July 2017 ENCALS statement on edaravone

Peer reviewe

Intravenous immunoglobulin treatment in a patient with adrenomyeloneuropathy

Abstract Background Adrenomyeloneuropathy (AMN) is one of several phenotypes of the adrenoleukodystrophy spectrum caused by mutations in the ABCD1 gene on the X chromosome. An inflammatory component is part of the disease complex ranging from severe childhood CNS demyelination to spinal cord and peripheral nerve degeneration. Case presentation We present a patient with clinical progressive AMN and severe lower limb pain. Longitudinal brain magnetic resonance spectroscopy showed a constant slightly elevated myoinositol/total creatine ratio during the five year treatment period, probably reflecting demyelination, microglial activation and gliosis, indicating an inflammatory response. The pain was refractory to conventional therapy but intravenous immunoglobulin (IVIG) treatment was highly efficient. Conclusion IVIG may be considered as a last resort for treatment of refractory pain in AMN patients with indications of an inflammatory component.</p

Intravenous immunoglobulin treatment in a patient with adrenomyeloneuropathy

BACKGROUND: Adrenomyeloneuropathy (AMN) is one of several phenotypes of the adrenoleukodystrophy spectrum caused by mutations in the ABCD1 gene on the X chromosome. An inflammatory component is part of the disease complex ranging from severe childhood CNS demyelination to spinal cord and peripheral nerve degeneration. CASE PRESENTATION: We present a patient with clinical progressive AMN and severe lower limb pain. Longitudinal brain magnetic resonance spectroscopy showed a constant slightly elevated myoinositol/total creatine ratio during the five year treatment period, probably reflecting demyelination, microglial activation and gliosis, indicating an inflammatory response. The pain was refractory to conventional therapy but intravenous immunoglobulin (IVIG) treatment was highly efficient. CONCLUSION: IVIG may be considered as a last resort for treatment of refractory pain in AMN patients with indications of an inflammatory component

High incidence of amyotrophic lateral sclerosis in the Faroe Islands 2010–2020

The Faroese population isolate harbors epidemiological and genetic characteristics that likely differ from outbred populations. This population‐based register study found that the Faroese 2010–2020 crude incidence of amyotrophic lateral sclerosis (ALS) was 4.9/100,000 person‐years (95% confidence interval [CI], 3.3–7.0) and the age‐ and sex‐standardized incidence (US 2010 Census Population) was 4.1/100,000 person‐years (95% CI, 2.7–6.0), which is a 68% increase from the 1987–2009 estimate. The 2020 crude prevalence was 9.5/100,000 (95% CI, 3.0–19.6) in a population of 52,912 inhabitants. Incidence and prevalence estimates of ALS in the Faroes are high and further research is warranted to uncover the genetic or environmental determinants of ALS in this population