Evidence map›Paper›PMID 41366634›Full record

Trial reportAmerican journal of hematology2026

Iptacopan for Immune Thrombocytopenia and Cold Agglutinin Disease: A Global Phase 2 Basket Clinical Trial.

Alexander Röth, Wilma Barcellini, Christine Ademokun, Junho Jang, Maria Luisa Lozano, David Valcarcel Ferreiras, Cristina Pascual-Izquierdo, Shripad Chitnis, Sofiya Matviykiv, Alessandra Vitaliti and 4 more

Abstract readMulticenter StudyClinical Trial, Phase II
In one paragraph

Trial report in American journal of hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Trial
  2. Review
  3. Cold agglutinin disease.British journal of haematology · 2026
    Review
  4. Article
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

14 authors.

Alexander RöthDepartment of Hematology and Stem Cell Transplantation, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Essen, Germany.ORCID 0000-0003-4414-7699
Wilma BarcelliniDepartment of Hematology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Christine AdemokunHammersmith Hospital, Imperial College Healthcare NHS Trust, London, UK.
Junho JangSamsung Medical Center, Seoul, Republic of Korea.
Maria Luisa LozanoServicio de Hematología, Hospital Universitario Morales Meseguer, Instituto Murciano de Investigación Sanitaria (IMIB)-Pascual Parrilla, Universidad de Murcia, Murcia, Spain.ORCID 0000-0003-3148-7037
David Valcarcel FerreirasHematology Department, Vall Hebron Institute of Oncology (VHIO), Vall d'Hebron University Hospital, Barcelona, Spain.
Cristina Pascual-IzquierdoServicio de Hematología, Hospital General Universitario Gregorio Marañon, Instituto de Investigación Gregorio Marañon, Madrid, Spain.ORCID 0000-0001-5442-4886
Shripad ChitnisNovartis Biomedical Research, Cambridge, Massachusetts, USA.
Sofiya MatviykivNovartis Biomedical Research, Basel, Switzerland.
Alessandra VitalitiNovartis Biomedical Research, Basel, Switzerland.
Chi ChenChina Novartis Institutes for Biomedical Research Co Ltd, Shanghai, China.
Vasiliki KatsanouNovartis Biomedical Research, Basel, Switzerland.
Raghav ChawlaNovartis Biomedical Research, Basel, Switzerland.
Hanny Al-SamkariDivision of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0001-6175-1383

Funding

Novartis
6 · The paper itself

Abstract

Iptacopan is a first-in-class, oral, selective inhibitor of complement factor B that has demonstrated positive efficacy across several complement-driven diseases. Here we evaluate the efficacy and safety of iptacopan monotherapy in adult patients with primary immune thrombocytopenia (ITP) and primary cold agglutinin disease (CAD). We performed a global, multicenter, phase 2 basket study enrolling patients with primary ITP or CAD after failure of ≥ 1 unique prior therapies. Primary endpoints were platelet response (≥ 50 × 10

Indexed as

Anemia, Hemolytic, AutoimmunePurpura, Thrombocytopenic, IdiopathicAdultAgedAged, 80 and overBenzoatesFemaleHemoglobinsHumansIndolesMaleMiddle AgedPiperidinesTreatment OutcomeBenzoatesHemoglobinsIndolesiptacopanPiperidinesalternative complement pathwaycold agglutinin diseasecomplement inhibitionhemolytic anemiaimmune thrombocytopeniaIptacopan

Identifiers

PMID41366634
PMCPMC12766356

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.