Real pandemic world results of pharmacokinetic-tailored personalized prophylaxis of bleeds in Polish children and adolescents with severe hemophilia A.

Introduction: In 2020, the new nationwide protocol of prophylaxis in Polish plasma-derived FVIII (pdFVIII) previously treated patients (PTPs) with severe hemophilia A (sHA) was introduced, resulting in the necessity of switching from pdFVIII to recombinant FVIII (octocog-alpha; rFVIII). The study aimed to: (1) assess the safety of switching from pdFVIII to rFVIII, (2) assess the safety and efficacy of pharmacokinetically based (PK-based) personalized prophylaxis in severe hemophilia A.

Patients And Methods: 151 children and adolescents receiving prophylaxis with a standard dose (40 U/kg 3 x weekly) of pdFVIII were included in this study.

[Xanthogranuloma juvenile (McDonagh disease) in a 4-month-old infant].

A rare case is presented of xanthogranuloma juvenile (McDonagh disease) in a four-month-old infant. The diagnosis was based on a characteristic appearance of skin rash, good general condition, absence of changes in laboratory investigations and typical histological pattern. In the differential diagnosis other conditions with similar skin lesions were considered (xanthoma tuberosum, eruptivum, disseminatum and planum).