Real-world clinical experience with Idebenone in the treatment of Leber hereditary optic neuropathy

Background: Leber hereditary optic neuropathy (LHON) leads to bilateral central vision loss. In a clinical trial setting, idebenone has been shown to be safe and to provide a trend toward improved visual acuity, but long-term evidence of effectiveness in real-world clinical practice is sparse. Methods: Open-label, multicenter, retrospective, noncontrolled analysis of long-term visual acuity and safety in 111 LHON patients treated with idebenone (900 mg/day) in an expanded access program. Eligible patients had a confirmed mitochondrial DNA mutation and had experienced the onset of symptoms (most recent eye) within 1 year before enrollment. Data on visual acuity and adverse events were collected as per normal clinical practice. Efficacy was assessed as the proportion of patients with either a clinically relevant recovery (CRR) or a clinically relevant stabilization (CRS) of visual acuity. In the case of CRR, time to and magnitude of recovery over the course of time were also assessed. Results: At time of analysis, 87 patients had provided longitudinal efficacy data. Average treatment duration was 25.6 months. CRR was observed in 46.0% of patients. Analysis of treatment effect by duration showed that the proportion of patients with recovery and the magnitude of recovery increased with treatment duration. Average gain in best-corrected visual acuity for responders was 0.72 logarithm of the minimal angle of resolution (logMAR), equivalent to more than 7 lines on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. Furthermore, 50% of patients who had a visual acuity below 1.0 logMAR in at least one eye at initiation of treatment successfully maintained their vision below this threshold by last observation. Idebenone was well tolerated, with most adverse events classified as minor. Conclusions: These data demonstrate the benefit of idebenone treatment in recovering lost vision and maintaining good residual vision in a real-world setting. Together, these findings indicate that idebenone treatment should be initiated early and be maintained more than 24 months to maximize efficacy. Safety results were consistent with the known safety profile of idebenone

Peripherally inserted central catheters (PICCs) in the management of oncohematological patients submitted to autologous stem cell transplantation

The aim of our study was to evaluate the feasibility and the safety of the use of peripherally inserted central catheters (PICCs) during autologous peripheral blood stem cell transplantation. Sixty PICCs were inserted in 57 patients (23 females and 34 males; mean age 48, range 19-68\ua0years) and remained in place for an overall period of 1,276\ua0days. All PICCs were positioned by a team of specifically trained physicians and nurses and utilized by specifically trained nurses of our hematology unit. No major insertion-related complications were observed; the only complication during insertion was one local hematoma (1.6\ua0%) due to accidental arterial puncture. Late complications were accidental catheter removal (5\ua0%, 2.3 per 1,000 PICC days), symptomatic catheter-related venous thrombosis (5\ua0%, 2.3 per 1,000 PICC days), and catheter-related bloodstream infection (CRBSI; 3.3\ua0%, 1.5 CRBSI per 1,000 PICC days). The reasons for catheter removal were completion of therapy (42 patients, 70\ua0%), fever of unknown origin (9 patients, 15\ua0%), catheter-related thrombosis (2 patients, 3.3\ua0%), CRBSI (2 patients, 3.3\ua0%), accidental removal (3 patients, 5\ua0%), lumen occlusion (1 patient, 1.6\ua0%), positive culture from peripheral blood (1 patient, 1.6\ua0%), and death (1 patient, 1.6\ua0%). Our data suggest that PICCs are a safe and effective alternative to conventional central venous catheters even in patients particularly prone to infective and hemorrhagic complications such as patients receiving autologous stem cell transplantation

Real-World Clinical Experience With Idebenone in the Treatment of Leber Hereditary Optic Neuropathy

Leber hereditary optic neuropathy (LHON) leads to bilateral central vision loss. In a clinical trial setting, idebenone has been shown to be safe and to provide a trend toward improved visual acuity, but long-term evidence of effectiveness in real-world clinical practice is sparse

Asthma in patients admitted to emergency department for COVID-19: prevalence and risk of hospitalization

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