A randomized, placebo-controlled trial of prednisone in early Henoch Schönlein Purpura [ISRCTN85109383]

BACKGROUND: Henoch Schönlein Purpura (HSP) is the most common systemic vasculitis of childhood. There is considerable controversy over whether children with HSP should be treated with corticosteroids. The goal of this study was to investigate whether early corticosteroid administration could reduce the rate of renal or gastrointestinal complications in children with HSP. METHODS: Forty children with HSP, seen in the emergency room of a tertiary-care, paediatric centre, entered a randomized, double-blind, placebo controlled study. The treatment group (n = 21) received oral prednisone, 2 mg/kg/day for one week, with weaning over a second week, while the placebo group (n = 19) received an identical appearing placebo. Co-primary outcomes were the rate of renal involvement at one year and the rate of acute gastrointestinal complications. Co-primary outcomes were analysed using Fisher's Exact test. RESULTS: At one year, there was no difference in the rate of renal involvement (3/21 prednisone group vs. 2/19 placebo group, P = 1.0). There was also no statistically significant difference in the rate of acute gastrointestinal complications (2/21 prednisone group vs. 3/19 placebo group, P = 0.7). Two children in the placebo group did experience intussusceptions compared with none in the prednisone group (P = 0.2). CONCLUSIONS: Early prednisone therapy in HSP does not appear to reduce the risk of renal involvement at one year, or the risk of acute gastrointestinal complications. There may be a reduced risk of intussusception. The routine, early use of prednisone in uncomplicated HSP cannot be recommended at this time

Optimizing the operation of an electrodiagnostic laboratory during the COVID-19 pandemic: A 6-month single-center experience.

INTRODUCTION/AIMS: The initial surge of the coronavirus disease-2019 (COVID-19) pandemic in early 2020 led to widespread cancellation of elective medical procedures in the United States, including nonurgent outpatient and inpatient electrodiagnostic (EDx) studies. As certain regions later showed a downtrend in daily new cases, EDx laboratories have reopened under the guidance of the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM). In our reopening experience guided by the AANEM, we measured relevant outcomes to determine further workflow adaptations. We aimed to detail our experience and share the lessons learned. METHODS: We reviewed the clinical volumes, billing data, diagnosis distributions, and rates of COVID-19 exposure and transmission among patients and staff in our EDx laboratory during the first 6 months of reopening, starting on June 1, 2020. For context, we detailed the recent AANEM guidelines we adopted at our laboratory, supplemented by other consensus statements. RESULTS: We completed 816 outpatient studies from June 1 to December 1, 2020, reaching 97% of the total volume and 97% of total billing compared with the same time period in 2019. The average relative value units per study were similar. There were no major shifts in diagnosis distributions. We completed 10 of 12 requested inpatient studies during this period. There were no known COVID-19 transmissions between patients and staff. DISCUSSION: Our experience suggests that it is possible to safely operate an EDx laboratory under the guidance of the AANEM and other experts, with clinical volume and billing rates comparable to pre-pandemic baselines

Disparate healthcare access and telehealth-based hybrid consultations during the COVID-19 pandemic.

BACKGROUND: The coronavirus disease-2019 pandemic led to rapid expansion of telehealth services. This was speculated to improve healthcare access among underserved populations, including individuals unable to take time off work or arrange transportation. OBJECTIVE: We completed a quality improvement project to evaluate the feasibility of hybrid consultations that combined televisits and abbreviated in-person visits for neuromuscular referrals. METHODS: Using a censoring date of August 5, 2021, we reviewed all outpatient neuromuscular consultations from August 5, 2020 to February 5, 2021. For both hybrid and traditional in-person consultations, we reviewed no-show rates, completion rates of ordered diagnostic workup, and billing codes. For hybrid consultations only, we also reviewed intervals between initial televisit and subsequent examination and rates of video-enhanced versus audio-only televisits. RESULTS: During the study period, we completed 153 hybrid and 59 in-person new-patient consultations (no-show rates 9% and 27% respectively.) For hybrid consultations, 77% and 73% of laboratory and imaging studies were completed respectively, compared to 89% and 91% for in-person consultations. For hybrid visits, average RVUs (a marker for reimbursement) per consultation depended on whether audio-only televisits were billed as telephone calls or E/M visits per insurance payer rules, while video-enhanced televisits were uniformly billed as E/M visits. This resulted in average RVUs between 2.09 and 2.26, compared to 2.30 for in-person consultations. CONCLUSIONS: Telehealth-based hybrid neuromuscular consultations are feasible with minor caveats. However, the future of telehealth may be restricted by decreasing reimbursement rates particularly for audio-only televisits, limiting its potential to improve healthcare access

Neurofilament light chain kinetics as a biomarker for polyneuropathy in V122I hereditary transthyretin amyloidosis

Neurofilament light chain kinetics as a biomarker for polyneuropathy in V122I hereditary transthyretin amyloidosi

Incobotulinum Toxin-A in Professional Musicians With Focal Task-Specific Dystonia: A Double Blind, Placebo Controlled, Cross-Over Study

BACKGROUND: Musician\u27s focal task-specific dystonia is a complex disorder of fine motor control, with incomplete understanding of its etiology. There have been relatively few trials of botulinum toxin in upper limb task-specific dystonia, and prior studies have yielded variable results, leading to skepticism regarding the utility of this approach in elite performers. METHODS: We conducted a double-blind, placebo-controlled, randomized, cross-over study of incobotulinum toxin-A in 21 professional musicians with focal upper extremity task-specific dystonia affecting performance on their instrument, using a novel paradigm of initial injections followed by booster injections at two- and four-week intervals. The primary outcome measure was the change in blinded dystonia rating of the active arm by two expert raters using a Clinical Global Impression numeric scale at week 8 compared to enrollment. FINDINGS: 19 men and 2 women with musicians\u27 dystonia were enrolled over a six-year period. Nineteen patients completed the study. Analysis of the primary outcome measure in comparison to baseline revealed a change in dystonia severity of P = 0.04 and an improvement in overall musical performance of P = 0.027. No clinically significant weakness was observed, and neutralizing antibodies to toxin were not found. INTERPRETATION: Despite its small sample size, our study demonstrated a statistically significant benefit of incobotulinum toxin-A injections as a treatment for musicians\u27 task-specific dystonia. Tailoring the use of toxin with booster injections allowed refinement of dosing strategy and outcomes, with benefits that were meaningful to patients clearly visible on videotaped evaluations. In addition to its application to musicians\u27 dystonia, this approach may have relevance to optimize application of botulinum toxin in other forms of focal dystonia such as blepharospasm, cervical dystonia, writer\u27s cramp, and spasmodic dysphonia