title: Transfusion-dependent low-risk myelodysplastic patients receiving deferasirox: Long-term follow-up.
abstract: Myelodysplastic syndromes (MDSs) are characterized by ineffective hematopoiesis that results in peripheral cytopenias. Anemia is the most common symptom of MDS and the majority of patients become transfusion-dependent with the risk of iron overload, which may lead to cardiac, hepatic and endocrine complications. Deferasirox is an orally available iron chelator administered once-daily in transfusion-dependent patients with various chronic anemias. Its efficacy has been established in controlled clinical trials. In the present study, we describe our experience with 55 consecutive MDS patients [International Prognostic Scoring System risk score of low (n=32) or intermediate-1 (n=23)] treated with deferasirox in a routine clinical setting following Consensus Guidelines on Iron Chelation Therapy. According to WHO classifications, patients had refractory anemia (n=30), refractory anemia with ringed sideroblasts (n=16), refractory cytopenia with multilineage dysplasia (n=8) or refractory cytopenia with multilineage dysplasia and ringed sideroblasts (n=1). The median monthly transfusion requirement at baseline was 3 units. Patients received a starting dosage of 10 mg/kg/day, subsequently titrated according to serum ferritin (SF) levels which were measured monthly. Safety assessment included monitoring of liver and renal parameters and recording adverse events (AE) during treatment. At the baseline, the mean ± SD SF level was 2,362±172 ng/ml and after 24 months, the mean ± SD decrease in SF was 1,679±209 ng/ml. Sixteen patients had sustained hematological improvement meeting International Working Group 2006 criteria. One patient became transfusion-independent. No severe AE were reported. In conclusion, deferasirox therapy was effective and safe in reducing transfusional iron overload and it reduces transfusion requirement in a subset of patients.
authors.affiliation: Hematology Division, P.O. San Gennaro ASL NA1 Centro, Naples I-80136, Italy.
authors.forename: Salvatore
authors.initials: S
authors.lastname: Improta
authors.forename: Maria Rosaria
authors.initials: MR
authors.lastname: Villa
authors.forename: Antonio
authors.initials: A
authors.lastname: Volpe
authors.forename: Angela
authors.initials: A
authors.lastname: Lombardi
authors.forename: Paola
authors.initials: P
authors.lastname: Stiuso
authors.forename: Nicola
authors.initials: N
authors.lastname: Cantore
authors.forename: Lucia
authors.initials: L
authors.lastname: Mastrullo
keywords: chelation; deferasirox; erythroid response; iron overload; myelodysplastic syndromes; transfusion dependence