Neuromesenchymal hamartoma of small bowel - an extremely rare entity: a case report

Neuromuscular and vascular hamartoma (NMVH) is a very rare stricturing condition of the small intestine, occurring focally and causing recurrent obstructive symptoms or occult chronic gastrointestinal bleeding. Salas et al. (Neuromesenchymal hamartoma of the small bowel. J Clin Gastroenterol. 1990, 12 (6): 705-9) proposed the term of "Neuromesenchymal hamartoma" for the cases of NMVH with participation of mesenchymal tissues

Patient adherence, compliance, and perspectives on evolocumab for the management of resistant hypercholesterolemia

Constantine E Kosmas,1 Delia Silverio,2 Julio Ovalle,2 Peter D Montan,2 Eliscer Guzman3 1Division of Cardiology, Department of Medicine, Mount Sinai Hospital, New York, NY, USA; 2Cardiology Clinic, Cardiology Unlimited, PC, New York, NY, USA; 3Division of Cardiology, Department of Medicine, Montefiore Medical Center, Bronx, NY, USA Abstract: Evolocumab is a PCSK9 inhibitor which is administered subcutaneously, and when added to statin therapy it has been shown to cause a significant incremental LDL-C reduction, leading to a reduction of cardiovascular risk. Evolocumab has a favorable side effect profile, and its self-administration at home appears to be safe and effective with the appropriate training and instructions from a health care provider. Current studies are showing encouraging results regarding adherence to evolocumab in real-life settings, and adherence rates to evolocumab appear to be better than those to statins. However, further larger studies are needed for a more definitive assessment of the short- and long-term patient adherence rates to evolocumab. In addition, reductions in the price of evolocumab may also be necessary to improve cost-effectiveness of the drug. Keywords: evolocumab, adherence, hypercholesterolemia, cardiovascular ris

Elicitation of health state utilities associated with the mode of administration of drugs acting on the prostacyclin pathway in pulmonary arterial hypertension

Evan W Davies,1 Samuel Llewellyn,2 Amélie Beaudet,1 Charlotte E Kosmas,2 Wendy Gin-Sing,3 Helen A Doll2 1Actelion Pharmaceuticals Ltd., Allschwil, Switzerland; 2ICON plc, Abingdon, UK; 3Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, UK Introduction: Pulmonary arterial hypertension (PAH) is a rare, incurable disease associated with decreased life expectancy and a marked impact on quality of life (QoL). There are three classes of drugs available for treatment: endothelin receptor antagonists (ERA), drugs acting on nitric oxide pathway (riociguat and phosphodiesterase type 5 inhibitors [PDE5i]), and drugs acting on prostacyclin pathway. The latter have widely different modes of administration – continuous intravenous infusion, continuous subcutaneous infusion, inhaled, and oral – each associated with variable treatment burden, and implications for health economic assessment. This study aimed to establish utility values associated with different modes of administration of drugs acting on the prostacyclin pathway for use in economic evaluations of PAH treatments. Methods: A UK general public sample completed the EQ-5D-5L and valued four health states in time trade-off interviews. The health states drafted from literature and interviews with PAH experts (n=3) contained identical descriptions of PAH and ERA/PDE5i treatment, but differed in description of administration including oral (tablets), inhaled (nebulizer), continuous subcutaneous infusion, and continuous intravenous infusion. Results: A total of 150 participants (63% female; mean age 37 years) completed interviews. Utilities are presented as values between 0 and 1, with 0 representing the state of being dead and 1 representing being in full health. The mean (SD) utility for oral health state was 0.85 (0.16), while all other health states were significantly lower at 0.74 (0.27) for inhaled (p=0.001), 0.59 (0.31) for subcutaneous (p<0.001) and 0.54 (0.32) for intravenous (p<0.001), indicating that there are disutilities (negative differences) associated with non-oral health states. Disutilities were -0.11 for inhaled, -0.26 for subcutaneous, and -0.31 for intravenous administration. Conclusion: The results demonstrate quantifiable QoL differences between modes of administration of drugs acting on the prostacyclin pathway. QoL burden should be considered for economic evaluation of drugs for PAH treatment. Keywords: pulmonary arterial hypertension, health-related quality of life, prostacyclin pathway, cost-utility, process utility, time trade-of