Evidence map›Paper›PMID 31890998›Full record

ArticleKidney international reports2019

Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry.

Eric Rondeau, Spero R Cataland, Imad Al-Dakkak, Benjamin Miller, Nicholas J A Webb, Daniel Landau

Open access · goldAbstract read
In one paragraph

Article in Kidney international reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
47citing papers in PubMed, 5 pooled it
3.8field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

47 citing papers in PubMed, 5 syntheses or guidelines pooled it, 75 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 5 institutions in 4 countries.

Eric RondeauUrgences Néphrologiques et Transplantation Rénale, Hôpital Tenon, and Sorbonne Université, Paris, France.
Spero R CatalandDivision of Hematology, The Ohio State University, Wexner Medical Center, Columbus, Ohio, USA.
Imad Al-DakkakGlobal Epidemiology, Alexion Pharmaceuticals, Inc., Boston, Massachusetts, USA.
Benjamin MillerGlobal Epidemiology, Alexion Pharmaceuticals, Inc., Boston, Massachusetts, USA.
Nicholas J A WebbDepartment of Paediatric Nephrology, University of Manchester, Royal Manchester Children's Hospital, Manchester, UK.
Daniel LandauSchneider Children's Medical Center, Sackler School of Medicine, Petach Tikva, Israel.
Alexion Pharmaceuticals (United States) · USRoyal Manchester Children's Hospital · GBSchneider Children's Medical Center · ILSorbonne Université · FRThe Ohio State University Wexner Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionEculizumab has transformed outcomes for patients with atypical hemolytic uremic syndrome (aHUS). Its efficacy and safety profile was well characterized in the clinical trial program. The long-term safety profile was not previously assessed or compared against nontreated patients in an observational registry setting.

methodsThe Global aHUS Registry recruits patients with clinical diagnoses of aHUS. This analysis includes baseline characteristics and targeted safety events from adult and pediatric patients who were "ever treated" versus "never treated" with eculizumab in the first 5 years of the registry, through January 26, 2017.

resultsOverall, 1321 patients (adult, n = 842; pediatric, n = 479; ever treated, n = 865; never treated, n = 456) were enrolled. A higher proportion of ever-treated versus never-treated adult and pediatric patients had renal, cardiovascular, pulmonary, central nervous system, gastrointestinal symptoms, and hepatic impairment. No differences in safety event rates between ever-treated and never-treated patients were observed, except serious infections in pediatric patients (5.15 versus 1.12 events/100 patient-years for ever- and never-treated patients, respectively). Deaths were more frequent in adult (4.7% and 9.9% of ever- and never-treated patients) compared with pediatric patients (1.8% of ever-treated patients; no deaths in never-treated patients).Three meningococcal infections were reported in ever-treated patients; 1 infection led to a fatal outcome.

conclusionIn this large observational dataset covering 5 years of registry enrollment, no new safety concerns were identified for adult or pediatric eculizumab-treated patients with aHUS, confirming a positive benefit-risk profile in a real-world setting.

Indexed as

atypical hemolytic uremic syndromecomplementsafetythrombotic microangiopathy

Identifiers

PMID31890998
PMCPMC6933459
OpenAlexW2966342106

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.