Validation of the revised International Prognostic Scoring System in treated patients with myelodysplastic syndromes

Abstract

The International Prognostic Scoring System (IPSS) was recently revised (IPSS-R) under the auspices of the MDS Foundation as a collaborative international effort to refine its prognostic power. Our purpose was to externally validate this new risk model using a large single-institution cohort, determine its prognostic power in patients receiving active treatment, and explore its utility in guiding therapeutic decisions. Data were collected retrospectively from our myelodysplastic syndrome (MDS) database and verified by chart review. Of the data available for 1,088 patients, 152 (14%), 353 (32%), 237 (22%), 190 (18%), and 156 (14%) patients were classified as very low, low, intermediate, high, and very high risk, respectively, with median overall survival (OS) of 90 (95%CI 71–109), 54 (95%CI 50–59), 34 (95%CI 26–43), 21 (95%CI 17–25), and 13 months (95%CI 11– 15), respectively (P < 0.005). We found that the IPSS-R further refined prognostic discrimination in all IPSS risk categories, particularly in the intermediate 1 and 2 groups. Among high and very high IPSS-R patients receiving azacitidine, OS was significantly improved versus patients not receiving azacitidine, with corresponding median OS of 25 versus 18 months (P = 0.028) and 15 versus 9 months (P = 0.005), respectively. Similarly, patients with IPSS-R high- and very high-risk disease who underwent allogeneic hematopoietic stem cell transplantation had significantly improved OS versus nontransplant approaches (P < 0.005). High and very high IPSS-R patients derived a survival advantage from disease-modifying therapies. Our data validate the prognostic value of the proposed IPSS-R and show that its refined IPSS prognostic discrimination can be applied to actively treated patients.

IPSS‐R in 555 Taiwanese patients with primary MDS: Integration of monosomal karyotype can better risk‐stratify the patients

“…In this study, we applied the IPSS-R to 555 patients with primary MDS in Taiwan. Compared with the previous reports, [4][5][6] fewer cases were in very low-and low-risk groups in this study (3.1% vs. 14-38% and 22.9% vs. 32-38%, respectively). In contrast, about one half of patients had high or very high-risk MDS in our cohort, compared with only 23-39% in the West [4][5][6].…”

“…Similarly, the IPSS-R could better discriminate patients with IPSS Int-2 into three distinct risk categories (P < 0.001). Similar findings were also reported by Greenberg et al [4] and Mishra et al [5]. The IPSS-R further refines the prognostic discrimination in MDS patients.…”

“…Recently, Mishra et al [5] demonstrated IPSS-R very high and high risk patients derived a survival advantages from disease-modifying agents. In short, IPSS-R is a useful predictor to help guide treatment in MDS patients.…”

“…Compared with IPSS, the IPSS-R split patients with fewer than 5% BM blast into those with 0%-2% and greater than 2% to fewer than 5%, incorporated more sophisticated cytogenetic classification to separate patients into five, rather than three risk subtypes, and scored the depth of cytopenias to improve prognosis assessment for primary MDS patients [4]. It has been shown that IPSS-R predicts survival and leukemic evolution in MDS patients significantly better than IPSS in Western countries [5][6][7][8]. Since the demographics and disease natures in MDS patients are different between Asian and Western countries [9][10][11][12], it is unclear whether this new risk model is similarly useful for risk stratification of MDS in Asia.…”

Myelodysplastic syndromes: Contemporary review and how we treat

“…In this regard, the IPSS-R has been validated in therapy-related MDS and patients having received disease-modifying therapy [59,60]. To overcome this limitation, in 2008, the MD Anderson group developed a very practical score that included both therapy-related MDS and patients previously treated with disease-modifying therapy [6].…”