Anna Klukowska

Clinical efficacy of a novel VWF-containing FVIII concentrate, Wilate®, in the prophylaxis and treatment of bleeding episodes in previously treated haemophilia A patients

The new-generation coagulation factor VIII (FVIII)/von Willebrand factor (VWF) concentrate, Wilate(®), is effective in the therapy of von Willebrand disease for which it was originally developed. The ratio of haemostatic components (FVIII to VWF) of this high-purity plasma-derived concentrate is 1:1, with VWF naturally stabilising FVIII. Bleeding episode control in patients with haemophilia A (HA) is generally managed by replacement therapy with recombinant or plasma-derived FVIII. When complexed with VWF as in the physiological situation, the product may also show a lower incidence of inhibitor development in patients with HA. The clinical efficacy, safety and tolerability of Wilate(®) were therefore evaluated in 81 previously treated individual patients with severe HA. Data from 5 good clinical practice (GCP) prospective clinical studies were pooled and assessed with regards to the prevention and treatment of bleeding and during surgical procedures. Haemostatic efficacy was excellent or good in 96.7% of 1495 rated treatments of bleeding episodes. The average dose per treatment was approximately 30 IU FVIII/kg. In surgical settings, the global haemostatic efficacy of Wilate(®) was generally rated excellent or good for use during and after operations with a mean total dose of FVIII of 848.6 IU/kg±578.5 IU/kg administered peri-operatively. Overall, tolerability of Wilate(®) was rated very good or good by patients and physicians. Rarely occurring adverse events were mild in intensity and no anti-FVIII inhibitors were detected. Wilate(®) also displayed a good viral safety profile with no seroconversions occurring in response to treatment. These data show that Wilate(®) is an efficacious treatment option in the management of patients with severe HA.

Immunogenicity, efficacy and safety of Nuwiq® (human-cl rhFVIII) in previously untreated patients with severe haemophilia A—Interim results from the NuProtect Study

Nuwiq® (Human‐cl rhFVIII) is a fourth generation recombinant FVIII, produced in a human cell line, without chemical modification or protein fusion. No inhibitors developed in studies with Nuwiq® in 201 previously treated patients with haemophilia A (HA). The immunogenicity, efficacy and safety of Nuwiq® in previously untreated patients (PUPs) with severe HA are being assessed in the ongoing NuProtect study.

Intracranial haemorrhage in a boy with severe haemophilia A and factor VIII inhibitor

BackgroundThis report describes the case of a boy with severe haemophilia A and factor VIII inhibitor who suffered subarachnoid haemorrhage at age 7 years, followed by bleeding in the right cerebellar hemisphere and an epidural haematoma at age 12 years.MethodThe patient received intensive replacement treatment leading to reversal of neurological signs and symptoms and resorption of haematomas, as demonstrated by CT scans.

Nadpłytkowość u dzieci – opis przypadku

Nadplytkowośc samoistna zaliczana jest do chorob mieloproliferacyjnych. Wystepuje u osob doroslych i bardzo rzadko u dzieci. Chorobe rozpoznaje sie na podstawie liczby plytek wyzszej od 1000 × 109/l, po wykluczeniu chorob prowadzących wtornie do nadplytkowości. Przedstawiono przypadek 12-letniej dziewczynki z nadplytkowością samoistną leczonej przez 52 miesiące anagrelidem.

Krwawienie do ośrodkowego układu nerwowego u niemowlęcia jako pierwszy objaw postępującej rodzinnej cholestazy wewnątrzwątrobowej – opis przypadku ☆ ☆☆ ◊

Przedstawiono historie choroby niemowlecia, u ktorego w 3. i 4. miesiącu zycia wystąpily masywne krwawienia do ośrodkowego ukladu nerwowego. Przyczyną krwawien byl niedobor osoczowych czynnikow krzepniecia zaleznych od witaminy K: II, VII, IX, X. Krwawienie wewnątrzczaszkowe bylo u tego dziecka pierwszym objawem niedoboru witaminy K, a jednocześnie pierwszym objawem choroby wątroby – postepującej rodzinnej cholestazy wewnątrzwątrobowej. Zastosowanie świezo mrozonego osocza, rekombinowanego aktywowanego czynnika VII i wlewow witaminy K unormowalo hemostaze. Od momentu wdrozenia substytucji witaminą K u dziecka nie powtorzyly sie powazne krwawienia.