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

J 2024

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

CIZNAR, Peter, Marion RODERICK, Helena SCHNEIDEROVÁ, Milos JESENAK, Gergely KRIVAN et. al.

Základní údaje

Originální název

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

Autoři

CIZNAR, Peter, Marion RODERICK, Helena SCHNEIDEROVÁ (203 Česká republika, domácí), Milos JESENAK, Gergely KRIVAN, Nicholas BRODSZKI, Stephen JOLLES, Charles ATISSO, Katharina FIELHAUER, Shumyla SAEED-KHAWAJA, Barbara MCCOY a Leman YEL

Vydání

ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY, London, BMC, 2024, 1710-1492

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30102 Immunology

Stát vydavatele

Velká Británie a Severní Irsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 2.700 v roce 2022

Organizační jednotka

Lékařská fakulta

DOI

http://dx.doi.org/10.1186/s13223-024-00904-9

UT WoS

001314942200001

Klíčová slova anglicky

Hyaluronidase; Immunoglobulins; Inborn errors of immunity (IEI); Primary immunodeficiency diseases; Patient safety; Pediatrics; Subcutaneous

Štítky

14110317, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 25. 10. 2024 09:44, Mgr. Tereza Miškechová

Anotace

V originále

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 < 18 years with a documented PID diagnosis who had received immunoglobulin therapy for >= 3 months before enrollment. New fSCIG 10% starters underwent fSCIG 10% dose ramp-up for <= 6 weeks (epoch 1) before receiving fSCIG 10% for <= 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 >= 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.

Citovat

CIZNAR, Peter, Marion RODERICK, Helena SCHNEIDEROVÁ, Milos JESENAK, Gergely KRIVAN, Nicholas BRODSZKI, Stephen JOLLES, Charles ATISSO, Katharina FIELHAUER, Shumyla SAEED-KHAWAJA, Barbara MCCOY a Leman YEL. fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study. ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY. London: BMC, 2024, roč. 20, č. 1, s. 1-9. ISSN 1710-1492. Dostupné z: https://dx.doi.org/10.1186/s13223-024-00904-9.

@article{2446199,
   author = {Ciznar, Peter and Roderick, Marion and Schneiderová, Helena and Jesenak, Milos and Krivan, Gergely and Brodszki, Nicholas and Jolles, Stephen and Atisso, Charles and Fielhauer, Katharina and SaeedandKhawaja, Shumyla and McCoy, Barbara and Yel, Leman},
   article_location = {London},
   article_number = {1},
   doi = {http://dx.doi.org/10.1186/s13223-024-00904-9},
   keywords = {Hyaluronidase; Immunoglobulins; Inborn errors of immunity (IEI); Primary immunodeficiency diseases; Patient safety; Pediatrics; Subcutaneous},
   language = {eng},
   issn = {1710-1492},
   journal = {ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY},
   title = {fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study},
   url = {https://aacijournal.biomedcentral.com/articles/10.1186/s13223-024-00904-9},
   volume = {20},
   year = {2024}
}

TY  - JOUR
ID  - 2446199
AU  - Ciznar, Peter - Roderick, Marion - Schneiderová, Helena - Jesenak, Milos - Krivan, Gergely - Brodszki, Nicholas - Jolles, Stephen - Atisso, Charles - Fielhauer, Katharina - Saeed-Khawaja, Shumyla - McCoy, Barbara - Yel, Leman
PY  - 2024
TI  - fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study
JF  - ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY
VL  - 20
IS  - 1
SP  - 1-9
EP  - 1-9
PB  - BMC
SN  - 17101492
KW  - Hyaluronidase
KW  - Immunoglobulins
KW  - Inborn errors of immunity (IEI)
KW  - Primary immunodeficiency diseases
KW  - Patient safety
KW  - Pediatrics
KW  - Subcutaneous
UR  - https://aacijournal.biomedcentral.com/articles/10.1186/s13223-024-00904-9
N2  - 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 < 18 years with a documented PID diagnosis who had received immunoglobulin therapy for >= 3 months before enrollment. New fSCIG 10% starters underwent fSCIG 10% dose ramp-up for <= 6 weeks (epoch 1) before receiving fSCIG 10% for <= 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 >= 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.
ER  -

CIZNAR, Peter, Marion RODERICK, Helena SCHNEIDEROVÁ, Milos JESENAK, Gergely KRIVAN, Nicholas BRODSZKI, Stephen JOLLES, Charles ATISSO, Katharina FIELHAUER, Shumyla SAEED-KHAWAJA, Barbara MCCOY a Leman YEL. fSCIG 10\%{} in pediatric primary immunodeficiency diseases: a European post-authorization safety study. \textit{ALLERGY ASTHMA AND CLINICAL IMMUNOLOGY}. London: BMC, 2024, roč.~20, č.~1, s.~1-9. ISSN~1710-1492. Dostupné z: https://dx.doi.org/10.1186/s13223-024-00904-9.

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