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.
Here, we report on response to Trametinib, a MEK inhibitor in children with NF‐1. Our experience presented in this report demonstrates that Trametinib is an effective therapy for advanced, life‐threatening PNF, and treatment‐refractory PLGG in patients with NF‐1 and is well tolerated in children.
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.
To assess the interobserver and intraobserver reliability of the Brodsky and Friedman tonsil size grading scales and a novel 3-grade scale.
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.
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.
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.
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.
Casting a look at pediatric plastic bronchitis Jasinovic, Tin; Kozak, Frederick K; Moxham, J. Paul ...
International journal of pediatric otorhinolaryngology,
10/2015, Letnik:
79, Številka:
10
Journal Article
Recenzirano
Abstract 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.
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.
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.
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.
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.
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).
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.
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.
Abstract 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 2 cm 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 3 French (F) flexible urological forceps through the side port of a 2.5 mm 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.
Abstract 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.
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.
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.
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.
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.
OK-432 is an excellent treatment for patients with macrocystic lymphatic malformations. However, it is ineffective for microcystic lesions.
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.
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.
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.
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).
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