Centers of Excellence: If We Build It, Will They Come?

Pediatric surgeons have been pursuing high quality, affordable care or value-based care for over 50 years. One approach to streamlining the clinical care for a complex problem was the development of a center of excellence (COE). The concept of COE focuses on a shared vision of providing high quality care through a multidisciplinary approach. The goal is to improve diagnostic accuracy as well as therapeutic outcomes using focused expertise within a group. COEs are often resource intensive before becoming fiscally viable and therefore require initial support from hospital leadership. This review discusses the key steps to consider before building a COE, strategies to help build one, and how to keep one successful as defined by quality, accessibility, equity, training, and maintaining teams within the group.

American society of pediatric otolaryngology vascular anomalies task force review of tongue venous malformations: Techniques, pearls, and pitfalls.

Management of tongue venous malformations can be challenging in the pediatric population due to their heterogeneity in presentation, extent of involvement and functional compromise. It is important to recognize the value of various treatment options in order to guide management of each patient in an individualized fashion. Here we describe a series of patients with tongue venous malformations that are managed using diverse modalities to illustrate the relative benefits and risks of each technique. The challenges of venous malformation treatment can be mitigated by tailoring the approach to each individual patient and malformation. This case series also emphasizes the need and importance of working in the setting of a multidisciplinary vascular anomalies team.

Sphincter Pharyngoplasty for Velopharyngeal Dysfunction: Impact of 22q11.2 Deletion Syndrome.

OBJECTIVE: Patients with 22q11.2 deletion syndrome (22q11DelS) often present with velopharyngeal dysfunction (VPD). VPD in patients with 22q11DelS is multifactorial beyond velopharyngeal insufficiency (VPI) alone, and differences in surgical outcomes are poorly understood. Our objective was to determine whether patients with 22q11DelS have an increased risk for persistent VPI after sphincter pharyngoplasty compared to patients without 22q11DelS. METHODS: We completed a retrospective cohort study of patients with 22q11DelS undergoing sphincter pharyngoplasty between 1995 and 2019 using a VPD clinic database. Patients with 22q11DelS were compared to a cohort of 2:1 frequency-matched (age, degree of velopharyngeal closure) patients without 22q11DelS. Variables included patient characteristics, surgical history, perceptual speech evaluation, and degree of closure on nasopharyngoscopic evaluations. Primary outcomes included postoperative VPI severity and hypernasality. Speech and nasopharyngoscopic characteristics were compared using Fisher's exact test. Postoperative VPI severity and hypernasality were compared between groups via relative risks (RR) from mixed effects Poisson regression models, with random effects of age and velopharyngeal closure. RESULTS: 134 patients (51 22q11DelS, 83 matched) were included, with mean age of 7.3 years (standard deviation 3.0) and 50% male. Cohorts had similar preoperative speech characteristics and nasopharyngoscopic findings. Patients with 22q11DelS had similar postoperative VP function as patients without 22q11DelS (RR 0.85, CI 0.46-1.57 for VPI severity, RR 0.83, CI 0.45-1.53 for hypernasality). Even after adjusting by preoperative variables, no differences were seen between both groups. CONCLUSION: Matched for age and pre-operative velopharyngeal closure, patients with and without 22q11DelS and VPI had similar benefits after sphincter pharyngoplasty. LEVEL OF EVIDENCE: Non-randomized controlled cohort study, 3 Laryngoscope, 133:2813-2820, 2023.

Delaying Invasive Treatment in Unilateral Head and Neck Lymphatic Malformation Improves Outcomes.

