InterLab Poznan

ChatGPT powiedział: Introduction: The population pharmacokinetic (PK) analysis of clotting factor VIII (FVIII) enables individual PK value estimation and hemophilia treatment personalization. Aim: To compare the number of bleeding episodes and standard half-life (SHL) FVIII (SHL-FVIII) consumption before and after PK-based dosing adjustment in adult patients with severe hemophilia A. Material and methods: The prospective, 18-month study had three phases: (1) before FVIII dosing adjustment, (2) dosing adjustment based on FVIII PK analysis, and (3) after FVIII dosing adjustment. During phases 1 and 3 of the study, patients frequently changed SHL-FVIII concentrates and recorded FVIII consumption and bleeding episodes in electronic diaries (myPKFiT® licensed for octocog alfa). In phase 2, PK analysis was performed following the infusion of octocog alfa. Annualized bleeding rates (ABRs) and SHL-FVIII consumption in phases 1 and 3 were compared. Results: The clearance of octocog alfa ranged from 0.014 to 0.051 dL/h/kg, half-life from 9.1 to 27.9 h, and time to reach 1 IU/dL from 51 to 179 h. Seventeen of 22 included male patients modified treatment according to the PK analysis, which resulted in a 3.1-fold decrease in total ABR (p = 0.02), a 3.3-fold decrease in spontaneous ABR (p = 0.06), and a 3.1-fold decrease in injury-related ABR (p = 0.072) compared to phase 1. The total consumption of SHL-FVIII increased by 16.8% (p = 0.052), while the consumption due to bleeds decreased by 77% (p = 0.034). Conclusions: Personalizing FVIII replacement therapy lowers bleeding risk, with a statistically non-significant increase in drug consumption in patients with severe hemophilia A. Treatment based on octocog alfa PK analysis was successfully applied to other SHL-FVIII products.

Patient adherence to a prophylactic regimen is important for optimal benefit of hemophilia treatment. Despite a growing number of adults with hemophilia in Poland receiving secondary prophylaxis, data on adherence to the regimen are limited. The aim of the study was to assess adherence to secondary prophylaxis in Polish adults with severe hemophilia. Patients were recruited in 18 hemophilia treatment centers in Poland. Adherence to prophylaxis was assessed with the Validated Hemophilia Regimen Treatment Adherence Scale Prophylaxis (VERITAS‑Pro) questionnaire. Data on 270 men on the prophylactic regimen (median [interquartile range, IQR] age, 37 [18-75] years; mean [SD], 38.2 [13.3] years) were analyzed. Median (IQR) VERITAS‑Pro score for the study population was 36 (24-76) years; mean (SD), 37.7 (9.9) years, indicating general adherence to the prophylactic regimen. The median subscale scores ranged from 4 for Dosing to 8 for Planning (means, 5.6 and 7.7, respectively). The most pronounced difference in the subscale scores between adherent and nonadherent patients was recorded for Dosing (median, 4 vs 10; mean, 5.3 vs 9.3) and Remembering (median, 5 vs 11; mean, 5.7 vs 10.7). The overall adherence rate was 94%. Our results show a high rate of adherence to hemophilia prophylaxis by Polish adults. Problems with the management of clotting factor stocks and remembering about the injection of the clotting factor were identified as potential barriers to adherence in adults with hemophilia in Poland.

Hereby we present case report of acquired von Willebrand syndrome (AVWS) in patient with indolent systemic mastocytosis (ISM). Systemic mastocytosis may be considered as a model disease for development of AVWS. Thereinafter, pathophysiology of the disease as well as diagnostic and therapeutic approach were extensively discussed.

DOI: 10.20452/pamw.15039, Published online: October 30, 2019, Authors: Anetta Undas^1,2, Krzysztof Chojnowski³, Anna Klukowska⁴, Magdalena Łętowska⁵, Andrzej Mital⁶, Wojciech Młynarski⁷, Jacek Musiał², Maria Podolak-Dawidziak⁸, Maria Sąsiadek⁹, Jacek Treliński³, Tomasz Urasiński¹⁰, Jerzy Windyga¹¹, Joanna Zdziarska¹², Krystyna Zawilska¹³