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.
Endocrine function was evaluated in 20 prepubertal patients with homozygous beta-thalassemia treated with frequent transfusions and long term iron chelation therapy. FSH, LH, PRL, and TSH secretion were evaluated by LRH and TRH testing and L-dopa and ACTH were used to assess GH and adrenocortical reserve. No statistically significant differences were found between FSH, LH, PRL, GH, and cortisol secretion in the patients and in normal subjects. There was a relatively high incidence (35%) of primary thyroid impairment since 1 patient had primary hypothyroidism and 6 others had evidence of subclinical hypothyroidism as manifested by increased TSH responses to TRH. However, no statistically significant correlations were found between either serum ferritin levels, total blood transfusions received, and thyroid function.
The purpose of this investigation was to determine the oral status in a group of patients with thalassemia major (TM). Eighteen TM patients (15 M, three F) and 18 healthy controls randomly matched for age and sex were examined for dental caries using the decayed, missing, and filled teeth (DMFT) index and for oral hygiene conditions using the oral hygiene index (OHI)-S. Spontaneous saliva was collected from each subject, and the biochemical composition (calcium, phosphorous, potassium, sodium, urea) was determined. Furthermore, salivary Streptococcus mutans levels were evaluated. Statistical analysis (Student's t-test) were performed for means comparison, while independence among categorical variables was assessed using the chi(2) test. Fisher's exact test was used when expected cell values were less than 5. Dental status (DMFT index) was almost equal in the two groups (10.3 in TM vs 9.4 in controls, P=0.34). The occurrence of plaque (OHI-S 2) was higher in the control group, but no statistically significant association was observed between oral hygiene conditions in the two groups (Fisher's exact test 0.47, P=0.79). Biochemical saliva composition was very similar in the two groups; only the urea concentration was lower in TM, and this difference was statistically significant ( P=0.002). The TM patients had an increased presence of mutans streptococci at detectable levels. Our findings confirm that, although no substantial differences were found between the two observed groups, further investigations are needed to determine the theoretical risk of oral diseases in thalassemic patients.
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