Evaluation of Discipline-Specific Outcomes Through a Multidisciplinary Team Clinic for Patients With Isolated Cleft Palate.

OBJECTIVE: To describe the incidence and timing of provider-specific interventions for children with isolated cleft palate. DESIGN: This was a retrospective cohort study involving review of medical records. SETTING: Multidisciplinary team care clinic at a tertiary academic children's hospital between January 2000 and July 2019. PATIENTS: Patients with isolated nonsyndromic cleft palate seen by an American Cleft Palate-Craniofacial Association-approved team; 138 children were included. MAIN OUTCOME MEASURES: Study outcomes included incidence of secondary velopharyngeal management, tympanostomy tube insertion, speech therapy, hearing loss, dental/orthodontic treatment, and psychology interventions. Provider-specific outcomes were calculated for patients at ages 0 to 3, 3 to 5, and >5 years. RESULTS: Median follow-up time was 7.0 years (interquartile range: 3.3-11.8 years). At their last team assessment, 42% of patients still had conductive hearing loss. The rate of tympanostomy tube insertions not done alongside a palatoplasty was highest for ages 3 to 5 and dropped after new American Academy of Otolaryngology-Head and Neck Surgery Foundation guidelines in 2013 (P = .015); 54% of patients received speech-language therapy during follow-up. Palatoplasty, psychology, and dental/orthodontic treatment were all less common than speech or ENT treatment (P < .01). Secondary palatoplasty was performed in 31 patients (22%). Patients who received speech, dental/orthodontic, or psychology intervention followed up longer than those who did not (9.8 vs 2.1 years, P < .001). CONCLUSION: Half of the patients terminated team follow-up by age 7, suggesting that burden of care outweighed perceived benefits of continued follow-up for many families. These results can be used to adjust protocols for children with isolated cleft palate.

Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.

INTRODUCTION: Glucocorticosteroids (GC) are effective in slowing weakness in boys with Duchenne muscular dystrophy (DMD). METHODS: This is a multisite, 1-year, open-label trial of twice-weekly prednisolone (5 mg/kg/dose) in infants/young boys (0.4-2.4 years) with DMD. We compared changes in Bayley III Scales of Infant Development (Bayley-III) with untreated boys followed for 1 year (historical control cohort [HCC]). Twenty-three of 25 participants completed the study. RESULTS: Treated boys gained an average of 0.5 points on the Bayley-III gross motor scaled score (GMSS) compared with the HCC who, on average, declined 1.3 points (P = 0.03). All boys maintained linear growth, and none developed Cushingoid features. Excessive weight gain occurred in 13 of 23 (56%) boys. DISCUSSION: This study provides evidence that twice-weekly GC is well tolerated in infants and young boys with DMD and improves GMSS. Excessive weight gain is a potential risk. Longer follow-up is required to determine whether early GC initiation is feasible in most infants/boys with DMD. Muscle Nerve 59:650-657, 2019.

One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development.

BACKGROUND: 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 ± 1.7; normal 10 ± 3; P < 0.0001) and revealed a further declining trend to 5.7 ± 1.7 (P = 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 ± 1.7; P < 0.0001), declined at 6 months (5.9 ± 1.8) and further at 12 months (5.3 ± 2.0) (P = 0.11). Cognitive and language scores were lower at baseline compared with normal children (range, P = 0.002-<0.0001) and did not change significantly at 6 or 12 months (range, P = 0.89-0.09). Fine motor skills, also low at baseline, improved >1 year (P = 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.

Motor and cognitive assessment of infants and young boys with Duchenne Muscular Dystrophy: results from the Muscular Dystrophy Association DMD Clinical Research Network.

Therapeutic trials in Duchenne Muscular Dystrophy (DMD) exclude young boys because traditional outcome measures rely on cooperation. The Bayley III Scales of Infant and Toddler Development (Bayley III) have been validated in developing children and those with developmental disorders but have not been studied in DMD. Expanded Hammersmith Functional Motor Scale (HFMSE) and North Star Ambulatory Assessment (NSAA) may also be useful in this young DMD population. Clinical evaluators from the MDA-DMD Clinical Research Network were trained in these assessment tools. Infants and boys with DMD (n = 24; 1.9 ± 0.7 years) were assessed. The mean Bayley III motor composite score was low (82.8 ± 8; p ≤ .0001) (normal = 100 ± 15). Mean gross motor and fine motor function scaled scores were low (both p ≤ .0001). The mean cognitive comprehensive (p=.0002), receptive language (p ≤ .0001), and expressive language (p = .0001) were also low compared to normal children. Age was negatively associated with Bayley III gross motor (r = -0.44; p = .02) but not with fine motor, cognitive, or language scores. HFMSE (n=23) showed a mean score of 31 ± 13. NSAA (n = 18 boys; 2.2 ± 0.4 years) showed a mean score of 12 ± 5. Outcome assessments of young boys with DMD are feasible and in this multicenter study were best demonstrated using the Bayley III.

Behavioral adjustment of toddler and preschool-aged children with single-suture craniosynostosis.

