Costs, quality of life, treatment satisfaction and compliance in patients with beta-thalassemia major undergoing iron chelation therapy: the ITHACA study.
OBJECTIVE
Iron chelation treatment (ICT) in beta-thalassemia major (beta-TM) patients undergoing blood transfusions can cause low satisfaction, low compliance, with possible negative consequences on treatment success, patients' wellbeing, and costs. The purpose was to estimate the societal burden attributable to beta-TM in terms of direct and indirect costs, health-related quality-of-life (HRQoL), satisfaction and compliance with ICT in patients undergoing transfusions and ICT.


METHODS
The naturalistic, multicenter, longitudinal Italian-THAlassemia-Cost-&-Outcomes-Assessment (ITHACA) cost-of-illness study was conducted involving patients of any age, on ICT for at least 3 years, who were enrolled at 8 Italian Thalassemia Care Centers. Costs were estimated from the societal perspective, quantified with tariffs, prices, or net earnings valid in 2006.


RESULTS
One-hundred and thirty-seven patients were enrolled (median age = 28.3, 3-48 years, 49.6% male) and retrospectively observed for a median of 11.6 months. Mean direct costs were euro1242/patient/month, 55.5% attributable to ICT, 33.2% attributable to transfusions. Relevant quantity and quality of productivity was lost. Both physical and mental components of HRQoL were compromised. Little difficulties remembering to take ICT and positive satisfaction with the perceived effectiveness of therapy were declared, but not good levels of satisfaction with acceptance, perception of side effects and burden of ICT.


CONCLUSIONS
The management of beta-TM patients undergoing transfusions and ICT is efficacious, although costly, but overall benefits were not always perceived as optimal by patients. Efforts must be focused to improve patients' acceptance and satisfaction with their therapy; this would contribute to a better compliance and hence an increase in treatment effectiveness and patients' overall wellbeing, with expected improved allocation of human and economic resources.
Scalone
Luciana
L
CIRFF - Centre of Pharmacoeconomics, University of Naples Federico II, Naples, Italy; CHARTA Foundation (member of COPERA), Milan, Italy. lorenzogiovanni.mantovani@unina.it
Mantovani
Lorenzo G
LG
Krol
Marieke
M
Rofail
Diana
D
Ravera
Simona
S
Bisconte
Maria Grazia
MG
Borgna-Pignatti
Caterina
C
Borsellino
Zelia
Z
Cianciulli
Paolo
P
Gallisai
Domenico
D
Prossomariti
Luciano
L
Stefàno
Ippazio
I
Cappellini
Maria D
MD
D000293:Adolescent; D000328:Adult; D001803:Blood Transfusion; D015913:Chelation Therapy / Q000191:economics* / Q000523:psychology*; D002648:Child; D002675:Child, Preschool; D003362:Cost-Benefit Analysis; D005260:Female; D006801:Humans; D007558:Italy; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D010349:Patient Compliance; D010549:Personal Satisfaction; D011788:Quality of Life; D012189:Retrospective Studies; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000188:drug therapy* / Q000191:economics*