Functional and symptom impacts of pediatric head and neck lymphatic malformations: developing a patient-derived instrument.
OBJECTIVE
Lymphatic malformations cause significant symptoms and functional deficits. Patients seek care for functional and symptomatic effects of their disease, but current disease burden and treatment outcome measures focus primarily on anatomy and histopathology. The authors describe disease impacts reported by patients and parents as a step toward more comprehensive disease burden assessments.


METHODS
Cross-sectional.


METHODS
Children's hospital vascular anomaly clinic.


METHODS
Participants were recruited through a pediatric vascular anomaly clinic. A panel of senior pediatric otolaryngologists and an outcomes scientist developed interview questions based on clinical and research experience and available literature. The outcomes scientist conducted parent and adolescent interviews. The panel reviewed responses to define relevant items within functional domains. Participants rated impact on daily life for each domain.


RESULTS
Thirty-one participants represented all 5 de Serres stages (mean [SD] age, 9 [6] years; n = 11 adolescents and 20 parents). Adolescents reported frequent sickness as the domain with greatest impact. Sleep was more affected in adolescents with higher stage lesions. Parents of younger children reported greatest impact on breastfeeding. For adolescents, lesion stage predicted perceived social stigma (controlling for age), whereas increasing age was associated with greater impact from swelling (controlling for stage). For parents, stage predicted breastfeeding impact (controlling for stage).


CONCLUSIONS
This is the first detailed assessment of patient- and parent-reported functional and symptomatic impacts of head and neck lymphatic malformations. Both adolescent patients and parents of younger children reported significant symptom and functional effects of this disease.
Department of Otolaryngology-Head and Neck Surgery, University of Washington, Seattle, WA, USA. karthikb@uw.edu
Karthik
K
Balakrishnan
Todd C
TC
Edwards
Jonathan A
JA
Perkins
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003430:Cross-Sectional Studies; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007407:Interviews as Topic; D044148:Lymphatic Abnormalities / Q000175:diagnosis* / Q000503:physiopathology*; D010290:Parents