Long-term follow-up of anti-TNF treatment in adult and pediatric DADA2 patients: Insights from real-world data.

Background/purpose: Our objective was to investigate real-world outcomes and treatment strategies in individuals affected by DADA2 using over 10-year period real-life experience.

Methods: This descriptive analysis encompassed all adult/pediatric patients with DADA2 from our Vasculitis Research Center prospective database. Patients on anti-TNF therapy have been specifically examined, analyzing the treatment's duration, indications, and outcomes. Treatment responses were based on physicians' assessments and categorized as full response (symptom-free with normal acute phase reactants) or partial/no response.

Results: Totally 32 patients (Adult/Childhood age: 19/13) were analyzed. Anti-TNF agents were prescribed to 27 of the 32 patients. Over a median follow-up of 58 months on anti-TNF therapy, 10 patients (35.7%) exhibited a complete response, predominantly in cases with nervous system or skin involvement. Partial responses were observed in the other 10 patients (35.7%). Currently, 20/ 27 patients remain on anti-TNF treatment. Among the seven who are not on anti-TNF now: five died (four of them with a late diagnosis, one could not continue due to cardiomyopathy), one refused treatment and one had a cure after bone marrow transplantation. We have become aware that four patients increased their dose interval and one returned to the normal interval after an increase in CRP. The first patient was diagnosed in 2013 and over the last 10 years, 6/32 (18.8%) of the patients died.

Conclusion: Anti-TNF treatment is beneficial for vasculitic and inflammatory lesions. The clinical course of patients is diverse, especially if the diagnosis is delayed, with a mortality rate of up to 20% over a 10-year period.