Pediatric Evans Syndrome: A 20-year experience from a tertiary center in Brazil

ABSTRACT

Abstract Introduction The Evans syndrome (ES) is a rare, often chronic, relapsing and treatment-refractory hematological disorder. We described the clinical features, diagnostic workup, treatment and outcome in patients with ES. Method We performed a retrospective chart review of patients aged < 18 years with ES admitted to a tertiary center in Brazil from 2001 to 2021. The analysis of the data was primarily descriptive, using median, interquartile range and categorical variables presented in absolute frequencies. Main results Twenty patients (12 female, 8 male) were evaluated in this study. The median age at the initial cytopenia was 4.98 years (1.30-12.57). The ES was secondary in nine cases (45%), of which six patients (30%) showed autoimmune disease (AID) or primary immunodeficiencies (PID) and one presented a spontaneous recovery. Steroids and intravenous immunoglobulin were first-line therapy in 19 cases. Twelve patients (63%) required second-line treatments (rituximab, cyclosporine, splenectomy, sirolimus, cyclophosphamide, mycophenolate mofetil, azathioprine and eltrombopag). The median follow-up period was 2.41 years (1.4 -7.52). One patient (5%) died of underlying neuroblastoma, one case (5%) was lost to follow-up and four patients (20%) received a medical discharge. The median age for the 14 remaining cases was 12.6 years. Twelve patients (85.7%) were in complete response (CR) with no therapies. Two patients (14.3%) were in CR with chronic therapy. Conclusion As ES may be a symptom of AID and PID, a thorough rheumatological, immunologic and genetic workup and a careful follow-up are essential. The second-line treatment remains a dilemma. Further prospective studies are needed to address the optimal therapeutic combinations, morbidity and mortality in this disorder.