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Immune tolerance induction in haemophilia A patients with inhibitors by treatment with recombinant factor VIII: a retrospective non-interventional study.
Rivard, G E; Rothschild, C; Toll, T; Achilles, K.
Afiliação
  • Rivard GE; CHU Sainte-Justine, Montreal, QC, Canada.
Haemophilia ; 19(3): 449-55, 2013 May.
Article em En | MEDLINE | ID: mdl-23510123
Immune tolerance induction (ITI) can overcome inhibitory factor VIII (FVIII) antibodies in haemophilia A patients receiving FVIII replacement therapy. The objective was to evaluate the use of sucrose-formulated, full-length recombinant FVIII (rFVIII-FS) for ITI therapy. Patients (<8 years at ITI start) with severe haemophilia A and a peak inhibitor titre >5 Bethesda units (BU) who underwent ITI with any rFVIII-FS dose for ≥ 9 months (or until success) were eligible for this retrospective study. Efficacy analyses included descriptions of ITI treatment regimens and outcomes; ITI success was determined solely at the discretion of the investigator. Safety analyses included assessment of adverse events. Of 51 enrolled patients, 32 [high dose (≥ 85 IU kg(-1) day(-1)), n = 21; low dose, n = 11] were eligible for analysis. ITI was successful in 69% (22/32) of patients (high dose, 66.7%; low dose, 72.7%) after a median of 1.4 years (range, 0.1-3.6 years). Influencing factors for ITI success were start of ITI <1 year after inhibitor detection and an inhibitor titre <10 BU at ITI start. All patients successfully tolerized with ITI continued to receive rFVIII-FS prophylaxis as maintenance therapy, with no inhibitor recurrence from the end of ITI until study enrolment. Use of rFVIII-FS for ITI was effective and well tolerated; success rates were similar to those in published studies.
Assuntos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Sacarose / Fator VIII / Inibidores dos Fatores de Coagulação Sanguínea / Hemofilia A / Tolerância Imunológica Tipo de estudo: Observational_studies / Prognostic_studies Limite: Child / Child, preschool / Humans / Infant / Male Idioma: En Ano de publicação: 2013 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Sacarose / Fator VIII / Inibidores dos Fatores de Coagulação Sanguínea / Hemofilia A / Tolerância Imunológica Tipo de estudo: Observational_studies / Prognostic_studies Limite: Child / Child, preschool / Humans / Infant / Male Idioma: En Ano de publicação: 2013 Tipo de documento: Article