Leniolisib treatment for people with activated PI3K delta syndrome (APDS): a plain language summary of the phase 3 study

This is a plain language summary of an article originally published in Blood. Leniolisib is a drug developed to treat activated PI3K delta syndrome (APDS). APDS is a rare disease in which the immune system does not work correctly. People with APDS have a wide range of symptoms including infections, certain organs associated with the immune system becoming larger, and worse quality of life. This summary reports the results from a clinical study that aimed to understand if leniolisib is effective at treating people with APDS. The study showed that leniolisib improved the amounts of immune cells and antibodies (proteins that help the immune system to function). Leniolisib also reduced the size of participants' enlarged organs, and lowered the activity of the protein that causes APDS. There were no major safety concerns for participants who took leniolisib. These results indicate that leniolisib helps the immune system to work in a way that is closer to those without APDS. By helping the immune system, leniolisib may be able to improve the lives of people with APDS by making them feel better. Leniolisib may also prevent difficulties in completing activities in the daily lives of people with APDS. <b>Clinical Trial Registration:</b>NCT02435173 (ClinicalTrials.gov)

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Publication Details

Journal
Future Rare Diseases
Published
2024-08-21
DOI
https://doi.org/10.1080/23995270.2024.2379239
Primary Topic
Chronic Lymphocytic Leukemia Research
Type
article
Field-Weighted Citation Impact
0.00

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article

Leniolisib treatment for people with activated PI3K delta syndrome (APDS): a plain language summary of the phase 3 study

Julia Körholz, Elaine Kulm, Alessandro Plebani, Anna Šedivá et al.
Future Rare Diseases
Chronic Lymphocytic Leukemia Research
article

Leniolisib treatment for people with activated PI3K delta syndrome (APDS): a plain language summary of the phase 3 study

Julia Körholz, Elaine Kulm, Alessandro Plebani, Anna Šedivá, Virgil A. S. H. Dalm, Jason Bradt, Vassilios Lougaris, Niall Conlon, Sharon Webster, Jo Luscombe, Catharina Schuetz
article en

Abstract

This is a plain language summary of an article originally published in Blood. Leniolisib is a drug developed to treat activated PI3K delta syndrome (APDS). APDS is a rare disease in which the immune system does not work correctly. People with APDS have a wide range of symptoms including infections, certain organs associated with the immune system becoming larger, and worse quality of life. This summary reports the results from a clinical study that aimed to understand if leniolisib is effective at treating people with APDS. The study showed that leniolisib improved the amounts of immune cells and antibodies (proteins that help the immune system to function). Leniolisib also reduced the size of participants' enlarged organs, and lowered the activity of the protein that causes APDS. There were no major safety concerns for participants who took leniolisib. These results indicate that leniolisib helps the immune system to work in a way that is closer to those without APDS. By helping the immune system, leniolisib may be able to improve the lives of people with APDS by making them feel better. Leniolisib may also prevent difficulties in completing activities in the daily lives of people with APDS. <b>Clinical Trial Registration:</b>NCT02435173 (ClinicalTrials.gov)

Future Rare DiseasesVol. 4(1)
National Institutes of Health (US), Trinity College Dublin (IE), Charles University (CZ), Erasmus MC (NL), Brescia University (US), Directorate of Health (IS), Scientific Research Institute of Experimental and Clinical Medicine (RU), University Hospital in Motol (CZ), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Pharming Group (Netherlands) (NL), Institute of Clinical and Experimental Medicine (CZ), Frederick National Laboratory for Cancer Research (US), National Institute of Allergy and Infectious Diseases (US), NIHR Wellcome Trust Southampton Clinical Research Facility (GB), St. James's Hospital (IE), University Hospital Carl Gustav Carus (DE), Technische Universität Dresden (DE), University of Brescia (IT)
National Institutes of Health, National Cancer Institute
Quality Education
Openalex Percentile: Top 78%
Chronic Lymphocytic Leukemia Research
DOIOpen Access PDFOpenAlex
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