Severity of disease and quality of life in parents of children with alopecia areata, totalis, and universalis: A prospective, cross-sectional study.

BACKGROUND: Caregiver-oriented quality of life (QoL) research in alopecia areata is limited. No study has used a parent-tailored survey to examine the relationship between QoL and severity of alopecia as measured by Severity of Alopecia Tool (SALT) scores. OBJECTIVES: This is a prospective study that describes QoL in parents of pediatric patients with all subtypes of alopecia areata and investigates the relationship between QoL and severity of disease, duration of disease, and age of patients. METHODS: Pediatric patients and their parents were invited to participate during clinic visits. Participating parents completed the Quality of Life in a Child's Chronic Disease Questionnaire (QLCCDQ) and the Family Dermatology Life Quality Index (FDLQI). A subset of children completed the Children's Dermatologic Life Quality Index (CDLQI). SALT scores at time of survey completion were recorded. RESULTS: In total, 153 patients were included. Significant mild-to-moderate negative correlations were found between SALT scores and FDLQI scores, QLCCDQ scores, and QLCCDQ emotional domain scores. Age of child correlated negatively with QLCCDQ scores but not FDLQI scores. No significant correlation was found between duration of disease and FDLQI scores, QLCCDQ scores, or QLCCDQ emotional domain scores. LIMITATIONS: This study is limited by its small sample size and cross-sectional design. CONCLUSIONS: Impaired parent QoL might be associated with increasing severity of disease and age of affected child but not duration of disease. Providers should tailor counseling accordingly and help parents set realistic expectations for long-term experience with the disease.

Treatment of severe pediatric atopic dermatitis with methotrexate: A retrospective review.

BACKGROUND/OBJECTIVES: Severe atopic dermatitis (AD) may require systemic immunomodulatory agents to control symptoms. A lack of evidence and guidelines for systemic AD therapy in children has led to variability in agents selected and uncertainty in their comparative efficacy and safety. Evaluation of the efficacy of methotrexate in children with severe AD was performed. METHODS: We performed a retrospective chart review of 55 pediatric patients seen at Children's Hospital of Philadelphia that measured improvement using the Investigator's Global Assessment (IGA), a scale that rates AD symptoms from 0 to 5. RESULTS: About 76% of patients showed improvement with methotrexate. Mean baseline IGA of all patients was 4.18. After 6-9 months of treatment, this improved to 2.94. There was additional improvement to a mean IGA score of 2.39 after 12-15 months of treatment. At the final visit before each patient stopped methotrexate, the mean IGA score was 2.71. Approximately 50% of patients experienced minor side effects with gastrointestinal side effects the most common. CONCLUSIONS: In a diverse patient population, safety and efficacy of methotrexate was demonstrated. Significant improvement in IGA was noted for the majority after 6-9 months of therapy with further improvement when continuing treatment to 12-15 months. Methotrexate remains an important option for long-term symptom control with a favorable side effect profile and low cost.

Extracorporeal Cardiopulmonary Resuscitation (E-CPR) During Pediatric In-Hospital Cardiopulmonary Arrest Is Associated With Improved Survival to Discharge: A Report from the American Heart Association's Get With The Guidelines-Resuscitation (GWTG-R) Registry.

BACKGROUND: Although extracorporeal cardiopulmonary resuscitation (E-CPR) can result in survival after failed conventional CPR (C-CPR), no large, systematic comparison of pediatric E-CPR and continued C-CPR has been reported. METHODS AND RESULTS: Consecutive patients <18 years old with CPR events ≥10 minutes in duration reported to the Get With the Guidelines-Resuscitation registry between January 2000 and December 2011 were identified. Hospitals were grouped by teaching status and location. Primary outcome was survival to discharge. Regression modeling was performed, conditioning on hospital groups. A secondary analysis was performed with the use of propensity score matching. Of 3756 evaluable patients, 591 (16%) received E-CPR and 3165 (84%) received C-CPR only. Survival to hospital discharge and survival with favorable neurological outcome (Pediatric Cerebral Performance Category score of 1-3 or unchanged from admission) were greater for E-CPR (40% [237 of 591] and 27% [133 of 496]) versus C-CPR patients (27% [862 of 3165] and 18% [512 of 2840]). Odds ratios (ORs) for survival to hospital discharge and survival with favorable neurological outcome were greater for E-CPR versus C-CPR. After adjustment for covariates, patients receiving E-CPR had higher odds of survival to discharge (OR, 2.80; 95% confidence interval, 2.13-3.69; P<0.001) and survival with favorable neurological outcome (OR, 2.64; 95% confidence interval, 1.91-3.64; P<0.001) than patients who received C-CPR. This association persisted when analyzed by propensity score-matched cohorts (OR, 1.70; 95% confidence interval, 1.33-2.18; P<0.001; and OR, 1.78; 95% confidence interval, 1.31-2.41; P<0.001, respectively]. CONCLUSION: For children with in-hospital CPR of ≥10 minutes duration, E-CPR was associated with improved survival to hospital discharge and survival with favorable neurological outcome compared with C-CPR.

