Efficacy, safety and pharmacokinetics of a new 10% normal human immunoglobulin for intravenous infusion, BT595, in children and adults with primary immunodeficiency disease.
Vox Sang
; 117(10): 1153-1162, 2022 Oct.
Article
em En
| MEDLINE
| ID: mdl-35944615
ABSTRACT
BACKGROUND AND OBJECTIVES:
To evaluate the efficacy, safety and pharmacokinetics of a new, highly purified 10% IVIg (BT595, Yimmugo®) administered in children and adults with Primary immunodeficiency diseases (PID). MATERIALS ANDMETHODS:
Prospective, uncontrolled, multicentre Phase III trial. Patients aged 2 to <76 years with PID were switched from their pre-trial IVIg replacement therapy to BT595. In all, 67 patients (49 adults, 18 children) received doses between 0.2 and 0.8 g/kg body weight for approximately 12 months at intervals of 3 or 4 weeks. Dosing and dosing intervals were based on each patient's pre-trial infusion schedule. The primary end point was the rate of acute serious bacterial infections (SBIs); secondary efficacy, safety and pharmacokinetic outcomes were also evaluated.RESULTS:
The primary efficacy end point was met, and the unadjusted SBI rate was 0.01 per subject-year (adjusted SBI rate 0.015 per subject-year, with an upper limit of the one-sided 99% confidence interval of 0.151). A single adult patient experienced one event classified as an SBI. All secondary end points, including those related to infections, supported the efficacy. Infusion rates were increased up to 8 ml/kg/h. Overall, 8% of infusions were associated with ≥1 infusional adverse event (AE) (start during or within 72 h post-infusion), comprising mainly headache (2.4%), fatigue (0.9%) and nausea (0.5%). There were no infusional AEs at infusion rates of >4.0 ml/kg/h, and only one patient required a single premedication. The observed patterns, severity and frequency of treatment-emergent adverse events are consistent with the established safety profile for IVIgs and did not show clinically relevant differences between all age groups.CONCLUSION:
BT595 is effective, safe and well tolerated for treating patients with PID.Palavras-chave
Texto completo:
1
Bases de dados:
MEDLINE
Assunto principal:
Doenças da Imunodeficiência Primária
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Síndromes de Imunodeficiência
Tipo de estudo:
Clinical_trials
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Observational_studies
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Risk_factors_studies
Limite:
Adult
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Child
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Humans
Idioma:
En
Revista:
Vox Sang
Ano de publicação:
2022
Tipo de documento:
Article
País de afiliação:
Hungria