Enteric tube position on preoperative radiographs predicts clinical outcomes in neonatal congenital diaphragmatic hernia with and without prenatal diagnosis.

OBJECTIVE: Congenital Diaphragmatic Hernia (CDH) is diagnosed prenatally in ~60% of cases. Prenatal measures typically guide management and prognostication. Simple postnatal prognosticators are needed when prenatal diagnosis is lacking. We hypothesized that preoperative orogastric tube (OGT) tip position relative to the contralateral diaphragm correlates with defect severity, resource utilization, and clinical outcomes regardless of diagnostic status. STUDY DESIGN: 150 neonates with left-posterolateral CDH were analyzed. Impact of intrathoracic and intraabdominal preoperative tip position on clinical outcomes was compared. RESULTS: Ninety-nine neonates were prenatally diagnosed. Overall, intrathoracic position significantly correlated with larger diaphragmatic defects, advanced postnatal pulmonary support requirements (HFOV, pulmonary vasodilators, and ECMO), operative complexity, longer hospitalization, and poorer survival to discharge. These observations persisted when analyzing only cases lacking prenatal diagnosis. CONCLUSIONS: Preoperative OGT tip position predicts defect severity, resource utilization, and outcomes in CDH. This observation enhances postnatal prognostication and care planning for neonates without a prenatal diagnosis.

Congenital Diaphragmatic Hernia Repair at the Bedside or Operating Theater.

BACKGROUND: For critically ill congenital diaphragmatic hernia (CDH) patients on high frequency oscillatory ventilation (HFOV), extracorporeal membrane oxygenation (ECMO), and/or inhaled nitric oxide (iNO), operative repair in the neonatal intensive care unit (NICU) has been proposed to avoid complications during transport to an operating room (OR). This study compared neonates with CDH who received herniorrhaphy in the NICU or OR, with a subgroup analysis considering only patients supported with ECMO. METHODS: Patients admitted to the NICU in the first 2 weeks of life at a free-standing children's hospital between July 2004 and September 2021 were examined. Patients were categorized according to location of CDH repair, and impact on operative complications and survival was compared. RESULTS: 185 patients were admitted to the NICU with posterolateral CDH and received operative repair. 48 cases were operated on at the bedside in the NICU and 137 in the OR. Patients repaired in the NICU had higher use of HFOV, pulmonary vasodilators, and ECMO (all P < .001). Children repaired in the NICU experienced significantly higher in-hospital death and overall mortality (P < .001). However, in multivariate analysis, repair location was not a significant predictor of survival to discharge in patients receiving ECMO. No significant difference in surgical site infection was detected for operative location (P = .773). DISCUSSION: Congenital diaphragmatic hernia repair in the NICU occurred more frequently among higher risk patients who experienced worse survival. The rate of surgical site infection appeared similar overall and across subgroups suggesting adequate sterility and technique for bedside procedures, when necessary, despite restricted access to advanced operative equipment.

Non-inferiority of fragmented care for high-risk pediatric neuroblastoma patients: a single institution analysis.

Pediatric neuroblastoma (NB) patients receive multi-modal therapy and may experience care fragmented among multiple institutions with a significant travel burden, which has been associated with poor outcomes for some adult cancers. We hypothesized that fragmented care for pediatric NB patients is associated with inferior outcomes compared to treatment consolidated at one location. We reviewed paper and electronic records for pediatric NB patients who received ≥1 hematopoietic stem cell transplant (HSCT) at Duke from 1990-2017. Fragmented care was defined by treatment at >1 institution and grouped by 2 institutions vs. 3+ institutions. Distances were calculated using Google Maps. To compare all care groups, we used Fisher's Exact and Kruskal-Wallis tests for demographic and treatment characteristics, Kaplan-Meier for unadjusted overall survival (OS), and Cox proportional hazards for factors associated with OS. Of 127 eligible patients, 102 (80.3%) patients experienced fragmented care, with 17 treated at 3+ facilities. Kaplan-Meier analysis did not associate fragmented care with increased mortality (log-rank p = 0.13). With multivariate analysis, only earlier diagnostic decade and greater distance to HSCT remained significantly associated with worsened OS. In this single institutional study, we found fragmented care did not impact overall survival. Worsened overall survival was associated with increased travel distance for HSCT and further research should aim to improve supportive processes for patients undergoing HSCT for high-risk neuroblastoma.

