Background Cerebrotendinous xanthomatosis (CTX) is a rare, chronic, progressive, neurodegenerative disorder requiring life-long care. Patients with CTX often experience a diagnostic delay. Although early diagnosis and treatment initiation can improve symptoms and prognosis, a standardised approach to diagnosis, treatment and management of patients is not yet established. Aim To assess expert opinion on best care practices for patients with CTX using a modified Delphi method. Methods A multidisciplinary group of healthcare professionals with expertise in CTX responded to a 3-round online questionnaire (n = 10 in Rounds 1 and 2; n = 9 in Round 3), containing questions relating to the diagnosis, treatment, monitoring, multidisciplinary care and prognosis of patients with CTX. Determination of consensus achievement was based on a pre-defined statistical threshold of ≥ 70% Delphi panellists selecting 1–2 (disagreement) or 5–6 (agreement) for 6-point Likert scale questions, or ≥ 70% Delphi panellists choosing the same option for ranking and proportion questions. Results Of the Round 1 (n = 22), Round 2 (n = 32) and Round 3 (n = 26) questions for which consensus was assessed, 59.1%, 21.9% and 3.8% reached consensus, respectively. Consensus agreement that genetic analyses and/or determination of serum cholestanol levels should be used to diagnose CTX, and dried bloodspot testing should facilitate detection in newborns, was reached. Age at diagnosis and early treatment initiation (at birth, where possible) were considered to have the biggest impact on treatment outcomes. All panellists agreed that chenodeoxycholic acid (CDCA) is a lifetime replacement therapy which, if initiated early, can considerably improve prognosis as it may be capable of reversing the pathophysiological process in CTX. No consensus was reached on the value of cholic acid therapy alone. Monitoring patients through testing plasma cholestanol levels and neurologic examination was recommended, although further research regarding monitoring treatment and progression of the disease is required. Neurologists and paediatricians/metabolic specialists were highlighted as key clinicians that should be included in the multidisciplinary team involved in patients’ care. Conclusions The results of this study provide a basis for standardisation of care and highlight key areas where further research is needed to inform best practices for the diagnosis, treatment and management of patients with CTX.

Based on the outcomes of our review, paediatric ocular and adnexal trauma are significant causes for hospital attendance in childhood. Identifying associated risk factors will help develop injury prevention strategies to promote eye safety for children.

ABSTRACT.Purpose: To identify the causes of sports-related eye and adnexal injuries in children in Perth, Western Australia, to determine which sporting activities pose the highest risk of eye and adnexal injury to children. Methods: We performed a 12-year retrospective review of children admitted to hospital from 2002 to 2013 with sports-related ocular and adnexal eye injuries. The main outcome measures were the cause and type of ocular and adnexal injuries, age and gender risk factors. Results: A total of 93 cases of sports-related ocular and adnexal injury were identified in the 12-year time period. A peak in injuries occurred for 12-to 14-year-olds with a second peak in 6-to 8-year-olds; the median age was 8.82 years (range = 1.59-16.47). Cycling, football (including soccer and Australian Rules Football), tennis, trampolining, fishing and swimming were the sports responsible for the greatest number of injuries, a total of 63%. More than one-third (35%) of injuries resulted from being struck by a blunt object, and more than a quarter (26%) were as a result of contact with a blunt projectile. Conclusion: Serious ocular and adnexal injuries have occurred in children as a result of participating in sports, with cycling and football being the largest contributors in the 12-year period we assessed. As we continue to encourage children to spend more time participating in sports and recreational activities, identifying associated risk factors will help us develop injury prevention strategies to promote eye safety for children.

This study provides a summary of EQ-5D HSUVs for patients with chronic hepatitis C infection, and demonstrates that clinically important disease stages associated with treatment decisions are associated with differences in HRQoL.

Eye injury remains the leading cause of monocular blindness in children despite 90% of injuries being potentially preventable. Children interact with animals in a variety of situations, and the associated dangers may be underestimated. Animals are capable of causing ocular and adnexal injuries that are cosmetically and visually devastating. We examine the current literature regarding the nature and severity of animal-inflicted ocular and adnexal injuries in children.

