Trametinib therapy for children with neurofibromatosis type 1 and life-threatening plexiform neurofibroma or treatment-refractory low-grade glioma.

PURPOSE: To describe a series of children with extensive PNF or treatment refractory PLGG treated on a compassionate basis with trametinib. METHODS: We report on six patients with NF-1 treated with trametinib on a compassionate basis at British Columbia Children's Hospital since 2017. Data were collected retrospectively from the patient record. RAPNO and volumetric criteria were used to evaluate the response of intracranial and extracranial lesions, respectively. RESULTS: Subjects were 21 months to 14 years old at the time of initiation of trametinib therapy and 3/6 subjects are male. Duration of therapy was 4-28 months at the time of this report. All patients had partial response or were stable on analysis. Two patients with life-threatening PNF had a partial radiographic response in tandem with significant clinical improvement and developmental catch up. One subject discontinued therapy after 6 months due to paronychia and inadequate response. The most common adverse effect (AE) was grade 1-2 paronychia or dermatitis in 5/6 patients. There were no grade 3 or 4 AEs. At the time of this report, five patients remain on therapy. CONCLUSION: Trametinib is an effective therapy for advanced PNF and refractory PLGG in patients with NF-1 and is well tolerated in children. Further data and clinical trials are required to assess tolerance, efficacy and durability of response, and length of treatment required in such patients.

Competency-Based Assessment Tool for Pediatric Esophagoscopy: International Modified Delphi Consensus.

OBJECTIVES/HYPOTHESIS: Create a competency-based assessment tool for pediatric esophagoscopy with foreign body removal. STUDY DESIGN: Blinded modified Delphi consensus process. SETTING: Tertiary care center. METHODS: A list of 25 potential items was sent via the Research Electronic Data Capture database to 66 expert surgeons who perform pediatric esophagoscopy. In the first round, items were rated as "keep" or "remove" and comments were incorporated. In the second round, experts rated the importance of each item on a seven-point Likert scale. Consensus was determined with a goal of 7 to 25 final items. RESULTS: The response rate was 38/64 (59.4%) in the first round and returned questionnaires were 100% complete. Experts wanted to "keep" all items and 172 comments were incorporated. Twenty-four task-specific and 7 previously-validated global rating items were distributed in the second round, and the response rate was 53/64 (82.8%) with questionnaires returned 97.5% complete. Of the task-specific items, 9 reached consensus, 7 were near consensus, and 8 did not achieve consensus. For global rating items that were previously validated, 6 reached consensus and 1 was near consensus. CONCLUSIONS: It is possible to reach consensus about the important steps involved in rigid esophagoscopy with foreign body removal using a modified Delphi consensus technique. These items can now be considered when evaluating trainees during this procedure. This tool may allow trainees to focus on important steps of the procedure and help training programs standardize how trainees are evaluated. LEVEL OF EVIDENCE: 5. Laryngoscope, 131:1168-1174, 2021.

Competency-Based Assessment Tool for Pediatric Tracheotomy: International Modified Delphi Consensus.

OBJECTIVES/HYPOTHESIS: Create a competency-based assessment tool for pediatric tracheotomy. STUDY DESIGN: Blinded, modified, Delphi consensus process. METHODS: Using the REDCap database, a list of 31 potential items was circulated to 65 expert surgeons who perform pediatric tracheotomy. In the first round, items were rated as "keep" or "remove," and comments were incorporated. In the second round, experts were asked to rate the importance of each item on a seven-point Likert scale. Consensus criteria were determined a priori with a goal of 7 to 25 final items. RESULTS: The first round achieved a response rate of 39/65 (60.0%), and returned questionnaires were 99.5% complete. All items were rated as "keep," and 137 comments were incorporated. In the second round, 30 task-specific and seven previously validated global rating items were distributed, and the response rate was 44/65 (67.7%), with returned questionnaires being 99.3% complete. Of the Task-Specific Items, 13 reached consensus, 10 were near consensus, and 7 did not achieve consensus. For the 7 previously validated global rating items, 5 reached consensus and two were near consensus. CONCLUSIONS: It is feasible to reach consensus on the important steps involved in pediatric tracheotomy using a modified Delphi consensus process. These items can now be considered to create a competency-based assessment tool for pediatric tracheotomy. Such a tool will hopefully allow trainees to focus on the important aspects of this procedure and help teaching programs standardize how they evaluate trainees during this procedure. LEVEL OF EVIDENCE: 5 Laryngoscope, 130:2700-2707, 2020.

