Caplacizumab in pediatric immune thrombotic thrombocytopenic purpura: the UK TTP Registry experience
Pediatric thrombotic thrombocytopenic purpura (TTP) is an ultrarare disease. Immune TTP (iTTP) is driven by anti-ADAMTS13 autoantibodies causing an imbalanced von Willebrand factor (VWF):ADAMTS13 axis, and rarer still in children, but potentially life-threatening. Caplacizumab is licensed for iTTP treatment in adults and adolescents aged ≥12 years who weigh ≥40 kg. There is a need to […]
Pharmacokinetics, pharmacodynamics, efficacy, and safety of ravulizumab in pediatric paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disease of uncontrolled terminal complement activation leading to intravascular hemolysis, thrombotic events and increased morbidity and mortality. This phase 3, open-label, single-arm, multicenter study evaluated ravulizumab treatment in eculizumab-naive or -experienced pediatric patients (aged Blood Adv. 2024 Jun 11;8(11):2813-2824. doi: 10.1182/bloodadvances.2023012267. ABSTRACT Paroxysmal nocturnal hemoglobinuria (PNH) is […]
Twelve-month prevalence of haemarthrosis and joint disease using the Haemophilia Joint Health score: evaluation of the UK National Haemophilia Database and Haemtrack patient reported data: an observational study
CONCLUSION: Despite therapeutic advances, only two-thirds of children and one-third of adults were bleed-free, even in a UK cohort selected for high compliance with prophylaxis. The median HJHS of zero in children suggests joint health is relatively unaffected during childhood. In adults, bleed rates were highest in ankles and elbows, but the ankles led to […]
Thirteen-year-old female with an unusual presentation of deep venous thrombosis
We report a case of a 13-year-old female presenting with deep venous thrombosis in her left lower limb. Investigations led by her symptoms and history revealed persistently positive lupus anticoagulant and anticardiolipin antibodies, indicating the diagnosis of antiphospholipid syndrome. Double-stranded DNA antibodies were detected positive pointing to the coexistence of systemic lupus erythematosus (SLE). Antiphospholipid […]