Prognostic Role of Transfusion Requirement and Validation of the IPSS Prognostic System in Myelodisplastic Syndromes.
Authors: Salvi Flavia , Gioia Daniela , Cilloni Daniela , Audisio Ernesta , Bonferroni Margherita , Cametti Gianni , Darbesio Antonella , D’Ardia Stefano , Dellacasa Chiara , Ferrero Dario , Freilone Roberto , Gatto Simona , Franceschetti Silvia , Marinone Carlo , Marmont Filippo , Scassa Enzo , Tonso Anna , Boccadoro Mario , Gaidano Gianluca , Gallamini Andrea , Gallo Eugenio , Girotto Mario , Saglio Giuseppe , Levis Alessandro 
Published: October 2019

Abstract
BACKGROUND. The International Prognostic Scoring System (IPSS) of myelodysplastic syndromes (MDS) is based on bone marrow blast count, cytogenetic features and number of peripheral cytopenias. Transfusion requirement has been recently proposed as an important risk factor (Malcovati et al JCO23, 7594, 2005), and a new prognostic system (WPSS) has been suggested, based on WHO diagnostic subgroups, cytogenetic abnormalities and transfusion requirements (2005 ASH meeting abs.789). AIM OF THE WORK. To confirm the prognostic role of transfusion requirement and to compare the prognostic value of WPSS to that of IPSS.
PATIENTS AND METHODS. The Piedmont MDS register is active since 1999. Clinical and laboratory data of patients were centrally recorded through our web site. Transfusion requirement data were retrospectively obtained from blood banks when possible. IPSS was calculated according to Greenberg et al. (Blood 89, 2079Blood 89, 1997). WPSS was calculated according to Malcovati et al. (2005 ASH meeting abst 789) by summing the score values of the following three variables:
cytogenetic abnormalities scored according to the IPSS: 0 for good; 1 for intermediate; 2 for poor; transfusion requirement: 0 for absent; 1 for regular; WHO category: 0 for RA, RARS, 5q-, and unclassifiable; 1 for RCMD and RCMD-RS; 2 for RAEB-I; 3 for RAEB-II.
WPSS score values were stratified into the proposed five risk groups: very low (score 0), low (1); intermediate (2); high (3–4); very high (5–6).
RESULTS. From June 1999 to December 2004, 762 MDS patients were registered from 37 different institutions. Transfusion information was available for 376 patients. Data on both cytogenetics and transfusion requirements were available for 202 patients who are the object of the present analysis. 132 patients (65 %) needed regular transfusions, while the remaining 70 ones (35 %) did not. In univariate analyses all variables of both IPSS and WPSS scoring systems statistically influenced overall survival (OS). The time from diagnosis to the first transfusion was variable: less than six months in 97 cases (48 %); 6 to 12 months in 15 (7 %); more than 12 months in 20 (10 %). The OS of the 132 transfused patients was significantly worse than that of the 70 non-transfused ones, but the OS of patients who began to require transfusions more than 12 months after diagnosis was not significantly worse than that of not transfused patients. Only patients with an early transfusion requirement (within the first 12 month) had a poor outcome. When considering the prognosis of WHO subgroups the difference in OS between the subgroups 0 and 1 did not reach a significant level (p>0.05). In stepwise multivariate analysis the best prognostic factors were blast count, peripheral cytopenias and cytogenetic abnormalities. When the IPSS variables were included into the Cox model, neither WHO subgroups nor transfusion requirement retained an independent prognostic value.
CONCLUSIONS. The prognostic value of transfusion requirement was confirmed, but only patients requiring transfusion within the first 12 months since diagnosis showed a poor outcome. In our experience IPSS model fits prognosis better than WPSS.