of uncertainty when deciding which agents to use and when, especially because MDS can vary widely in its aggressiveness. Our objective to is compare the effectiveness of these new MDS therapies--taking into account baseline disease aggressiveness--by capitalizing on resources to undertake such investigations that are now maturing. First, MDS became reportable to the National Cancer Institute's Surveillance Epidemiology and End Results (SEER) Program in 2001, and the eleven years of cases available, when linked with Medicare insurance claims (SEER-Medicare), represent an unparalleled opportunity to study well over 14,000 patients. Second, since 2006, we have captured diagnostic and treatment information on every MDS patient seen at our institution in the Dana-Farber MDS Clinical Research Information System (DF/MDS CRIS). This highly detailed source of data on approximately 500 MDS patients will be extremely useful to help validate the SEER-Medicare analyses, and also to fill in knowledge gaps that arise from our use of claims data. Using the two datasets, we will first characterize the influence of patient, provider, and institutional variables on choice of MDS treatment. Analyses will be performed in subgroups determined by a score for baseline MDS aggressiveness that we have created specifically for use with SEER-Medicare data, the "SEER-Medicare MDS Risk Score" (SMMRS). Next, utilizing the above datasets, we will perform several analyses to assess the effectiveness of these treatments in increasing overall survival, reducing transfusion dependence, limiting progression to AML, and reducing hospitalizations. Effects will again be assessed in disease risk subgroups using the SMMRS. Finally, we will also collect and analyze pilot data as to how each treatment affects MDS-specific quality of life using a new measure we have recently developed and validated (the "QUALMS-1"). Ultimately, we expect the results of our analyses will allow clinicians to make better choices when treating MDS patients, and also provide sorely needed data to inform MDS-related health care policy in the United States. |
| MeSH Terms: |
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| Country: | United States |
| State: | Massachusetts |
| Zip Code: | 02215 |
| UI: | 20163001 |
| Project Status: | Completed |