PB2047 COMPARATIVE ANALYSIS OF DEFERASIROX DISPERSIBLE TABLETS VS FILM‐COATED TABLETS IN SEVERE CONGENITAL ANEMIAS: RESTROSPECTIVE STUDY IN A TERTIARY HOSPITAL
Authors: C. Insua Vilas, R.M. Rodríguez Nuñez, P.M. García Cereijo, Ó. Domínguez Muñiz, C. Albo López, C. Fonte Feal
Journal: HemaSphere (2019)

Background: Several congenital anemias are defined by ineffective erythropoiesis, sometimes even exarcerbated by chronic hemolysis, which in combination with periodic transfusions required on the most severe forms lead to iron overload in the organism, which settle on body tissues leading to their disfunction, with significant impact on morbimortality. Iron chelators aim to decrease iron load, as well as prevent or delay long term complications. Deferasirox (DF) is an oral chelator whose efficacy and security had been well defined since its approval. New film‐coated formulation proved similar efficacy as well as better palatability and tolerance on the ECLIPSE trial, achieving better treatment compliance. Aims: Conducting a comparative study between deferasirox dispersable tablets (DF‐D) versus film‐coated tablets (DF‐FC) amidst severe congenital anemias, analyzing chelating capacity as well as treatment compliance and side effects in both formulations. Methods: Retrospective case series. We collected data from patients diagnosed with severe congenital anemias and active follow up visits in our hospital, who were treated with DF‐D as well as DF‐FC over 12 months each. Qualitative variables are presented as absolute frequencies and percentages; and the quantitative ones as median and standard deviation. Exact Fisher test was performed when comparing qualitative variables, and Mann Whitney test when analyzing quantitative ones. Data was analized by SPSS Statistics. Results: We collected data from 7 patients: 6 of them diagnosed as thalassemia major and 1 as Blackfan Diamond anemia. 85,7% of our patients were female and 14,3% were male, median age 44 years old. Mean red blood cells concentrates tranfused were 20 units per year for both formulations. Median DF dosing was 1500 mg over DF‐D treatment and 1080 mg over DF‐FC treatment, which translates into equivalent dosing given DF‐FC better bioavailability. We got 6,2% mean increasing percentage of serum ferritin levels with DF‐D versus 1,3% with DF‐FC; however, when analyzing liver iron concentration (LIC) we observed an 8,3% decrease in iron load with DF‐D as opposite to 39,7% with DF‐FC, as well as a similar decrease in cardiac iron concentration (CIC) comparing both formulations (9,5% with DF‐D vs 6,5% with DF‐FC). 71,4% patients showed poor treatment compliance over DF‐D treatment, due to the development of side effects, outlining digestive disturbances (80%) and less frequently renal impairment or nonspecific disturbances (10% each), conditioning a change in therapy in 40% of cases. We did not find adherence problems with DF‐FC formulation, side effects rate decreased to 28,6% and no patient required a change in therapy. Summary/Conclusion: Our data revealed a high rate of side effects associated with DF‐D, mainly digestive disturbances, conditioning poor therapeutic adherence as well as changes to treatment in 40% of our patients. New formulation substantially decreased side effects rate, no patients required a change in treatment and we found a 100% therapy compliance. image Serum ferritin levels showed an slight increase over both treatments, with hardly any diffence between the two, which did not correlate with LIC levels, that showed a significant decrease in iron load, or cardiac deposits. In our opinion this data could be explained given ferritin low specificity quantifying iron load, whose levels fluctuate with inflammatory or infectious states; as compared to LIC and CIC estimation by magnetic resonance imaging, which have shown higher reliability when quantifying iron burden.