دورية أكاديمية

Identification of the Profile of the Patients with Hemophilia B Eligible for Treatment with Nonacog Alfa Once-Weekly

التفاصيل البيبلوغرافية
العنوان: Identification of the Profile of the Patients with Hemophilia B Eligible for Treatment with Nonacog Alfa Once-Weekly
المؤلفون: Dorina Cultrera, Raimondo De Cristofaro, Paola Giordano, Silvia Linari, Silvia Macchi, Renato Marino, Angelo Claudio Molinari, Angiola Rocino, Cristina Santoro, Piercarla Schinco, Sergio Siragusa, Giuseppe Tagariello, Annarita Tagliaferri, Ezio Zanon, Massimo Morfini
المصدر: Reports; Volume 3; Issue 1; Pages: 3
بيانات النشر: Multidisciplinary Digital Publishing Institute
سنة النشر: 2020
المجموعة: MDPI Open Access Publishing
مصطلحات موضوعية: hemophilia B, factor IX, nonacog alfa, once-weekly prophylaxis
الوصف: This study aimed to identify the characteristics of patients with hemophilia B eligible for once-weekly treatment with Nonacog alfa. Methods: A survey was conducted in 14 Hemophilia (HCs) of Italy. These centers were given a questionnaire consisting of ten closed multiple-choice questions. The centers were asked: (a) the percentages of their hemophilia B (HB) patients undergoing replacement therapy, “On-demand”, or weekly prophylaxis, (b) the criteria guiding the monitoring of patients, the advantages according to the age of patients, and (c) the obstacles to prophylaxis. The percentage of patients receiving “On-demand” (OD) treatment or continuous prophylaxis (prophy) differed depending on patient age and the severity of the disease. Only 57% of HCs provided “On-demand” therapy to the mild HB patients, about 93% to moderate ones, of whom 43% on prophylaxis. About 78% of patients <6 years old, were on treatment in 9 out of 14 HCs, by prophylaxis 66.7% and 33.3% by On-demand. In the 6–18 age group, 90.1% of HCs treated HB patients with prophylaxis, 42.8% in the 18–30 age range. On-demand treatment was the therapy of choice in 61.5% of HCs for patients aged 30–65 years. In total, 64% of the HCs assigned the maximum score to bleeding frequency, especially in the <6 and 6–18 age groups. Bleeding severity was also taken into significant consideration, particularly in subjects up to 30 years old. The scores regarding venous access were distributed relatively evenly throughout all age groups. The majority of the centers attributed a medium-high score to treatment compliance, especially in the 6–65 age range. In actuality, 55% of HCs attributed pro-thrombotic comorbidity a low score in the 18–30 age group, whereas 81% gave pro-hemorrhagic comorbidity a high rating in patients aged >65 years old. Many centers assigned a medium-high score to the baseline concentration of FIX level at diagnosis in all age groups. Most HCs attributed a medium-high score to type of genetic mutation in the younger age groups. As for ...
نوع الوثيقة: text
وصف الملف: application/pdf
اللغة: English
العلاقة: https://dx.doi.org/10.3390/reports3010003Test
DOI: 10.3390/reports3010003
الإتاحة: https://doi.org/10.3390/reports3010003Test
حقوق: https://creativecommons.org/licenses/by/4.0Test/
رقم الانضمام: edsbas.A0CF101A
قاعدة البيانات: BASE