A Simple Model to Assess and Improve Adherence to Iron Chelation Therapy with Deferoxamine in Patients with Thalassemia

Abstract

Adherence to deferoxamine (DFO) is vital for the long-term survival of patients with thalassemia; however, currently no measure exists to quantify adherence directly. In this study, 90 patients with thalassemia major underwent liver iron concentration (LIC) assessment by SQUID biosusceptometer, were asked to rate their adherence to DFO using a Numerical Likert Scale (NLS), and were educated about complications of iron overload. Of 38% (n = 28) of patients who rated themselves as very compliant, 19 had elevated LIC related to inadequate dosing of DFO and nine reported nonadherence in the past. Adherence improved after counseling and LIC decreased by 25% (7-60%) in eight previously noncompliant patients who returned for subsequent LIC over 15 months. In conclusion, the NLS seems to be a simple but reliable tool to assess patients' adherence to DFO. Education and frequent noninvasive LIC assessments can improve adherence and iron burden. Elevated LIC does not necessarily reflect concurrent noncompliance; however, it can be an indication of nonadherence in the past.

Spin density projection‐assisted R2 magnetic resonance imaging of the liver in the management of body iron stores in patients receiving multiple red blood cell transfusions: an audit and retrospective study in South Australia

“…The body iron burden of the cohort as measured by the geometrical mean LIC decreased significantly during the period of access to non‐invasive LIC measurement. The significant decrease in the body iron burden together with the documented clinical decisions regarding chelation therapy based on the LIC results are consistent with previous observations that introduction of non‐invasive monitoring of LIC can contribute to a decreased body iron burden through improved clinical decision making and improved feedback to patients and hence improved adherence to chelation therapy 14 . Other factors could also have an effect on the body iron burden of the cohort, such as the introduction of the newly available orally administered chelators, deferiprone and deferasirox.…”

“…Pakbaz and co‐workers 14 concluded that frequent non‐invasive measurements of LIC using biomagnetic susceptometry together with patient education can improve patient adherence to chelation regimens and result in reduced body iron burdens. Thus, we hypothesise that non‐invasive monitoring of LIC with the R2‐MRI technique 11,12 would aid in reducing the body iron burden in transfused patients through improved quantitative feedback of response of LIC to chelator to both clinician and patient.…”

Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia

“…Chelation guidelines for sickle-cell syndromes are similar to those for other iron overload situations as thalassemia major and multi-transfused patients [14,15]. Until recently, desferrioxamine and deferiprone were the only active iron-chelating agent approved for clinical use, mainly in the thalassemia setting [16,17]. Recent advances have led to the development of new oral iron chelators, such as deferasirox, which has changed the clinical practice in several transfusiondependent anemias with iron overload.…”

“…We have clarified now in the "study design" section that "The starting deferasirox dose was 10 or 20 mg/kg/day depending on previous transfusion requirements: if the patient had received 20-30 red blood cell (RBC) units the dose of 10 mg/kg/day was used; if the patients had received more than 30 RBC units the dose of 20 mg/kg/day was used." [page 4, lines [15][16][17].…”

MRI Measurements of Iron Load in Transfusion-Dependent Patients: Implementation, Challenges, and Pitfalls

“…Likewise, serial LIC measurements that show decreasing LIC can also demonstrate the benefits of chelation to asymptomatic patients and reinforce adherence. Routine education about disease states and chelation therapy has been shown to correlate with improvements in compliance and decreases in LIC . Therefore, patient education and engagement needs to be incorporated into every visit.…”