Results from a 1-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload

摘要: The majority of patients with myelodysplastic syndrome (MDS) present anemia and will become dependent on regular transfusions packed red blood cells (PRBC) the risk iron overload (IOL). Liver content best reflects total body content, measurement liver concentration (LIC) by MRI is a validated tool for detection, but data in MDS rather limited. Here we results multi-center trial evaluating efficacy safety deferasirox (DFX) low intermediate-1 transfusion-dependent IOL. Three transfusion frequency > 4 units PRBC per month were initially treated 30 mg/kg/day while 46 lower burden was initiated at 20 mg/kg/day, due to patient related reasons one received DFX dose 6 mg/kg/day only. LIC measured baseline end study using method St. Pierre et al. intention treat population consisted 50 (28 male; 22 female) median age 69 years who duration 354 days. Mean daily 19 mg/kg/day. Median serum ferritin level (SF) 2,447 ng/mL decreased 1,685 ng/mL (reduction 31 %) (p = 0.01). In 7 (13 %) chosen had be increased unsatisfactory chelation therapy. For 21 patients, performed 19 these study: mean significantly from 16,8 mg/g dry tissue weight (± 8.3 mg/g weight) entry 10,8 mg/g 10.4 mg/g Of all exposed drug (n = 54), 28 (52 %) did not complete 12 month period most commonly AEs 28 % (n = 15) abnormal laboratory values 7 % (n = 4), respectively. common adverse events (≥ 10 % patients) suspected relationship diarrhea (n = 25, 46 %), nausea (n = 13, 24 %), upper abdominal pain (n = 8, 15 %), creatinine increase (n = 16, 30 %) rash (n = 5, 9 %). Adverse making adjustments or interruption necessary occurred 33 (61 %). Hematologic improvement according IWG criteria (2006) observed 6 (11 %). Initiation treatment IOL depending yields sufficient reduction excess indicated levels importantly MRI. profile comparable previous observations.