1. Inicio
  2. Publicaciones
  3. Safety and efficacy of intravenous belimumab in children with systemic lupus erythematosus: results from a randomised, placebo-controlled trial

Safety and efficacy of intravenous belimumab in children with systemic lupus erythematosus: results from a randomised, placebo-controlled trial

Fecha de publicación: Fecha Ahead of Print:

Autores de IIS La Fe

Participantes ajenos a IIS La Fe

  • Brunner, HI
  • Abud-Mendoza, C
  • Viola, DO
  • Levy, D
  • Anton, J
  • Calderon, JE
  • Chasnyk, VG
  • Ferrandiz, MA
  • Keltsev, V
  • Gastanaga, MEP
  • Shishov, M
  • Boteanu, AL
  • Henrickson, M
  • Bass, D
  • Clark, K
  • Hammer, A
  • Ji, BN
  • Nino, A
  • Roth, DA
  • Struemper, H
  • Wang, ML
  • Martini, A
  • Lovell, D
  • Ruperto, N
  • Paediat Rheumatology
  • Pediat Rheumatology Collab

Grupos

Abstract

Objectives This ongoing Phase-2, randomised, placebo-controlled, double-blind study evaluated the efficacy, safety and pharmacokinetics of intravenous belimumab in childhood-onset systemic lupus erythematosus (cSLE). Methods Patients (5 to 17 years) were randomised to belimumab 10 mg/kg intravenous or placebo every 4 weeks, plus standard SLE therapy. Primary endpoint: SLE Responder Index (SRI4) response rate (Week 52). Key major secondary endpoints: proportion of patients achieving the Paediatric Rheumatology International Trials Organisation/American College of Rheumatology (PRINTO/ACR) response using 50 and '30 alternative' definitions (Week 52), and sustained response (Weeks 44 to 52) by SRI4 and Parent Global Assessment of well-being (Parent-global). Safety and pharmacokinetics were assessed. Study not powered for statistical testing. Results Ninety-three patients were randomised (belimumab, n=53; placebo, n=40). At Week 52, there were numerically more SRI4 responders with belimumab versus placebo (52.8% vs 43.6%; OR 1.49 (95% CI 0.64 to 3.46)). PRINTO/ACR 30 alternative (52.8% vs 27.5%; OR 2.92 (95% CI 1.19 to 7.17)) and PRINTO/ACR 50 (60.4% vs 35.0%; OR 2.74 (95% CI 1.15 to 6.54)) responses were more frequent with belimumab than placebo, as were sustained responses for SRI4 (belimumab, 43.4%; placebo, 41.0%; OR 1.08 (95% CI 0.46 to 2.52)) and Parent-global (belimumab, 59.1%; placebo, 33.3%; OR 3.49 (95% CI 1.23 to 9.91)). Serious adverse events were reported in 17.0% of belimumab patients and 35.0% of placebo patients; one death occurred (placebo). Week-52, geometric mean (95% CI) belimumab trough concentration was 56.2 (45.2 to 69.8) mu g/mL. Conclusion The belimumab intravenous pharmacokinetics and benefit-risk profile in cSLE are consistent with adult belimumab studies and the 10 mg/kg every 4 weeks dose is appropriate.

Datos de la publicación

ISSN/ISSNe:
0003-4967, 1468-2060

ANNALS OF THE RHEUMATIC DISEASES  ELSEVIER

Tipo:
Article
Páginas:
1340-1348
PubMed:
32699034
Factor de Impacto:
6,333 SCImago ℠
Cuartil:
Q1 SCImago ℠

Citas Recibidas en Web of Science: 162

Documentos

  • No hay documentos

Métricas

Filiaciones mostrar / ocultar

Keywords

  • systemic lupus erythematosus; treatment; DMARDs (biologic)

Campos de Estudio

Cita

Compartir