Casting a look at pediatric plastic bronchitis.

OBJECTIVES: To review clinical presentations and management strategies for children with plastic bronchitis. METHODS: Retrospective chart review. RESULTS: Seven patients required rigid bronchoscopy to remove bronchial casts over a 17-year study period. Mean age at presentation was 60 months. Mean follow-up was 53 months. Co-morbidities included: congenital heart disease (n=3), chronic pulmonary disorders (n=2) and sickle cell disease (n=1). 4 patients required multiple bronchoscopies for recurrent casts. Adjunctive topical therapies were administered in all 7 patients, without complication. Rigid bronchoscopy for cast removal was performed in 2 patients who were on extra-corporal membrane oxygenation (ECMO), using special precautions to safeguard the ECMO catheters. CONCLUSIONS: Bronchial casts in children may present acutely or sub-acutely. Recurrent casts are unusual; however, in combination with severe cardiac disease may lead to mortality. Adjunctive topical therapies are still under investigation. Special safeguards for ECMO catheters are imperative. This case series complements and adds to the International Plastic Bronchitis Registry.

False carina: a distinct variant of tracheal bronchus.

Tracheal-bronchus is an aberrant bronchus arising from the lateral tracheal wall, superior to the carina. A "False-carina" can be classified as a sub-type. This clinical entity will be defined and the clinical presentation, diagnosis and management of five patients with variations of the anatomical definition of a False-carina, identified at our institution, will be reviewed. Congenital bronchial abnormalities, including False-carina, have important implications in the overall management of the airway. Management can range from expectant in asymptomatic patients to surgical intervention in cases of recurrent respiratory infections. Awareness and understanding of this clinical entity allows for timely investigation, diagnosis and appropriate intervention.

The reliability of clinical tonsil size grading in children.

IMPORTANCE: Because tonsillar enlargement can have substantial ill health effects in children, reliable monitoring and documentation of tonsil size is necessary in clinical settings. Tonsil grading scales potentially allow clinicians to precisely record and communicate changes in tonsil size, but their reliability in a clinical setting has not been studied. OBJECTIVE: To assess the interobserver and intraobserver reliability of the Brodsky and Friedman tonsil size grading scales and a novel 3-grade scale. DESIGN, SETTING, AND PARTICIPANTS: Cross-sectional study between June 2012 and August 2013 at a tertiary pediatric otolaryngology outpatient clinic at British Columbia Children's Hospital. We recruited 116 children, aged 3 to 14 years, with no major craniofacial abnormalities. For each child, 2 separate tonsil assessments (with at least a 5-minute interval in between) were conducted by 4 independent observers: 2 staff pediatric otolaryngologists, 1 otolaryngology trainee (fellow or resident), and 1 medical student. Each observer assessed and graded tonsil sizes using 3 different scales. MAIN OUTCOMES AND MEASURES: Interobserver and intraobserver reliabilities were assessed by deriving the intraclass correlation coefficients (ICCs) and Pearson correlation coefficients, respectively. To discount for any asymmetric scores, all data analysis was conducted on the left tonsil measurement only. RESULTS: Mean interobserver reliability was highest for the Brodsky grading scale (ICC, 0.721; Cronbach α, 0.911), followed by the Friedman grading scale (ICC, 0.647; Cronbach α, 0.879) and the 3-grade scale (ICC, 0.599; Cronbach α, 0.857). The mean intraobserver reliabilities for the Brodsky, Friedman, and modified 3-grade scales were 0.954, 0.932, and 0.927, respectively. CONCLUSIONS AND RELEVANCE: The Brodsky grading scale offered the highest interobserver and intraobserver reliability when compared with the Friedman and novel 3-grade scales. The results of this study would support the uniform use of the Brodsky scale for future clinical and research work.

