One Year Outcome of Boys With Duchenne Muscular Dystrophy Using the Bayley-III Scales of Infant and Toddler Development

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• 作者：Anne M. Connolly ; MD^a ; ^b ; ^{connollya@wustl.edu" class="auth_mail} ; Julaine M. Florence ; PT ; DPT^a ; Mary M. Cradock ; PHD^b ; Michelle Eagle ; PhD ; MSc ; MCSPⁱ ; ^j ; Kevin M. Flanigan ; MD^d ; ^e ; Craig M. McDonald ; MD^f ; Peter I. Karachunski ; MD^g ; Basil T. Darras ; MD^h ; Kate Bushby ; MDⁱ ; ^j ; Elizabeth C. Malkus ; PT ; MHS^a ; Paul T. Golumbek ; MD ; PhD^a ; ^b ; Craig M. Zaidman ; MD^a ; ^b ; J. Philip Miller ; PhD^c ; Jerry R. Mendell ; MD^d ; ^e ; MDA DMD Clinical Research Network
• 关键词：Duchenne muscular dystrophy ; Bayley-III ; infant development ; clinical trial outcomes
• 刊名：Pediatric Neurology
• 出版年：June 2014
• 年：2014
• 卷：50
• 期：6
• 页码：557-563
• 全文大小：367 K

文摘

The pathogenesis of Duchenne muscular dystrophy starts before birth. Despite this, clinical trials exclude young boys because traditional outcome measures rely on cooperation. We recently used the Bayley-III Scales of Infant and Toddler Development to study 24 infants and boys with Duchenne muscular dystrophy. Clinical evaluators at six centers were trained and certified to perform the Bayley-III. Here, we report 6- and 12-month follow-up of two subsets of these boys.

Patients

Nineteen boys (1.9 ± 0.8 years) were assessed at baseline and 6 months. Twelve boys (1.5 ± 0.8 years) were assessed at baseline, 6, and 12 months.

Results

Gross motor scores were lower at baseline compared with published controls (6.2 &plusmn; 1.7; normal 10 &plusmn; 3; m>Pm> < 0.0001) and revealed a further declining trend to 5.7 &plusmn; 1.7 (m>Pm> = 0.20) at 6 months. Repeated measures analysis of the 12 boys monitored for 12 months revealed that gross motor scores, again low at baseline (6.6 &plusmn; 1.7; m>Pm> < 0.0001), declined at 6 months (5.9 &plusmn; 1.8) and further at 12 months (5.3 &plusmn; 2.0) (m>Pm> = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, m>Pm> = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, m>Pm> = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (m>Pm> = 0.05).

Conclusion

Development can reliably be measured in infants and young boys with Duchenne muscular dystrophy across time using the Bayley-III. Power calculations using these data reveal that motor development may be used as an outcome measure.