Costs, quality of life, treatment satisfaction and compliance in patients with β-thalassemia major undergoing iron chelation therapy: the ITHACA study

Abstract

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.

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

“…The mean costs were € 1242/patient/month, 55.5% attributed to iron chelation therapy and 33.2% attributable to transfusions. 98 Moreover, proper medical therapy for TM requires advanced technologies such as cardiac or hepatic MRI. 30,99 With the combined cost of blood transfusions, chelation and management of complications, the requirements for optimal thalassemia care clearly exceed the health resources available in most non-industrialized countries.…”

Allogeneic bone marrow transplant in a child with thalassaemia

“…This number has by and large remained static over the past decade. An Italian group reported in 2006, that the mean cost of treatment for thalassaemia major was € 1242/patient/month with 55.5% of it attributed to iron chelation therapy and 33.2% attributed to blood transfusion [2]. Bone marrow transplant (BMT) is the only established cure for thalassaemia at present.…”

“…This paper fails to portray the cardinal symptoms affecting the patients with MUO that the authors set out to palliate, the severity of such symptoms and the onset, duration and the degree of symptom improvement after RUS. Palliation should be viewed as a three-dimensional concept, namely onset, duration and degree of symptom relief [2]. Prophylaxis before surgery (9 cases), certainly not ureteric obstruction, should be excluded.…”

Study on Safety and Efficacy of Deferasirox in the treatment of Thalassemia in a South Indian Tertiary Care Hospital

“…If patients are not feeling the beneficial effects of their therapy and particularly if they experience adverse events, they are not motivated to comply with the proposed treatment. One longitudinal multicenter, Italian-THAlassemia-Cost-and-outcomes-Assessment (ITHACA) 17 has previously reported a decrease in patient satisfaction and acceptance of their chelation therapy by deferoxamine, as the overall benefits were not always perceived as optimal. Another prospective, open-label, 1-year, multicenter study in the Middle East (ESCALATOR) 18 demonstrated that patients had greater satisfaction and convenience with Deferasirox compared to deferoxamine, which improved compliance and overall patient well-being.…”