Abstract
Background: Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) characterized by platelet consumption, hemolysis, and organ damage. Eculizumab (ECU), a humanized antibody that blocks complement activity, has been successfully used in aHUS, but the best treatment schedule is not yet clear. Methods: Here, we report our experience with ECU maintenance treatment and the interval between subsequent doses being extended based on global classical complement pathway (CCP) activity aimed at <30% for maintaining aHUS into remission. Results: We report on 38 patients with aHUS, 13 children, 21 female, with a median age of 25.0 years (range 0.5–60) at disease onset treated with ECU standard schedule for a median of 2.6 months (range 0.4–24.6). Once stable TMA remission was obtained, the interval between ECU doses was extended based on complement function, with a target CCP activity of <30%. With this approach, 22 patients regularly receive ECU infusion every 28 days and 16 every 21. During a median observation period on ECU, an extended interval of 26.9 months (range 0.8–80.9), with a cumulative observation period of 1,208 months, none of the patients relapsed. Conclusion: Monitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.
| Original language | English |
|---|---|
| Pages (from-to) | 457-461 |
| Number of pages | 5 |
| Journal | Pediatric Nephrology |
| Volume | 33 |
| Issue number | 3 |
| DOIs | |
| Publication status | Published - 2018 |
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Keywords
- aHUS
- Complement activity
- Eculizumab
- Maintenance
- Remission
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Nephrology
Cite this
Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome : an update. / Ardissino, Gianluigi; Tel, Francesca; Sgarbanti, Martina; Cresseri, Donata; Giussani, Antenore; Griffini, Samantha; Grovetto, Elena; Possenti, Ilaria; Perrone, Michela; Testa, Sara; Paglialonga, Fabio; Messa, Piergiorgio; Cugno, Massimo.
In: Pediatric Nephrology, Vol. 33, No. 3, 2018, p. 457-461.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome
T2 - an update
AU - Ardissino, Gianluigi
AU - Tel, Francesca
AU - Sgarbanti, Martina
AU - Cresseri, Donata
AU - Giussani, Antenore
AU - Griffini, Samantha
AU - Grovetto, Elena
AU - Possenti, Ilaria
AU - Perrone, Michela
AU - Testa, Sara
AU - Paglialonga, Fabio
AU - Messa, Piergiorgio
AU - Cugno, Massimo
PY - 2018
Y1 - 2018
N2 - Background: Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) characterized by platelet consumption, hemolysis, and organ damage. Eculizumab (ECU), a humanized antibody that blocks complement activity, has been successfully used in aHUS, but the best treatment schedule is not yet clear. Methods: Here, we report our experience with ECU maintenance treatment and the interval between subsequent doses being extended based on global classical complement pathway (CCP) activity aimed at <30% for maintaining aHUS into remission. Results: We report on 38 patients with aHUS, 13 children, 21 female, with a median age of 25.0 years (range 0.5–60) at disease onset treated with ECU standard schedule for a median of 2.6 months (range 0.4–24.6). Once stable TMA remission was obtained, the interval between ECU doses was extended based on complement function, with a target CCP activity of <30%. With this approach, 22 patients regularly receive ECU infusion every 28 days and 16 every 21. During a median observation period on ECU, an extended interval of 26.9 months (range 0.8–80.9), with a cumulative observation period of 1,208 months, none of the patients relapsed. Conclusion: Monitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.
AB - Background: Atypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) characterized by platelet consumption, hemolysis, and organ damage. Eculizumab (ECU), a humanized antibody that blocks complement activity, has been successfully used in aHUS, but the best treatment schedule is not yet clear. Methods: Here, we report our experience with ECU maintenance treatment and the interval between subsequent doses being extended based on global classical complement pathway (CCP) activity aimed at <30% for maintaining aHUS into remission. Results: We report on 38 patients with aHUS, 13 children, 21 female, with a median age of 25.0 years (range 0.5–60) at disease onset treated with ECU standard schedule for a median of 2.6 months (range 0.4–24.6). Once stable TMA remission was obtained, the interval between ECU doses was extended based on complement function, with a target CCP activity of <30%. With this approach, 22 patients regularly receive ECU infusion every 28 days and 16 every 21. During a median observation period on ECU, an extended interval of 26.9 months (range 0.8–80.9), with a cumulative observation period of 1,208 months, none of the patients relapsed. Conclusion: Monitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.
KW - aHUS
KW - Complement activity
KW - Eculizumab
KW - Maintenance
KW - Remission
UR - http://www.scopus.com/inward/record.url?scp=85031760281&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85031760281&partnerID=8YFLogxK
U2 - 10.1007/s00467-017-3813-2
DO - 10.1007/s00467-017-3813-2
M3 - Article
AN - SCOPUS:85031760281
VL - 33
SP - 457
EP - 461
JO - Pediatric Nephrology
JF - Pediatric Nephrology
SN - 0931-041X
IS - 3
ER -