Pharmacokinetics, efficacy and safety of a novel fibrinogen concentrate in pediatric patients with congenital afibrinogenemia.

Introduction: Congenital afibrinogenemia treatment with plasma-derived fibrinogen concentrates in pediatric patients is limited. This study investigated the pharmacokinetics, surrogate efficacy, and safety of a plasma-derived fibrinogen concentrate (FIB Grifols) in pediatric patients with congenital afibrinogenemia.

Methods: Patients aged <18 years old diagnosed with congenital afibrinogenemia were included in this prospective, multinational, phase 1-2, single-arm study.

Pharmacokinetics, surrogate efficacy and safety evaluations of a new human plasma-derived fibrinogen concentrate (FIB Grifols) in adult patients with congenital afibrinogenemia.

Background And Aims: Congenital afibrinogenemia is a rare coagulation disorder resulting from a deficiency in fibrinogen. This study assessed the pharmacokinetics, surrogate efficacy and safety of FIB Grifols, a new human plasma-derived fibrinogen concentrate, to treat congenital afibrinogenemia.

Methods: Eleven adult patients from a multinational, phase 1-2, prospective, open-label, single-arm, uncontrolled clinical study received a single infusion of FIB Grifols, 70 mg/kg bw.

Safety and efficacy of intravenous immunoglobulin (Flebogamma 10% DIF) in patients with immune thrombocytopenic purpura.

Aim: To evaluate the safety and efficacy of 10% intravenous immunoglobulin (IVIG; Flebogamma 10% DIF) in individuals with chronic immune thrombocytopenic purpura (ITP).

Patients & Methods: Patients aged 3-70 years, diagnosed with chronic ITP, received 1 g/kg IVIG over two consecutive days.

Results: 64 evaluable patients (51 adults, 13 children) with chronic ITP received IVIG.

Reconditioning After Exertional Rhabdomyolysis.

In brief Prompt recognition and treatment of rhabdomyolysis may prevent further muscle damage and serious renal complications. The patient presented here, an unconditioned rugby player with moderately severe exertional rhabdomyolysis, had thigh pain, thigh and knee swelling, and myoglobinuria. His rehabilitation focused on a well-monitored, progressive exercise program after his muscle injury resolved.