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Measuring the benefits of growth hormone therapy in children: a role for preference-based approaches?
  1. Petrou1,2,
  2. McIntosh1
  1. 1
    Health Economics Research Centre, Department of Public Health, University of Oxford, Oxford, UK
  2. 2
    National Perinatal Epidemiology Unit, Department of Public Health, University of Oxford, Oxford, UK
  1. Dr S Petrou, National Perinatal Epidemiology Unit, University of Oxford (Old Road Campus), Old Road, Headington, Oxford OX3 7LF, UK; stavros.petrou{at}npeu.ox.ac.uk

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Recombinant human growth hormone has revolutionised the management of children with growth hormone deficiency, chronic renal insufficiency, Turner syndrome, Prader-Willi syndrome, small-for-gestational-age status, idiopathic short stature and other growth disorders in recent decades. The benefits of administering growth hormone to children have commonly been measured in terms of acceleration in linear growth.1 Although informative, lines of enquiry that measure the benefits of recombinant human growth hormone in natural or physical units, such as acceleration in linear growth, suffer from a number of limitations. Most notably, they are unable to incorporate the several health changes that can result from growth hormone therapy, including the increased risk of a number of adverse effects1 such as pigmentation and growth of nevi, gynecomastia, pancreatitis and benign intracranial hypertension, within a single measure. In addition, they overlook the broader effects that the treatment may have on children’s physical, mental and social well-being, as well individual preferences for those health changes. A number of preference-based approaches for measuring the benefits of healthcare interventions have been developed by health economists and might usefully be applied to growth hormone therapy in children. One such approach is conjoint analysis, the application of which is illustrated by Ahmed et al2 in this issue of the journal in relation to preferences for growth hormone injection devices. This is contrasted with the study by Kapoor et al,3 also published in this issue of the journal, which measures the impact of growth hormone-prescribing policies on non-preference-based outcomes, namely treatment concordance and height velocity.

Preference-based approaches that can be used to measure the benefits of growth hormone therapy in children include quality adjusted life year (QALY)-based approaches, contingent valuation-based approaches and conjoint analysis-based approaches. QALY-based approaches combine estimates of life years gained and enhancement of health-related quality of life …

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