Responsiveness of clinical outcome measures in Charcot-Marie-Tooth disease.

BACKGROUND AND PURPOSE: Charcot-Marie-Tooth disease (CMT) is a very slowly progressive neuropathy which makes it difficult to detect disease progression over time and to assess intervention efficacy. Experience from completed clinical trials with ascorbic acid and natural history studies confirm difficulties in detecting such changes. Consequently, sensitive-to-change outcome measures (OMs) are urgently needed. METHODS: The relative responsiveness of clinical scales of the Italian-UK ascorbic acid trial (placebo arm) were assessed by using the standardized response mean (SRM), which is the ratio of the paired scores mean change over time to the standard deviation of the score change (0 is worst responsiveness). RESULTS: Little worsening of OM scores was found over 2 years. In detail, the primary OM of the trial, the CMT Neuropathy Score version 1 (CMTNSv1), showed low responsiveness (SRM 0.13). Some CMTNS items showed slightly greater responsiveness (CMT Examination Score 0.17; CMTNS Signs 0.19). Myometric assessments of handgrip and foot dorsiflexion strength were the most responsive (SRM -0.31 and -0.38, respectively). Amongst the other measures, the nine-hole peg test, which assesses upper limb functioning, showed the best sensitivity to change (SRM 0.28). CONCLUSIONS: Overall these OMs showed low or negligible responsiveness, confirming the need to improve current OMs and to develop novel ones for prognostic and interventional studies. However, handgrip and foot dorsiflexion myometry are worth retaining for future trials as they were the most responsive and are likely to be clinically relevant for patients.

Is overwork weakness relevant in Charcot-Marie-Tooth disease?

BACKGROUND: In overwork weakness (OW), muscles are increasingly weakened by exercise, work or daily activities. Although it is a well-established phenomenon in several neuromuscular disorders, it is debated whether it occurs in Charcot-Marie-Tooth disease (CMT). Dominant limb muscles undergo a heavier overload than non-dominant and therefore if OW occurs we would expect them to become weaker. Four previous studies, comparing dominant and non-dominant hand strength in CMT series employing manual testing or myometry, gave contradictory results. Moreover, none of them examined the behaviour of lower limb muscles. METHODS: We tested the OW hypothesis in 271 CMT1A adult patients by comparing bilateral intrinsic hand and leg muscle strength with manual testing as well as manual dexterity. RESULTS: We found no significant difference between sides for the strength of first dorsal interosseous, abductor pollicis brevis, anterior tibialis and triceps surae. Dominant side muscles did not become weaker than non-dominant with increasing age and disease severity (assessed with the CMT Neuropathy Score); in fact, the dominant triceps surae was slightly stronger than the non-dominant with increasing age and disease severity. DISCUSSION: Our data does not support the OW hypothesis and the consequent harmful effect of exercise in patients with CMT1A. Physical activity should be encouraged, and rehabilitation remains the most effective treatment for CMT patients.

Importancia de la terapia básica pre-tratamiento ortodóncico / The importance of pre-orthodontic treatment basic therapy

Introducción: la capacidad de los microorganismos para formar y mantenerse en el biofilm tiene un alto impacto en las infecciones crónicas, pues el mismo protege y nutre a comunidades de microorganismos que pueden influir en el tratamiento ortodóncico, aumentando la incidencia de caries y enfermedad periodontal. Objetivo: comparar la condición periodontal inicial y la alcanzada luego de la terapia básica periodontal de los pacientes que concurren a la consulta para iniciar un tratamiento ortodóncico. Materiales y métodos: se evaluaron 10 pacientes entre 14 y 30 años que concurrieron al servicio de la Cátedra de Ortodoncia de la Facultad de Odontología de la UBA. Se tomaron el índice de placa de Silness y Loe y gingival de Loe y Silness, la profundidad de sondaje y hemorragia gingival al sondaje en todas las piezas dentarias presentes en la boca del paciente. De la zona de los primeros premolares superiores se eliminó la placa supragingival y se tomaron muestras subgingivales, las cuales fueron colocadas en solución fisiológica y medio de transporte VMGAIII. Simultáneamente se realizaron extendidos del materiales y se coloreó con la técnica de Gram y de Giemsa. Luego de que los pacientes recibieran enseñanza de técnicas de higiene bucal y/o terapia básica periodontal, se volvieron a registrar dichos índices. Resultados: el índice de placa inicial presentó una mediana de 2.5 y post terapia básica, una mediana de 1.0 con una diferencia estadísticamente significativa (p=0.005) según Wilcoxon Signed Rank Test. El indice gingival inicial presentó una mediana de 2.0 y post terapia básica fue de 1.0 con una diferencia estadísticamente significativa (p<0.005 Wilcoxon Signed Test). La profundidad de sondaje pretratamiento presentó una mediana de 3.0mm con sangrado al sondaje positivo y microbiota compatible con gingivitis y periodontitis leve. Luego de enseñanza de técnicas de higiene bucal la misma fue de 1 mm con sangrado al sondaje negativo.