OBJECTIVES: Large (De Serres stage [IV-V]) head and neck lymphatic malformations (HNLMs) often have multiple, high-risk, invasive treatments (ITs) to address functional compromise. Logically reducing HNLM ITs should reduce treatment risk. We tested whether delaying HNLM ITs reduces total IT number. MATERIALS: Consecutive HNLM patients (n = 199) between 2010 and 2017, aged 0-18 years. METHODS: ITs (surgery or sclerotherapy) were offered for persistent or dysfunction causing HNLMs. Treatment effectiveness categorized by IT number: optimal (0-1), acceptable (2-5), or suboptimal (>5). Clinical data were summarized, and outcome associations tested (χ2 ). Relative risk (RR) with a Poisson working model tested whether HNLM observation or IT delay (>6 months post-diagnosis) predicts treatment success (i.e., ≤1 IT). RESULTS: Median age at HNLM diagnosis was 1.3 months (interquartile range [IQR] 0-45 m) with 107/199(54%) male. HNLM were stage I-III (174 [88%]), IV-V (25 [13%]). Initial treatment was observation (70 [35%]), invasive (129 [65%]). Treatment outcomes were optimal (137 [69%]), acceptable (36 [18%]), and suboptimal (26 [13%]). Suboptimal outcome associations: EXIT procedure, stage IV-V, oral location, and tracheotomy (p < 0.001). Stage I-III HNLMs were initially observed compared with stage I-III having ITs within 6 months of HNLM diagnosis, had a 82% lower relative treatment failure risk ([i.e., >1 IT], RR = 0.09, 95% CI 0.02-0.36, p < 0.001). Stage I-III HNLMs with non-delayed ITs had reduced treatment failure risk compared with IV-V (RR = 0.47, 95% CI 0.33-0.66, p < 0.001). CONCLUSION: Observation and delayed IT in stage I-III HNLM ("Grade 1") is safe and reduces IT (i.e., ≤1 IT). Stage IV-V HNLMs ("Grade 2") with early IT have a greater risk of multiple ITs. LEVEL OF EVIDENCE: 4 Laryngoscope, 133:956-962, 2023.

Primary targeted medical therapy for management of bilateral head and neck lymphatic malformations in infants.

OBJECTIVES: Patients born with bilateral head and neck lymphatic malformations (BHNLMs) often require multiple invasive treatments, including tracheostomy. We hypothesized that primary targeted medical therapy (pTMT) with diagnostic needle aspiration reduces the need for invasive therapy such as surgical resection and/or sclerotherapy. METHODS: Retrospective case review was performed of infants with BHNLMs (Grade 2 or De Serres stage IV and V) treated only at our institution from 2000 to 2021. Patients were divided into two cohorts: those managed with pTMT and those managed with observation, sclerotherapy, or surgical intervention (non-pTMT). Data regarding interventions, clinical outcomes, morbidity, and mortality were analyzed with descriptive statistics. RESULTS: Nine children with BHNLMs met inclusion criteria. Three (33%) were in the pTMT cohort and six (66%) were non-pTMT. Eight (89%) malformations were genotyped, and all demonstrated hotspot PIK3CA variants. All pTMT patients had sirolimus initiated in the first month of life and underwent needle aspiration of malformation cyst fluid for cell-free DNA samples. All pTMT patients tolerated medical therapy. For the non-pTMT cohort, primary treatment included none (deceased, n = 1, 17%), observation with needle aspiration (n = 1, 17%), surgical resection (n = 2, 33%), or combination surgery and sclerotherapy (n = 2, 33%). Intubation duration, intensive care and initial hospital length of stay were not different between cohorts. Four non-pTMT patients (67%) required tracheostomy, and two (33%) died prior to discharge. All pTMT patients survived and none required tracheostomy. Non-pTMT patients required a median of two invasive therapies prior to discharge (IQR 1-4) and a mean total of 13 over the course of their lifetime (IQR 1-16), compared to the pTMT group who did not require any lifetime invasive therapy, even after initial pTMT and discharge home. CONCLUSION: This study compares patients with BHNLMs (Grade 2) treated with pTMT versus those treated with observation or invasive therapy. Patients treated with pTMT required no surgical or invasive procedural treatment of their malformations, no tracheostomy placement, no unplanned readmissions after discharge, and had no mortalities. Needle aspiration was useful as a therapeutic adjunct for cell-free DNA diagnosis of PIK3CA variants, which guided TMT.

Alpelisib for the treatment of PIK3CA-related head and neck lymphatic malformations and overgrowth.

