Evaluation of an Intensive Program for Children With Co-occurring Medical and Emotional Disorders.

The study objective was to assess clinical outcomes and cost avoidance of an intensive day treatment program for children with co-occurring chronic medical disease and emotional problems. Intensive day treatment programs for this population are uncommon, and their effectiveness has not been previously reported. A total of 175 children were enrolled during the 3-year study period. Children had more than 30 medical diagnoses including chronic pain, dysautonomia, neurologic disorders, and diabetes. Complete utilization data were available for 118 patients, and demonstrated decreased hospitalizations and increased behavioral health visits during the 12 months post program compared with 12 months prior. Private insurance and female sex were associated with reduced utilization costs after program participation. Estimated avoided cost for the 118 children was $1 111 485. Patients reported significant improvements in somatic symptoms, sleep problems, inattention, depression, anger, and anxiety. Limited data indicated improvements in school attendance. Additional research addressing other outcomes, such as school-related symptoms, would be helpful.

Autologous cellular therapy for cerebral palsy: a randomized, crossover trial.

We examined an autologous mononuclear-cell-therapy-based approach to treat cerebral palsy using autologous umbilical cord blood or bone-marrow-derived mononuclear cells. The primary objective was to determine if autologous cells are safe to administer in children with cerebral palsy. The secondary objectives were to determine if there was improvement in motor function of patients 12 months after infusion using the Gross Motor Function Measure and to evaluate impact of treatment on corticospinal tract microstructure as determined by radial diffusivity measurement. This Phase 1/2a trial was a randomized, blinded, placebo-controlled, crossover study in children aged 2-10 years of age with cerebral palsy enrolled between November 2013 and November 2016. Participants were randomized to 2:1 treatment:placebo. Treatment was either autologous bone-marrow-derived mononuclear cells or autologous umbilical cord blood. All participants who enrolled and completed their baseline visit planned to return for follow-up visits at 6 months, 12 months and 24 months after the baseline visit. At the 12-month post-treatment visit, participants who originally received the placebo received either bone-marrow-derived mononuclear cell or umbilical cord blood treatment. Twenty participants were included; 7 initially randomized to placebo, and 13 randomized to treatment. Five participants randomized to placebo received bone-marrow-derived mononuclear cells, and 2 received umbilical cord blood at the 12-month visit. None of the participants experienced adverse events related to the stem cell infusion. Cell infusion at the doses used in our study did not dramatically alter motor function. We observed concordant bilateral changes in radial diffusivity in 10 of 15 cases where each corticospinal tract could be reconstructed in each hemisphere. In 60% of these cases (6/10), concordant decreases in bilateral corticospinal tract radial diffusivity occurred post-treatment. In addition, 100% of unilateral corticospinal tract cases (3/3) exhibited decreased corticospinal tract radial diffusivity post-treatment. In our discordant cases (n = 5), directionality of changes in corticospinal tract radial diffusivity appeared to coincide with handedness. There was a significant improvement in corticospinal tract radial diffusivity that appears related to handedness. Connectivity strength increased in either or both pathways (corticio-striatal and thalamo-cortical) in each participant at 12 months post-treatment. These data suggest that both stem cell infusions are safe. There may be an improvement in myelination in some groups of patients that correlate with small improvements in the Gross Motor Function Measure scales. A larger autologous cord blood trial is impractical at current rates of blood banking. Either increased private banking or matched units would be required to perform a larger-scale trial.

Neurocognitive functioning in individuals with congenital central hypoventilation syndrome.

BACKGROUND: Congenital central hypoventilation syndrome (CCHS) is a rare disorder characterized by respiratory system abnormalities, including alveolar hypoventilation and autonomic nervous system dysregulation. CCHS is associated with compromised brain development and neurocognitive functioning. Studies that evaluate cognitive skills in CCHS are limited, and no study has considered cognitive abilities in conjunction with psychosocial and adaptive functioning. Moreover, the roles of pertinent medical variables such as genetic characteristics are also important to consider in the context of neurocognitive functioning. METHODS: Seven participants with CCHS ranging in age from 1 to 20 years underwent neuropsychological evaluations in a clinic setting. RESULTS: Neurocognitive testing indicated borderline impaired neurocognitive skills, on average, as well as relative weaknesses in working memory. Important strengths, including good coping skills and relatively strong social skills, may serve as protective factors in this population. CONCLUSION: CCHS was associated with poor neurocognitive outcomes, especially with some polyalanine repeat expansion mutations (PARMS) genotype. These findings have important implications for individuals with CCHS as well as medical providers for this population.

Screening Children with a Family History of Central Congenital Hypoventilation Syndrome.

