Ropeginterferon Alfa-2b versus Standard Therapy for Polycythaemia Vera; a Randomised, Controlled, Phase III Trial
| العنوان: | Ropeginterferon Alfa-2b versus Standard Therapy for Polycythaemia Vera; a Randomised, Controlled, Phase III Trial |
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| المؤلفون: | Heinz Gisslinger, Christoph Klade, Pencho Georgiev, Dorota Krochmalczyk, Liana Gercheva-Kyuchukova, Miklos Egyed, Viktor Rossiev, Petr Dulicek, Arpad Illes, Halyna Pylypenko, Lylia Sivcheva, Jiri Mayer, Vera Yablokova, Kurt Krejcy, Barbara Grohmann-Izay, Hans C. Hasselbalch, Robert Kralovics, Jean-Jacques Kiladjian, PROUD-PV Study Group |
| المصدر: | SSRN Electronic Journal. |
| بيانات النشر: | Elsevier BV, 2019. |
| سنة النشر: | 2019 |
| مصطلحات موضوعية: | Polycythaemia, medicine.medical_specialty, education.field_of_study, business.industry, Population, Institutional review board, medicine.disease, Tolerability, Internal medicine, Good clinical practice, Clinical endpoint, medicine, education, business, Adverse effect, Disease burden |
| الوصف: | Background: Ropeginterferon alfa-2b (ropeg) is a novel, mono-pegylated interferon (IFN) for treatment of polycythemia vera (PV). The PROUD-PV/CONTINUATION-PV trial compared ropeg with hydroxyurea (HU), the standard therapy for PV, over three years of treatment. Methods: This phase III, randomised, controlled, open-label trial was conducted in 48 clinics in Europe in patients aged =18 years, with early-stage PV (cytoreduction-naive or less than three years of prior HU treatment), diagnosed by WHO 2008 criteria. In the PROUD-PV part, patients were randomised 1:1 with block-stratification by prior HU treatment, age (=60 or >60 years) and history of thromboembolic events to receive ropeg (subcutaneously every two weeks, starting at 100 µg) or HU (orally starting at 500 mg/day). After one year, patients rolled over to the CONTINUATION-PV part. The primary endpoint was disease response rate at 3 monthly assessment visits from 12 months onwards. Composite evaluation criteria were applied: complete haematological response (CHR) with normalisation of spleen size (PROUD-PV/CONTINUATION-PV), and CHR with improved disease burden (signs and symptoms) (CONTINUATION-PV only). The trial was registered on EudraCT (2012-005259-18; 2014-001357-17). Findings: Recruitment took place from October 2013 to March 2015; 257 patients were randomised, three withdrew consent and 127 per arm were treated. Overall, 171 patients rolled over to the ongoing CONTINUATION-PV part. All patients treated were analysed for safety; the intent-to-treat population was analysed for efficacy. After three years of treatment, response rates were significantly higher in the ropeg arm compared to control for CHR (67/95 [70·5%] versus 38/74 [51·4%]; p=0·01) and CHR with improved disease burden (50/95 [52·6%] versus 28/74 [37·8%]; p=0·04). Only 27/254 patients had splenomegaly at baseline precluding meaningful assessment of spleen size normalisation. JAK2V617F allele burden at three years showed a sustained decline in the ropeg arm only (molecular response in ropeg arm: 62/94 [66·0%]; control: 20/74 [27·0%]; p |
| تدمد: | 1556-5068 2012-0052 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_________::37569d2e3dc4a85c590828407b37f479 https://doi.org/10.2139/ssrn.3426089 |
| رقم الأكسشن: | edsair.doi...........37569d2e3dc4a85c590828407b37f479 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 15565068 20120052 |
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