PURPOSE: PIK3CA-related overgrowth spectrum (PROS) conditions of the head and neck are treatment challenges. Traditionally, these conditions require multiple invasive interventions, with incomplete malformation removal, disfigurement, and possible dysfunction. Use of the PI3K inhibitor alpelisib, previously shown to be effective in PROS, has not been reported in PIK3CA-associated head and neck lymphatic malformations (HNLMs) or facial infiltrating lipomatosis (FIL). We describe prospective treatment of 5 children with PIK3CA-associated HNLMs or head and neck FIL with alpelisib monotherapy. METHODS: A total of 5 children with PIK3CA-associated HNLMs (n = 4) or FIL (n = 1) received alpelisib monotherapy (aged 2-12 years). Treatment response was determined by parental report, clinical evaluation, diary/questionnaire, and standardized clinical photography, measuring facial volume through 3-dimensional photos and magnetic resonance imaging. RESULTS: All participants had reduction in the size of lesion, and all had improvement or resolution of malformation inflammation/pain/bleeding. Common invasive therapy was avoided (ie, tracheotomy). After 6 or more months of alpelisib therapy, facial volume was reduced (range 1%-20%) and magnetic resonance imaging anomaly volume (range 0%-23%) were reduced, and there was improvement in swallowing, upper airway patency, and speech clarity. CONCLUSION: Individuals with head and neck PROS treated with alpelisib had decreased malformation size and locoregional overgrowth, improved function and symptoms, and fewer invasive procedures.

Somatic activating BRAF variants cause isolated lymphatic malformations.

Somatic activating variants in PIK3CA, the gene that encodes the p110α catalytic subunit of phosphatidylinositol 3-kinase (PI3K), have been previously detected in ∼80% of lymphatic malformations (LMs).1 , 2 We report the presence of somatic activating variants in BRAF in individuals with LMs that do not possess pathogenic PIK3CA variants. The BRAF substitution p.Val600Glu (c.1799T>A), one of the most common driver mutations in cancer, was detected in multiple individuals with LMs. Histology revealed abnormal lymphatic channels with immunopositivity for BRAFV600E in endothelial cells that was otherwise indistinguishable from PIK3CA-positive LM. The finding that BRAF variants contribute to low-flow LMs increases the complexity of prior models associating low-flow vascular malformations (LM and venous malformations) with mutations in the PI3K-AKT-MTOR and high-flow vascular malformations (arteriovenous malformations) with mutations in the RAS-mitogen-activated protein kinase (MAPK) pathway.3 In addition, this work highlights the importance of genetic diagnosis prior to initiating medical therapy as more studies examine therapeutics for individuals with vascular malformations.

Avoidance of surgery for head and neck infantile myofibromatosis using imatinib monotherapy.

Describe a novel use for a kinase inhibitor, imatinib, in young children with a known activated somatic mutation in PDGFR-beta. Two patients with infantile myofibromatosis treated with imatinib. Case description of evaluation, diagnosis and treatment decisions for infantile myfibromatosis of the head and neck. Description of medical therapy for infantile myofibromatosis in these patients. For function threatening myofibromas of a known genotype, in infants, targeted medical therapy is a treatment option.

Complex Airway Management in Patients with Tracheal Cartilaginous Sleeves.

