School of Medicine and Health Sciences Poster Presentations

Title

Retrospective Analysis of Compliance and Efficacy of a Novel Formulation of Deferasirox (Jadenu)

Document Type

Poster

Abstract Category

Clinical Specialties

Keywords

Sickle Cell Disease, Chronic Transfusion Therapy, Iron overload, Deferasirox, Pediatric Quality of Life

Publication Date

Spring 5-1-2019

Abstract

Background: Transfusion-related iron overload is a complication of chronic transfusion therapy in patients with sickle cell disease (SCD). Iron overload can cause hepatic, cardiac and other end-organ dysfunctions, and greater iron burden has been associated with increased mortality in this population. Several medications are available to chelate iron, and adherence to chelation medication is critical to prevent iron-related damages. We assessed the impact of Jadenu®, a novel film-coated tablet formulation of deferasirox, versus dissolvable deferasirox on adherence, iron control, patient/parent reported preferences, and quality of life (QoL) in chronically-transfused patients with SCD. Methods: Pediatric patients with SCD receiving chronic transfusion therapy and chelation were invited to participate in this single-institution trial. Subjects and parents were administered a survey on medication preference and adherence. Then, they completed PedsQL™ Sickle Cell Disease Module 3.0, and PedsQL™ Quality of Life Short Form 4.0. Retrospective measures of iron burden (laboratory values and imaging) were abstracted from the electronic health record. In subjects who transitioned to tablet deferasirox, iron measures were compared during the time on their prior chelation and while they were taking tablet deferasirox. Results: Twenty-one subjects were enrolled including 15 subjects prescribed tablet deferasirox and 6 prescribed dissolvable deferasirox. 92% on tablet deferasirox reported missing more doses with dissolvable formulation than with tablet, with 50% reporting missing 3-4 doses per week of dissolvable formulation. Patient-reported barriers to taking dissolvable formulation included side effects, need to take on empty stomach, taste, and forgetfulness. 64% reported no side effects from either formulation. Cost did not appear to be a barrier to taking or obtaining either formulation. There were no statistically significant differences in QoL measures between subjects taking either formulations of deferasirox, except patient-reported psychosocial QoL was higher in 8-13y cohort taking tablet deferasirox. For subjects on both formulations, average ferritin was comparable during the time periods on dissolvable vs tablet within subjects (3358ng/dL vs 3395ng/dL, p>0.05), but there was a trend towards improved LIC on the tablet formulation (15.6mg/g dry weight vs 14.9mg/g dry weight, p>0.05). Discussion: Film-coated tablets were the patient-preferred formulation of deferasirox, and subjects reported improved adherence with this formulation. Though chelation had little impact on general and sickle cell specific measures of QoL, there was a trend towards improved iron burden as measured by LIC. Long term evaluations of chelation adherence and impact of iron burden on mortality are needed in patients with SCD receiving chronic transfusion.

Open Access

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Presented at Research Days 2019.

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Retrospective Analysis of Compliance and Efficacy of a Novel Formulation of Deferasirox (Jadenu)

Background: Transfusion-related iron overload is a complication of chronic transfusion therapy in patients with sickle cell disease (SCD). Iron overload can cause hepatic, cardiac and other end-organ dysfunctions, and greater iron burden has been associated with increased mortality in this population. Several medications are available to chelate iron, and adherence to chelation medication is critical to prevent iron-related damages. We assessed the impact of Jadenu®, a novel film-coated tablet formulation of deferasirox, versus dissolvable deferasirox on adherence, iron control, patient/parent reported preferences, and quality of life (QoL) in chronically-transfused patients with SCD. Methods: Pediatric patients with SCD receiving chronic transfusion therapy and chelation were invited to participate in this single-institution trial. Subjects and parents were administered a survey on medication preference and adherence. Then, they completed PedsQL™ Sickle Cell Disease Module 3.0, and PedsQL™ Quality of Life Short Form 4.0. Retrospective measures of iron burden (laboratory values and imaging) were abstracted from the electronic health record. In subjects who transitioned to tablet deferasirox, iron measures were compared during the time on their prior chelation and while they were taking tablet deferasirox. Results: Twenty-one subjects were enrolled including 15 subjects prescribed tablet deferasirox and 6 prescribed dissolvable deferasirox. 92% on tablet deferasirox reported missing more doses with dissolvable formulation than with tablet, with 50% reporting missing 3-4 doses per week of dissolvable formulation. Patient-reported barriers to taking dissolvable formulation included side effects, need to take on empty stomach, taste, and forgetfulness. 64% reported no side effects from either formulation. Cost did not appear to be a barrier to taking or obtaining either formulation. There were no statistically significant differences in QoL measures between subjects taking either formulations of deferasirox, except patient-reported psychosocial QoL was higher in 8-13y cohort taking tablet deferasirox. For subjects on both formulations, average ferritin was comparable during the time periods on dissolvable vs tablet within subjects (3358ng/dL vs 3395ng/dL, p>0.05), but there was a trend towards improved LIC on the tablet formulation (15.6mg/g dry weight vs 14.9mg/g dry weight, p>0.05). Discussion: Film-coated tablets were the patient-preferred formulation of deferasirox, and subjects reported improved adherence with this formulation. Though chelation had little impact on general and sickle cell specific measures of QoL, there was a trend towards improved iron burden as measured by LIC. Long term evaluations of chelation adherence and impact of iron burden on mortality are needed in patients with SCD receiving chronic transfusion.