Patient Characteristics, Quality-of-Life and Compliance with Deferoxamine in Patients with Transfusional Iron Overload: Results of ‘ISOSFER’ Study.
Authors: Thuret I. , Brun-Strang C. , Bachir D. , Hacini M. , Pegourie-Bandelier B. , Gardembas-Pain M. 
Published: October 2019

Abstract
Purpose and Methods
ISOSFER is an epidemiological cross-sectional study with prospective recruitment whose objectives are to evaluate patient characteristics, quality of life (QoL), compliance and patient satisfaction with deferoxamine (DFO) therapy.
Results
Among 278 consecutive patients receiving regular transfusions for thalassemia major (TM), sickle cell disease (SCD) or myelodysplastic syndromes (MDS) who consulted between October 2005 and February 2006 in 24 French centers, 161 were on chelation therapy. 124 patients were treated with DFO alone for more than 1 year. Among them, 67 aged 14 years or more agreed to participate. QoL was studied using the Short Form-36 scale. Compliance was assessed with the Morisky scale and a specific questionnaire. DFO was administered via subcutaneous (sc) infusion for 70% of patients, mainly nightly and with a mean duration of 10 hours. Other ways of administering DFO included intravenous (iv) infusion (15%), sc bolus (9%) and combined sc and iv treatment (5%). Patient characteristics are summarized in the table below.
In comparison with the French general population, all dimensions of QoL are impaired in all three diseases and in all dimensions (physical activities, emotional problems, social activities, bodily pain, and vitality). TM and SCD patients had a better physical activities score than MDS patients but experienced more bodily pain than MDS patients. The global physical score was significantly influenced by age, marital status and occurrence of a co-morbidity related to hemosiderosis. Good compliance scores (0 and 1 on the Morisky scale) were found in only 74%, 67% and 87% of TM, SCD, MDS patients, respectively. In addition, DFO infusion was missed at least once a week during the past month in 17%, 44% and 4% of TM, SCD, MDS patients, respectively. Compliance was influenced by age, duration and number of weekly DFO infusions.
Iron chelation with DFO was generally found to be inconvenient and more than half of the patients were unsatisfied with its parenteral mode of administration. Nonetheless, patients, particularly those with TM, were convinced that DFO was an effective treatment for iron overload and a very important drug for their health.
Conclusions
These results suggest that QoL is severely compromised in patient on DFO and that compliance to DFO is poor. These results could help ascertain the improvement in QoL and compliance, which would result from the use of oral chelation in patients with transfusional iron overload.
TM (n=24) SCD (n=17) MDS (n=26) *Cardiac, liver and endocrine diseases, lens opacities, osteoporosis Median age in years (range) 30 (15–70) 32 (14–57) 69 (45–85) Sex, M/F 11/13 6/11 14/12 Geographic origin: EU/subS; Africa/North; Africa/others 16/0/6/2 1/12/3/1 26/0/0/0 Employment/university (%) 45 27 8 Marital status + (patients >18 yrs old) 9/21 7/15 25/26 Organ dysfunction potentially related to hemosiderosis* (%) 75 47 54 Median ferritin level (μg/L) 1049 2653 2627 Nb of TF >100 (%) 100 82 42 Mean DFO frequency/week 3.7 4.5 4 Mean dose 40 17 43