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

Kód výsledku v 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>

Nalezeny alternativní kódy

RIV/00216208:11130/24:10483658

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

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).

Název v anglickém jazyce

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

Popis výsledku anglicky

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).

Druh

J_SC - Článek v periodiku v databázi SCOPUS

CEP obor

—

OECD FORD obor

30102 - Immunology

Projekt

—

Návaznosti

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2024

Kód důvěrnosti údajů

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

Název periodika

Future Rare Diseases

ISSN

—

e-ISSN

2399-5270

Svazek periodika

4

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

12

Strana od-do

2379239

Kód UT WoS článku

—

EID výsledku v databázi Scopus

2-s2.0-85201950871