Hepatic vascular malignancies in children are associated with increased rates of surgical resection and improved overall survival compared with adults.

BACKGROUND: Hepatic vascular malignancies (HVMs) are rare malignancies, with no standardized treatment regimens. The most common HVMs, angiosarcoma and malignant epithelioid hemangioendothelioma (EHE), are often grouped together in the literature complicating our ability to achieve reliable survival data and treatment strategies. OBJECTIVE: To compare the disease characteristics of HVMs, with a subanalysis on pediatric patients. METHODS: The 2016 National Cancer Database was queried for patients with HVMs using international classification of diseases-oncology-3 (ICD-O-3) codes yielding 699 patients. Descriptive statistics, chi-square, Kaplan-Meier, and log-rank analyses were performed. RESULTS: We found 478 patients (68%) with angiosarcoma and 221 (32%) with EHE. The median (Q1, Q3) age for angiosarcoma patients was 65 years (56, 75) versus 54 years (37, 65) in EHE patients (P < .001). The rate of resection was lower in patients with angiosarcoma than EHE (13% vs 32%, P < .001). The mean 1-, 3-, and 5-year overall survival for angiosarcoma patients was 17%, 8%, and 6%, respectively, versus 80%, 65%, and 62% in EHE patients (P < .0001). A subgroup analysis was performed on pediatric patients demonstrating six with angiosarcoma and 10 with EHE. The mean 1-, 3-, and 5-year overall survival for pediatric angiosarcoma patients was 67%, 50%, and 50%, respectively, and 90%, 90%, and 90% for pediatric EHE patients. CONCLUSION: In the largest study of HVMs to date, we found angiosarcoma has significantly worse overall survival than EHE. Pediatric patients appear to have improved survival and higher rates of resection. Larger studies of HVMs are needed to clearly differentiate tumor types, standardize care, and improve survivorship.

Thrombosis in infants in the neonatal intensive care unit: Analysis of a large national database.

BACKGROUND: Thrombosis in the neonatal population is rare, but increasing. Its incidence and management are not well understood. OBJECTIVES: To investigate the incidence, associated factors, and management of thrombosis in the neonatal intensive care unit (NICU) population. PATIENTS/METHODS: We performed a retrospective cohort study of infants admitted to a Pediatrix Medical Group-affiliated NICU from 1997 through 2015. We determined the prevalence of venous and arterial thrombosis, and assessed demographic characteristics and known risk factors. Categorical variables were compared with the Pearson χ2 test and continuous variables with Wilcoxon rank-sum tests. Stepwise logistic regression was used to identify associated factors. The primary outcome was incidence of thrombosis. Secondary analyses investigated correlations between clinical and demographic characteristics and thrombosis. RESULTS: Among 1 158 755 infants, we identified 2367 (0.20%) diagnosed with thrombosis. In a multivariable regression analysis, prematurity, male sex, congenital heart disease, sepsis, ventilator support, vasopressor receipt, central venous catheter, invasive procedures, and receipt of erythropoietin were associated with increased risk of thrombosis, while Black race and Hispanic ethnicity were associated with reduced risk. The majority of infants diagnosed with thrombosis (73%) received no anticoagulation, but anticoagulant use in infants with thrombosis was higher than those without (27% versus 0.2%, P < .001). Thrombosis in infants was associated with higher mortality (11% versus 2%, P < .001) and longer hospital stays (57 days, [interquartile range (IQR) 28--100] versus 10 days, [IQR 6--22], P < .001). CONCLUSIONS: In the largest national study to date, we found that thrombosis in NICU patients is associated with prematurity, low birth weight, sepsis, and invasive procedures.

The Developing Balance of Thrombosis and Hemorrhage in Pediatric Surgery: Clinical Implications of Age-Related Changes in Hemostasis.

