Clinical efficacy of the enzyme replacement therapy in patients with late-onset Pompe disease: a systematic review and a meta-analysis.

In patients with late-onset Pompe disease (LOPD), the efficacy of the enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA) is difficult to evaluate, due to the clinical heterogeneity and the small sample sizes in published studies. Therefore, we conduct a systematic literature review and meta-analysis of the literature to evaluate the efficacy of ERT in LOPD patients considering the walking distance, respiratory function and muscle strength. Particularly, six-minute walk test (6MWT), forced vital capacity (FVC), medical research council (MRC) grading, quantitative muscle testing (QMT), and quick motor function test (QMFT) were outcomes of interest. Overall, 619 studies were identified in PubMed, EMBASE and by manual search on July 18th, 2020. After an initial assessment, 16 studies were included in the meta-analysis, containing clinical data from 589 patients with LOPD. For the 6MWT, 419 patients were analyzed. Walking distance improved on average, 32.2 m greater during the observed period (p = 0.0003), compared to the distance at the baseline. The meta-analysis did not show any improvement in FVC and only a tendency towards better muscle strength after treatment with ERT, but the difference was not statistically significant. In conclusion, the available data showed that ERT has a significant beneficial efficacy in the improvement of walking distance in LOPD patients and a non-significant improvement of muscle strength. No improvement in respiratory capacity was found. More prospective and controlled trials are needed to demonstrate a clear clinical benefit of ERT.

Regional Versus General Anesthesia: Effect of Anesthetic Techniques on Clinical Outcome in Lumbar Spine Surgery: A Prospective Randomized Controlled Trial.

BACKGROUND: There are only a few prospective clinical trials investigating the effects of different anesthetic techniques on clinical outcomes after lumbar spine surgery. The purpose of this study was to evaluate clinical outcomes in patients receiving general (GA) and regional anesthesia (RA) for lumbar spine surgery. METHODS: This was a single-center, 2-arm, trial in which 100 patients undergoing lumbar spine surgery were randomized to receive either RA or GA (50 per group). The primary endpoint was morphine consumption during the first postoperative 48 hours. In addition, anesthesia time, transition time (defined as time from end of surgery to admission to the postoperative anesthesia care unit), visual analogue scale (VAS) for pain, and patient satisfaction at hospital discharge were recorded. RESULTS: There was no difference in the primary endpoint (cumulative morphine consumption at 48 h) between the 2 anesthesia types. Anesthesia and transition times were significantly shorter in the RA compared with the GA group-anesthesia time 125.4±23.6 minutes for GA versus 99.4±13.5 minutes for RA, transition time 22.5 minutes for GA versus 10.0 minutes for RA (both P<0.001). The VAS for pain on arrival to the postoperative anesthetic care unit was lower for patients who received RA compared with GA (crude and adjusted, both <0.001). 84% of patients in the RA group were completely satisfied compared with 74% in the GA group (P<0.001). There was a significant difference in the sex analysis for VAS for pain over time; females reported higher VAS for pain from the preoperative assessment to 6 weeks after the operation (P<0.001). CONCLUSIONS: There was no difference in postoperative morphine consumption in patients receiving GA and RA for lumbar spine surgery. RA was associated with shorter anesthesia and transition times, lower VAS for pain at arrival at the postoperative anesthesia care unit, and higher patient satisfaction at hospital discharge.

Sexual and urinary function following anterior lumbar surgery in females.

AIMS: Anterior lumbar interbody fusion procedures (ALIF) and total disc replacement (TDR) with anterior exposure of the lumbar spine entail a risk of a vascular injury and dysfunction of the sympathetic and parasympathetic nerves due to disturbance of the inferior and superior hypogastric plexus. While retrograde ejaculation is a known complication of the anterior spinal approach in males, post-operative sexual as well as urinary function in females has not yet been thoroughly investigated and was hence the aim of this study. METHODS: Fifteen female patients documented their sexual and urinary function preoperatively, 3 months and 6 months postoperatively, using the validated questionnaires FSFI (Female Sexual Function Index) and ICIQ (International Consultation of Incontinence Questionnaire). Randomization tests were used to statistically analyze expectation values over time (two-sided, P < 0.05). RESULTS: While no statistically significant change in the total FSFI score occurred over time, a significant increase in FSFI desire score was noted between preoperative (2.95 ± 0.8) and 6 months follow-up (3.51 ± 0.6, P = 0.02). Urinary continence remained unchanged over time. CONCLUSION: In summary, ALIF and lumbar TDR do not seem to negatively influence sexual and urinary function in females. In contrast, increased sexual desire was noted, likely secondary to post-surgical pain relief.