Primary surgery vs primary sclerotherapy for head and neck lymphatic malformations

Abstract

Importance: The optimal treatment for head and neck lymphatic malformations (LMs) is unknown. To our knowledge, this is the first head-to-head comparison of primary surgery and sclerotherapy for this condition.

Objective: To compare surgery and sclerotherapy as initial treatment for head and neck LMs.

Design, setting, and participants: Retrospective cohort study including patients in 2 pediatric vascular anomaly programs receiving treatment for head and neck LMs.

Interventions: Primary surgery or primary sclerotherapy and any subsequent therapy within 1 year.

Main outcomes and measures: Treatment effectiveness was measured by (1) need for further therapy after first treatment and within 1 year and (2) change in Cologne Disease Score (CDS). Resource utilization was reflected by total intervention number, hospital and intensive care unit (ICU) days, and tracheostomy placement.

Results: A total of 174 patients were studied. Their mean (SD) age at presentation was 4.2 (4.7) years; 45.1% were female. The initial treatment was surgery in 55.8%, sclerotherapy in 35.1%, and other interventions in 9.1%. The LM stage ranged from 1 to 5, with similar distributions (P = .15) across initial treatment types; 31.2% of LMs were macrocystic, 34.8% were microcystic, and 33.9% were mixed, with similar distributions across treatment types. Patients receiving sclerotherapy had worse pretreatment CDS subscores for respiration, nutrition, and speech (all P ≤ .02). In univariate analysis, initial surgery and initial sclerotherapy had similar effectiveness after the first intervention (P = .21) and at 1 year (P = .30). In multivariate analysis controlling for lesion stage and type, initial surgery and sclerotherapy did not differ in effectiveness after the first intervention (P = .28) or at 1 year (P = .97). Total CDS and subscale changes were similar between treatment types except for the nutrition subscale. Treatment type did not predict total number of interventions (P = .64), total hospital days (P = .34), total ICU days (P = .59), or higher likelihood of subsequent tracheostomy (P = .36). Higher LM stage predicted more hospital and ICU days and higher likelihood of tracheostomy (all P ≤ .02).

Conclusions and relevance: In this multisite comparison, initial surgery and sclerotherapy for head and neck LMs were similar in effectiveness and resource utilization. Higher stage predicted greater resource utilization.