Purpose: Congenital hemophilia A and B are bleeding disorders characterized by deficiency of factors VIII and IX, respectively. This study aimed to collect health-related quality-of-life (HRQoL) and health-utility data from hemophilia patients with differing disease severity. Methods: Individuals with hemophilia aged ≥12 years living in France or the UK completed a series of questionnaires, including the EQ-5D-3L and -5L and SF-36 version 2. Association with demographic and clinical variables was explored using linear regression, and health-utility comparison was completed using Pearson and intraclass correlation coefficients. Results: A total of 122 patients in France and 62 in the UK completed the survey. The combined sample primarily consisted of hemophilia A patients, mean age of 41 years, 70% had severe hemophilia, and 56% were on long-term prophylaxis. Similar HRQoL and utility scores were observed across the French and UK samples. The presence of more than two target joints, occurrence of joint surgery, and increased joint-pain frequency were independent predictors of lower SF-36 — physical health summary scores and lower health-utility scores. No statistically significant reductions in SF-36 — mental health summary scores were observed, except for participants with target joints. Strong correlations were observed between health- utility values derived from the three instruments ( r =0.69–0.79). Conclusion: Results of this study reinforce the importance of appropriate treatment to limit the physical burden and long-term joint damage associated with hemophilia. Further, utility values collected here reflect real-world data, and can serve as health-state weights in future cost–utility analyses.

Background Prophylactic treatment regimens lead to improvements in health-related quality-of-life (HRQoL) among individuals with hemophilia. Turoctocog alfa pegol (N8-GP) provides the benefit of extending the duration of protection from bleeding and reducing the number of injections, which is expected to impact HRQoL and treatment satisfaction (TS). Aim To investigate the HRQoL and TS of patients with severe hemophilia A from two phase III trials evaluating the safety and efficacy of N8-GP. Methods HRQoL was assessed using the Haemo-QoL (reported by children and their parents) and Haem-A-QoL (reported by adults). TS was assessed using Hemo-Sat. Domain and total scores for all questionnaires ranged from 0 to 100, with lower scores indicating a better HRQoL or TS. A negative change in score indicates an improvement in HRQoL/TS. Results Mean changes in HRQoL scores were reported for 14 children aged 4–7 years, 21 children aged 8–11 years, 10 adolescents aged 13–16 years, and 163 adults (17 years and above). Mean changes in children/adolescents-reported Haemo-QoL total score were -14.0 for ages 4–7 years, -3.6 for ages 8–11 years, and -0.1 for ages 13–16 years. Mean changes in parent-reported Haemo-QoL total scores were -11.5 for 4–7 years, -8.6 for ages 8–11 years, and -4.0 for 13–16 years. Adults’ mean change in Haem-A-QoL total score was -3.1 for those receiving on-demand treatment and -2.3 for those receiving prophylaxis treatment. High levels of TS with N8-GP were reported by parents of children/adolescents and the adults at the end of the trial. Conclusion While most patients reported a relatively good baseline HRQoL when entering the respective trials, the HRQoL of patients was either maintained or further improved when treated with N8-GP. Adults and parents of children and adolescents reported a high level of treatment satisfaction with N8-GP.

This study was aimed to produce a stable kenaf seed oil-nanostructured lipid carrier (KSO-NLC) sunscreen, which can help in the photoprotective effect. The nanostructured lipid carrier (NLC) formulation was optimized and selected based on the results of mean particle size, polydispersity index (PDI), and storage stability of formulas at both chilled (4 AE 2 C) and room (25 AE 2 C) temperatures. Uvinul A plus B was added to KSO-NLC with the optimized formula (80% w/w aqueous phase, 20% w/w lipid phase, and 7% w/w of surfactants with a ratio of 70:15:15 of Tween 20: poloxamer 188: lecithin). The mean particle size distribution (224.73 AE 1.56 nm) and PDI (0.41 AE 0.01) of KSO-NLC were determined and were found to be stable against storage without creaming or phase separation. The 2,2-Diphenyl-1-picrylhydrazyl (DPPH) radical-scavenging and 2,2 0 -Azino-bis(3-ethylbenzothiazoline-6-sulfonic acid) diammonium salt (ABTS) radical-scavenging activities of KSO-NLC were 5.43 AE 1.00 mg Trolox equiv. g −1 of NLC and 6.70 AE 0.31 mg Trolox equiv. g −1 of NLC, respectively. The Sun Protection Factor (SPF) of KSO-NLC, 41.38 AE 6.03 with a UVA/UVB ratio 0.64 AE 0.01, suggested a good photoprotective effect. The sustained release of Univul A plus B from KSO-NLC accompanied by its entrapment efficiency up to 64.09 AE 0.98% and drug loading (DL) of 32.05 AE 0.49% (maximum 50% DL capacity) proved that the degradation of the ultraviolet (UV) filter could be reduced. Therefore, the KSO-NLC sunscreen was a feasible solution for the photoprotective approach by using unconventional plant seed oil with a significant enhancement (P < 0.05) in many aspects compared to the formula without KSO incorporation. Keywords Particle size Á Polydispersity index (PDI) Á Uvinul a plus B Á Entrapment efficiency (EE) Á Drug loading (DL) J Am Oil Chem Soc (2019) 96: 201-211.

