C o p y r i g h t I n f o r m a U K L i m i t e d 2 0 0 8 N o t f o r S a l e o r C o m e r c i a l D i s t r i b u t i o n U n a u t h o r i z e d u s e p r o h i b i t e d . A u t h o r i s e d u s e r s c a n d o w n l o a d ,Objectives: Iron chelation treatment (ICT) in -thalassemia major (-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 -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.Research design and 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 ¼ 28.3, 3-48 years, 49.6% male) and retrospectively observed for a median of 11.6 months. Mean direct costs were E1242/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 -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.

Thromboembolic events, which are associated with significant morbidity and mortality, occur in beta-thalassaemia. We studied the expression of the platelet selectin PADGEM/GMP-140 on intact cells from thalassaemic patients, as a marker of in vivo platelet activation. The mean of positive cells (%) was 38.143 +/- 20.65 in the patients versus 5.048 +/- 1.8 in the controls, n = 21, P < 0.001. No correlation was found between GMP-140 expression and splenectomy, platelet counts, plasma ferritin and natural coagulation inhibitors. Instead an indirect correlation was found between GMP-140 expression and HDL-cholesterol. Moreover platelet activation was directly correlated with pre-beta lipoproteins. Our data indicate that thalassaemic patients present an in vivo platelet activation, which possibly depends on the dyslipidaemia, which is now regarded as a frequent feature of this disease.

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