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OBJECTIVES: Abdominal Aortic Aneurysms (AAA) in females are less prevalent, have higher expansion rates and experience rupture at smaller diameters than in males. Studies have compared outcomes of the retroperitoneal (RP) and transperitoneal (TP) approach in open aortic aneurysm repair (OAR) with conflicting results. No study to date has compared the two approaches solely in females. In this study we compare midterm outcomes of the RP and TP approach in females undergoing OAR. METHODS: Single-center, retrospective review of all females undergoing OAR from 2010 to 2021. Patients undergoing elective, symptomatic and ruptured OAR were included. The cohort was stratified by surgical approach RP versus TP and midterm outcomes were compared amongst the groups. Outcomes included mortality, graft related, and non-graft related complications. RESULTS: A total of 244 patients (RP n = 133; TP n = 111) were identified. Follow-up period was 28 ± 30.7 months. Baseline perioperative characteristics were similar except that more people in the RP group had ejection fraction ((EF) > 50% (82% vs 68%), p = .037). Patients who underwent RP repair had longer visceral/renal ischemia time (p = .01), larger graft diameter (18 vs 16 mm; p = <0.001), were more likely to have a suprarenal clamp placed(70.5 vs 48.2; p < .001), and had decreased autotransfusion volume (611 vs 861 mL; p < .01) compared to those who underwent TP repair. Number of deaths was higher in the TP group during study follow-up period (36.4 vs 23.8; p = .035), but the difference of the time to event analysis was not significant. There was no difference in all-cause survival at 36 months between RP and TP (77.8 vs 76.8; p = .045). Overall midterm complications were 9.5% in both groups. Any graft related complication was 1.8% in TP versus 3% RP (p = .69). In a multivariable model, after adjusting for age, urgency, smoking, prior aneurysm repair, and ASA level, the hazard ratio decreases with the RP approach, however this did not reach significance (p = .052). CONCLUSION: In a 12-year period of OAR in females, TP and RP results were comparable at midterm analysis. The RP approach appeared to be used more often for OAR requiring suprarenal clamping. Although the TP group had increased mortality, the difference of the time to event analysis was not significant. Midterm postoperative complications in both groups were low. This suggests that both approaches are safe in the female population and decision should be driven by anatomy and surgeon's preference.
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BACKGROUND: There is significant underutilisation of allocated health service resources when a scheduled flexible cystoscopy (FC) is cancelled because a pre-cystoscopy urinalysis (PCU) suggests "infection", despite patients being asymptomatic for urinary tract infection (UTI). OBJECTIVE: To evaluate the risk of UTI or urinary sepsis when FC is performed in asymptomatic patients with a PCU positive for leucocyte esterase and/or nitrites. DESIGN SETTING AND PARTICIPANTS: A prospective cohort study was conducted in a high-volume UK centre recruiting all patients undergoing outpatient FC. INTERVENTION: A protocol was developed to guide response to PCU performed prior to FC, which was performed regardless of the result, unless patients were symptomatic for UTI. All patients completed a questionnaire to identify risk factors and were followed up via a telephone survey and a review of electronic clinical records. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: Post-FC UTI was defined as hospital admission with UTI/urinary sepsis or if patients were symptomatic for UTI with receipt of antibiotics or with positive urine culture and sensitivity. An analysis of the association was performed. RESULTS AND LIMITATIONS: An initial pilot study confirmed the safety and feasibility of our protocol. Of 1996 patients, 136 (6.8%) developed a UTI by our definition, with 51 (2.6%) having a culture-proven infection. The risk was higher in patients with a positive PCU (odds ratio [OR] 1.61, 95% confidence interval [CI] = 1.07-2.40, p = 0.02), history of UTI (OR 1.72, 95% CI = 1.09-2.73, p = 0.02), or a bladder tumour on FC (OR 2.22, 95% CI = 1.27-3.90, p = 0.005). No patient with a positive PCU developed urinary sepsis. The main limitation of this study was the lack of pre-protocol control. CONCLUSIONS: We observed a clinically low and acceptable risk of UTI, with no incidence of sepsis, when FC was performed in asymptomatic patients with a PCU suggesting "infection". Routine cancellation of these patients is unnecessary and may worsen the burden on health service resources. PATIENT SUMMARY: We evaluated the safety of performing flexible cystoscopy when the urine dipstick on the day suggested presence of an "infection" but the patient had no symptoms of urinary tract infection (UTI). Our study in over 2000 patients demonstrated a low incidence of UTI, and none of these patients developed sepsis. We therefore recommend that flexible cystoscopy should not be cancelled automatically on the basis of the dipstick result alone, as it might delay a time-sensitive crucial diagnosis.
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AIMS: The success of the Regulation on Orphan Medicinal Products in the European Union is evidenced by the 127 orphan drugs that have had market authorization since 2000. However, the incentives aimed at stimulating research and development have had the unintended consequence of increasing drug cost, resulting in many orphan drugs not being cost-effective. Orphan drugs command an increasing share of the pharmaceutical market and account for a disproportionate amount of healthcare expenditure. Orphan drug ownership by socially motivated, not-for-profit organizations may facilitate access to more affordable orphan drugs, for the benefit of patients and healthcare systems alike. This study aims to describe opportunities for such organizations to become orphan drug Market Authorization Holders. METHODS: We reviewed data on the ownership of EMA designated and approved orphan drugs, identified funding opportunities and business models for not-for-profit organizations, and summarised relevant legal and policy documents concerning intellectual property rights and drug regulation. RESULTS: Using repurposed drugs as a paradigm, this narrative review navigates the regulatory hurdles, describes the legal context and identifies funding opportunities, in a bid to facilitate and encourage not-for-profit organizations to lead on the development of affordable orphan drugs. CONCLUSIONS: Although the regulatory steps required to obtain an MA for an orphan drug are numerous and challenging, they are not insurmountable and can be achieved by not-for-profit organizations that are socially motivated to reduce the costs of orphan drugs to the payers of healthcare. Opportunities for orphan drug development resulting in affordable products lie mainly with repurposed drugs.
