Validation of International Working Group response criteria in higher ‐risk myelodysplastic syndromes: A report on behalf of the MDS Clinical Research Consortium

In higher risk MDS patients responses associated with restoration of effective hematopoiesis namely complete remission (CR) are associated with better outcomes.Marrow CR without hematological improvement outside context of allogeneic hematopoietic stem cell transplant should not be included in response criteria assessing new therapeutic agents in MDS. AbstractThe utility of the International Working Group (IWG) 2006 response criteria for myelodysplastic syndromes (MDS) as a surrogate endpoint for outcomes is unclear. We assessed the validity of the IWG 2006 response criteria in a large cohort of higher ‐risk MDS patients (pts) treated at centers from the MDS Clinical Research Consortium. The best overall response rate (ORR) by IWG 2006 criteria to first‐line therapy among 597 evaluable pts was 38% and include complete response (CR) 16%, marrow CR (mCR) 2%, partial response (PR) 10%, hematologi cal improvement (HI) 10%, stable disease (SD) 33%, and progressive disease (PD) 24%. CR was associated with a better overall survival (OS) compared to all other response groups (P <  0.001). Among 470 pts treated with hypomethylating agent (HMA) as first‐line therapy, the overall Response Rate, defined as HI or better was 39%. The median OS from time of best response was 21 mo, 8 mo, 14 mo, 12 mo, 13 mo, and 8 mo for CR, mCR, PR, HI, SD, and PD, respectively (P <  0.001). We validated those results in a separate cohort of 539 higher‐risk MDS pts treated at Moffitt Canc...
Source: Cancer Medicine - Category: Cancer & Oncology Authors: Tags: ORIGINAL RESEARCH Source Type: research