Evidence map›Paper›PMID 40795230›Full record

SynthesisBlood advances2025

Efficacy of eculizumab discontinuation in atypical hemolytic uremic syndrome: a systematic review and meta-analysis.

Amy Hockman, Sydney Anuskiewicz, Emily Brennan, Saifur R Chowdhury, Alexander Coltoff, Jacqueline N Poston, Charles Greenberg, Benjamin Djulbegovic

Erratum issuedAbstract readSystematic ReviewMeta-Analysis
In one paragraph

Synthesis in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

8 authors.

Amy HockmanDivision of Hematology/Oncology, Medical University of South Carolina, Charleston, SC.ORCID 0009-0005-9841-1506
Sydney AnuskiewiczDivision of Hematology/Oncology, Medical University of South Carolina, Charleston, SC.
Emily BrennanDivision of Hematology/Oncology, Medical University of South Carolina, Charleston, SC.ORCID 0000-0001-8463-1499
Saifur R ChowdhuryDepartment of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada.ORCID 0000-0003-4361-0792
Alexander ColtoffDivision of Hematology/Oncology, Medical University of South Carolina, Charleston, SC.
Jacqueline N PostonDivision of Hematology/Oncology, Larner College of Medicine at the University of Vermont, Burlington, VT.ORCID 0000-0003-2431-5106
Charles GreenbergDivision of Hematology/Oncology, Medical University of South Carolina, Charleston, SC.
Benjamin DjulbegovicDivision of Hematology/Oncology, Medical University of South Carolina, Charleston, SC.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

abstractAtypical hemolytic uremic syndrome (aHUS), a life-threatening complement-mediated disorder, is now treatable with terminal complement inhibitors like eculizumab. Although effective, these therapies are costly, and increase susceptibility to infections, notably meningococcal disease, raising concerns about long-term use. The optimal duration of complement inhibition remains unclear, prompting efforts to explore the possibility of treatment discontinuation. We conducted a systematic review and meta-analysis to evaluate the benefits and risks of stopping terminal complement inhibitor therapy in aHUS. We searched PubMed, Scopus, and CINAHL for studies of continuing vs stopping anticomplement treatment in aHUS. Of 3303 identified studies, 13 observational studies (3 case control and 10 cohort) comprising 584 patients were included. Overall, continuing treatment was associated with an ∼76% reduction in the odds of relapse (odds ratio [OR], 0.24; 95% confidence interval [CI], 0.09-0.62; P = .01). Study design influenced results: cohort studies showed a more modest effect (OR, 0.40 [95% CI, 0.15-1.09]), whereas case-control studies reported inflated estimates (OR, 0.04; 95% CI, 0.02-0.08; subgroup interaction P = .03). When the analysis was restricted to cohort studies, the effects became uncertain (statistically nonsignificant with large CIs, indicating the possibility that outcomes with continued treatment could be either superior or inferior to those observed after treatment withdrawal, or that there may be no true difference in relapse rates between the 2 therapies). Although current evidence is insufficient to provide personalized guidance on which patients with aHUS can safely discontinue anticomplement therapy, findings from higher-quality studies, which show no statistical difference between continued and discontinued treatment, suggest that discontinuation may be possible for at least some patients.

Indexed as

Antibodies, Monoclonal, HumanizedAtypical Hemolytic Uremic SyndromeComplement Inactivating AgentsHumansTreatment OutcomeWithholding TreatmentAntibodies, Monoclonal, HumanizedComplement Inactivating Agentseculizumab

Identifiers

PMID40795230
PMCPMC12719173

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Registered trials

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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.