Gold Coast criteria expand clinical trial eligibility in amyotrophic lateral sclerosis.

INTRODUCTION/AIMS: Consensus criteria to formalize the diagnosis of amyotrophic lateral sclerosis (ALS) and refine clinical trial populations have evolved. The recently proposed Gold Coast consensus criteria are intended to simplify use and increase sensitivity. We aimed to evaluate the potential impact of these criteria on clinical trial eligibility. METHODS: We performed a single-center, retrospective study of people diagnosed with ALS between 2016 and 2021 to determine the numbers of those meeting Gold Coast, revised El Escorial (rEEC) criteria, and Awaji criteria. We identified the proportion of those who would have been eligible for participation in three major ALS clinical trials if Gold Coast were used in place of rEEC definite/probable criteria. (rEEC D/P). RESULTS: Two hundred six people with ALS were included in our study. 48.5% met Gold Coast criteria but not rEEC D/P. Using the Gold Coast criteria would result in higher rates of clinical trial eligibility after other inclusion criteria were met: 95.2% vs 42.5% (P < .001) in a phase III study of riluzole; 100% vs 31.0% (P = .002) in a phase III study of edaravone; and 95.6% vs 45.3% (P < .001) in an ongoing phase III study of sodium phenylbutyrate and taurursodiol. The sensitivity of the Gold Coast criteria (96.1%; 95% confidence interval [CI], 92.2%-98.2%) was significantly higher than that of rEEC D/P (47.6%; 95% CI, 40.6%-54.6%; for difference, χ2  = 117.6; P < .001). DISCUSSION: Until robust biomarkers are available to diagnose ALS, consensus diagnostic criteria remain necessary. Gold Coast criteria would expand research and clinical trial eligibility and improve external validity of clinical trial results.

Is There an Association Between Ulnar Nerve Hypermobility and Neuropathy at the Elbow?

OBJECTIVE: To systematically review the association between ulnar nerve hypermobility (UNH) at the elbow and ulnar neuropathy (UNE). DATA SOURCES: Cumulative Index to Nursing and Allied Health, MEDLINE, and Embase databases were searched for English language studies published up to July 4, 2020. STUDY SELECTION: We included case-control, cohort, and randomized controlled studies that established the presence or absence of UNH and UNE. Twenty out of 654 studies identified met the inclusion criteria. DATA EXTRACTION: Two reviewers independently extracted data for analysis. Risk of bias and applicability were assessed with the QUADAS-2 tool. DATA SYNTHESIS: We compared rates of UNH between patients diagnosed with and without UNE and found no significant difference. The meta-analysis pooled rate of UNH was 0.37 (95% confidence interval, 0.20-0.57) for those without UNE and 0.33 (95% confidence interval, 0.23-0.45) for those with UNE. CONCLUSIONS: The clinical finding of UNH is unhelpful when assessing for UNE, as the presence of UNH does not make the diagnosis of UNE more likely.

A systematic review and meta-analysis of patient-reported outcomes following nerve transfer surgery for brachial plexus injury.

OBJECTIVE: The purpose of this systematic review and meta-analysis was to determine the effect of nerve transfer surgery for brachial plexus injury (BPI) on patient-reported outcomes. LITERATURE SURVEY: MEDLINE, EMBASE, and CINAHL were searched and screened in duplicate for relevant studies on September 25, 2019 and repeated June 24, 2020. METHODOLOGY: The patient population included male and female patients who received nerve transfer surgery for BPI. Further inclusion criteria were: (1) all levels of evidence; (2) published in English; (3) conducted in humans; and (4) report of patient-reported outcome. Two reviewers independently abstracted data regarding injury type, surgical technique, surgical timing, follow-up duration, strength, and patient-reported outcome measures. Quality was assessed in duplicate using the Methodological Index for Non-Randomized Studies criteria. SYNTHESIS: Twenty-one studies involving 464 participants were included in analyses. The mean age ± SD was 32.4 ± 1.8 (range: 5-77) and 89.2% of included participants were male. The mean time to surgery was 5.6 ± 0.6 months and the mean follow-up time was 48.1 ± 8.4 months. The most used patient-reported outcome was the Disabilities of Arm, Shoulder and Hand (DASH), where scores variably improved postoperatively, although the degree of disability remained high. Return to work was reported in five studies, with 66.0% of participants returning to work. Patient satisfaction was assessed in four studies with an overall satisfaction proportion of 64.0%. Pain was assessed in 12 studies using various measures. In studies reporting pain intensity postoperatively, 29.3% of patients were "pain-free" or had "no pain." CONCLUSIONS: Patient-reported outcomes following nerve transfer for BPI are infrequently reported in the literature. Although patient-reported outcomes demonstrate variable postoperative improvement, there is evidence of ongoing limitations and postoperative DASH scores report a high degree of ongoing disability. Future studies with greater reporting as well as validation of patient-reported outcomes within BPI are warranted.

Paediatric lumbar disc herniation in the very young: a case-based update.

BACKGROUND: A lumbar disc prolapse is commonly seen in the adult population and as a result, the signs and symptoms are well recognised and easily treated. A disc prolapse, in the adolescent and particularly the paediatric age group, is very rare indeed. The signs and symptoms are atypical in this age group, and there is often difficulty in recognising the pathology. This can lead to delays in the diagnosis and treatment of a very painful condition. DISCUSSION: Lumbar disc herniation in very young children can be hard to diagnose. There is limited information currently available in the literature on the topic of lumbar disc herniation in very young children. Overall, the surgical option in these children appears to have a favourable outcome. ILLUSTRATIVE CASE: This case highlights an 18-month-old child who was not able to walk, following a fall. The child had a normal neurological examination and normal bladder and bowel function. A magnetic resonance imaging (MRI) scan of her lumbar sacral spine indicated a large right paracentral L4/5 disc prolapse indenting the right anterolateral margin of the thecal sac and abutting the right L5 nerve root. She subsequently underwent a laminoplasty and excision of a sub-ligamentous L4/5 disc prolapse under an operating microscope. CONCLUSIONS: This case report highlights the problems in diagnosing an acute lumbar disc prolapse in an 18-month-old child and reviews the current literature dealing with an acute disc prolapse in very young children.

Perioperative Botulinum Toxin Injections to Enhance Surgical Outcomes in Patients With Spasticity: Preoperative, Intraoperative, and Postoperative Case Reports.

Spasticity causes an array of disabilities, which in turn may lead to the need for surgical intervention. Spasticity itself may also negatively affect surgical outcomes. This report reviews the potential benefit of perioperative (before, during, or after surgery) botulinum toxin (BoNT) injections for 3 patients with spasticity due to spinal cord injury, stroke, or multiple sclerosis. We discuss perioperative BoNT in 3 time periods: preoperatively, intraoperatively, and postoperatively. The cases demonstrate the use of perioperative BoNT in decreasing pain, improving wound healing, and improving surgical outcomes. We conclude by discussing the potential use of perioperative BoNT for surgical interventions in patients with spasticity and the need for further high-quality research in this field.