Immediate-release granule formulation of hydrocortisone, Alkindi®, for treatment of paediatric adrenal insufficiency (Infacort development programme)

INTRODUCTION: Treatment of paediatric patients with adrenal insufficiency is challenging due to the lack of appropriate glucocorticoid preparations for children, and the use of either pharmacy- or parent-compounded hydrocortisone tablets. Alkindi (hydrocortisone granules in capsules for opening) is a new therapeutic option for paediatric adrenal insufficiency. Areas Covered: Drawbacks of current therapy and formulation and clinical trial programme for Alkindi. Expert Commentary: Compounding hydrocortisone has multiple issues including inconsistent dosing with under and over treatment and practical problems for parents who compound the drug themselves or travel long distances to a compounding pharmacy and the cost of compounding by the pharmacy. Alkindi® is a novel paediatric formulation of immediate release hydrocortisone licensed for use in paediatric adrenal insufficiency. Alkindi® is formulated to address the needs of neonates, infants and young children, being available at appropriate paediatric doses of 0.5, 1.0, 2.0 and 5.0 mg, is multiparticulate, allowing either direct oral dosing or dosing mixed with food, is taste masked to obscure the bitter taste of hydrocortisone and is bioequivalent to current hydrocortisone preparations. Clinical trials in young children with adrenal insufficiency demonstrated cortisol levels after dosing similar to those seen in healthy children and the drug was well tolerated and favoured over current therapy by parents. Alkindi® will provide a licenced treatment option for accurate dosing in children with adrenal insufficiency where compounded adult tablets of hydrocortisone are unsuitable

Poor compliance and increased mortality, depression and healthcare costs in patients with congenital adrenal hyperplasia

Objectives To evaluate risks of depression and all-cause mortality, healthcare utilisation costs, and treatment adherence in congenital adrenal hyperplasia (CAH) in the UK. Design and Methods A retrospective, matched-cohort study using UK primary-care data from the Clinical Practice Research Datalink linked to hospital and death-certification data. Patients diagnosed with CAH and having ≥1 corticosteroid prescription were matched 1:10 to reference subjects. Risk of death and lifetime prevalence of depression were compared using Cox regression models. Direct financial costs were estimated for healthcare contacts. Treatment adherence was measured by medical possession ratio (MPR). Results 605 patients with CAH were identified; 562 were matched. 270 CAH patients (2,700 controls) were linkable to death-certificate data, with adjusted hazard ratio for all-cause mortality 5.17 (95%CI 2.81 to 9.50). Mean (SD) age at death in CAH patients was 54.8 (23.9) versus 72.8 (18.0) years in control patients. The prevalence ratio of depression in CAH versus control patients was 1.28 (95%CI 1.13 to 1.45). Mean (SD) annual healthcare costs were higher in CAH than controls: at age 0-6 years, £7,038 (£14,846) versus £2,879 (£13,972, p<0.001); 7-17 years, £3,766 (£7,494) versus £1,232 (£2,451, p<0.001); 18-40 years, £1,539 (£872) versus £1,344 (£1,620, p=0.007); and ≥41 years, £4,204 (£4,863) versus £1,651 (£2,303, p<0.001). Treatment adherence was lowest in adults, with 141 (36%) of 396 eligible patients having an MPR <80%. Conclusions This first analysis of CAH in routine UK healthcare suggests that patients with CAH have increased mortality, depression, and healthcare utilisation, and low treatment adherence

Morphinofobia: the situation among the general population and health care professionals in North-Eastern Portugal

<p>Abstract</p> <p>Background</p> <p>Morphinofobia among the general population (GP) and among health care professionals (HP) is not without danger for the patients: it may lead to the inappropriate management of debilitating pain. The aim of our study was to explore among GP and HP the representation and attitudes concerning the use of morphine in health care.</p> <p>Methods</p> <p>A cross-sectional study was done among 412 HP (physicians and nurses) of the 4 hospitals and 10 community health centers of Beira Interior (Portugal)and among 193 persons of the GP randomly selected in public places. Opinions were collected through a translated self-administered questionnaire.</p> <p>Results</p> <p>A significant difference of opinion exists among GP and HP about the use of morphine. The word morphine first suggests drug to GP (36,2%) and analgesia to HP (32,9%.). The reasons for not using morphine most frequently cited are: for GP morphine use means advanced disease (56%), risk of addiction (50%), legal requirements (49,7%); for HP it means legal risks (56,3%) and adverse side effects of morphine such as somnolence - sedation (30,5%) The socio-demographic situation was correlated with the opinions about the use of morphine.</p> <p>Conclusions</p> <p>False beliefs about the use of morphine exist among the studied groups. There seems to be a need for developing information campaigns on pain management and the use of morphine targeting. Better training and more information of HP might also be needed.</p