Costs and outcomes over 36 years of patients with phenylketonuria who do and do not remain on a phenylalanine-restricted diet.

BACKGROUND: To quantify the costs and consequences of managing phenylketonuria (PKU) in the UK and to estimate the potential implications to the UK's National Health Service (NHS) of keeping patients on a phenylalanine-restricted diet for life. METHOD: A computer-based model was constructed depicting the management of PKU patients over the first 36 years of their life, derived from patients suffering from this metabolic disorder in The Health Improvement Network database (a nationally representative database of patients registered with general practitioners in the UK). The model was used to estimate the incidence of co-morbidities and the levels of healthcare resource use and corresponding costs over the 36 years. RESULTS: Patients who remained on a phenylalanine-restricted diet accounted for 38% of the cohort. Forty-seven per cent of patients discontinued their phenylalanine-restricted diet between 15 and 25 years of age. Of these, 73% remained off diet and 27% restarted a restricted diet at a mean 30 years of age. Fifteen per cent of the cohort had untreated PKU. Eleven per cent of patients who remained on a phenylalanine-restricted diet for 36 years received the optimum amount of prescribed amino acid supplements. Patients had a mean 12 general practitioner visits per year and one hospital outpatient visit annually, but phenylalanine levels were only measured once every 18 to 24 months. The mean NHS cost (at 2007/08 prices) of managing a PKU sufferer over the first 36 years of their life was estimated to range between £21 000 and £149 000, depending on the amount of prescribed nutrition they received. CONCLUSION: The findings suggest that the majority of patients with PKU were under-treated. The NHS cost of patient management should not be an obstacle to encouraging patients to remain on a restricted diet until further information becomes available about the long-term clinical impact of stopping such a diet. Nevertheless, patients require counselling and managed follow up regardless of the choices they make about their diet.

Modelling the cost-effectiveness of electric stimulation therapy in non-healing venous leg ulcers.

OBJECTIVE: To estimate the cost-effectiveness of using electric stimulation (ES) therapy (Accel-Heal) plus dressings and compression bandaging compared with dressings and compression bandaging alone in treating chronic, non-healing venous leg ulcers (VLUs) of >6 months' duration from the perspective of the National Health Service (NHS) in the UK. METHOD: A 5-month Markov model was constructed, depicting the management of a chronic, non-healing VLU of >6 months' duration. The model considers the decision by a clinician to continue with a patient's previous care plan (comprising dressings and compression bandaging) or treating with ES therapy plus dressings and compression bandaging. The model was used to estimate the relative cost-effectiveness of ES therapy at 2008-2009 prices. RESULTS: According to the model, 38% of VLUs are expected to heal within 5 months after starting ES therapy, with a further 57% expected to improve. This improvement in clinical outcome is expected to lead to a 6% health gain of 0.017 QALYs (from 0.299 to 0.316 QALYs) over 5 months. The model also showed that using ES therapy instead of continuing with a patient's previous care plan is expected to reduce the NHS cost of managing them by 15%, from £880 to £749, due in part to a 27% reduction in the requirement for nurse visits (from mean 49.0 to 35.9 visits per patient) over the first 5 months after the start of treatment. Hence, use of ES therapy was found to be a dominant treatment (improved outcome for less cost). CONCLUSION: Within the model's limitations, use of ES therapy potentially affords the NHS a cost-effective treatment, compared with patients remaining on their previous care plan in managing chronic, non-healing VLUs of >6 months' duration. However, this is dependant on the number of ES therapy units per treatment, the unit cost of the device, and the number of nurse visits required to manage patients in clinical practice. DECLARATION OF INTEREST: This study was sponsored by Synapse Microcurrent Ltd., manufacturers of Accel-Heal. The authors have no other conflicts of interest that are directly relevant to the content of this manuscript. In particular, Synapse Microcurrent Ltd. had no role in the study design, in the collection, analysis and interpretation of data, or in writing the manuscript.