OBJECTIVES/HYPOTHESIS: A tracheal cartilaginous sleeve (TCS) is a rare anomaly characterized by anterior fusion of tracheal cartilages. TCS is associated with syndromic craniosynostoses including Apert, Crouzon and Pfeiffer syndromes and FGFR2, FGFR3, and TWIST1 variants. This study presents a 30-year review of patients with syndromic craniosynostosis and TCS and describes diagnostic methods, genetic variants, surgical interventions, and long-term outcomes. STUDY DESIGN: Retrospective, single-institution review. METHODS: This review included patients with syndromic craniosynostosis and TCS treated at Seattle Children's Hospital from 1990 to 2020. Tracheostomy, genetic variants, and additional surgery were primary measures. Fisher's exact test compared need for tracheostomy in patients with proposed high-risk (FGFR2 p.W290 or FGFR2 p.C342) versus low-risk genetic variants. RESULTS: Thirty patients with TCS were identified. Average age at diagnosis was 12 months (range 2-weeks to 7.9-years; standard deviation 19.8 months). Syndromes included Pfeiffer (37%), Apert (37%), and Crouzon (26%). Severe obstructive sleep apnea was present in 76% of patients. Tracheostomy was performed in 17 patients (57%); five were successfully decannulated. Additional interventions included adenotonsillectomy (57%), nasal (20%), laryngeal (17%), and craniofacial skeletal surgery (87%). All patients with Pfeiffer syndrome and FGFR2 p.W290C variants and 83% of patients with FGFR2 p.C342 variants required tracheostomy, differing from other variants (P = .02, odds ratio 33, 95% confidence interval 1.56-697.96). One patient (3%) died. CONCLUSION: TCS contributes to multilevel airway obstruction in patients with syndromic craniosynostosis. Genetic testing in patients with FGFR2-related syndromic craniosynostoses may identify those at risk of TCS and facilitate early intervention. A better understanding of this patient population may foster individualized airway management strategies and improve outcomes. LEVEL OF EVIDENCE: 4 Laryngoscope, 132:215-221, 2022.

Accuracy and Reliability of 4D-CT and Flexible Laryngoscopy in Upper Airway Evaluation in Robin Sequence.

OBJECTIVES: To evaluate the performance of 4-dimensional computed tomography (4D-CT) in assessing upper airway obstruction (UAO) in patients with Robin sequence (RS) and compare the accuracy and reliability of 4D-CT and flexible fiber-optic laryngoscopy (FFL). STUDY DESIGN: Prospective survey of retrospective clinical data. SETTING: Single, tertiary care pediatric hospital. METHODS: At initial and 30-day time points, a multidisciplinary group of 11 clinicians who treat RS rated UAO severity in 32 sets of 4D-CT visualizations and FFL videos (dynamic modalities) and static CT images. Raters assessed UAO at the velopharynx and oropharynx (1 = none to 5 = complete) and noted confidence levels of each rating. Intraclass correlation and Krippendorff alpha were used to assess intra- and interrater reliability, respectively. Accuracy was assessed by comparing clinician ratings with quantitative percentage constriction (QPC) ratings, calculated based on 4D-CT airway cross-sectional area. Results were compared using Wilcoxon rank-sum and signed-rank tests. RESULTS: There was similar intrarater agreement (moderate to substantial) with 4D-CT and FFL, and both demonstrated fair interrater agreement. Both modalities underestimated UAO severity, although 4D-CT ratings were significantly more accurate, as determined by QPC similarity, than FFL (-1.06 and -1.46 vs QPC ratings, P = .004). Overall confidence levels were similar for 4D-CT and FFL, but other specialists were significantly less confident in FFL ratings than were otolaryngologists (2.25 and 3.92, P < .0001). CONCLUSION: Although 4D-CT may be more accurate in assessing the degree of UAO in patients with RS, 4D-CT and FFL assessments demonstrate similar reliability. Additionally, 4D-CT may be interpreted with greater confidence by nonotolaryngologists who care for these patients.

Predictors of Success After Furlow Palatoplasty for Repair of Cleft Palate: Does Syndrome Matter?

