Adolescence-onset atypical hemolytic uremic syndrome: is it different from infant-onset?

Abstract

Atypical hemolytic uremic syndrome (aHUS) is a rare, mostly complement-mediated thrombotic microangiopathy. The majority of patients are infants. In contrast to infantile-onset aHUS, the clinical and genetic characteristics of adolescence-onset aHUS have not been sufficiently addressed to date. A total of 28 patients (21 girls, 7 boys) who were diagnosed as aHUS between the ages of ≥10years and <18years were included in this study. All available data in the Turkish Pediatric aHUS registry were collected and analyzed. The mean age at diagnosis was 12.8±2.3years. Extra-renal involvement was noted in 13 patients (46.4%); neurological involvement was the most common (32%). A total of 21 patients (75%) required kidney replacement therapy. Five patients (17.8%) received only plasma therapy and 23 (82%) of the patients received eculizumab. Hematologic remission and renal remission were achieved in 25 (89.3%) and 17 (60.7%) of the patients, respectively. Compared with the infantile-onset aHUS patients, adolescent patients had a lower complete remission rate during the first episode (p=0.002). Genetic analyses were performed in all and a genetic variant was detected in 39.3% of the patients. The mean follow-up duration was 4.9±2.6years. At the last visit, adolescent patients had lower eGFR levels (p=0.03) and higher rates of chronic kidney disease stage 5 when compared to infantile-onset aHUS patients (p=0.04). Adolescence-onset aHUS is a rare disease but tends to cause more permanent renal dysfunction than infantile-onset aHUS. These results may modify the management approaches in these patients.