Table 1. Patient demographics and occupation category. SD, standard deviation. Table 2. Patient disease history and symptom characteristics. Data are expressed as mean (SD), unless otherwise stated. SD, standard deviation. • Most severely affected patients (UHDRS independence scale score of 10%–50%, i.e. the patient requires 24-hour supervision) comprised 20% of the overall population sample. Figure 1. Distribution of disease severity. Data are from the Unified Huntington’s Disease Rating Scale independence scale, rated by the patient’s caregiver. • Based on the mobility, self-care, usual activities and anxiety/depression sections of the EQ-5D scale, patients’ HRQoL was significantly different compared with that of the general population (p < 0.01, when comparing distribution of the categories 'no problem' versus any problem [‘moderate' and 'extreme'] between the two populations; Figure 2). • The difference was not significant in the EQ-5D pain/discomfort section (p = 0.10). • Mean (SD) number of monthly healthcare professional visits was 1.10 (1.66) for GPs and 2.10 (5.85) for specialists, and ranged from 4.38 (13.76) to 3.58 (9.12) visits for paramedical services. Burden of Huntington’s disease in Poland D Zielonka, 1 J Dorey, 2 M Mraidi, 2 M Lamure, 2 G Witkowski, 4 A Gogol, 5 J Marcinkowski, 1 M Toumi 3 1 Poznan University of Medical Sciences, Poznan, Poland; 2 Creativ Ceutical SA, Paris, France; 3 University Claude Bernard Lyon I, Villeurbanne, France; 4 Institute of Psychiatry and Neurology, Warsaw, Poland; 5 Warsaw Medical University, Warsaw, Poland Background • Huntington’s disease (HD) is a rare, progressive, genetic neurodegenerative disorder, with symptom onset typically occurring between the ages of 30 and 50 years. • The disease incurs a substantial burden on the health- related quality of life (HRQoL) of patients and their caregivers, as well as on the financial expenditure of affected families. • Identifying determinants of the cost of illness of HD, and the HRQoL of patients and caregivers, is critical to policy decision makers. • It is, therefore, essential to measure HD burden accurately, using a suitable battery of scales and in a large enough patient population. • The burden of HD in patients and their caregivers has not been measured previously in the Polish HD population. Objective • The aim of this study was to evaluate patient health status, patient and caregiver HRQoL, and the cost of illness of HD in Poland. Methods • The European HD burden (Euro-HDB) study is a comprehensive, observational, cross-sectional study being conducted in eight European countries (France, Germany, Italy, Spain, Poland, Portugal, Sweden and the UK), which has recently been extended to Australia* and the USA. 1 – In Poland, the Polish HD Association was responsible for providing patients with the self-reported questionnaires. • In the Euro-HDB study, data are being collected from patients and their caregivers, using two validated self- reported questionnaires. • The questionnaires include the Huntington Self- Assessment Instrument, a comprehensive and specific tool assessing patient’s clinical characteristics, HRQoL and healthcare resource utilization. – The patient’s questionnaire assesses mainly clinical characteristics, HRQoL and healthcare resource utilization. – The caregiver’s questionnaire describes the patient’s motor and functional ability and behaviour, as well as caregiver’s QoL and resource utilization. • Health outcomes, utility and well-being are assessed by the 5-dimension EuroQol questionnaire (EQ-5D) 2−4 and the 36-item Short-Form Health Survey (SF-36), 5,6 allowing HRQoL comparisons with other disorders to be made. Results • In total, 60 patients and 55 caregivers from Poland were enrolled into the Euro-HDB study. • Population characteristics are presented in Table 1 and Table 2. • The mean (standard deviation, SD) age of patients was 48.02 (13.06) years and 56% were female (Table 1). • 12% of patients were working, 49% were retired and 37% were unemployed (Table 