KH.M. EMIROVA^1, 2, O.M. ORLOVA^1, 2, V.K. VAKHITOV³, E.M. CHICHUGA⁴, O.V. ZAITSEVA¹, A.L. MUZUROV⁵, T.P. MAKAROVA³, P.V. AVDONIN⁶, P.P. AVDONIN⁶

 ¹Russian University of Medicine, Moscow

²St. Vladimir Children’s Clinical Hospital, Moscow

³Kazan State Medical University, Kazan

⁴Voronezh State Medical University named after N. N. Burdenko, Voronezh

⁵Russian Medical Academy for Continuing Professional Education, Moscow

⁶Koltzov Institute of Developmental Biology of the Russian Academy of Sciences, Moscow

  Contact details:

Emirova Kh.M. — PhD (Medicine), Associate Professor, Professor of the Department of Pediatrics, nephrologist

Address: 1/3 Rubtsovsko-Dvortsovaya St., Moscow, Russian Federation, 107014, tel.: +7-926-605-15-31, е-mail: kh.emirova@outlook.com

Atypical hemolytic-uremic syndrome (aHUS) is an ultra-rare disease based on the development of complement-mediated thrombotic microangiopathy, which has an unfavorable prognosis in its natural course.

The purpose — to determine predictors of unfavorable prognosis of aHUS in children.

Material and methods. 70 patients (aged 2.5 months — 16 years) were examined at the debut of aHUS and 54 in the catamnesis (1–6 years). The activity of metalloproteinase ADAMTS13 was determined in all children in the acute period by the FRET method. The peculiarities of the course and outcomes of the disease were analyzed.

Results. 62.9% of children had decreased ADAMTS13 activity. ADAMTS13 deficiency (57.2 vs 15.6%) was associated with prolonged thrombocytopenia (p = 0.03), development of anuric acute kidney injury (p = 0.0005), increased risk of anuria (OR 8.4; 95% CI 2.2–32.1) with the need for dialysis (OR 5.25; 95% CI 1.77–15.57). In the catamnesis group, 61.1% of cases had CKD, 53.7% had proteinuria, and 38.9% had arterial hypertension. The development of CKD directly correlated with CNS damage (r_pb = 0.32, p = 0.02), duration of anuria (R = 0.74, p = 0.001), thrombocytopenia (R = 0.5, p = 0.0009), duration of dialysis (R = 0.45, p = 0.008) and late start of complement-blocking therapy with eculizumab (r_pb = 0.54, p = 0.0004). CКD C2-C5 in the distant catamnesis (3–6 years) developed in children who had thrombocytopenia > 10 days at the debut of aНUS (p = 0.0005), anuria > 7 days (p = 0.03) and needed dialysis > 10 days (р = 0.02).

Conclusion. ADAMTS13 activity deficiency is associated with a more severe course of acute аHUS episode, but does not affect the long-term prognosis. The risk of developing CKD is associated with the severity of acute aHUS. To improve the prognosis of renal and overall survival of patients with aHUS, long-term monitoring of the condition and preventive measures aimed at ensuring long-term remission are necessary.

Key words: children, atypical hemolytic-uremic syndrome, ADAMTS13 metalloproteinase, prognosis.

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