Purpose This systematic literature review investigated whether extended dosing intervals (EDIs) of pharmacological acromegaly treatments reduce patient burden and costs compared with standard dosing, while maintaining effectiveness. Methods MEDLINE/Embase/the Cochrane Library (2001–June 2021) and key congresses (2018–2021) were searched and identified systematic literature review bibliographies reviewed. Included publications reported on efficacy/effectiveness, safety and tolerability, health-related quality of life (HRQoL), and patient-reported and economic outcomes in longitudinal/cross-sectional studies in adults with acromegaly. Interventions included EDIs of pegvisomant, cabergoline, and somatostatin receptor ligands (SRLs): lanreotide autogel/depot (LAN), octreotide long-acting release (OCT), pasireotide long-acting release (PAS), and oral octreotide; no comparator was required. Results In total, 35 publications reported on 27 studies: 3 pegvisomant monotherapy, 11 pegvisomant combination therapy with SRLs, 9 LAN, and 4 OCT; no studies reported on cabergoline, PAS, or oral octreotide at EDIs. Maintenance of normal insulin-like growth factor I (IGF-I) was observed in ≥ 70% of patients with LAN (1 study), OCT (1 study), and pegvisomant monotherapy (1 study). Achievement of normal IGF-I was observed in ≥ 70% of patients with LAN (3 studies) and pegvisomant in combination with SRLs (4 studies). Safety profiles were similar across EDI and standard regimens. Patients preferred and were satisfied with EDIs. HRQoL was maintained and cost savings were provided with EDIs versus standard regimens. Conclusions Clinical efficacy/effectiveness, safety, and HRQoL outcomes in adults with acromegaly were similar and costs lower with EDIs versus standard regimens. Physicians may consider acromegaly treatment at EDIs, especially for patients with good disease control.

objeCtives: Assess the consumer perception of communication with their HCPs and the information clarity during their recent medical visit when a new medication was prescribed. Methods: A cross-sectional online survey of consumers (patients and caregivers) was conducted (FDA cooperative-agreement #5U18FD004653-01) in Nov-Dec 2013 using a consumer panel in the U.S. A geographically diverse sample of consumers taking prescription medication(s) daily in the past 6 months to manage > = 1 chronic disease condition, or who had a primary responsibility for helping another adult taking prescription medication was recruited. Survey collected medication-taking and information-seeking behavior, and personal experience with and receipt of medication warnings/safety information. Descriptive statistics are reported. Results: Two thousand consumers (1600 patients/400 caregivers) participated. Positive attributes concerning HCP communication and information clarity (specific to medication being prescribed and their risk/safety included: comfortable asking HCP questions about the medication (90%), HCP made me feel confident the medication was the best to treat my condition (86%), HCP addressed fears/concerns about medication (84%), HCP informed me of medication side-effects (75%). Negative attributes/perceptions included: I had concerns about medication safety (44%), it was hard to follow-up with HCP via email/phone with medication-related questions (44%), HCP did not spend enough time to discuss medication (33%), HCP seemed too quick to prescribe medication (31%), I don't know what do to if I miss medication dose or took too much (27%), HCP used medical terms that were confusing (26%). These perceptions varied by age, number of comorbidities and whether the consumer was a patient or a caregiver. ConClusions: A diversity of positive and negative perceptions of HCP communication and information clarify was observed concerning recently prescribed medications. Factors influencing the negative perceptions and the modalities to address them warrants scrutiny.objeCtives: To evaluate the perceptions of consumers and HCPs concerning the receipt and delivery of medication risk/safety information. Methods: A crosssectional online survey of consumers (patients and caregivers) and HCPs was conducted (FDA cooperative-agreement #5U18FD004653-01) in Nov-Dec 2013 using online U.S panels. Two geographically diverse samples were recruited: consumers taking prescription medication(s) daily in the past 6 months to manage > = 1 chronic disease condition, or who had a primary responsibility for helping another adult taking prescription medication; and HCPs (primary care physicians (PCPs), pharmacists, nurse practitioners (NPs) and physician assistants (PAs)) spending > = 50% of time in direct ambulatory patient care (PCP/PA/NP) or working in a retail pharmacy (pharmacists), with > = 2 yrs of practice experience and seeing/consulting > = 20 patients/wk. HCP survey collected beliefs about medication risks, information seeking behavior and communication o...

discrete choice data was used. The comparison between the models was performed using the criteria of logical consistency, goodness of fit, and parsimony. Results: From 1056 participants who completed the interview, 2 met criteria that excluded them from the primary analyses. The characteristics of remaining 1054 respondents were very similar to those of the Indonesian population. Descriptive analysis showed that anxiety/depression and pain/discomfort are the two dimensions with most reported health problems. In the value set, values are ranged from -0.8506 for health state 55555 to 0,9426 for health state 11112. Mobility dimension affects most quality of life utility values, while Pain and Discomfort the least. Comparisons with healthy states values in other countries, and associations with demographic characteristics will be presented. ConClusions: This is the first value set of the EQ-5D-5L for Indonesia, and Indonesian preference on health states. We expect our survey to promote and facilitate research on health economic evaluations in Indonesia. PHP36Cost Variations of inPatient, DayCare anD outPatient HysterosCoPy in a tertiary-Care HosPital in Malaysia