Treatments and Outcomes of Pediatric Head and Neck Lymphatic Malformations: A 20-Year Single Institution Experience

Introduction: The management of pediatric head and neck lymphatic malformations (HNLMs) is complex due to heterogeneity in malformations, variability in patient age, and multidisciplinary care. The goal of this study was to evaluate the management and outcomes of pediatric HNLM patients at a single institution to investigate HNLM treatments across different conditions.

Methods: We performed a retrospective chart review on children aged 0-18 years treated for HNLMs at Johns Hopkins Hospital between January 1, 2000 and August 1, 2023. We collected data on patient demographics, classifications of HNLMs, symptoms, and comorbidities. We classified treatments as unimodal or multimodal, including surgery, sclerotherapy, and sirolimus. Outcome measures include estimated size reduction of LM posttreatment and complications by treatment type.

Results: We identified 101 children (50% male) with HNLMs, 44% of whom first received treatment before the age of 2 years. 50 patients received unimodal treatment, 11 received multimodal treatment, and 40 underwent active observation only without initiation of treatment. Of them, 53% received sclerotherapy. It was associated (p < 0.01) with greater size reduction of low-stage (de Serres stage 1-3) HNLMs and complete resolution in 25.5% of them. Sirolimus was given to 63% of high-stage (de Serres stage 4-5) HNLMs, achieving partial reduction in 80%. Sclerotherapy was the most common treatment in macrocystic (77%) and mixed (67%) HNLMs, achieving 29.6% and 3.7% resolution rates, respectively. Multimodal treatments were most common for microcystic HNLMs. Sclerotherapy had a similar all-inclusive complication rate (14%) to sirolimus therapy (11%). Surgery had the highest all-inclusive complication rate (27%).

Conclusions: Sclerotherapy is a safe and effective treatment modality for low-stage and macrocystic pediatric HNLMs. Observation alone was chosen in many patients. Few high-stage HNLMs had a significant response to treatment. As newer mutation-specific treatments emerge, there are opportunities for improved treatment algorithms for all stages, types, and patient ages.