1). • Mean (SD) age of HD onset was 38.02 (15.55) years and a clinical diagnosis was made, on average, 6 years after onset (according to patients and caregivers; Table 2). • All levels of disease severity, as measured by the Unified Huntington’s Disease Rating Scale (UHDRS) independence scale, were represented (Figure 1). Figure 2. Distribution of EQ-5D responses in patients with HD and in the general population. Source for general population: Golicki D et al. Pol Arch Med Wewn 2010;120:276−81. Gen. Pop, general population; HD, Huntington’s disease. • Total third-party payer perspective costs were evaluated to be 6718 Polish zloty (see Table 3 for full cost details). Table 3. Third-party, societal and caregiver costs of HD. Unless otherwise stated, all figures are presented as Polish zloty. TPP, third- party perspective. • Caregivers spent a substantial amount of time (an average of 21 hours/day) and money (mean of 289 Polish zloty/year) caring for their patients. • Generally, caregivers presented with a lower QoL than that of the general population, with 49% reporting dissatisfaction with their overall quality of life. Discussion • Results presented here, based on findings from a large, European cross-sectional study examining the burden of HD in patients and carers, are the first of their kind to be reported in a Polish population. • Whereas patients with the most severe forms of HD comprised only 20% of the total patients analysed, the overall HRQoL of patients was considerably reduced. • The significant financial expenditure incurred by caregivers is exacerbated further by the fact that patients are receiving low incomes, due to a decreased capacity to work. • These results are consistent with findings from other countries, namely that HD is associated with a significant costs of illness. 1 Our findings also highlight the substantial burden that HD has on patients and caregivers, which has been measured in detail using three different HRQoL scales. Acknowledgments We would like to thank the Polish Huntington’s disease Association for contributing to the study, especially Danuta Lis, Magdalena Boczarska–Jedynak, Witold Soltan and Monika Rudzinska. We also want to thank all patients and carers who participated, without whom the study would not have been possible. We are also grateful to NeuroSearch A/S for providing financial support for the study, in the form of an unrestricted academic grant. References 1. Dorey J et al. J Neurol Neurosurg Psychiatry 2010;81 Suppl 1:A46. 2. Brazier J et al. Qual Life Res 1993;2:169–80. 3. van Agt HM et al. Soc Sci Med 1994;39:1537–44. 4. Hurst NP et al. Br J Rheumatol 1994;33:655–62. 5. Garratt AM et al. BMJ 1993;306:1440–4. 6. Jenkinson C et al. BMJ 1993;306:1437–40 Corresponding author: Daniel Zielonka, Department of Social Medicine, Poznan University of Medical Sciences, Poznan, Poland. E-mail: daniel.zielonka@gmail.com *Project still under initiation. Poster 250 presented at the World Congress on Huntington's Disease, Melbourne, Australia, 11–14 September 2011. Patients from Poland N = 60 Demographic Male, n (%) 25 (42%) Age, years, mean (SD) 48.02 (13.06) Occupational category Workers, n (%) 7 (12%) Retired, n (%) 28 (49%) Unemployed, n (%) 21 (37%) Patients from Poland N = 60 Disease history Age at onset of symptoms, years 38.02 (15.55) Age at clinical diagnosis, years 44.25 (12.91) Tested genetically, n (%) 52 (90%) Motor score Patient motor score 31.34 (16.91) Patient chorea score 6.75 (3.99) Modified motor score 24.24 (13.75) Behavioural Score Depression-anxiety [0–20] 9.31 (6.04) Impulsivity [0–8] 3.98 (2.50) Psychotic disorder [0–12] 1.46 (1.80) Cognition [0–16] 3.98 (3.94) Functional score [0–8] 3.51 (1.96) Cost Mean Consultation 1136.00 Paramedical 612.12 Services (societal) 247.57 (377.26) Hospitalization 821.61 Facilities (societal) 3900.32 (4426.63) Total third-party payer costs 6717.64 Total societal costs 7373.64 Caregiver expenses per year 289.00 Caregiver time involvement, cost 1346.00 Caregiver time involvement, hours per day 21.23