Pregnancy-triggered atypical hemolytic uremic syndrome (aHUS) : a Global aHUS Registry analysis
© 2021. The Author(s)..
BACKGROUND: Atypical hemolytic uremic syndrome (aHUS) is a rare disease in which uncontrolled terminal complement activation leads to systemic thrombotic microangiopathy (TMA). Pregnancy can trigger aHUS and, without complement inhibition, many women with pregnancy-triggered aHUS (p-aHUS) progress to end-stage renal disease (ESRD) with a high risk of morbidity. Owing to relatively small patient numbers, published characterizations of p-aHUS have been limited, thus the Global aHUS Registry (NCT01522183, April 2012) provides a unique opportunity to analyze data from a large single cohort of women with p-aHUS.
METHODS: The demographics and clinical characteristics of women with p-aHUS (n = 51) were compared with those of women of childbearing age with aHUS and no identified trigger (non-p-aHUS, n = 397). Outcome evaluations, including renal survival according to time to ESRD, were compared for patients with and without eculizumab treatment (a complement C5 inhibitor) in both aHUS groups.
RESULTS: Baseline demographics and clinical characteristics were broadly similar in both groups. The proportion of women with p-aHUS and non-p-aHUS with pathogenic variant(s) in complement genes and/or anti-complement factor H antibodies was similar (45% and 43%, respectively), as was the proportion with a family history of aHUS (12% and 13%, respectively). Eculizumab treatment led to significantly improved renal outcomes in women with aHUS, regardless of whether aHUS was triggered by pregnancy or not: adjusted hazard ratio for time to ESRD was 0.06 (p = 0.006) in the p-aHUS group and 0.20 (p < 0.0001) in the non-p-aHUS group.
CONCLUSION: Findings from this study support the characterization of p-aHUS as a complement-mediated TMA.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2021 |
|---|---|
| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:34 |
|---|---|
| Enthalten in: |
Journal of nephrology - 34(2021), 5 vom: 07. Okt., Seite 1581-1590 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Fakhouri, Fadi [VerfasserIn] |
|---|
| Links: |
|---|
| Anmerkungen: |
Date Completed 13.10.2021 Date Revised 12.11.2021 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1007/s40620-021-01025-x |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM32381395X |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM32381395X | ||
| 003 | DE-627 | ||
| 005 | 20231225185002.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231225s2021 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1007/s40620-021-01025-x |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1079.xml |
| 035 | |a (DE-627)NLM32381395X | ||
| 035 | |a (NLM)33826112 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Fakhouri, Fadi |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Pregnancy-triggered atypical hemolytic uremic syndrome (aHUS) |b a Global aHUS Registry analysis |
| 264 | 1 | |c 2021 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 13.10.2021 | ||
| 500 | |a Date Revised 12.11.2021 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2021. The Author(s). | ||
| 520 | |a BACKGROUND: Atypical hemolytic uremic syndrome (aHUS) is a rare disease in which uncontrolled terminal complement activation leads to systemic thrombotic microangiopathy (TMA). Pregnancy can trigger aHUS and, without complement inhibition, many women with pregnancy-triggered aHUS (p-aHUS) progress to end-stage renal disease (ESRD) with a high risk of morbidity. Owing to relatively small patient numbers, published characterizations of p-aHUS have been limited, thus the Global aHUS Registry (NCT01522183, April 2012) provides a unique opportunity to analyze data from a large single cohort of women with p-aHUS | ||
| 520 | |a METHODS: The demographics and clinical characteristics of women with p-aHUS (n = 51) were compared with those of women of childbearing age with aHUS and no identified trigger (non-p-aHUS, n = 397). Outcome evaluations, including renal survival according to time to ESRD, were compared for patients with and without eculizumab treatment (a complement C5 inhibitor) in both aHUS groups | ||
| 520 | |a RESULTS: Baseline demographics and clinical characteristics were broadly similar in both groups. The proportion of women with p-aHUS and non-p-aHUS with pathogenic variant(s) in complement genes and/or anti-complement factor H antibodies was similar (45% and 43%, respectively), as was the proportion with a family history of aHUS (12% and 13%, respectively). Eculizumab treatment led to significantly improved renal outcomes in women with aHUS, regardless of whether aHUS was triggered by pregnancy or not: adjusted hazard ratio for time to ESRD was 0.06 (p = 0.006) in the p-aHUS group and 0.20 (p < 0.0001) in the non-p-aHUS group | ||
| 520 | |a CONCLUSION: Findings from this study support the characterization of p-aHUS as a complement-mediated TMA | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a Atypical hemolytic uremic syndrome (aHUS) | |
| 650 | 4 | |a Complement C5 inhibitor | |
| 650 | 4 | |a Complement-mediated TMA | |
| 650 | 4 | |a End-stage renal disease (ESRD) | |
| 650 | 4 | |a Pregnancy | |
| 650 | 7 | |a Complement Inactivating Agents |2 NLM | |
| 650 | 7 | |a Complement System Proteins |2 NLM | |
| 650 | 7 | |a 9007-36-7 |2 NLM | |
| 700 | 1 | |a Scully, Marie |e verfasserin |4 aut | |
| 700 | 1 | |a Ardissino, Gianluigi |e verfasserin |4 aut | |
| 700 | 1 | |a Al-Dakkak, Imad |e verfasserin |4 aut | |
| 700 | 1 | |a Miller, Benjamin |e verfasserin |4 aut | |
| 700 | 1 | |a Rondeau, Eric |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Journal of nephrology |d 1995 |g 34(2021), 5 vom: 07. Okt., Seite 1581-1590 |w (DE-627)NLM08977650X |x 1724-6059 |7 nnns |
| 773 | 1 | 8 | |g volume:34 |g year:2021 |g number:5 |g day:07 |g month:10 |g pages:1581-1590 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1007/s40620-021-01025-x |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 34 |j 2021 |e 5 |b 07 |c 10 |h 1581-1590 | ||