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

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00064203%3A_____%2F24%3A10483658" target="_blank" >RIV/00064203:_____/24:10483658 - isvavai.cz</a>

Alternative codes found

RIV/00216208:11130/24:10483658

Result on the web

<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=m_sSP6GOj9" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=m_sSP6GOj9</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1080/23995270.2024.2379239" target="_blank" >10.1080/23995270.2024.2379239</a>

Result language

angličtina

Original language name

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

Original language description

What is this summary about?: 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. What were the results?: 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&apos; enlarged organs, and lowered the activity of the protein that causes APDS. There were no major safety concerns for participants who took leniolisib. What do the results mean?: 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. Clinical Trial Registration:NCT02435173 (ClinicalTrials.gov).

Czech name

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Czech description

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Type

J_SC - Article in a specialist periodical, which is included in the SCOPUS database

CEP classification

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OECD FORD branch

30102 - Immunology

Project

—

Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2024

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Future Rare Diseases

ISSN

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e-ISSN

2399-5270

Volume of the periodical

4

Issue of the periodical within the volume

1

Country of publishing house

GB - UNITED KINGDOM

Number of pages

12

Pages from-to

2379239

UT code for WoS article

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EID of the result in the Scopus database

2-s2.0-85201950871