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ROPEGINTERFERON ALFA-2B ACHIEVES PATIENT-SPECIFIC TREATMENT GOALS IN POLYCYTHEMIA VERA: FINAL RESULTS FROM THE PROUD-PV/CONTINUATION-PV STUDIES
Author(s): ,
Heinz Gisslinger
Affiliations:
Department of Internal Medicine I, Division of Hematology and Blood Coagulation,Medical University Vienna,Vienna,Autriche;Department of Internal Medicine I, Division of Hematology and Blood Coagulation,Medical University Vienna,Vienna,Österreich;Department of Internal Medicine I, Division of Hematology and Blood Coagulation,Medical University Vienna,Vienna,Austria;Department of Internal Medicine I
,
Christoph Klade
Affiliations:
AOP Orphan Pharmaceuticals GmbH,Vienna,Autriche;AOP Orphan Pharmaceuticals GmbH,Vienna,Österreich;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Oostenrijk;AOP Orphan Pharmaceuticals GmbH,Vienna,Áustria;AOP Orphan Pharmaceuticals GmbH,Vienna,Австрия;AOP Orphan Pharma
,
Pencho Georgiev
Affiliations:
Medical University of Plovdiv,Plovdiv,Bulgarie; Medical University of Plovdiv,Plovdiv,Bulgarien; Medical University of Plovdiv,Plovdiv,Bulgaria; Medical University of Plovdiv,Plovdiv,Bulgaria; Medical University of Plovdiv,Plovdiv,Bulgaria; Medical University of Plovdiv,Plovdiv,Bulgarije; Medical University of Plovdiv,Plovdiv,Bulgária; Medical University of Plovdiv,Plovdiv,Болгария; Medical Unive
,
Dorota Krochmalczyk
Affiliations:
Teaching Unit of the Hematology Department,University Hospital in Krakow,Krakow,Pologne;Teaching Unit of the Hematology Department,University Hospital in Krakow,Krakow,Polen;Teaching Unit of the Hematology Department,University Hospital in Krakow,Krakow,Polonia;Teaching Unit of the Hematology Department,University Hospital in Krakow,Krakow,Poland;Teaching Unit of the Hematology Department,Universi
,
Liana Gercheva-Kyuchukova
Affiliations:
Clinical Hematology Clinic,Multiprofile Hospital for Active Treatment "Sveta Marina",Varna,Bulgarie;Clinical Hematology Clinic,Multiprofile Hospital for Active Treatment "Sveta Marina",Varna,Bulgarien;Clinical Hematology Clinic,Multiprofile Hospital for Active Treatment "Sveta Marina",Varna,Bulgaria;Clinical Hematology Clinic,Multiprofile Hospital for Active Treatment "Sveta Marina",Varna,Bulgaria
,
Miklos Egyed
Affiliations:
Department of Internal Medicine II,Kaposi Mor County Teaching Hospital,Kaposvar,Hongrie;Department of Internal Medicine II,Kaposi Mor County Teaching Hospital,Kaposvar,Ungarn;Department of Internal Medicine II,Kaposi Mor County Teaching Hospital,Kaposvar,Ungheria;Department of Internal Medicine II,Kaposi Mor County Teaching Hospital,Kaposvar,Hungary;Department of Internal Medicine II,Kaposi Mor Co
,
Petr Dulicek
Affiliations:
Department of Clinical Hematology,University Hospital Hradec Kralove,Hradec Kralove,Tchèque, République;Department of Clinical Hematology,University Hospital Hradec Kralove,Hradec Kralove,Tschechische Republik;Department of Clinical Hematology,University Hospital Hradec Kralove,Hradec Kralove,Rep. Ceca;Department of Clinical Hematology,University Hospital Hradec Kralove,Hradec Kralove,Czech;Depart
,
Arpad Illes
Affiliations:
Department of Hematology, Faculty of Medicine,University of Debrecen,Debrecen,Hongrie;Department of Hematology, Faculty of Medicine,University of Debrecen,Debrecen,Ungarn;Department of Hematology, Faculty of Medicine,University of Debrecen,Debrecen,Ungheria;Department of Hematology, Faculty of Medicine,University of Debrecen,Debrecen,Hungary;Department of Hematology, Faculty of Medicine,University
,
Halyna Pylypenko
Affiliations:
Department of Hematology, Regional Treatment and Diagnostics Hematology Centre,Cherkasy Regional Oncology Centre,Cherkasy,Ukraine;Department of Hematology, Regional Treatment and Diagnostics Hematology Centre,Cherkasy Regional Oncology Centre,Cherkasy,Ukraine;Department of Hematology, Regional Treatment and Diagnostics Hematology Centre,Cherkasy Regional Oncology Centre,Cherkasy,Ucraina;Department
,
Lylia Sivcheva
Affiliations:
First Department of Internal Medicine,Multiprofile Hospital for Active Treatment - HristoBotev,Vratsa,Bulgarie;First Department of Internal Medicine,Multiprofile Hospital for Active Treatment - HristoBotev,Vratsa,Bulgarien;First Department of Internal Medicine,Multiprofile Hospital for Active Treatment - HristoBotev,Vratsa,Bulgaria;First Department of Internal Medicine,Multiprofile Hospital for Ac
,
Jiří Mayer
Affiliations:
Clinic of Internal Medicine - Hematology and Oncology,University Hospital Brno,Brno,Tchèque, République;Clinic of Internal Medicine - Hematology and Oncology,University Hospital Brno,Brno,Tschechische Republik;Clinic of Internal Medicine - Hematology and Oncology,University Hospital Brno,Brno,Rep. Ceca;Clinic of Internal Medicine - Hematology and Oncology,University Hospital Brno,Brno,Czech;Clinic
,
Vera Yablokova
Affiliations:
Department of Hematology,Yaroslavl Regional Clinical Hospital,Yaroslavl,Russie, Fédération De;Department of Hematology,Yaroslavl Regional Clinical Hospital,Yaroslavl,Russia;Department of Hematology,Yaroslavl Regional Clinical Hospital,Yaroslavl,Russia;Department of Hematology,Yaroslavl Regional Clinical Hospital,Yaroslavl,Russische Federatie;Department of Hematology,Yaroslavl Regional Clinical Hos
,
Victoria Empson
Affiliations:
AOP Orphan Pharmaceuticals GmbH,Vienna,Autriche;AOP Orphan Pharmaceuticals GmbH,Vienna,Österreich;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Oostenrijk;AOP Orphan Pharmaceuticals GmbH,Vienna,Áustria;AOP Orphan Pharmaceuticals GmbH,Vienna,Австрия;AOP Orphan Pharma
,
Kurt Krejcy
Affiliations:
AOP Orphan Pharmaceuticals GmbH,Vienna,Autriche;AOP Orphan Pharmaceuticals GmbH,Vienna,Österreich;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Austria;AOP Orphan Pharmaceuticals GmbH,Vienna,Oostenrijk;AOP Orphan Pharmaceuticals GmbH,Vienna,Áustria;AOP Orphan Pharmaceuticals GmbH,Vienna,Австрия;AOP Orphan Pharma
,
Hans Hasselbalch
Affiliations:
Department of Hematology,Zealand University Hospital, University of Copenhagen,Roskilde,Danemark;Department of Hematology,Zealand University Hospital, University of Copenhagen,Roskilde,Dänemark;Department of Hematology,Zealand University Hospital, University of Copenhagen,Roskilde,Danimarca;Department of Hematology,Zealand University Hospital, University of Copenhagen,Roskilde,Danemark;Department
,
Robert Kralovics
Affiliations:
Department of Laboratory Medicine,Medical University of Vienna,Vienna,Autriche;Department of Laboratory Medicine,Medical University of Vienna,Vienna,Österreich;Department of Laboratory Medicine,Medical University of Vienna,Vienna,Austria;Department of Laboratory Medicine,Medical University of Vienna,Vienna,Austria;Department of Laboratory Medicine,Medical University of Vienna,Vienna,Austria;Depart
Jean-Jacques Kiladjian
Affiliations:
Centre d'Investigations Cliniques, INSERM, CIC1427,Université de Paris, AP-HP, Hôpital Saint-Louis,Paris,France;Centre d'Investigations Cliniques, INSERM, CIC1427,Université de Paris, AP-HP, Hôpital Saint-Louis,Paris,Frankreich;Centre d'Investigations Cliniques, INSERM, CIC1427,Université de Paris, AP-HP, Hôpital Saint-Louis,Paris,Francia;Centre d'Investigations Cliniques, INSERM, CIC1427,Un
(Abstract release date: 05/12/22) EHA Library. Gisslinger H. 06/11/22; 357060; S196
Prof. Dr. Heinz Gisslinger
Prof. Dr. Heinz Gisslinger
Contributions
Abstract
Presentation during EHA2022: All Oral presentations will be presented between Friday, June 10 and Sunday, June 12 and will be accessible for on-demand viewing from Monday, June 20 until Monday, August 15, 2022 on the Congress platform.