IntroductionCurrently, there is limited knowledge about the experiences and challenges type 2 diabetes (T2D) patients face when intensifying from basal insulin to more complex regimens. The purpose of this study was to examine the experiences of adults with T2D who have been intensified to a basal-bolus insulin regimen, including challenges related to intensification, medication adherence issues, non-persistence, and healthcare resource use related to intensification.MethodsA web-based survey of adults diagnosed with T2D and currently treated with basal insulin was conducted in the UK and the USA. The analysis sample was restricted to respondents with current/recent basal-bolus treatment (n = 398) and divided into three analysis groups: (1) “basal-bolus adherent” (current basal-bolus treatment with at least 90% adherence); (2) “basal-bolus non-adherent” (current basal-bolus treatment with less than 80% adherence); and (3) “stopped bolus” (discontinued bolus in past 12 months).ResultsBasal-bolus non-adherent respondents reported fewer discussions with their healthcare providers (HCPs) before starting bolus and more frequent difficulties with and worries about taking bolus insulin compared to basal-bolus adherent and stopped bolus groups. The most frequently reported reasons for discontinuing bolus were related to the complicated nature of regimen, including too complicated to calculate bolus doses (25.7%), too complicated to regulate food in relation to bolus (20.7%), and too complicated to keep track of taking two different insulins (18.6%). Respondents who stopped bolus reported more frequent HCP visits related to diabetes compared to the basal-bolus adherent and basal-bolus non-adherent groups.ConclusionResults suggest that the complicated nature of basal-bolus therapy contributes to the difficulties that T2D patients have with the regimen and to non-persistence. Physician and patient education may help patients address these treatment challenges to improve basal-bolus adherence and persistence, which could reduce healthcare resource use and costs. Less complex regimens may be appropriate for patients with persistent treatment difficulties.FundingNovo Nordisk A/S.

AimTo evaluate the cost‐effectiveness of IDegLira versus basal‐bolus therapy (BBT) with insulin glargine U100 plus up to 4 times daily insulin aspart for the management of type 2 diabetes in the UK.MethodsA Microsoft Excel model was used to evaluate the cost‐utility of IDegLira versus BBT over a 1‐year time horizon. Clinical input data were taken from the treat‐to‐target DUAL VII trial, conducted in patients unable to achieve adequate glycaemic control (HbA1c <7.0%) with basal insulin, with IDegLira associated with lower rates of hypoglycaemia and reduced body mass index (BMI) in comparison with BBT, with similar HbA1c reductions. Costs (expressed in GBP) and event‐related disutilities were taken from published sources. Extensive sensitivity analyses were performed.ResultsIDegLira was associated with an improvement of 0.05 quality‐adjusted life years (QALYs) versus BBT, due to reductions in non‐severe hypoglycaemic episodes and BMI with IDegLira. Costs were higher with IDegLira by GBP 303 per patient, leading to an incremental cost‐effectiveness ratio (ICER) of GBP 5924 per QALY gained for IDegLira versus BBT. ICERs remained below GBP 20 000 per QALY gained across a range of sensitivity analyses.ConclusionsIDegLira is a cost‐effective alternative to BBT with insulin glargine U100 plus insulin aspart, providing equivalent glycaemic control with a simpler treatment regimen for patients with type 2 diabetes inadequately controlled on basal insulin in the UK.