fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216224%3A14110%2F24%3A00137419" target="_blank" >RIV/00216224:14110/24:00137419 - isvavai.cz</a>

Výsledek na webu

<a href="https://aacijournal.biomedcentral.com/articles/10.1186/s13223-024-00904-9" target="_blank" >https://aacijournal.biomedcentral.com/articles/10.1186/s13223-024-00904-9</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1186/s13223-024-00904-9" target="_blank" >10.1186/s13223-024-00904-9</a>

Jazyk výsledku

angličtina

Název v původním jazyce

fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study

Popis výsledku v původním jazyce

Background The safety, tolerability, and immunogenicity of hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) 10% (dual-vial unit of human immunoglobulin 10% and recombinant human hyaluronidase [rHuPH20]) were assessed in children with primary immunodeficiency diseases (PIDs). Methods This phase 4, post-authorization, prospective, interventional, multicenter study (NCT03116347) conducted in the European Economic Area, enrolled patients aged 2 to &lt; 18 years with a documented PID diagnosis who had received immunoglobulin therapy for &gt;= 3 months before enrollment. New fSCIG 10% starters underwent fSCIG 10% dose ramp-up for &lt;= 6 weeks (epoch 1) before receiving fSCIG 10% for &lt;= 3 years (epoch 2); patients pretreated with fSCIG 10% entered epoch 2 directly. The primary outcome was the number and rate (per infusion) of all noninfectious treatment-related serious and severe adverse events (AEs). Results In total, 42 patients were enrolled and dosed (median [range] age: 11.5 [3-17] years; 81% male; 23 new starters; 19 pretreated). Overall, 49 related noninfectious, treatment-emergent AEs (TEAEs) were reported in 15 patients; most were mild in severity (87.8%). No treatment-related serious TEAEs were reported. Two TEAEs (infusion site pain and emotional distress) were reported as severe and treatment-related in a single new fSCIG 10% starter. The rate of local TEAEs was lower in pretreated patients (0.1 event/patient-year) versus new starters (1.3 events/patient-year). No patients tested positive for binding anti-rHuPH20 antibodies (titer of &gt;= 1:160). Conclusions No safety signals were identified, and the incidence of local AEs declined over the duration of fSCIG 10% treatment. This study supports fSCIG 10% long-term safety in children with PIDs.

Název v anglickém jazyce

fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study

Popis výsledku anglicky

Background The safety, tolerability, and immunogenicity of hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) 10% (dual-vial unit of human immunoglobulin 10% and recombinant human hyaluronidase [rHuPH20]) were assessed in children with primary immunodeficiency diseases (PIDs). Methods This phase 4, post-authorization, prospective, interventional, multicenter study (NCT03116347) conducted in the European Economic Area, enrolled patients aged 2 to &lt; 18 years with a documented PID diagnosis who had received immunoglobulin therapy for &gt;= 3 months before enrollment. New fSCIG 10% starters underwent fSCIG 10% dose ramp-up for &lt;= 6 weeks (epoch 1) before receiving fSCIG 10% for &lt;= 3 years (epoch 2); patients pretreated with fSCIG 10% entered epoch 2 directly. The primary outcome was the number and rate (per infusion) of all noninfectious treatment-related serious and severe adverse events (AEs). Results In total, 42 patients were enrolled and dosed (median [range] age: 11.5 [3-17] years; 81% male; 23 new starters; 19 pretreated). Overall, 49 related noninfectious, treatment-emergent AEs (TEAEs) were reported in 15 patients; most were mild in severity (87.8%). No treatment-related serious TEAEs were reported. Two TEAEs (infusion site pain and emotional distress) were reported as severe and treatment-related in a single new fSCIG 10% starter. The rate of local TEAEs was lower in pretreated patients (0.1 event/patient-year) versus new starters (1.3 events/patient-year). No patients tested positive for binding anti-rHuPH20 antibodies (titer of &gt;= 1:160). Conclusions No safety signals were identified, and the incidence of local AEs declined over the duration of fSCIG 10% treatment. This study supports fSCIG 10% long-term safety in children with PIDs.

Druh

J_imp - Článek v periodiku v databázi Web of Science

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

ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY

ISSN

1710-1492

e-ISSN

1710-1492

Svazek periodika

20

Číslo periodika v rámci svazku

1

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

9

Strana od-do

1-9

Kód UT WoS článku

001314942200001

EID výsledku v databázi Scopus

2-s2.0-85204308515