Bleeding and thrombosis in critically ill infants and children is a vexing clinical problem. Despite the relatively low incidence of bleeding and thrombosis in the overall pediatric population relative to adults, these critically ill children face unique challenges to hemostasis due to extreme physiologic derangements, exposure of blood to foreign surfaces and membranes, and major vascular endothelial injury or disruption. Caring for pediatric patients on extracorporeal support, recovering from solid organ transplant or invasive surgery, and after major trauma is often complicated by major bleeding or clotting events. As our ability to care for the youngest and sickest of these children increases, the gaps in our understanding of the clinical implications of developmental hemostasis have become increasingly important. We review the current understanding of the development and function of the hemostatic system, including the complex and overlapping interactions of coagulation proteins, platelets, fibrinolysis, and immune mediators from the neonatal period through early childhood and to young adulthood. We then examine scenarios in which our ability to effectively measure and treat coagulation derangements in pediatric patients is limited. In these clinical situations, adult therapies are often extrapolated for use in children without taking age-related differences in pediatric hemostasis into account, leaving clinicians confused and impacting patient outcomes. We discuss the limitations of current coagulation testing in pediatric patients before turning to emerging ideas in the measurement and management of pediatric bleeding and thrombosis. Finally, we highlight opportunities for future research which take into account this developing balance of bleeding and thrombosis in our youngest patients.

Virtual visits for Parkinson disease: A multicenter noncontrolled cohort.

BACKGROUND: Previous small-scale studies have demonstrated the feasibility of providing remote specialty care via virtual visits. We assessed the feasibility and benefits of a one-time consultation between a remote Parkinson Disease (PD) specialist and an individual with PD at home on a larger scale. METHODS: We conducted a multicenter noncontrolled cohort of virtual visits administered over videoconferencing between remote PD specialists and individuals with PD in their home. Specialists performed a patient history and a PD-specific physical examination and provided recommendations to patients and their local physicians. The primary outcome measures were feasibility, as measured by the proportion of visits completed as scheduled, and the 6-month change in quality of life, as measured by the Parkinson's Disease Questionnaire 39. Additional outcomes included satisfaction with visits and interest in future virtual visits. RESULTS: A total of 277 participants from 5 states enrolled, 258 participants completed virtual visits with 14 different physicians, and 91% of visits were completed as scheduled. No improvement in quality of life was observed at 6 months (0.4-point improvement; 95% confidence interval -1.5 to 0.6; p = 0.39). Overall satisfaction with virtual visits was high among physicians (94% satisfied or very satisfied) and patients (94% satisfied or very satisfied), and 74% of participants were interested in receiving future care via virtual visits. CONCLUSIONS: Providing specialty care remotely into the homes of individuals with PD is feasible, but a one-time visit did not improve quality of life. Satisfaction with the visits was high among physicians and patients, who were interested in receiving such care in the future. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with PD, remote specialty care is feasible but does not improve quality of life. CLINICALTRIALSGOV IDENTIFIER: NCT02144220.

National randomized controlled trial of virtual house calls for Parkinson disease.

OBJECTIVE: To determine whether providing remote neurologic care into the homes of people with Parkinson disease (PD) is feasible, beneficial, and valuable. METHODS: In a 1-year randomized controlled trial, we compared usual care to usual care supplemented by 4 virtual visits via video conferencing from a remote specialist into patients' homes. Primary outcome measures were feasibility, as measured by the proportion who completed at least one virtual visit and the proportion of virtual visits completed on time; and efficacy, as measured by the change in the Parkinson's Disease Questionnaire-39, a quality of life scale. Secondary outcomes included quality of care, caregiver burden, and time and travel savings. RESULTS: A total of 927 individuals indicated interest, 210 were enrolled, and 195 were randomized. Participants had recently seen a specialist (73%) and were largely college-educated (73%) and white (96%). Ninety-five (98% of the intervention group) completed at least one virtual visit, and 91% of 388 virtual visits were completed. Quality of life did not improve in those receiving virtual house calls (0.3 points worse on a 100-point scale; 95% confidence interval [CI] -2.0 to 2.7 points; p = 0.78) nor did quality of care or caregiver burden. Each virtual house call saved patients a median of 88 minutes (95% CI 70-120; p < 0.0001) and 38 miles per visit (95% CI 36-56; p < 0.0001). CONCLUSIONS: Providing remote neurologic care directly into the homes of people with PD was feasible and was neither more nor less efficacious than usual in-person care. Virtual house calls generated great interest and provided substantial convenience. CLINICALTRIALSGOV IDENTIFIER: NCT02038959. CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that for patients with PD, virtual house calls from a neurologist are feasible and do not significantly change quality of life compared to in-person visits. The study is rated Class III because it was not possible to mask patients to visit type.

