Evidence map›Paper›PMID 40837563›Full record

ArticleFrontiers in medicine2025

Case Report: Iptacopan in a paroxysmal nocturnal hemoglobinuria patient with severe renal insufficiency.

Ting Wu, Xiaoqin Wang

Abstract readCase Reports
In one paragraph

Article in Frontiers in medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

2 authors.

Ting WuDepartment of Hematology, Huashan Hospital, Fudan University, Shanghai, China.
Xiaoqin WangDepartment of Hematology, Huashan Hospital, Fudan University, Shanghai, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure. Iptacopan, an oral factor B inhibitor, has demonstrated efficacy in managing PNH but has not been studied in patients with severe renal insufficiency. We report a case of a PNH patient with end-stage renal disease who required renal replacement therapy and had a peritoneal dialysis catheter placed during treatment. After switching from eculizumab to iptacopan, the patient achieved transfusion independence, sustained hematologic improvement, and resolution of both intravascular and extravascular hemolysis. Iptacopan was well tolerated, with only mild adverse effects and no breakthrough hemolysis or infections. This case highlights the potential of iptacopan as a therapeutic option in PNH patients with severe renal impairment requiring dialysis.

Indexed as

complement inhibitionextravascular hemolysisiptacopanparoxysmal nocturnal hemoglobinuriarenal insufficiency

Identifiers

PMID40837563
PMCPMC12361199

What OpenQuestion holds

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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.