Intranasal topical local anesthetic and decongestant for flexible nasendoscopy in children: a randomized, double-blind, placebo-controlled trial.

IMPORTANCE: To our knowledge, the present study is the first double-blind, randomized, placebo-controlled trial in children to compare nasal preparation sprays administered before flexible nasendoscopy with placebo. OBJECTIVE: To compare the degree of pain experienced by children undergoing flexible nasendoscopy after 1 of 3 intranasal sprays: placebo, decongestant with topical local anesthetic (TLA), or decongestant without TLA. DESIGN, SETTING, AND PARTICIPANTS: A randomized placebo-controlled trial with blinding of participants, caregivers, observers, and otolaryngologists was conducted in a tertiary pediatric otolaryngology ambulatory clinic. Participants included a consecutive sample of children aged 3 to 12 years requiring flexible nasendoscopy. Exclusion criteria included concomitant respiratory tract infection, known allergy to a trial agent, or previous flexible nasendoscopy. One hundred fifty-one children were assessed for eligibility; 24 eligible children refused participation and 69 were included and block-randomized. All completed the study, and there were no adverse events. INTERVENTIONS: Nasal spray administration of placebo (normal saline); xylometazoline hydrochloride, 0.05% (decongestant); or lidocaine hydrochloride, 1%, with xylometazoline hydrochloride, 0.05% (TLA with decongestant) was performed 10 minutes before flexible nasendoscopy. MAIN OUTCOMES AND MEASURES: Primary outcome measure was the child-reported Wong-Baker Faces Pain (WBFP) scale. Secondary outcomes included the caregiver-proxy WBFP scale; the Face, Legs, Activity, Cry, and Consolability (FLACC) scale; and the physician-reported Difficulty of Procedure Visual Analog Scale (DPVAS). RESULTS: Twenty-three children were recruited in each of the intervention arms. Baseline characteristics were comparable between groups. The mean child-rated WBFP scale scores were 2.4, 1.8, and 2.2 for the placebo, decongestant, and TLA with decongestant groups, respectively (P = .45). Although the finding was statistically nonsignificant, decongestant had the lowest mean caregiver-proxy WBFP scale score, lowest observer-rated FLACC scale score, and highest physician-rated DPVAS score. Subgroup analysis did not demonstrate any correlation between the outcomes and age or sex. CONCLUSIONS AND RELEVANCE: This study revealed no statistically significant difference in the discomfort experienced by children undergoing flexible nasendoscopy after placebo, decongestant, or TLA with decongestant. Decongestant was associated with the least discomfort (on child, caregiver, and observer-rated pain scale scores) and the lowest rating for difficulty of procedure. With these findings, the study suggests that there is no significant benefit of topical decongestant with or without TLA compared with placebo in reducing pain associated with pediatric flexible nasendoscopy. TRIAL REGISTRATION clinicaltrials.gov Identifier: NCT01351298.

Best management of ultra-small tracheobronchial foreign bodies in neonates.