National Randomized Controlled Trial of Virtual House Calls for People with Parkinson's Disease: Interest and Barriers.

BACKGROUND: Delivering specialty care remotely directly into people's homes can enhance access for and improve the healthcare of individuals with chronic conditions. However, evidence supporting this approach is limited. MATERIALS AND METHODS: Connect.Parkinson is a randomized comparative effectiveness study that compares usual care of individuals with Parkinson's disease in the community with usual care augmented by virtual house calls with a Parkinson's disease specialist from 1 of 18 centers nationally. Individuals in the intervention arm receive four virtual visits from a Parkinson's disease specialist over 1 year via secure, Web-based videoconferencing directly into their homes. All study activities, including recruitment, enrollment, and assessments, are conducted remotely. Here we report on interest, feasibility, and barriers to enrollment in this ongoing study. RESULTS: During recruitment, 11,734 individuals visited the study's Web site, and 927 unique individuals submitted electronic interest forms. Two hundred ten individuals from 18 states enrolled in the study from March 2014 to June 2015, and 195 were randomized. Most participants were white (96%) and college educated (73%). Of the randomized participants, 73% had seen a Parkinson's disease specialist within the previous year. CONCLUSIONS: Among individuals with Parkinson's disease, national interest in receiving remote specialty care directly into the home is high. Remote enrollment in this care model is feasible but is likely affected by differential access to the Internet.

Vocal Hygiene Habits and Vocal Handicap Among Conservatory Students of Classical Singing.

OBJECTIVES: This study sought to assess classical singing students' compliance with vocal hygiene practices identified in the literature and to explore the relationship between self-reported vocal hygiene practice and self-reported singing voice handicap in this population. The primary hypothesis was that increased attention to commonly recommended vocal hygiene practices would correlate with reduced singing voice handicap. STUDY DESIGN: This is a cross-sectional, survey-based study. METHODS: An anonymous survey assessing demographics, attention to 11 common vocal hygiene recommendations in both performance and nonperformance periods, and the Singing Voice Handicap Index 10 (SVHI-10) was distributed to classical singing teachers to be administered to their students at two major schools of music. RESULTS: Of the 215 surveys distributed, 108 were returned (50.2%), of which 4 were incomplete and discarded from analysis. Conservatory students of classical singing reported a moderate degree of vocal handicap (mean SVHI-10, 12; range, 0-29). Singers reported considering all 11 vocal hygiene factors more frequently when preparing for performances than when not preparing for performances. Of these, significant correlations with increased handicap were identified for consideration of stress reduction in nonperformance (P = 0.01) and performance periods (P = 0.02) and with decreased handicap for consideration of singing voice use in performance periods alone (P = 0.02). CONCLUSIONS: Conservatory students of classical singing report more assiduous attention to vocal hygiene practices when preparing for performances and report moderate degrees of vocal handicap overall. These students may have elevated risk for dysphonia and voice disorders which is not effectively addressed through common vocal hygiene recommendations alone.

Feasibility of Virtual Research Visits in Fox Trial Finder.

BACKGROUND: Fox Trial Finder is an online registry for individuals with and without Parkinson disease (PD) interested in participating in PD research. However, distance or disability could prevent such individuals from participating in traditional, clinic-based research at major centers. OBJECTIVE: Use videoconferencing to connect participants to specialists to: (1) demonstrate feasibility of virtual research visits within this population (2) collect phenotypic data of the participants, (3) validate self-reported diagnosis, and (4) gauge interest in virtual research visits. METHODS: We solicited volunteers throughout the United States through Fox Trial Finder. Interested individuals with PD provided consent, were given web cameras if needed, completed baseline surveys, and downloaded videoconferencing software remotely. Participants had a test connection and assessment appointment which included the Montreal Cognitive Assessment (MoCA), then a virtual research visit with a neurologist who reviewed their history and assessed their PD using a modified Movement Disorders Society Unified Parkinson's Disease Rating Scale. Neurologists assessed PD diagnosis and symptomatology. Physicians and participants were surveyed about their experience. RESULTS: Of 204 individuals who consented, 166 (81% ) individuals from 39 states completed all visits. The mean age was 62 and mean disease duration was 8.0 years. Mean MoCA score was 26.5, and mean modified MDS-UPDRS motor score was 22.8 (out of a possible 124). Neurologists judged PD as the most likely diagnosis in 97% of cases. Overall satisfaction with the visits was 79% (satisfied or very satisfied) among neurologists and 93% among participants. CONCLUSIONS: Through virtual research visits, neurologists engaged, characterized, and validated self-reported diagnosis in individuals with PD over a broad geography. This model may facilitate future research participation.

