Anthropometric measurement standardization for a multicenter nutrition survey in children with spinal muscular atrophy

Abstract

Spinal muscular atrophy (SMA) is a neuromuscular disease associated with nutritional status derangement and altered body composition. New drugs are changing the natural history of the disease, so now more than ever is important to focus on the correct assessment of nutritional status in SMA. We implemented a standardization process for the anthropometric measurement as part of our ongoing longitudinal study of growth patterns in SMA patients. It features a procedural manual, included in this communication, observers training and reliability assessment, of which we publish the values obtained in our pilot study. The standardization process was able to produce inter-observer reliability values in agreement with the literature and a procedure manual is now available for multicentre studies of nutritional status and body composition in SMA and possibly other pediatric neuromuscular disorders.

Predictive energy equations for spinal muscular atrophy type I children

“…The greatest strength of this study is both the relatively large sample size of treated and naïve SMA patients, and the quality of data; it included patients from an ongoing longitudinal study on nutritional aspects in SMA children which enabled us to prospectively collect the parameters with standardized procedures by the same dietitians (28). In addition, REE was measured under strictly controlled conditions and by a single center.…”

“…All anthropometric measurements were collected by 2 welltrained dietitians and had been standardized by applying conventional criteria (27) and recognized measuring procedures, as described previously (28). Specifically, BW was measured using a wheelchair scale to the nearest 100 g: the child and wheelchair were weighed together, then the wheelchair was weighed alone, and the difference in the 2 measurements gave the weight of the subject.…”

“…TL, approximated to the nearest 0.5 cm, was taken along the child's left side by a nonelastic tape and with the child lying supine on the exam table, as with the SL measurement. TL was measured from the proximal end of the medial border of the tibia to the distal tip of the medial malleolus (28).…”

“…To test whether age, BW, SL, TL, ventilatory status, and nusinersen treatment had a significant impact on energy expenditure, we plotted in Figure 1 the relations between these variables and 19 (16,25) 19 (16,41) 20 (17,28) Sex Girls 15 (12,20) 12 (11,15) 14 (14,15) 14 (13,17) 14 (13,17) BMI, kg Relation between age, weight, body, and tibia length and measured resting energy expenditure (mREE) according to the ventilatory status (yes compared with no) and nusinersen treatment (yes compared with no). A) Plots the relation between age, weight, supine and tibia length, and mREE according to the ventilatory status (yes compared with no).…”

The SMA Clinical Trial Readiness Program: creation and evaluation of a program to enhance SMA trial readiness in the United States

“…Major advances began with identification of the genes SMN1 and SMN2, the latter of which is a partially functional analog whose copy number is inversely correlated with disease severity [3,16]. Subsequent advances included development of mouse models; therapeutic target identification; emergence of new therapeutic modalities; natural history studies; development of standard of care; and establishment of reliable, sensitive, and meaningful outcome measures for SMA [4,5,7,8,15,[17][18][19][20][21][22][23][24][25][26][27][28]. These advances enabled therapeutic pipeline growth and paved the way for clinical trials in SMA, the first of which began in 2011 [5,17,29,30].…”