OBJECTIVES: To develop (1) a practical and comprehensive algorithm of instruments and techniques available to treat neonates with tracheobronchial foreign bodies, based on the patient's subglottic diameter and (2) primary and secondary prevention strategies for neonatal tracheobronchial foreign bodies. METHODS: (1) Analysis of the case of a severely premature infant who presented with the incidental radiological finding of a 2cm suction catheter tip, which, over a two week period, had migrated between her main bronchi; the foreign body was removed with a previously unreported combination of instruments: a 3French (F) flexible urological forceps through the side port of a 2.5mm rigid bronchoscope. (2) In vitro testing of typical foreign bodies and readily available endoscopic instruments. RESULTS: We have developed a practical and comprehensive algorithm for the treatment of neonates with tracheobronchial foreign bodies, as well as primary and secondary prevention strategies. CONCLUSIONS: The treatment algorithm and prevention strategies may reduce morbidity and mortality from neonatal tracheobronchial foreign bodies.

Management of congenital midline cervical cleft.

Congenital midline cervical cleft (CMCC) is a rare developmental defect of the anterior neck normally characterized by an atrophic mucosal plaque with a cranial nipple-like skin tag, a short caudal sinus, and may be attached to a subcutaneous fibrous cord of variable length. Clinically, patients present at an early age with, white females being the most commonly affected population. In addition to aesthetic concerns, CMCC can prevent full extension of the neck, result in micrognathia and torticollis, predispose patients to infection, and can coexist with other clefting defects or cysts. Fewer than 50 cases have been published in the English-language literature. Herein, we report a case of CMCC that also presented with a mild contracture of the right sternohyoid muscle. The embryopathogenesis, histopathology, diagnosis, and treatment of this rare condition are also discussed.

Assessment of vocal fold mobility before and after cardiothoracic surgery in children.

OBJECTIVES: To assess the incidence of vocal fold immobility (VFI) after cardiothoracic surgery in children and to determine the factors potentially associated with this outcome. METHODS: Flexible laryngoscopy to assess vocal fold mobility was performed before surgery and within 72 hours after extubation in 100 pediatric patients who underwent cardiothoracic procedures. The 2 operating surgeons recorded the surgical technique and their impression of possible injury to the recurrent laryngeal nerve. The presence of laryngeal symptoms, such as stridor, hoarseness, and strength of cry, after extubation was documented. RESULTS: Of 100 children included in this study, 8 had VFI after surgery. Univariate analyses showed that these 8 patients were younger and weighed less than the patients with normal vocal fold movement. Monopolar cautery was used in all patients with VFI. On univariate analysis, factors statistically significantly associated with VFI were circulatory arrest and dissection or ligation of the patent ductus arteriosus, left pulmonary artery, right pulmonary artery, or descending aorta. However, multivariate analyses failed to show these associations. CONCLUSIONS: The incidence of VFI after cardiothoracic surgery in our population of children was 8.0% (8 of 100). Of several factors found to be potentially associated with VFI on univariate analysis, none were significant on multivariate analysis. This may be a result of the few patients with VFI. A larger multicenter prospective study would be needed to definitively identify factors associated with the outcome of VFI.

Choking prevention: shortcomings of traditional public education campaigns, prompting the development of web-based interactive teaching tools for preteens, teenagers, and adults.

OBJECTIVES: To evaluate the effectiveness of the BC Children's Hospital (BCCH) Safe Start Choking Prevention campaign, which began in 2002 and used newspaper, magazine, radio, and television interviews of parents, injury prevention experts, and physicians to educate the public about choking risks, prevention, and treatment (CRPT); to compare our campaign with other campaigns; and to determine if a new strategy is needed to educate the public about CRPT. METHODS: A retrospective analysis of the number of cases of laryngeal, bronchial, and esophageal foreign bodies (LBEFBs) that required rigid endoscopy under general anesthesia at BCCH and/or admission to BCCH was conducted. Data from January 1997 to December 2002 to January 2003 to December 2006 were compared. The world literature on choking prevention and analysis of barriers to CRPT public education was reviewed. RESULTS: The data demonstrate the ineffectiveness of our traditional media campaign. The number of total operating room visits at BCCH for LBEFBs increased marginally after our choking prevention campaign began. Published reports from Israel and Crete indicate that educational campaigns that included direct teaching of CRPT by otolaryngologists and other health educators to parents and their children are effective; however, such campaigns would be difficult to replicate within larger populations. CONCLUSION: A new strategy for CPRT public education is required. We are creating an animated video and a comprehensive, interactive website to teach CRPT to preteens, teenagers, and adults (with prospective validation of knowledge transfer and long-term outcome measurement).

