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Blood. 2015 Oct 8;126(15):1762-9. doi: 10.1182/blood-2015-04-637280. Epub 2015 Aug 10.

Ropeginterferon alfa-2b, a novel IFNα-2b, induces high response rates with low toxicity in patients with polycythemia vera.

Author information

1
Department of Internal Medicine I, Division of Hematology and Blood Coagulation, Medical University of Vienna, Vienna, Austria;
2
AOP Orphan Pharmaceuticals AG, Vienna, Austria;
3
Sozialmedizinisches Zentrum Ost - Donauspital, Vienna, Austria; Krankenhaus der Elisabethinen Linz, Linz, Austria;
4
Department of Internal Medicine IV, Wels-Grieskirchen Hospital, Wels, Austria;
5
Third Medical Department, Hanusch Hospital, Vienna, Austria;
6
Department of Internal Medicine V, Hematology & Oncology, Innsbruck Medical University, Innsbruck, Austria;
7
Department of Internal Medicine V, Hematology & Oncology, Innsbruck Medical University, Innsbruck, Austria; Medical Clinic 3, Oncology, Hematology and Rheumatology, University Hospital of Bonn, Bonn, Germany;
8
Department of Internal Medicine I, Division of Hematology and Blood Coagulation, Medical University of Vienna, Vienna, Austria; Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria;
9
Sozialmedizinisches Zentrum Ost - Donauspital, Vienna, Austria;
10
Laboratory for Immunological and Molecular Cancer Research, Department of Internal Medicine III with Hematology, Medical Oncology, Hemostaseology, Infectious Diseases, Rheumatology, Oncologic Center, Paracelsus Medical University, Salzburg, Austria; and.
11
Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria;
12
Advanced Drug and Device Services SAS, Brno, Czech Republic.

Abstract

In this prospective, open-label, multicenter phase 1/2 dose escalation study, we used a next-generation, mono-pegylated interferon (IFN) α-2b isoform, ropeginterferon alfa-2b. The unique feature of ropeginterferon alfa-2b is a longer elimination half-life, which allows administration every 2 weeks. We present data from 51 polycythemia vera patients. The main goal was to define the maximum tolerated dose and to assess safety and efficacy. A dose range of 50 to 540 µg was tested without the appearance of dose-limiting toxicities. All drug-related adverse events were known toxicities associated with IFN-α. The cumulative overall response rate was 90%, comprising complete response in 47% and partial response in 43% of patients; the best individual molecular response level was a complete response in 21% of patients and partial response in 47%. Notably, we did not observe any correlation between the dose level and the response rate or response duration, suggesting that already low levels of ropeginterferon alfa-2b are sufficient to induce significant hematologic and molecular responses. These data suggest promising efficacy and safety of ropeginterferon alfa-2b and support the development of the drug in a randomized phase 3 clinical trial. The study was disclosed at www.clinicaltrials.gov as #NCT01193699 before including the first patient.

PMID:
26261238
PMCID:
PMC4608390
DOI:
10.1182/blood-2015-04-637280
[Indexed for MEDLINE]
Free PMC Article

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