Direct and Indirect Cost of Beta Thalassemia Major: Resuts from the ITHACA Study.
Authors: Mantovani Lorenzo Giovanni , Scalone Luciana , Ravera Simona , Krol Marieke , Uyl-de Groot Carin , Cappellini Maria Domenica ,   the ITHACA Study Group
Published: October 2019

Background: Patients with beta Thalassemia Major (TM) require life-long blood transfusions and, to avoid iron overload, Iron Chelation Treatment (ICT), based on 8–12 hour infusions of Deferoxamine (DFO) for 5–7 days/week, and/or Deferiprone (L1) orally administered. ICT regimen often causes low satisfaction and low compliance, with potentially negative consequences on patients’ health, wellbeing and costs.

Aims: to investigate direct and indirect costs for TM patients on ICT.

Methods: The Italian-THAlassemia-Cost-&-Outcomes-Assessment (ITHACA) was a naturalistic multicentre study conducted to evaluate costs, quality of life, compliance and treatment satisfaction in TM patients undergoing ICT. Patients of any age, on ICT for at least 3 years, were sequentially enrolled at 8 Italian Thalassemia Care Centers. Direct and indirect costs were estimated from retrospective data: those on direct costs were drawn from medical records, referred to a median of 11.6 months before enrolment. As regards as indirect costs, patients completed modified versions of the Health and Labour Questionnaire (HLQ). Questions on indirect costs were referred to a 2 weeks (adults) or a 12 weeks (children) time horizon. Costs were estimated from a societal perspective, using tariffs, prices, average net earning applied in 2006, as appropriate.

Results: 137 patients were enrolled and aged a median of 28.3 years (2.7–48.5), 49.6% were male. Overall, mean direct cost was 1,245.33euro/patient/month, with ICT representing 55.4% of costs, followed by transfusions (33.1%), hospitalizations and surgery (3.3%), laboratory and instrumental tests and medical visits (3.1%), concomitant medications (1.6%), non-medical costs (transportation, 3.4%). Patients treated with DFO were 51.5%, 31.6% were treated with L1, 16.9% with DFO+L1. Treatment in patients taking DFO cost on average 552.88euro/patient/month plus 211.20 euro for pump and consumables; patients taking L1 cost 383.25 euro/patient/month, patients taking DFO+L1 cost 918.41 euro. Concerning indirect costs, 116 adults (> 16 years old) completed the HLQ. Sixty-one patients (52.7%) were in paid employment. Twenty-one patients (34.4%) had experienced absenteeism from work in the previous 2 weeks due to ill health. The average absenteeism among patients in paid employment was 0.7 days/week. Indirect costs related to absenteeism were 27.6 euro/patient/week. Fifteen patients (26.6% of patients in paid employment) were hindered by health problems at work. Indirect costs per patient based on hours needed to catch up on tasks neglected due to health problems were 9.4 euro/week. Patients received 1.24 hours of household help/week. Twenty-one children (<15 years old) or their caregivers completed the HLQ children-part. Eighteen children (85.7%) missed schooldays in previous twelve weeks (mean=0.66 days/week) and 6 missed regular activities.

Discussion: Transfusions and ICT account for 90% of total costs in TM patients, corresponding to approximately 1,000 euro/patient/month. The disease and the current treatment adopted have a high socio-economic impact on both TM patients and the healthcare system.