Abstract
Historically, head and neck lymphatic malformations (HNLM) have been managed through surgical and interventional procedures. Sirolimus was introduced in 2016 and has aided in symptomatic control of HNLM. The study objective was to assess healthcare utilization with the introduction of sirolimus for HNLM.
An observational cohort study of LM patients treated between 2008 and 2022 at a tertiary care children's hospital was performed. 588 charts were reviewed; patients with isolated, non-syndromic HNLM and at least 2 years of follow-up were included (n = 45). Data included sirolimus use, complications, presence of tracheostomy and/or gastrostomy-tube, and number and costs of HNLM-related sclerotherapies, procedures, hospitalizations, and emergency room visits. For patients who received sirolimus, encounters two years prior to and after sirolimus initiation were recorded. For the non-sirolimus group, encounters two years after the initial clinic visit for HNLM were recorded. Statistical analysis was used to compare the groups.
Median age at first clinic visit was 1.8 years (range 2 days–41 years). Tracheostomy was present in 43 % of sirolimus patients compared with 3 % of the non-sirolimus group (OR: 24.0, 95%CI: 1.55–1490, p = 0.02). Patients on sirolimus experienced significantly fewer sclerotherapy visits (z = 2.08, p = 0.03) compared to the non-sirolimus group. Minimal sirolimus-related side effects were reported. Total HNLM-related costs were significantly less in the sirolimus group during treatment (median $448.13, range $0–$7041.28) compared with before treatment (median $17,069.24, range $1999.16–$211,848.50, z = 2.20, p = 0.03). Median costs associated with sclerotherapy were less for the sirolimus groups during treatment compared with the non-sirolimus group (z = 1.97, p = 0.04). In the sirolimus group, costs associated with HNLM-related hospitalizations were significantly less during sirolimus treatment compared with before (z = 2.20, p = 0.03).
Sirolimus has improved the clinical course for HNLM patients by decreasing number of procedures and healthcare costs, with limited side effects. Larger cohorts matching type of HNLM and age are needed to assess healthcare utilization benefits of sirolimus.