ASH 2023: Using VALIDATE Database Results to Inform MDS Management

At ASH 2023, we had the opportunity to interview David Sallman, MD, from Moffitt Cancer Center. The interview refers to the abstract “Validation of the Composite Complete Response (cCR) Definitions in the International Working Group (IWG) 2023 Criteria in Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) Treated with Hypomethylating Agents (HMA) – a Large, Multicenter, Retrospective Analysis from the Validate Database”.  Watch below or continue reading here to learn more about this exciting study.

“We are looking at very big and central questions and the management of MDS patients, trying to identify and validate, for example, response criteria outcomes in certain patient populations to really give us the best historical subset when we’re looking at new therapies,” said Dr. David Salliman to our interviewer, “I think when we have really small cohorts, this can be very challenging, and you get sometimes controversial or conflicting data.” 

An enriched, validated database can contribute to more accurate data and, therefore, more opportunities for researching a tailor-made therapy for each patient.

From the abstract, the VALIDATE database includes high-risk MDS patients treated with hypomethylating agent-based therapies in frontline settings from 14 specialized centers. Best responses were assessed based on IWG 2006 and 2023 criteria. An independent research grant from AbbVie supported this study. 

The study showed the following results: of 1,223 patients in the VALIDATE database, 629 met eligibility. The median age was 68 years, 27.7% and 27.4% were red blood cell and 27.4% were platelet transfusion-dependent, respectively; 38.1% had TP53 mutations, and 45.3% had allogeneic transplantation (allo-HCT). Most patients (71.6%) received hypomethylating agent monotherapy (51.7% azacitidine, 19.9% decitabine), while 28.4% received hypomethylating agent-based combinations. The median duration of therapy was four cycles (Range: 1-94).

In this real-world analysis of high-risk patients treated with hypomethylating agent-based therapy, clinical complete remission, according to IWG 2023, was associated with improved overall survival. In particular, complete remission and complete remission with limited hematologic recovery (including complete remission with bilineage recovery and complete remission with unilineage recovery) were associated with improved overall survival, supporting their inclusion in the overall response rate in clinical trials.