Cochlear nerve aplasia detected through kindergarten hearing screening.

OBJECTIVES: To describe the importance of imaging with the use of magnetic resonance (MR) or computed tomography (CT) during the diagnostic workup of a patient with sensorineural hearing loss to determine the status of the cochlear nerve. DESIGN: Retrospective review. SETTING: Tertiary care academic institution. METHODS: A pediatric database was used to find patients with diagnoses of absent cochlear nerve. A retrospective chart review was performed from July 1999 to July 2004 to evaluate the route to diagnosis and any concomitant factors. Patients who had presented at kindergarten screening were included. Review was made of the audiologic investigations undertaken: routine audiometry, auditory brainstem response (ABR), and distortion product otoacoustic emission (OAE). Radiologic investigations were also reviewed consisting of CT and/or MR scans. RESULTS: The database yielded 12 cases of cochlear agenesis. Four patients were excluded because they had absence of the entire inner ear structures ipsilateral to the aplastic cochlear nerve (two cases) and because they had multiple congenital anomalies (two cases). There were equal numbers of males and females. There was a slight left-sided preponderance (5:3), and ages ranged from 5 through 7 years. All children had failed the initial screening audiogram. Follow-up audiologic evaluation revealed either profound loss or dead ear or a failed ABR in the presence of normal OAE testing. All patients had internal auditory canals less than 1.4 mm or MR-compatible findings. CONCLUSIONS: Agenesis of the cochlear nerve may be more common than previously thought, especially in an otherwise healthy, nonsyndromic, school-aged child. Although audiometric evaluation alone usually strongly suggests the diagnosis, definitive evaluation with MR remains the gold standard.

Utility and safety of methylene blue demarcation of preauricular sinuses and branchial sinuses and fistulae in children.

OBJECTIVE: To report our experience at British Columbia's Children's Hospital (BCCH) with the use of methylene blue (MB) to demarcate preauricular sinuses (PASs) and branchial sinuses and fistulae (BSF) in children under general anesthesia just prior to surgical excision. METHODS: A retrospective chart review was performed of all cases at BCCH between March 2003 and April 2006 in which MB was used to demarcate PASs and BSF. A review of the literature regarding the utility and safety of topical MB was performed. RESULTS: MB demarcation of 20 PASs enabled precise resection with the involved auricular cartilage and obviated the need for en bloc resection. In 11 BSF, MB differentiated between sinuses and fistulae, identified the branchial cleft involved, demonstrated (in two patients) a thin fistula that was not visible on high-resolution contrast-enhanced computed tomography, and enabled excision with the use of small incisions and minimal dissection. In our series, there have been no recurrences and no complications from the use of MB. CONCLUSIONS: MB demarcation of PASs and BSF enables minimally invasive surgery and helps ensure complete resection. This is an extremely useful and relatively safe technique that has been underreported in the otolaryngology literature.

Accuracy of the Weber and Rinne tuning fork tests in evaluation of children with otitis media with effusion.

