Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module.

BACKGROUND: Detecting change in health status over time and ascertaining meaningful changes are critical elements when using health-related quality of life (HRQL) instruments to measure patient-centered outcomes. The PedsQL™ Sickle Cell Disease module, a disease specific HRQL instrument, has previously been shown to be valid and reliable. Our objectives were to determine the longitudinal validity of the PedsQL™ Sickle Cell Disease module and the change in HRQL that is meaningful to patients. METHODS: An ancillary study was conducted utilizing a multi-center prospective trial design. Children ages 4-21 years with sickle cell disease admitted to the hospital for an acute painful vaso-oclusive crisis were eligible. Children completed HRQL assessments at three time points (in the Emergency Department, one week post-discharge, and at return to baseline (One to three months post-discharge). The primary outcome was change in HRQL score. Both distribution (effect size, standard error of measurement (SEM)) and anchor (global change assessment) based methods were used to determine the longitudinal validity and meaningful change in HRQL. Changes in HRQL meaningful to patients were identified by anchoring the change scores to the patient\u27s perception of global improvement in pain. RESULTS: Moderate effect sizes (0.20-0.80) were determined for all domains except the Communication I and Cognitive Fatigue domains. The value of 1 SEM varied from 3.8-14.6 across all domains. Over 50% of patients improved by at least 1 SEM in Total HRQL score. A HRQL change score of 7-10 in the pain domains represented minimal perceived improvement in HRQL and a HRQL change score of 18 or greater represented moderate to large improvement. CONCLUSIONS: The PedsQL™ Sickle Cell Disease Module is responsive to changes in HRQL in patients experiencing acute painful vaso-occlusive crises. The study data establish longitudinal validity and meaningful change parameters for the PedsQL™ Sickle Cell Disease Module. TRIAL REGISTRATION: ClinicalTrials.gov (study identifier: NCT01197417 ). Date of registration: 08/30/2010

Pediatric Emergency Care Research Networks: A Research Agenda

BackgroundPediatric emergency care research networks have evolved substantially over the past two decades. Some networks are specialized in specific areas (e.g., sedation, simulation) while others study a variety of medical and traumatic conditions. Given the increased collaboration between pediatric emergency research networks, the logical next step is the development of a research priorities agenda to guide global research in emergency medical services for children (EMSC).ObjectivesAn international group of pediatric emergency network research leaders was assembled to develop a list of research priorities for future collaborative endeavors within and between pediatric emergency research networks.MethodsBefore an in‐person meeting, we used a modified Delphi approach to achieve consensus around pediatric emergency research network topic priorities. Further discussions took place on May 15, 2018, in Indianapolis, Indiana, at the Academic Emergency Medicine (AEM) consensus conference “Aligning the Pediatric Emergency Medicine Research Agenda to Reduce Health Outcome Gaps.” Here, a group of 40 organizers and participants met in a 90‐minute “breakout” session to review and further develop the initial priorities.ResultsWe reached consensus on five clinical research priorities that would benefit from collaboration among the existing and future emergency networks focused on EMSC: sepsis, trauma, respiratory conditions, pharmacology of emergency conditions, and mental health emergencies. Furthermore, we identified nonclinical research priorities categorized under the domains of technology, knowledge translation, and organization/administration of pediatric emergency care.ConclusionThe identification of pediatric emergency care network research priorities within the domains of clinical care, technology, knowledge translation and organization/administration of EMSC will facilitate and help focus collaborative research within and among research networks globally. Engagement of essential stakeholders including EMSC researchers, policy makers, patients, and their caregivers will stimulate advances in the delivery of emergency care to children around the globe.Peer Reviewedhttps://deepblue.lib.umich.edu/bitstream/2027.42/147119/1/acem13656.pdfhttps://deepblue.lib.umich.edu/bitstream/2027.42/147119/2/acem13656_am.pd

Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial.

