A Survey of Pediatric Acute-Onset Neuropsychiatric Syndrome Characteristics and Course.

OBJECTIVE To date, studies in the area of pediatric acute-onset neuropsychiatric syndrome (PANS; including pediatric autoimmune neuropsychiatric disorder associated with streptococcal infection [PANDAS] and pediatric infection-triggered neuropsychiatric disorder [PITAND]) have been relatively small and hence unable to comprehensively address questions of disease heterogeneity (e.g., by age, gender), comorbidities, and progression. In this study, we investigated an internet survey sample to more fully characterize the phenotypic traits; medical, family, and developmental history; functional challenges; and clinical course associated with PANS. METHODS Six hundred and ninety-eight patients with clinical diagnoses of PANS were included in this study. Participants, who included parents and legal guardians (for minors) or the PANS patients themselves (for those ages 18 and older), were asked to complete a 146-question survey designed to ascertain medical, developmental, and family history; PANS symptomatology; medical and nonmedical interventions for PANS; PANS course; PANS outcomes; and access to PANS care. RESULTS Our results agree with previous findings concerning the core symptoms of PANS as well as its male predominance (65% in this survey) and infection-triggered onset, thus validating the study population. Infection was implicated as the primary inciting factor in 65% of patients; 54% of patients reported an association with group A streptococcus specifically. The results of this survey also revealed new findings, including a surprisingly strong impact of gender and pubertal status on symptom course and chronicity, a high rate of medical comorbidity suggesting generalized immune dysfunction, a profound impact of PANS episodes on functional status, and a role for early resolution of infection through antibiotic treatment in disease course. CONCLUSIONS This study serves as the first survey of its size to provide insight into the global clinical picture and range of phenotypes of PANS patients. Significant results included the impact of gender and pubertal status on phenotype, affirmation of the role of the immune system in PANS pathology, and the role of timely resolution of infection in clinical outcomes. Understanding how PANS presents in a broad population-based sample, within the limitations of a self-selected and administered online survey, is an important step toward improving diagnosis, creating more targeted treatment options, educating the clinical and research community, and generating hypotheses for future prospective research.