Congenital central hypoventilation syndrome (CCHS) is a rare genetic disorder of an autonomic nervous disorder that affects breathing. It is characterized by respiratory insufficiency secondary to insensitivity to hypoxemia and hypercarbia, particularly during sleep leading to persistent apnea. We report four individuals across two generations harboring heterozygous 25 polyalanine repeats mutations (PARMs) in PHOX2B with a varying degree of phenotypic clinical manifestations. Two family members who reported to be "asymptomatic" were subsequently diagnosed with CCHS, based on genetic testing, obtained because of their family history. Genetic studies in the family including a mother and three offsprings revealed in-frame five amino acid PARMs of PHOX2B consistent with CCHS in addition to full clinical assessment. All affected individuals had evidence of hypercapnia on blood gas analysis with PCO2 in the range of 32-70 (mean; 61). Nocturnal polysomnogram revealed evidence of hypoventilation in two individuals (1 offspring and mother) with the end-tidal CO2 median of 54. Magnetic resonance imaging of brain revealed no abnormalities in the brain stem. There was no evidence of cor pulmonale on echocardiograms in all individuals. Neuropsychological testing was conducted on all four patients; two patients (mother and 1 offspring) had normal results, while the other two offspring exhibited some impairments on neuropsychological testing. This case series emphasizes the importance of screening first-degree relatives of individuals with confirmed CCHS to minimize complications associated with long-term ventilatory impairment. It also suggests that some patients with CCHS should undergo neuropsychological evaluations to assess for cognitive weaknesses secondary to their CCHS.

Clinical outcomes of an inpatient pediatric obesity treatment program in the USA.

Background When treating recalcitrant and severe childhood obesity, pharmaceutical options are limited and few patients qualify for bariatric surgery. A prolonged inpatient program serves as an alternative treatment. The purpose of this project was to describe the development of a medically supervised inpatient weight management program and evaluate its effectiveness. Methods This is a retrospective chart review of 18 patients [4-18 years, mean body mass index (BMI) 50.2 kg/m2] admitted to an inpatient pediatric weight management program from October 2011 through December 31, 2012 to evaluate the biometric, laboratory, sleep and behavioral changes that occurred from admission to discharge from the program. Results Average weight loss was 15% (6.9%-21.5%, p = 0.0001), the decrease in BMI was 15.1% (1.61-21.57, p = 0.0001), systolic blood pressure and diastolic blood pressure decreased by 7.2% (p = 0.003) and 10.3% (p = 0.040), respectively. The reduction in heart rate was 15% (p = 0.013). Upon admission, nine patients had obstructive sleep apnea syndrome (OSAS), of which one was treated with tonsillectomy and six were not compliant with home positive airway pressure (PAP) therapy. At discharge, three patients no longer required PAP and five required decreased PAP settings. Upon admission, seven patients met the criteria for an internalizing disorder. At discharge, symptom reduction was noted. Conclusion An intensive pediatric inpatient weight management program leads to successful weight loss, improvement in hemodynamic parameters, reduction in OSA treatment requirements and symptom improvement in anxiety and depressive disorders in obese children.

The Test of Variables of Attention (TOVA): internal consistency (Q1 vs. Q2 and Q3 vs. Q4) in children with attention deficit/hyperactivity disorder (ADHD).

The internal consistency of the Test of Variables of Attention (TOVA) was examined in a cohort of 6- to 12-year-old children (N = 63) strictly diagnosed with ADHD. The internal consistency of errors of omission (OMM), errors of commission (COM), response time (RT), and response time variability (RTV) of different test conditions (stimulus infrequent condition [Q1 vs. Q2] and stimulus frequent condition [Q3 vs. Q4]) was assessed via correlation analyses. All TOVA index scores under investigation assessing its internal consistency exhibited statistically significant correlations. All correlations fell in the moderate-high range.

Validity of the child health questionnaire for use in children with sickle cell disease.

OBJECTIVE: To provide accurate data on health-related quality of life (HRQL), there must be a valid tool to measure this outcome. The objective of this study was to determine the validity of the Child Health Questionnaire (CHQ) as a measure of HRQL in sickle cell disease (SCD) by examining the relationship between HRQL and disease severity. METHODS: This was a cross-sectional study of children conducted at two urban, hospital-based clinics. The study participants were children with SCD ages 5 to 18 years who presented for a routine visit to the comprehensive SCD clinic. The main outcome was HRQL, as measured by the CHQ-Parent Form 28 (PF28). A t test was used to compare HRQL between those with mild and severe disease. RESULTS: Parents/caretakers of 95 children completed the CHQ-PF28. Children with mild SCD had a significantly better HRQL, as evidenced by a higher mean physical summary score (39.1), than those with severe disease (28.0) (difference=11.1, 95% confidence interval 5.03-18.11). There was no significant difference in psychosocial summary scores between groups. CONCLUSIONS: The CHQ is a valid tool to assess HRQL in children with SCD and could serve as an important adjunct to determine the effect of SCD on the lives of children.