OBJECTIVE: To determine predictors of success following Veau 1 and 2 cleft palate repair in patients with and without syndromes. STUDY DESIGN: Retrospective review of prospectively collected data. SETTING: Tertiary care children's hospital. METHODS: All children <18 months of age undergoing Furlow palatoplasty for Veau 1 and 2 cleft repair between 2000 and 2014 with postoperative perceptual speech assessment (PSA). RESULTS: In total, 368 consecutive patients were identified; 95 were excluded, resulting in 273 patients. Median age at surgery was 13.0 months (interquartile range [IQR], 11-15 months) with postoperative PSA at a median of 32.3 months (IQR, 26.3-44.5 months). Fifty patients (18.3%) had syndrome diagnosis; 59 patients (21.6%) had nonsyndromic Robin sequence. Velopharyngeal insufficiency (VPI) occurred in 27 patients (10.5%); 13 underwent secondary speech surgery. Cleft-related speech errors occurred in 46 patients (17.6%). Non-cleft-related speech errors occurred in 155 patients (59.6%) and reduced intelligibility in 127 patients (47.9%). Oronasal fistula occurred in 23 patients (8.8%) and was exclusive to Veau 2 clefts. In multivariate analysis, age >13 months at palatoplasty demonstrated a 6-fold higher rate of VPI (hazard ratio [HR], 6.64; P < .01), worse speech outcomes (HR, 6.04; P < .01; HR, 1.60; P < .01; HR, 1.57; P = .02), and greater speech therapy utilization (HR, 2.18; P < .01). CONCLUSION: VPI occurred in 10% of patients undergoing Furlow palatoplasty repair of Veau 1 or 2 clefts. Age <13 months at palatoplasty was associated with improved speech outcomes and lower VPI incidence (2.8% vs 16.2%). Syndromic diagnosis was associated with noncleft speech errors and reduced intelligibility on univariate analysis but not velopharyngeal function after palatoplasty.

Migration of n-BCA glue as a complication of venous malformation treatment in children.

Preoperative n-butyl cyanoacrylate (n-BCA) embolization of venous malformations facilitates surgical resection. Although embolization is generally well-tolerated, central venous n-BCA migration can occur. The purpose of this article is to describe 3 cases of glue migration requiring glue embolectomy. Strategies for prevention and treatment of glue migration during embolization of venous malformations are reviewed.

Acetylsalicylic acid suppression of the PI3K pathway as a novel medical therapy for head and neck lymphatic malformations.

OBJECTIVES: Head and neck lymphatic malformations (HNLM) are caused by gain-of-function somatic mutations in PIK3CA. Acetylsalicylic acid (ASA/aspirin) is thought to limit growth in PIK3CA-mutated neoplasms through PI3K pathway suppression. We sought to determine if ASA could be beneficial for HNLM. METHODS: Retrospective case series of patients (0-18 years) offered ASA (3-5 mg/kg/day) for HNLM treatment (2010-2018). Clinical and treatment characteristics, patient-reported symptom improvement, medication tolerance, compliance, and complications were recorded. Treatment response was determined by change in patient/caregiver-reported symptoms, or HNLM size [complete (resolved), partial (decreased), or stable]. RESULTS: Fifty-three patients were offered ASA, 23 (43%) accepted (median age 10 years, IQR 6-14). Compared to patients who declined, patients receiving ASA were more likely to have extensive malformations: ex-utero intrapartum treatment procedure, bilateral malformations, oral cavity location, ≥2 invasive treatments, or tracheotomy (p < 0.05). All patients with tissue available had PIK3CA mutations (13/23). Treatment indications included oral pain/blebs (12, 52%), recurrent pain/swelling (6, 26%), or sudden/persistent swelling (5, 22%). Treatment plan was commonly one 81 mg tablet daily (19, 83%) for 3-12 months (8, 42%). Therapeutic adherence was reported by 18 patients (78%). Symptoms improved in 18 patients [78%; decreased pain (9, 39%) and swelling (8, 35%)]. Treatment resulted in partial (14, 61%) or complete response (4, 17%). Three patients developed oral bleb bleeding, which resolved with medication discontinuation. CONCLUSION: ASA seems to be a well-tolerated, low-risk medication for HNLM treatment. This pilot study suggests that it often improves symptoms and reduces HNLM size. Further prospective, randomized studies are warranted to comprehensively assess indications, safety, and efficacy. LEVEL OF EVIDENCE: Level 4.

Impact of Eliminating Local Anesthesia on Immediate Postoperative Analgesia in Pediatric Ambulatory Adenotonsillectomy.