Virtual research visits and direct-to-consumer genetic testing in Parkinson's disease.

OBJECTIVE: The purpose of this study was to conduct a proof-of-concept study to evaluate remote recruitment and assessment of individuals ("virtual research visits") with Parkinson's disease who have pursued direct-to-consumer genetic testing. METHODS: Participants in 23andMe's "Parkinson's Research Community" were contacted by 23andMe. Fifty willing participants living in 23 states underwent a remote, standardized assessment including cognitive and motor tests by a neurologist via video conferencing and then completed a survey. Primary outcomes assessed were (a) proportion of participants who completed the remote assessments; (b) level of agreement (using Cohen's kappa coefficient) of patient-reported data with that of a neurologist; and (c) interest in future virtual research visits. RESULTS: The self-reported diagnosis of Parkinson's disease was confirmed in all cases (k = 1.00). The level of agreement for age of symptom onset (k = 0.97) and family history (k = 0.85) was very good but worse for falling (k = 0.59), tremor (k = 0.56), light-headedness (k = 0.31), and urine control (k = 0.15). Thirty-eight (76%) of the 50 participants completed a post-assessment survey, and 87% of respondents said they would be more or much more willing to participate in future clinical trials if they could do research visits remotely. CONCLUSION: Remote clinical assessments of individuals with known genotypes were conducted nationally and rapidly from a single site, confirmed self-reported diagnosis, and were received favorably. Direct-to-consumer genetic testing and virtual research visits together may enable characterization of genotype and phenotype for geographically diverse populations.

Virtual house calls for Parkinson disease (Connect.Parkinson): study protocol for a randomized, controlled trial.

BACKGROUND: Interest in improving care for the growing number of individuals with chronic conditions is rising. However, access to care is limited by distance, disability, and distribution of doctors. Small-scale studies in Parkinson disease, a prototypical chronic condition, have suggested that delivering care using video house calls is feasible, offers similar clinical outcomes to in-person care, and reduces travel burden. METHODS/DESIGN: We are conducting a randomized comparative effectiveness study (Connect.Parkinson) comparing usual care in the community to usual care augmented by virtual house calls with a Parkinson disease specialist. Recruitment is completed centrally using online advertisements and emails and by contacting physicians, support groups, and allied health professionals. Efforts target areas with a high proportion of individuals not receiving care from neurologists. Approximately 200 individuals with Parkinson disease and their care partners will be enrolled at 20 centers throughout the United States and followed for one year. Participants receive educational materials, then are randomized in a 1:1 ratio to continue their usual care (control arm) or usual care and specialty care delivered virtually (intervention arm). Care partners are surveyed about their time and travel burden and their perceived caregiver burden. Participants are evaluated via electronic survey forms and videoconferencing with a blinded independent rater at baseline and at 12 months. All study activities are completed remotely.The primary outcomes are: (1) feasibility, as measured by the proportion of visits completed, and (2) quality of life, as measured by the 39-item Parkinson's Disease Questionnaire. Secondary outcomes include measures of clinical benefit, quality of care, time and travel burden, and caregiver burden. DISCUSSION: Connect.Parkinson will evaluate the feasibility and effectiveness of using technology to deliver care into the homes of individuals with Parkinson disease. The trial may serve as a model for increasing access and delivering patient-centered care at home for individuals with chronic conditions. TRIAL REGISTRATION: This trial was registered on clinicaltrials.gov on January 8, 2014 [NCT02038959].