BACKGROUND: The purpose of this study was to confirm initial reports of elevated behavior problems in children with single-suture craniosynostosis, using multiple informants, longitudinal analyses, and a control group. The authors hypothesized that children with single-suture craniosynostosis would have higher levels of maladjustment than comparison children, particularly at the older age and in selected areas of previously observed vulnerability: attention and social adjustment. METHODS: The Child Behavior Checklist was completed by 436 mothers (219 with single-suture craniosynostosis) and 371 fathers (177 with single-suture craniosynostosis) when children were aged approximately 19 months, and by 361 mothers (175 with single-suture craniosynostosis) and 303 fathers (142 with single-suture craniosynostosis) when children were aged approximately 37 months. A minimum of one caregiver/teacher report was available for 169 of these children (74 with single-suture craniosynostosis) using the Caregiver-Teacher Report Form. RESULTS: Average Child Behavior Checklist/Caregiver-Teacher Report Form externalizing, internalizing, and total scores for all informants were consistently higher (worse) for children with single-suture craniosynostosis than for control group children, but most differences were small and statistically nonsignificant. No differences associated with suture site were found. At the oldest age point, both mothers and fathers (but not teachers) generated higher average scores for patients than for controls on scales measuring attention and social problems, with small to medium effect sizes (0.20 to 0.32). CONCLUSIONS: On average, toddlers/preschoolers with single-suture craniosynostosis show behavioral development that is largely indistinguishable from same-aged peers of similar socioeconomic background. The predictive significance of small group differences in attention and social adjustment will be assessed in a follow-up of this cohort at age 7.

Memory and response inhibition in young children with single-suture craniosynostosis.

Using two versions of the A-not-B task, memory and response inhibition were assessed in 17- to 24-month-old children with surgically corrected single-suture craniosynostosis (cases) and unaffected children (controls). Children's development and language were initially assessed on average at 6-7 months of age and again at this second visit. Cases and controls performed at equivalent levels on average, with cases performing slightly better than controls on several of the variables measured. However, fewer cases than controls were able to complete the more challenging of the two tasks, which may have predictive significance for later functioning. Children's age and cognitive ability were related to successful performance on the A-not-B task. Among cases, age of cranioplastic surgery was unrelated to performance. Our findings suggest that children with single-suture craniosynostosis show normal development of visual memory and response inhibition in the age range studied here.

Neurodevelopment of infants with single-suture craniosynostosis: presurgery comparisons with case-matched controls.

BACKGROUND: The hypothesized association between single-suture craniosynostosis and neurodevelopment remains unclear, given the methodologic limitations of previous studies, most notably the absence of control groups. METHODS: Standardized measures were used to assess the neurodevelopment of 125 matched case-control pairs shortly after cases were first diagnosed with isolated fusions of the sagittal, metopic, lambdoid, or right or left coronal sutures. Participants varied in age from 2 to 24 months. RESULTS: Cases had significantly lower mean standardized scores than controls on measures of cognitive ability and motor functioning (p < 0.02). These differences were unaffected by the location of synostosis, age of diagnosis, infant sex, and maternal IQ. Measures of early language functions revealed no group differences. CONCLUSIONS: Before cranioplasty, single-suture craniosynostosis is associated with modest but reliable neurodevelopmental delays that cannot be attributed to maternal intelligence and family sociodemographic variables. Follow-up of this sample will determine the predictive significance of these delays. In the meantime, routine neurodevelopmental screening of infants with isolated craniosynostosis is recommended.

Cognitive and behavioral outcomes of school-aged children who were born preterm: a meta-analysis.

CONTEXT: The cognitive and behavioral outcomes of school-aged children who were born preterm have been reported extensively. Many of these studies have methodological flaws that preclude an accurate estimate of the long-term outcomes of prematurity. OBJECTIVE: To estimate the effect of preterm birth on cognition and behavior in school-aged children. DATA SOURCES: MEDLINE search (1980 to November 2001) for English-language articles, supplemented by a manual search of personal files maintained by 2 of the authors. STUDY SELECTION: We included case-control studies reporting cognitive and/or behavioral data of children who were born preterm and who were evaluated after their fifth birthday if the attrition rate was less than 30%. From the 227 reviewed studies, cognitive data from 15 studies and behavioral data from 16 studies were selected. DATA EXTRACTION: Data on population demographics, study characteristics, and cognitive and behavioral outcomes were extracted from each study, entered in a customized database, and reviewed twice to minimize error. Differences between the mean cognitive scores of cases and controls were pooled. Homogeneity across studies was formally tested using a general variance-based method and graphically using Galbraith plots. Linear meta-analysis regression models were fitted to explore the impact of birth weight and gestational age on cognitive outcomes. Study-specific relative risks (RRs) were calculated for the incidence of attention-deficit/hyperactivity disorder (ADHD) and pooled. Quality assessment of the studies was performed based on a 10-point scale. Publication bias was examined using Begg modified funnel plots and formally tested using the Egger weighted-linear regression method. DATA SYNTHESIS: Among 1556 cases and 1720 controls, controls had significantly higher cognitive scores compared with children who were born preterm (weighted mean difference, 10.9; 95% confidence interval [CI], 9.2-12.5). The mean cognitive scores of preterm-born cases and term-born controls were directly proportional to their birth weight (R(2) = 0.51; P<.001) and gestational age (R(2) = 0.49; P<.001). Age at evaluation had no significant correlation with mean difference in cognitive scores (R(2) = 0.12; P =.20). Preterm-born children showed increases in externalizing and internalizing behaviors in 81% of studies and had more than twice the RR for developing ADHD (pooled RR, 2.64; 95% CI, 1.85-3.78). No differences were noted in cognition and behaviors based on the quality of the study. CONCLUSIONS: Children who were born preterm are at risk for reduced cognitive test scores and their immaturity at birth is directly proportional to the mean cognitive scores at school age. Preterm-born children also show an increased incidence of ADHD and other behaviors.