Our goal was to standardize intraoperative analgesic regimens for pediatric ambulatory tonsillectomy by eliminating local anesthetic use and to determine its impact on postoperative pain measures, while controlling for other factors. METHODS: We assembled a quality improvement team at an ambulatory surgery center. They introduced a standardized anesthetic protocol, involving American Society of Anesthesiologists Classification 1 and 2 patients undergoing adenotonsillectomy. Local anesthesia elimination was the project's single intervention. We collected pre-intervention data (79 cases) from July 5 to September 17, 2019 and post-intervention data (59 cases) from September 25 to December 17, 2019. The intervention requested that surgeons eliminate the use of local anesthetics. The following outcomes measures were evaluated using statistical process control charts and Shewhart's theory of variation: (1) maximum pain score in the post-anesthesia care unit, (2) total post-anesthesia care unit minutes, and (3) postoperative opioid rescue rate. RESULTS: No special cause variation signal was detected in any of the measures following the intervention. CONCLUSIONS: Our data suggest that eliminating intraoperative local anesthetic use does not worsen postoperative pain control at our facility. The intervention eliminated the added expenses and possible risks associated with local anesthetic use. This series is unique in its standardization of anesthetic regimen in a high-volume ambulatory surgery center with the exception of local anesthesia practices. The study results may impact the standardized clinical protocol for pediatric ambulatory adenotonsillectomy at our institution and may hold relevance for other centers.

Computational fluid dynamics modeling aiding surgical planning in a toddler with Parkes Weber syndrome.

Parkes Weber syndrome is a fast-flow and slow-flow vascular anomaly with limb overgrowth that can lead to congestive heart failure and limb ischemia. Current management strategies have focused on symptom management with focal embolization. A pediatric case with early onset heart failure is reported. We discuss the use of computational fluid dynamics (CFD) modeling to guide a surgical management strategy in a toddler with an MAP2K1 mutation.

Cell-free DNA as a diagnostic analyte for molecular diagnosis of vascular malformations.

PURPOSE: Vascular malformations (VM) are primarily caused by somatic activating pathogenic variants in oncogenes. Targeted pharmacotherapies are emerging but require molecular diagnosis. Since variants are currently only detected in malformation tissue, patients may be ineligible for clinical trials prior to surgery. We hypothesized that cell-free DNA (cfDNA) could provide molecular diagnoses for patients with isolated VM. METHODS: cfDNA was isolated from plasma or cyst fluid from patients with arteriovenous malformations (AVM), venous malformations (VeM), or lymphatic malformations (LM), and assayed for known pathogenic variants using droplet digital polymerase chain reaction (ddPCR). Cyst fluid cfDNA from an independent cohort of LM patients was prospectively screened for variants using a multiplex ddPCR assay. RESULTS: Variants were detected in plasma cfDNA in patients with AVM (2/8) and VeM (1/3). Variants were detected in cyst fluid cfDNA (7/7) but not plasma (0/26) in LM patients. Prospective testing of cyst fluid cfDNA with multiplex ddPCR identified variants in LM patients who had never undergone surgery (4/5). CONCLUSION: Variants were detected in plasma from AVM and VeM patients, and in cyst fluid from patients with LM. These data support investigation of cfDNA-based molecular diagnostics for VM patients, which may provide opportunities to initiate targeted pharmacotherapies without prior surgery.

Airway Hemangiomas in PHACE Syndrome: A Multicenter Experience.

OBJECTIVE: To describe the prevalence and clinical characteristics of airway findings in a multi-institutional cohort of PHACE patients. STUDY DESIGN: Multicenter retrospective case series. SETTING: Multidisciplinary vascular anomalies clinics at 2 institutions. METHODS: Data were collected from the electronic medical record, including clinical presentation, airway findings, treatment, and outcomes. RESULTS: Of 55 PHACE patients, 22 (40%) had airway hemangiomas. Patients with airway involvement were more commonly female (P = .034, odds ratio [OR] 23, 95% confidence interval [CI] 1.3-410) and of Caucasian ethnicity (P = .020, OR 5.3, 95% CI 1.3-21). Anatomically, patients with bilateral S3 involvement had higher rates of airway disease (P = .0012, OR 15, 95% CI 2.9-77). Most patients with airway hemangiomas had stridor (68%). Of the patients managed in the propranolol era (2008 or later, n = 35), 14 had airway involvement. All 14 were treated with propranolol, whereas 13 (62%) of 21 nonairway patients were treated with propranolol. The average treatment duration was longer in the airway patients (22.1 vs 16.7 months). All patients who underwent tracheostomy (n = 4) did so before 2008. CONCLUSION: Risk factors for airway involvement in PHACE include female gender, Caucasian ethnicity, and stridor. Since the widespread use of propranolol, fewer patients have required surgical management of their airway disease. Given the high prevalence of airway involvement even in patients without stridor, assessment of the airway is a crucial component of a comprehensive PHACE workup.

