Introduction: Crohn's disease (CD) is a chronic inflammatory disease of the gastrointestinal tract. This real-world study evaluated persistence, dose titration, health care resource utilization (HCRU) and associated costs, and medication use among CD patients treated with ustekinumab (UST) in several pooled US commercial database populations. Methods: CD patients aged C 18 years with medical or pharmacy claims for UST were selected from pooled data from 3 large, national commercial databases. The first observed medical or pharmacy claim for UST was the index date. Patients were required to have had C 1 medical claim with a CD diagnosis during the 12 months prior to the index date and continuous health plan enrollment for a minimum of 12 months prior to and 12 months after the index date. Comparisons of outcomes during the baseline and follow-up periods were conducted using inferential statistical tests. Results: A total of 214 eligible UST patients were selected. The majority (74.8%) were biologic experienced (mean age: 41 years), and 83.6% remained treatment persistent during the 12-month post-index period. Among discontinuers, 25.7% restarted UST, and 8.6% switched from UST in the 12-month observation period. The mean treatment duration was 329 days. Most patients (77%) used the recommended UST dose, as defined as being within a 20% dose variation from label (90 mg/ 8 weeks ± 20%), 17.9% experienced dose escalation, and 5.1% experienced dose reduction. Post-index immunomodulator and corticosteroid use reduced by 20% and 28%, respectively, as compared with pre-index use among CD patients using UST. Annual all-cause ER visits and inpatient stays decreased by 20.5% and 30.3%, respectively, with similar downward trends for annual CD-related HCRU. Conclusions: The majority of CD patients prescribed UST were biologic experienced, and persistence was high over the 1-year follow-up. UST treatment initiation was associated with reductions in ER visits, inpatient stays, and steroid and other medication use.

Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment promising to reduce morbidity associated with hemophilia. While multiple clinical trials continue to evaluate efficacy and safety, limited cost-effectiveness data have been published. This study compared the potential cost-effectiveness of AAV-mediated factor IX(FIX)-Padua gene therapy for severe hemophilia B patients in the United States (US) to on-demand FIX replacement and primary FIX prophylaxis, using either standard or extended half-life FIX products. A microsimulation Markov model was constructed and transition probabilities between health states and utilities were informed by published data. Costs were aggregated using a micro-costing approach. An 18-years-old till death time-horizon from the perspective of a third-party payer in the US was conducted. Gene therapy was more cost-effective than both alternatives considering a $150,000/QALY threshold. The price for gene therapy was assumed $2,000,000 in the base-case scenario, yet one of the one-way sensitivity analyses was conducted using observed manufacturing, administration and five-year follow-up cost of $87,198 for AAV-mediated gene therapy vector as derived from the manufacturing facility and clinical practice at St. Jude Children's Research Hospital. One-way sensitivity analyses showed 10/102 scenarios in which gene therapy was not cost-effective compared to alternative treatments. Notably, gene therapy remained cost-effective in a hypothetical scenario in which we estimated that the discounted factor concentrate price was 20% of the wholesale acquisition cost in the US. Probabilistic sensitivity analysis estimated gene therapy cost-effective at 92% of simulations considering $150,000/QALY threshold. In conclusion, based on detailed simulation inputs and assumptions, gene therapy was more cost-effective than on-demand treatment and prophylaxis for patients with severe hemophilia B.