OBJECTIVE: The purpose of this study was to determine the accuracy of the Rinne and Weber tuning fork tests in predicting the degree of hearing loss secondary to otitis media with effusion (OME) in children compared with pure-tone audiometry. DESIGN: A blinded prospective study was performed on cases of unilateral and bilateral OME in children between ages 2 and 11 years. SETTING: Patients were recruited from the tertiary Pediatric Otolaryngology Clinic at British Columbia's Children's Hospital in Vancouver. METHODS: The subjects consisted of 58 children with unilateral or bilateral OME. The Rinne and Weber tests were performed using a 256 Hz and a 512 Hz tuning fork. Pure-tone audiometry was performed to establish the presence and degree of an air-bone gap (ABG). MAIN OUTCOME MEASURES: The tuning fork responses were correlated with the ABG to determine their accuracy. RESULTS: The Weber test did not show an association between the ABG and the lateralization response using either the 256 Hz (p = .484) or the 512 Hz tuning fork (p = .120). For the Rinne test, an increase in ABG was significantly associated with a higher rate of response air conduction < bone conduction using either the 256 Hz or the 512 Hz fork (p < .04). Age was not associated with outcome for any of the tuning fork tests. CONCLUSIONS: The overall accuracy of the Rinne and Weber tuning fork tests in predicting conductive hearing loss associated with OME in children is poor.

Return to home, school, and sports after electrosurgical adenoidectomy: when is it safe?

OBJECTIVE: To determine when it is safe for healthy children to return to home, school, and sports after uncomplicated electrosurgical adenoidectomy (EA). DESIGN: (1) A survey of Canadian otolaryngologists (COs) regarding their EA complication rates and their opinions regarding best practice standards for patient care after EA. (2) Informal retrospective analysis of approximately 200 children who have been allowed to resume sports 3 days after uncomplicated EA. RESULTS: (1) Thirteen COs responded to the survey. The total estimated EA cases performed were 8617 over 10 years. Four patients were known to have bled mildly; none required reoperation. Eleven of 13 COs supported discharge home 2 hours after uncomplicated EA if other standard discharge criteria were met. Seven of 13 COs supported a return to school the next day if the child had returned to his or her usual state of health; however, 2 COs suggested 7 to 10 days at home. Four of 13 COs supported a return to sports 3 days after uncomplicated EA. (2) None of the 200 children who had been allowed to resume sports 3 days after uncomplicated EA have had any known bleeding. At least 10 toddlers have actually engaged in unplanned strenuous exercise within 4 to 6 hours of EA without adverse effect. CONCLUSIONS: Considerable variation exists in Canada in terms of current practice standards regarding return to home, school, and sports after uncomplicated EA. Survey data suggest that, after uncomplicated EA, it is safe for healthy children to return home after 2 hours and to school the next day. Preliminary data suggest that it may be safe for children to resume sports 3 days after uncomplicated EA; however, ideally, this should be studied prospectively, on a large scale.

Treatment of cystic lymphatic vascular malformations with OK-432 sclerotherapy.

BACKGROUND: Cystic lymphatic vascular malformations are benign lesions that can cause disfigurement and functional impairment. Complete surgical resection is often difficult, and clinical recurrence is common. Sclerotherapy has been used as an alternative to excision. OK-432 is a lyophilized mixture of Streptococcus pyogenes and benzylpenicillin which, when injected into a lesion, has shown significant ability to reduce its size or obliterate it completely. METHODS: The authors report a series of 12 patients treated in this fashion at the Vascular Anomalies Clinic, British Columbia Children's Hospital, between 1999 and 2004. All patients underwent imaging of the lesion: 10 had magnetic resonance imaging, one had a computed tomographic scan, and one had ultrasound examination. Six patients had macrocystic malformations (cysts > or = 2 cm) and six had microcystic or combined lymphaticovenous malformations. Patients were treated with intralesional injection of OK-432. The position of the injection was confirmed by angiography and/or ultrasound in 10 cases. Response to treatment was assessed clinically. RESULTS: All patients with macrocystic malformations had complete resolution or good response to treatment. None required any additional treatment. In contrast, those with microcystic or combined malformations responded poorly. All of these patients underwent subsequent excision without adverse consequences. The size and location of the lesion did not correlate with response to treatment. Seventy-five percent of patients experienced pyrexia. Local swelling is an expected phenomenon and must be anticipated, particularly for lesions near the airway. CONCLUSIONS: OK-432 is an excellent treatment for patients with macrocystic lymphatic malformations. However, it is ineffective for microcystic lesions.