Evidence map›Paper›PMID 39561927›Full record

Trial reportClinical immunology (Orlando, Fla.)2025

A randomised, placebo-controlled, phase III trial of leniolisib in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS): Adolescent and adult subgroup analysis.

V Koneti Rao, Anna Šedivá, Virgil A S H Dalm, Alessandro Plebani, Catharina Schuetz, Anna Shcherbina, Antonino Trizzino, Yulia Zharankova, Alanvin Orpia, Elaine Kulm and 9 more

Abstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Clinical immunology (Orlando, Fla.), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.

0numbers the graph read from it
0cells of the map it votes in
14citing 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

14 citing papers in PubMed.

  1. Trial
  2. Review
  3. Article
  4. A renaissance in targeting the PI3K/AKT/mTOR pathway.Nature reviews. Drug discovery · 2026
    Review
  5. From the gene to the clinic: Key milestones in the PI3Kδ journey.Journal of immunology (Baltimore, Md. : 1950) · 2026
    Review
  6. Navigating primary and secondary immunodeficiency intersections: how to find IEI hidden within SID.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2026
    Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Review
  12. Article
  13. Review
  14. Observational
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

19 authors.

V Koneti RaoNational Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States. Electronic address: korao@niaid.nih.gov.
Anna ŠediváDepartment of Immunology, Motol University Hospital, 2nd Faculty of Medicine, Charles University, Prague, Czech Republic.
Virgil A S H DalmDepartment of Internal Medicine, Division of Allergy and Clinical Immunology; Department of Immunology, Erasmus University Medical Center, Rotterdam, the Netherlands.
Alessandro PlebaniPediatrics Clinic, Department of Clinical and Experimental Sciences, University of Brescia, Azienda Socio Sanitaria Territoriale Spedali Civili di Brescia, Brescia, Italy.
Catharina SchuetzPediatric Immunology, Department of Pediatrics, Faculty of Medicine and University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Anna ShcherbinaDepartment of Immunology, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
Antonino TrizzinoDepartment of Pediatric Hematology and Oncology, ARNAS Ospedali Civico Di Cristina Benfratelli Hospital, Palermo, Italy.
Yulia ZharankovaBelarusian Research Center for Pediatric Oncology, Hematology and Immunology, Minsk, Belarus.
Alanvin OrpiaNational Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.
Elaine KulmClinical Research Directorate, Frederick National Laboratory for Cancer Research, Bethesda, MD, United States.
Sharon WebsterNational Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.
Julia KörholzPediatric Immunology, Department of Pediatrics, Faculty of Medicine and University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany.
Vassilios LougarisPediatrics Clinic, Department of Clinical and Experimental Sciences, University of Brescia, Azienda Socio Sanitaria Territoriale Spedali Civili di Brescia, Brescia, Italy.
Yulia RodinaDepartment of Immunology, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.
Niall ConlonWellcome Trust Clinical Research Facility, St. James's Hospital and School of Medicine, Trinity College Dublin, Ireland.
Tanya CoulterRegional Immunology Services of Northern Ireland, Belfast Health and Social Care Trust, United Kingdom.
Jason BradtPharming Healthcare Inc, Warren, NJ, United States.
Anurag RelanPharming Healthcare Inc, Warren, NJ, United States.
Gulbu UzelNational Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.

Funding

Clinical, Immunological and Genetic Analyses of ALPS and Related Immunedysregulatory DisordersZIAAI000732 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI RAO, V KONETI · 2009 to 2025
$10.5M
Intramural NIH HHS Z99 AI999999Intramural NIH HHS ZIA AI000732
6 · The paper itself

Abstract

Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) is an ultra-rare, progressive genetic disease, characterised by immune deficiency and dysregulation, affecting individuals from birth. In a 12-week phase III randomised placebo-controlled trial, leniolisib, a selective PI3Kδ inhibitor, was well-tolerated and met both co-primary endpoints (change from Baseline in log

Indexed as

Class I Phosphatidylinositol 3-KinasesPrimary Immunodeficiency DiseasesAdolescentAdultB-LymphocytesChildDouble-Blind MethodFemaleHumansMaleMiddle AgedYoung AdultClass I Phosphatidylinositol 3-KinasesPIK3CD protein, humanAdolescentsAPDSLeniolisibPASLIPediatricPI3KSubgroups

Identifiers

PMID39561927
PMCPMC11631659

What Socratic holds

Textmetadata
LicenceTDM
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.