Reliability and Accuracy of Remote Fiberoptic Nasopharyngolaryngoscopy in the Pediatric Population.

INTRODUCTION: Telemedicine is an increasingly prevalent component of medical practice. In otolaryngology, there is the potential for telemedicine services to be performed in conjunction with device use, such as with a nasolaryngoscope. This study evaluates the reliability of remote examinations of the upper airway through an iPhone recording using a coupling device attached to a nasopharyngolaryngoscope (NPL). METHODS: A prospective, blinded study was performed for pediatric patients requiring an NPL during an office visit. The NPL was performed using a coupling device attached to a smartphone to record the examination. A second, remote otolaryngologist then evaluated the recorded examination. Both otolaryngologists evaluated findings of anatomic sites including nasopharynx, oropharynx, base of tongue, larynx including subsites of epiglottis, arytenoids, aryepiglottic folds, false vocal cords, true vocal cords, patency of airway, and diagnostic impression, all of which were documented through a survey. Results of the survey were evaluated through inter-rater agreement using the κ statistic. RESULTS: Forty-five patients underwent an NPL, all of which were included in the study. The average age was 4.9 years. The most common complaint requiring NPL was noisy breathing (n = 16). The inter-rater agreement for overall diagnosis was 0.74 with 80% percent agreement, rated as "good." Other anatomic subsites with "good" or better inter-rater agreement were nasopharynx (0.75), oropharynx (0.75), and true vocal cords (0.71), with strong percentage agreement of 89%, 91%, and 87%, respectively. Both users of the adaptor found the recording setup to run smoothly. CONCLUSION: A telemedicine device for NPL use demonstrates strong diagnostic accuracy across providers and good overall evaluation. It holds potential for use in remote settings.

Utility of repeat testing in the evaluation for von Willebrand disease in pediatric patients.

BACKGROUND: Von Willebrand disease (VWD) is the most common inherited bleeding disorder and is caused by quantitative and qualitative defects in von Willebrand factor (VWF). The laboratory diagnosis of VWD in pediatric patients is complicated by VWF interassay and intra-assay variability, stress-induced elevations in VWF levels, and a lack of significant bleeding history with which to correlate test results. OBJECTIVE: Guidelines recommend repeat testing in patients with a high suspicion of VWD and unclear laboratory assay results; however, no studies have evaluated the utility of repeat VWF testing in pediatric patients. METHODS: This retrospective single-center cohort study aimed to determine clinical variables associated with requiring more than one test to diagnose VWD and to establish a cutoff VWF value above which further testing is not informative. RESULTS: Of 811 patients evaluated for a suspected bleeding disorder, 22.2% were diagnosed with VWD, with ~70% diagnosed on the first test. Patients with VWD were younger (5.8 vs. 8.5 years, P = .002) and more likely to have a family history of VWD (38% vs. 22%, P < .001) than those without VWD. Univariate analysis failed to identify any clinical variables that correlated with needing multiple tests for a VWD diagnosis. A cutoff of 100 IU/dL for VWF antigen or activity on the first test yielded negative predictive values >95%. CONCLUSIONS: We demonstrate that the majority of pediatric patients had diagnostic VWF values on the first set of testing. Pediatric patients without a family history of VWD and VWF levels >100 IU/dL may not need further testing to rule out the diagnosis of VWD.

Heart Retransplant Recipients Have Better Survival With Concurrent Kidney Transplant Than With Heart Retransplant Alone.

BACKGROUND: Heart retransplant (HRT) recipients represent a growing number of transplant patients. The impact of concurrent kidney transplants (KTs) in this population has not been well studied. We tested the hypothesis that recipients of HRT with concurrent KT (HRT-KT) would have worse survival than recipients of HRT alone. METHODS AND RESULTS: A retrospective analysis of the United Network of Organ Sharing database was performed for all patients undergoing HRT from 1987 to 2011. There were 1660 HRT patients, of which 116 (7%) received concurrent KT. Those who received HRT-KT had older age, longer wait-list time, worse kidney function, and more known diabetes. Survival among recipients of HRT-KT was significantly better than that of recipients of HRT alone (P=0.005). A subgroup of 323 HRT patients with severe kidney dysfunction (estimated glomerular filtration rate <30 mL/min per 1.73 m(2) or on dialysis) was studied in more detail, and 76 (24%) received concurrent KT. Those on dialysis at the time of HRT had better survival with versus without concurrent KT (P<0.0001). On multivariable analysis, concurrent KT was independently associated with better outcomes for all patients with HRT and for the subgroup of patients with severe kidney dysfunction. CONCLUSIONS: Recipients of HRT-KT have better survival than recipients of HRT alone. Further research is needed to determine which HRT patients may benefit the most from concurrent KT.