BACKGROUND: Despite substantial illness burden and healthcare utilization conferred by pain from vaso-occlusive episodes (VOE) in children with sickle cell disease (SCD), disease-modifying therapies to effectively treat SCD-VOE are lacking. The aim of the Sickle Cell Disease Treatment with Arginine Therapy (STArT) Trial is to provide definitive evidence regarding the efficacy of intravenous arginine as a treatment for acute SCD-VOE among children, adolescents, and young adults. METHODS: STArT is a double-blind, placebo-controlled, randomized, phase 3, multicenter trial of intravenous arginine therapy in 360 children, adolescents, and young adults who present with SCD-VOE. The STArT Trial is being conducted at 10 sites in the USA through the Pediatric Emergency Care Applied Research Network (PECARN). Enrollment began in 2021 and will continue for 5 years. Within 12 h of receiving their first dose of intravenous opioids, enrolled participants are randomized 1:1 to receive either (1) a one-time loading dose of L-arginine (200 mg/kg with a maximum of 20 g) administered intravenously followed by a standard dose of 100 mg/kg (maximum 10 g) three times a day or (2) a one-time placebo loading dose of normal saline followed by normal saline three times per day at equivalent volumes and duration as the study drug. Participants, research staff, and investigators are blinded to the participant\u27s randomization. All clinical care is provided in accordance with the institution-specific standard of care for SCD-VOE based on the 2014 National Heart, Lung, and Blood Institute guidelines. The primary outcome is time to SCD-VOE pain crisis resolution, defined as the time (in hours) from study drug delivery to the last dose of parenteral opioid delivery. Secondary outcomes include total parental opioid use and patient-reported outcomes. In addition, the trial will characterize alterations in the arginine metabolome and mitochondrial function in children with SCD-VOE. DISCUSSION: Building on the foundation of established relationships between emergency medicine providers and hematologists in a multicenter research network to ensure adequate participant accrual, the STArT Trial will provide definitive information about the efficacy of intravenous arginine for the treatment of SCD-VOE for children. TRIAL REGISTRATION: The STArT Trial was registered in ClinicalTrials.gov on April 9, 2021, and enrollment began on June 21, 2021 (NCT04839354)

Acute Pediatric Pain Management In The Primary Care Office

Pain is a chief complaint in children seeking medical care, yet it may also be experienced in evaluation and treatment during office visits. Inadequate relief of children’s procedural pain and distress not only affects the experience of the children and their parents, but also adversely affects procedural outcomes. Despite increasing awareness and research, management of procedural pain and anxiety in children is often inadequate. In addition, parent and patient satisfaction is often tied to pain management. Development of a pain management plan must be systematic, individualized, and multimodal. We present a review of nonpharmacologic modalities, topical and oral analgesic agents, and intranasal adjuncts for use in routine outpatient practice

A Sublingual Mass in a 5-Year-Old Male

History of present illness: A 5-year-old male presented to the emergency department with a mass under his tongue and tactile fever for one week. The mass had not grown noticeably since first noted by his mother. The child had no difficulty breathing, stridor, or odynophagia. He was eating and drinking well and had no other symptoms. There was no history of trauma. He was seen by his pediatrician three days prior and received a prescription for a 10-day course of amoxicillin. On physical exam, he was afebrile and had normal vital signs. Oral exam was notable for a small non-tender mass in the floor of the oral mucosa. He had no cervical swelling or lymphadenopathy. The rest of his physical examination was unremarkable. Significant findings: Oral ranula. On oral inspection he had a 3 x 1.5 cm, soft, non-tender, mobile, cystic, sublingual mass on the right aspect of the floor of his mouth that did not move with swallowing (Image 1). There was mild associated submandibular swelling on the right side of his face. Discussion: A ranula is a benign lesion caused by damage to the sublingual duct leading to the accumulation of mucoid material and cystic swelling in the floor of the oral mucosa.1,2 It presents as a non-tender, translucent cystic mass under the tongue primarily in adolescents.1 The diagnosis is made by physical examination alone and does not require further imaging.2 However, some otolaryngologists prefer ultrasound or computed tomography to further characterize the lesion prior to surgery.1 Though ranulas may arise secondary to previous trauma due to scarring, they are often idiopathic.1 Ranulas are generally asymptomatic, though they can lead to difficulty swallowing and even airway compromise in severe cases.1 Referral to otolaryngology often results in conservative management for up to three months followed by resection of a symptomatic or persistent ranula larger than 2 cm in diameter.3 Alternative approaches include early removal of the involved portion of the sublingual gland or sclerotherapy as an effective first-line treatment.4 The patient in this case was referred to otolaryngology as an outpatient and underwent transoral excision of the mass. Ten days after excision, he developed oral hemorrhage from the excision site and was observed overnight after his bleeding spontaneously subsided. Since that time, he has had no other complications and the mass has not recurred

