Adolescence-onset atypical hemolytic uremic syndrome: is it different from infant-onset?
Küçük Resim Yok
Tarih
2024
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Springer
Erişim Hakkı
info:eu-repo/semantics/closedAccess
Özet
Background: 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. Methods: A total of 28 patients (21 girls, 7 boys) who were diagnosed as aHUS between the ages of >= 10 years and <18 years were included in this study. All available data in the Turkish Pediatric aHUS registry were collected and analyzed. Results: The mean age at diagnosis was 12.8 +/- 2.3 years. 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 +/- 2.6 years. 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). Conclusions: 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.
Açıklama
Anahtar Kelimeler
Atypical hemolytic uremic syndrome, Adolescence, Genetics, Complement, Turkish aHUS registry
Kaynak
Clinical and Experimental Nephrology
WoS Q Değeri
N/A
Scopus Q Değeri
Q2