BackgroundPaediatric Early Warning Systems (PEWSs) improve identification of deterioration, however, their sustainability has not been studied. Sustainability is critical to maximise impact of interventions like PEWS, particularly in low-resource settings. This study establishes the reliability and validity of a Spanish-language Clinical Sustainability Assessment Tool (CSAT) to assess clinical capacity to sustain interventions in resource-limited hospitals.MethodsParticipants included PEWS implementation leadership teams of 29 paediatric cancer centres in Latin America involved in a collaborative to implement PEWS. The CSAT, a sustainability assessment tool validated in high-resource settings, was translated into Spanish and distributed to participants as an anonymous electronic survey. Psychometric, confirmatory factor analysis (CFA), and multivariate analyses were preformed to assess reliability, structure and initial validity. Focus groups were conducted after participants reviewed CSAT reports to assess their interpretation and utility.ResultsThe CSAT survey achieved an 80% response rate (n=169) with a mean score of 4.4 (of 5; 3.8–4.8 among centres). The CSAT had good reliability with an average internal consistency of 0.77 (95% CI 0.71 to 0.81); and CFAs supported the seven-domain structure. CSAT results were associated with respondents’ perceptions of the evidence for PEWS, its implementation and use in their centre, and their assessment of the hospital culture and implementation climate. The mean CSAT score was higher among respondents at centres with longer time using PEWS (p<0.001). Focus group participants noted the CSAT report helped assess their centre’s clinical capacity to sustain PEWS and provided constructive feedback for improvement.ConclusionsWe present information supporting the reliability and validity of the CSAT tool, the first Spanish-language instrument to assess clinical capacity to sustain evidence-based interventions in hospitals of variable resource levels. This assessment demonstrates a high capacity to sustain PEWS in these resource-limited centres with improvement over time from PEWS implementation.

PURPOSE Although > 90% of children with cancer live in low- and middle-income countries, little is known about communication priorities and experiences of families in these settings. We examined communication priorities and the quality of information exchange for Guatemalan caregivers of children with cancer during diagnostic communication. METHODS A cross-sectional survey including items used in pediatric communication studies from high-income countries and novel questions was verbally administered to 100 caregivers of children with cancer in Guatemala. RESULTS Guatemalan caregivers prioritized communication functions of exchanging information (99%), fostering healing relationships (98%), decision making (97%), enabling self-management (96%), and managing uncertainty (94%) over responding to emotions (66%) and cultural awareness (48%). Almost all caregivers wanted as many details as possible about their child's diagnosis and treatment (96%), likelihood of cure (99%), and late effects (97%). Only 67% were always given the information they needed without asking for it, and most caregivers sometimes (56%) or always (18%) had questions they wanted to discuss but did not. Approximately half of the caregivers (54%) correctly identified their child's diagnosis, primary site, disease extent (localized v metastatic), proposed treatment length, and treatment intent (curative v palliative). Caregivers of children with leukemia were more likely to correctly identify all attributes than those whose children had solid tumors ( P < .001). CONCLUSION Caregivers in Guatemala prioritize many of the same aspects of diagnostic communication as parents in the United States, and experience similar challenges. Shared communication values offer potential for adaptation of communication interventions across settings with varying resources and diverse cultures.

ObjectivesTo compare cardiopulmonary resuscitation (CPR) quality between manual CPR and miniaturized chest compressor (MCC) CPR. To improve CPR quality through evaluating the quality of our clinical work of resuscitation by real-time video recording system.MethodsThe study was a retrospective observational study of adult patients who experienced CPR at the emergency department of Shanghai Tenth People’s Hospital from March 2013 to August 2014. All the performance of CPR were checked back by the record of “digital real-time video recording system”. Average chest compression rate, actual chest compression rate, the percentage of hands-off period, time lag from patient arrival to chest compression, time lag from patient arrival to manual ventilation, time lag from patient arrival to first IV establish were compared. Causes of chest compression hands-off time were also studied.Results112 cases of resuscitation attempts were obtained. Average chest compression rate was over 100 compression per minute (cpm) in the majority of cases. However, indicators such as percentage of hands-off periods, time lag from patient arrival to the first manual ventilation and time lag from patient arrival to the first IV establish seemed to be worse in the manual CPR group compared to MCC CPR group. The saving of operators change time seemed to counteract the time spent on MCC equipment. Indicators such as percentage of hands-off periods, time lag between patient arrival to the first chest compression, time lag between patient arrival to the first manual ventilation and time lag from patient arrival to the first IV establish may influence the survival.ConclusionOur CPR quality remained to be improved. MCC may have a potentially positive role in CPR.