Online assessment of ALS functional rating scale compares well to in-clinic evaluation: A prospective trial

Self-assessment of symptom progression in chronic diseases is of increasing importance in clinical research, patient management and specialized outpatient care. Against this background, we developed a secure internet platform (ALShome.de) that allows online assessment of the revised ALS Functional Rating Scale (ALSFRS-R) and other established self-assessment questionnaires. We developed a secure and closed internet portal to assess patient reported outcomes. In a prospective, controlled and stratified study, patients conducted a web-based self-assessment of ALSFRS-R compared to on-site assessment. On-site and online assessments were compared at baseline (n = 127) and after 3.5 months (n = 81, 64%). Results showed that correlation between on-site evaluation and online testing of ALSFRS-R was highly significant (r = 0.96; p < 0.001). The agreement of both capturing methods (online vs. on-site) was excellent (mean interval, 8.8 days). The adherence to online rating was high; 75% of patients tested on-site completed a follow-up online visit (mean 3.5 months, SD 1.7). We conclude that online self-assessment of ALS severity complements the well-established face-to-face application of the ALSFRS-R during on-site visits. The results of our study support the use of online administration of ALSFRS-R within clinical trials and for managing the care of ALS patients

ALSFRS and appel ALS scores: discordance with disease progression

Progression of disease and effectiveness of therapy in patients with amyotrophic lateral sclerosis (ALS) are determined by both questionnaire- and examination-based measures. To determine whether both types of measurement tools are equally predictive at all stages of disease, we compared questionnaire-based ALS Functional Rating Scale (ALSFRS) scores to the examination-based Appel ALS (AALS) scores at different stages of disease. Same-day scores were obtained during 174 visits in 62 patients with definite or probable ALS. Using normalized scores, correlation between the scales and predictability were best in mildly affected patients. Predictions of ALSFRS based on AALS scores were less than half as precise in the later stages of disease. Both scales showed significant change with disease progression, but ALSFRS consistently underestimated disease severity defined by AALS (P > 0.001). Questionnaire-based measurements should be compared against objective scales at all stages of disease severity before they are accepted as primary endpoint measures

High blood pressure and decreased heart rate variability in the Cuban epidemic neuropathy

Autonomic cardiovascular reflexes were investigated in patients with an epidemic optic and peripheral neu-ropathy, which affected more than 50 000 people in Cuba between 1991 and 1994 and was probably caused by nutritional deficiency. Affected patients had signifi-cantly higher blood pressure than age matched controls, both while supine and standing, and significantly lower heart rate variability during paced breathing, suggesting reduced cardiac parasympathetic innervation. Between 1991 and 1994, an epidemic optic and peripheralneuropathy affected more than 50 000 people in Cubaamong a population of 10.8 million.1 2 The neuropathy coincided with a period of dramatic worsening of the island’s economic situation and has been linked to deficiency of nutri-ents, including methionine, vitamin B-12, riboflavin, and carotenoids, because widespread distribution of vitamin sup-plements, which began in April 1993, effectively halted the occurrence of new cases.3 A strong association with cigar smoking and increased cassava consumption, both of which contain cyanide, has also been found.3 Affected patients presented with retrobulbar optic neu-ropathy and a predominantly sensory peripheral neuropathy.4 Most patients also had diarrhoea, palpitations, and genitouri-nary disorders of varying severity suggesting involvement of autonomic fibres.5 Autonomic involvement was also suggested by low amplitude sympathetic skin responses.5 To determine whether the neuropathy produced long lasting effects on autonomic function, we investigated autonomic cardiovas-cular reflexes in patients who had been severely affected dur-ing the epidemic