Myelodysplastic Syndrome (MDS) in France: Results of a One-Week Cross-Sectional Survey on Daily Practice Management in 919 Patients by the GFM

Background: Epidemiological data on MDS is scarce in France, and registries from other countries do not provide data on the daily practice management of MDS in 2008. Methods: GFM centers were asked to collect characteristics of ongoing or recent treatments in all MDS patients (pts) seen at their clinic (as in or outpatients) during the Jan 28th–Feb 3rd, 2008 period (one week).Results: 919 pts from 74 centers were included, 57% males, mean age (+/− SD), 73 (±11) years, with 2.8%, 19% and 28% of pts aged <50, <65 and >80 years, respectively (resp).13% of pts were hospitalized >24h (4.5% for infections or bleeding and 8.5 % for “active” treatments), 46% were seen in the day care facility (40% for transfusions), and 41% as consultations (for staging, follow up or ambulatory treatment). 93% of patients had PS ≤2. Median interval from diagnosis to survey was 29.2 months. FAB at time of survey was: 35.1% RA, 18.5% RARS, 39.1% RAEB, 7.4% CMML; WHO was: 17.4% RA, 13.3% RARS, 14% RCMD, 4.5% RCMD-RS, 18.5% RAEB-1, 15.9% RAEB-2, 7.7% CMML, 4.9% 5q-syndrome and 3.9% unclassifiable. Cytogenetic analysis had been performed at least once in 77.4 % pts: favorable (498 pts), intermediate (88 pts), unfavorable (96 pts). IPSS (determined in 75.4% of pts) was: 41.6% low, 33.3% Int-1, 16.4% Int-2 and 8.7% high. Significant differences between pts <65 years and >65 years were, respectively, % of unfav karyotype (25.8% vs. 12.7%, p=0.0004), of isolated +8 (5.1% vs. 2.1%, p=0.04), of isolated −7 (6.2% vs. 1.1%, p=0.0003), and, with borderline significance, of CMML (4.5% vs. 9.5%, p=0.06), of 5q-syndrome (1.5% vs. 5%, p=0.07). EPO level, assessed in 359 (39.1%) of pts at diagnosis and 252 (27.4%) of pts at time of survey) was >200UI/l in 24.5% and 26.6% resp, and >500U/l in 13.5% and 14.7% pts resp and was significantly correlated with interval from diagnosis. At the time of survey, treatment received in the last 6 months (IPSS: high-int 2 vs low–int1) included: no active treatment 66.5% (IPSS: 42% vs. 72.9%), chemotherapy 12.8% (IPSS: 22.6% vs. 9.1%) including 2.7% intensive and 0.7% LD AraC, allogeneic SCT 1.7% (IPSS: 3.8% vs. 2.6 %) including 0.3% classical and 1.4% NMA, azacytidine 6.5%, (IPSS: 21.6% vs. 2.3%), decitabine 0.8%, lenalidomide 4%, thalidomide 0.5%, ATG 0.2 %, androgens 2.2% while 64.8% pts required RBC transfusions (IPSS: 81% vs. 61%) and 39.7% pts received an Erythropoiesis-Stimulating Agent (ESA) (IPSS: 40.3% vs. 37.2%), alone in 314 pts (epoetin alfa or beta in 92 pts, darbepoetin in 222 pts), and with G-CSF (61 pts). Response rates to ESAs were 58.6% and 33.8% in low int-1 and int-2-high risk MDS, resp (p=0.0009). Iron chelation therapy was administered in 17.6% pts (5.8% desferroxamine, 11% deferasirox) including 22.1% and 13.6% low-int-1 and int-2-high risk MDS, resp (p=0.009). Conclusions: Our survey provides a better knowledge of the characteristics and of the daily management of MDS in France. Of particular note are the more frequent unfavorable karyotypes in MDS pts <65 years and the generally low EPO levels that may increase the indications for ESAs in low and int 1 risk MDS with anemia. Apart from ESAs, active treatments of MDS still only reach a minority of pts, and transfusions account for as many as 40 % of the hospital visits/stays for MDS.