Pediatric Emergency Departments and Urgent Care Visits in Houston after Hurricane Harvey

Introduction: Natural disasters are increasingly common and devastating. It is essential to understand children’s health needs during disasters as they are a particularly vulnerable population. The objective of this study was to evaluate pediatric disease burden after Hurricane Harvey compared to the preceding month and the same period in the previous year to inform pediatric disaster preparedness.Methods: This was a retrospective cross-sectional study of patients seen at pediatric emergency departments (ED) and urgent care centers (UCC) 30 days before (late summer) and after (early fall) the hurricane and from the same time period in 2016. We collected demographic information and the first five discharge diagnoses from a network of EDs and UCCs affiliated with a quaternary care children’s hospital in Houston, Texas. We calculated the odds of disease outcomes during various timeframes using binary logistic regression modeling.Results: There were 20,571 (median age: 3.5 years, 48.1% female) and 18,943 (median age: 3.5 years, 47.3% female) patients in 2016 and 2017, respectively. Inpatient admission rates from the ED a month after Harvey were 20.5%, compared to 25.3% in the same period in 2016 (P<0.001). In both years, asthma and other respiratory illnesses increased from late summer to early fall. After controlling for these seasonal trends, the following diseases were more commonly seen after the hurricane: toxicological emergencies (adjusted odds ratio [aOR]: 2.61, 95% [confidence interval] CI, 1.35-5.05); trauma (aOR: 1.42, 95% CI, 1.32-1.53); and dermatological complaints (aOR: 1.34, 95% CI, 1.23-1.46).Conclusion: We observed increases in rashes, trauma, and toxicological diagnoses in children after a major flood. These findings highlight the need for more medication resources and public health and education measures focused on pediatric disaster preparedness and management

Pediatric Emergency Departments and Urgent Care Visits in Houston after Hurricane Harvey

Introduction: Natural disasters are increasingly common and devastating. It is essential to understand children’s health needs during disasters as they are a particularly vulnerable population. The objective of this study was to evaluate pediatric disease burden after Hurricane Harvey compared to the preceding month and the same period in the previous year to inform pediatric disaster preparedness.Methods: This was a retrospective cross-sectional study of patients seen at pediatric emergency departments (ED) and urgent care centers (UCC) 30 days before (late summer) and after (early fall) the hurricane and from the same time period in 2016. We collected demographic information and the first five discharge diagnoses from a network of EDs and UCCs affiliated with a quaternary care children’s hospital in Houston, Texas. We calculated the odds of disease outcomes during various timeframes using binary logistic regression modeling.Results: There were 20,571 (median age: 3.5 years, 48.1% female) and 18,943 (median age: 3.5 years, 47.3% female) patients in 2016 and 2017, respectively. Inpatient admission rates from the ED a month after Harvey were 20.5%, compared to 25.3% in the same period in 2016 (P<0.001). In both years, asthma and other respiratory illnesses increased from late summer to early fall. After controlling for these seasonal trends, the following diseases were more commonly seen after the hurricane: toxicological emergencies (adjusted odds ratio [aOR]: 2.61, 95% [confidence interval] CI, 1.35-5.05); trauma (aOR: 1.42, 95% CI, 1.32-1.53); and dermatological complaints (aOR: 1.34, 95% CI, 1.23-1.46).Conclusion: We observed increases in rashes, trauma, and toxicological diagnoses in children after a major flood. These findings highlight the need for more medication resources and public health and education measures focused on pediatric disaster preparedness and management