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FINAL RESULTS FROM PEN-PV STUDY, A SINGLE-ARM PHASE 3 TRIAL ASSESSING THE EASE OF SELF-ADMINISTRATING ROPEGINTERFERON ALFA-2B USING A PRE-FILLED PEN IN POLYCYTHEMIA VERA PATIENTS
Author(s):
Heinz Gisslinger
,
Heinz Gisslinger
Affiliations:
Hematology and Hemostaseology,Medical University Vienna,Vienna,Austria
Barbara Grohmann-Izay
,
Barbara Grohmann-Izay
Affiliations:
AOP Orphan Pharmaceuticals AG,Vienna,Austria
Pencho Georgiev
,
Pencho Georgiev
Affiliations:
Clinic of Hematology,University Multiprofile Hospital for Active Treatment 'Sveti Georgi',Plovdiv,Bulgaria
Aleksander Skotnicki
,
Aleksander Skotnicki
Affiliations:
Teaching Unit of the Hematology Department,University Hospital in Krakow,Krakow,Poland
Liana Gercheva-Kyuchukova
,
Liana Gercheva-Kyuchukova
Affiliations:
Clinical Hematology Clinic,Multiprofile Hospital for Active Treatment 'Sveta Marina',Varna,Bulgaria
Miklos Egyed
,
Miklos Egyed
Affiliations:
Department of Internal Medicine II,Kaposi Mor County Teaching Hospital,Kaposvar,Hungary
Viktor Rossiev
,
Viktor Rossiev
Affiliations:
Samara Kalinin Regional Clinical Hospital,Samara,Russian Federation
Petr Dulicek
,
Petr Dulicek
Affiliations:
Department of Clinical Hematology,University Hospital Hradec Kralove,Hradec Kralove,Czech Republic
Arpad Illes
,
Arpad Illes
Affiliations:
Medical and Health Science Center,University of Debrecen,Debrecen,Hungary
Halyna Pylypenko
,
Halyna Pylypenko
Affiliations:
Department of Hematology,Cherkasy Regional Oncology Center, Regional Treatment and Diagnostics Hematology Center,Cherkasy,Ukraine
Lylia Sivcheva
,
Lylia Sivcheva
Affiliations:
First Department of Internal Medicine,Multiprofile Hospital for Active Treatment - Hristo Botev,Vratsa,Bulgaria
Jiri Mayer
,
Jiri Mayer
Affiliations:
Clinic of Internal Medicine - Hematology and Oncology,University Hospital Brno,Brno,Czech Republic
Hans Hasselbalch
,
Hans Hasselbalch
Affiliations:
Department of Hematology,Roskilde Hospital, University of Copenhagen,Copenhagen,Denmark
Christoph Klade
,
Christoph Klade
Affiliations:
AOP Orphan Pharmaceuticals AG,Vienna,Austria
Jean-Jacques Kiladjian
Jean-Jacques Kiladjian
Affiliations:
Centre d'Investigations Cliniques,Hôpital Saint-Louis and Université Paris Diderot,Paris,France
(Abstract release date: 05/18/17) EHA Library. Gisslinger H. 05/18/17; 182767; PB2053
Prof. Dr. Heinz Gisslinger
Prof. Dr. Heinz Gisslinger
Contributions
PDF Abstract
Abstract

Abstract: PB2053

Type: Publication Only

Background
Interferon-alpha (IFNa) based therapies have been successfully used in myeloproliferative neoplasms for over thirty years. A known burden for long-term therapy applying IFNa in otherwise fit outpatients is the necessity of frequent hospital visits for product administration. Ropeginterferon alfa-2b (AOP2014) is a novel long-acting monopegylated IFNa allowing initially bi-weekly and, in long-term maintenance, monthly administration. To further improve on convenience and compliance, a pre-filled, dose-adjustable pen was developed for patient self-administration at home.

Aims
Open-label, single arm, multicenter phase III trial assessing the self-administration of AOP2014 using a pre-filled, dose-adjustable pen (NCT: 2014-001356-31).

Methods
The study was performed in 18 sites in 8 European countries. Patients were eligible who completed the AOP2014-arm in the PROUD-PV study (12 months of treatment). A total of 7 visits was scheduled within 3 months (two supervised self-administrations at site, followed by four self-administrations in the home-setting, and a final assessment visit at study site).

Results
A total of 36 patients were enrolled and received the AOP2014 pen for self-administration. The mean age was 58.5 years (range 37 to 77 years), 23/36 (63.9%) were male patients and a large proportion of patients (15/36 [41.7%]) entering the study received a baseline dose of 500 µg AOP2014. 72.2% (26/36) used 2 pens (dose > 250 µg) and 27.8% of patients (10/36) used one pen (dose up to 250 µg) to administer the appropriate dose.

