The revised IPSS is a powerful tool to evaluate the outcome of MDS patients treated with azacitidine: the GFM experience.

The revised IPSS is a powerful tool to evaluate the outcome of MDS patients treated with azacitidine: the GFM experience The International Prognostic Scoring System (IPSS), which is based on cytogenetics, BM blast percentage, and number of cytopenias, has played a major role in prognosis assessment in myelodysplastic syndrome (MDS) since its publication in 1997. The recently published revised IPSS (IPSS-R), 1 which uses the same parameters as the original except for using 5 rather than 3 cytogenetic subgroups, 2 new cutoff values for cytopenias and BM blast percentages, and different weighing of parameters, refines the original IPSS prognostic value. However, like the original IPSS, the IPSS-R was also established in patients who had received no disease-modifying drugs. We previously reported that peripheral blast percentages, performance status, RBC transfusion requirement , and original IPSS cytogenetic risk independently predicted overall survival (OS) in 282 IPSS high-risk and intermediate 2–risk MDS patients treated with azacitidine (AZA) in a compassionate patient-named program. 3 In that series (Table 1), cytogenetics could be reclassified using IPSS-R cytogenetic groups in 265 patients: 1% very good, 37% good, 18% intermediate, 12% poor, and 32% very poor. the patients, respectively. IPSS-R risk score could be calculated in 259 patients and was found to be low in 1 patient, intermediate in 11%, high in 34%, and very high in 55%. The single patient in the low-risk group was excluded from further analysis. With the classic IPSS score, high-risk and intermediate 2–risk patients treated with AZA had significantly different responses (37% vs 49%, P ϭ .05) and OS (median 9.4 vs 16 months, P ϭ .004). Using the IPSS-R, 46%, 47%, and 39% of patients responded (complete response, partial response, or hematologic improvement) to AZA in the intermediate-, poor-, and very-poor-risk groups, respectively (P ϭ .463). Individual IPSS-R parameters, including cytogenetics (P ϭ .646), hemoglobin (P ϭ .948), plate-lets (P ϭ .10), absolute neutrophil count (P ϭ .465), and BM blast percentage stratified according to IPSS-R (P ϭ .287) had no significant impact on response (Table 1). The 55% of patients with very-high-risk IPSS-R scores could be further subdivided by our AZA scoring system 3 into 3%, 67%, and 30% low-, intermediate-, and high-risk patients with significantly different OS (median not reached at 12.7 and 5.9 months, respectively, P Ͻ 10 Ϫ4). Similarly, the 34% of patients with high-risk IPSS-R scores could be further subdivided by our AZA scoring system …