Speech Outcomes After Sphincter Pharyngoplasty for Velopharyngeal Insufficiency.

OBJECTIVES/HYPOTHESIS: To investigate perceptual speech outcomes following sphincter pharyngoplasty (SP) and to identify patient characteristics associated with velopharyngeal insufficiency (VPI) resolution or improvement. METHODS: Retrospective review of prospectively collected data was performed of consecutive patients that underwent SP for management of VPI between 1994 and 2016 at a single tertiary care pediatric hospital. Demographic data, nasendoscopic findings, and speech characteristics were recorded using a standardized protocol. Pre- and post-operative VPI was graded on a five-point Likert scale. Frequency of post-operative VPI resolution and improvement was assessed and associations with patient characteristics were analyzed. The association between odds of VPI resolution or improvement and five patient characteristics identified a priori was performed controlling for confounding factors. RESULTS: Two-hundred ninety-six subjects were included. All patients had at least minimal VPI pre-operatively; 72% were graded moderate or severe. Sixty-four percent experienced resolution and 83% improved at least one point on the VPI-severity scale. Of the five patient characteristics, only history of cleft palate repair was significantly associated with decreased odds of VPI improvement but not resolution when controlling for other variables. CONCLUSIONS: Sphincter pharyngoplasty resulted in resolution of VPI in 64% and improvement in 83% of subjects. Children with a history of cleft palate had significantly decreased odds of VPI improvement compared to those without a history of cleft palate. Neither syndrome diagnosis nor 22q11 deletion had a significant association with speech outcomes after sphincter pharyngoplasty. LEVEL OF EVIDENCE: 4 Laryngoscope, 131:E2046-E2052, 2021.

Active Observation as an Alternative to Invasive Treatments for Pediatric Head and Neck Lymphatic Malformations.

OBJECTIVES: An increasing number of treatment modalities for lymphatic malformations are being described, complicating therapeutic decisions. Understanding lymphatic malformation natural history is essential. We describe management of head and neck lymphatic malformations where decisions primarily addressed lesion-induced functional compromise (ie, breathing, swallowing) to identify factors associated with invasive treatment and active observation. We hypothesize that non-function threatening malformations can be observed. STUDY DESIGN: Retrospective case series. METHODS: Retrospective case series of consecutive head and neck lymphatic malformation patients (2000-2017) with over 2 years of follow-up. Patient characteristics were summarized and associations with invasive treatment (surgery or sclerotherapy) tested using Fisher's exact. In observed patients, factors associated with spontaneous regression were assessed with Fisher's exact test. RESULTS: Of 191 patients, 101 (53%) were male, 97 (51%) Caucasian, and 98 (51.3%) younger than 3 months. Malformations were de Serres I-III 167 (87%), or IV-V 24 (12%), and commonly located in the neck (101, 53%), or oral cavity (36, 19%). Initial treatments included observation (65, 34%) or invasive treatments such as primary surgery (80, 42%), staged surgery (25, 13%), or primary sclerotherapy (9, 5%). Of 65 initially observed malformations, 8 (12%) subsequently had invasive treatment, 36 (58%) had spontaneous regression, and 21 (32%) elected for no invasive therapy. Spontaneous regression was associated with location in the lateral neck (P = .003) and macrocystic malformations (P = .017). CONCLUSION: Head and neck lymphatic malformation treatment selection can be individualized after stratifying by stage, presence of functional compromise, and consideration of natural history. Recognizing the spectrum of severity is essential in evaluating efficacy of emerging treatments, as selected malformations may respond to observation. LEVEL OF EVIDENCE: 4 Laryngoscope, 131:1392-1397, 2021.