At the first supervised visit, 80.6 % (29/36) of patients had achieved full success, defined as no technical problems with the pen experienced by the patient during the injection, and no early withdrawal of the pen (before injection was complete), both observed by the investigator. At the second supervised visit the full success rate was 91.7% (33/36). The majority of observations resolved after the second supervised visit. Only 5 patients (13.9%) needed one additional supervised visit prior using the pen correctly in a home-setting. All patients had achieved full success at Visit P7 (supervised assessment visit at study end).
The patients responded favourably to the use of the pre-filled pen for the administration of AOP2014 and the accompanying instructions. Based on the Investigator’s assessment, no patients exhibited any visible pain or physical discomfort, appeared to be dissatisfied when using the pen or exhibited any frustration using the pen, nor did the patient report any pain arising from the use of the pen. The majority of patients (32/36 patients) rated the instructions for the AOP2014 pen (i.e. scope and structure of the leaflet, clarity and comprehensibility of the text, clarity of the images and design of the leaflet), and the AOP2014 pen itself (i.e. setting the dose, user-friendliness, injection procedure) as “very good” or “good”.
The haematological parameters and spleen size remained stable throughout the study, and the rate of responders (haematological response with and without spleen size) was maintained during the entire study, suggesting that the use of the pen device did not affect drug activity. Of the 47 adverse events (AE) reported during the study, 19 were related. Most AEs were mild to moderate in intensity. One serious AE (mild atrial fibrillation, unrelated), one pen-related AE (mild nervousness reported prior first administration in the home setting), and one Grade 3 TEAE (pain in extremity, related) were recorded, but none led to a dose reduction.

Conclusion
The AOP2014 pen was well accepted and no major difficulties were reported. The study drug performed as expected and there were no safety concerns arising from the administration of AOP2014 using the pen device. The AOP2014 pen allows for individual dosing and a patient-convenient mode of self-administration of ropeginterferon alfa-2b at home and is expected to support adherence and compliance in the long-term treatment of PV patients.

Session topic: 16. Myeloproliferative neoplasms - Clinical

Abstract: PB2053

Type: Publication Only

Background
Interferon-alpha (IFNa) based therapies have been successfully used in myeloproliferative neoplasms for over thirty years. A known burden for long-term therapy applying IFNa in otherwise fit outpatients is the necessity of frequent hospital visits for product administration. Ropeginterferon alfa-2b (AOP2014) is a novel long-acting monopegylated IFNa allowing initially bi-weekly and, in long-term maintenance, monthly administration. To further improve on convenience and compliance, a pre-filled, dose-adjustable pen was developed for patient self-administration at home.

Aims
Open-label, single arm, multicenter phase III trial assessing the self-administration of AOP2014 using a pre-filled, dose-adjustable pen (NCT: 2014-001356-31).

Methods
The study was performed in 18 sites in 8 European countries. Patients were eligible who completed the AOP2014-arm in the PROUD-PV study (12 months of treatment). A total of 7 visits was scheduled within 3 months (two supervised self-administrations at site, followed by four self-administrations in the home-setting, and a final assessment visit at study site).

Results
A total of 36 patients were enrolled and received the AOP2014 pen for self-administration. The mean age was 58.5 years (range 37 to 77 years), 23/36 (63.9%) were male patients and a large proportion of patients (15/36 [41.7%]) entering the study received a baseline dose of 500 µg AOP2014. 72.2% (26/36) used 2 pens (dose > 250 µg) and 27.8% of patients (10/36) used one pen (dose up to 250 µg) to administer the appropriate dose.

At the first supervised visit, 80.6 % (29/36) of patients had achieved full success, defined as no technical problems with the pen experienced by the patient during the injection, and no early withdrawal of the pen (before injection was complete), both observed by the investigator. At the second supervised visit the full success rate was 91.7% (33/36). The majority of observations resolved after the second supervised visit. Only 5 patients (13.9%) needed one additional supervised visit prior using the pen correctly in a home-setting. All patients had achieved full success at Visit P7 (supervised assessment visit at study end).
The patients responded favourably to the use of the pre-filled pen for the administration of AOP2014 and the accompanying instructions. Based on the Investigator’s assessment, no patients exhibited any visible pain or physical discomfort, appeared to be dissatisfied when using the pen or exhibited any frustration using the pen, nor did the patient report any pain arising from the use of the pen. The majority of patients (32/36 patients) rated the instructions for the AOP2014 pen (i.e. scope and structure of the leaflet, clarity and comprehensibility of the text, clarity of the images and design of the leaflet), and the AOP2014 pen itself (i.e. setting the dose, user-friendliness, injection procedure) as “very good” or “good”.
The haematological parameters and spleen size remained stable throughout the study, and the rate of responders (haematological response with and without spleen size) was maintained during the entire study, suggesting that the use of the pen device did not affect drug activity. Of the 47 adverse events (AE) reported during the study, 19 were related. Most AEs were mild to moderate in intensity. One serious AE (mild atrial fibrillation, unrelated), one pen-related AE (mild nervousness reported prior first administration in the home setting), and one Grade 3 TEAE (pain in extremity, related) were recorded, but none led to a dose reduction.

Conclusion
The AOP2014 pen was well accepted and no major difficulties were reported. The study drug performed as expected and there were no safety concerns arising from the administration of AOP2014 using the pen device. The AOP2014 pen allows for individual dosing and a patient-convenient mode of self-administration of ropeginterferon alfa-2b at home and is expected to support adherence and compliance in the long-term treatment of PV patients.

Session topic: 16. Myeloproliferative neoplasms - Clinical

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