Abstract: S196

Type: Oral Presentation

Session title: Treatments and complications in MPN

Background
Treatment of polycythemia vera (PV) aims to prevent thromboembolic complications, reduce the risk of progression to acute leukemia or myelofibrosis - of particular concern to patients - and ameliorate the symptom burden; specifically, to improve quality of life, therapy should address the most clinically important symptoms while reducing phlebotomies to avoid iron-deficiency symptoms. Long-term efficacy and safety of ropeginterferon alfa-2b have been demonstrated in PROUD-PV/CONTINUATION-PV; the final analysis applied a patient-focused approach.

Aims
To analyze the patient-relevant benefit of ropeginterferon alfa-2b versus hydroxyurea (HU)/best available treatment (BAT) over 6 years.

Methods

Patients diagnosed with PV according to WHO 2008 criteria who were cytoreduction-naïve or hydroxyurea pre-treated and gave written informed consent were randomized 1:1 to ropeginterferon alpha-2b or control treatment (HU) for one year in PROUD-PV. In CONTINUATION-PV, control arm patients could switch from HU to BAT. Patient-reported PV symptom burden was assessed based on adverse events documented in the patient diary and recorded at each visit; items defined in the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; fatigue, concentration problems, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers) and medical synonyms were evaluated post-hoc. Efficacy assessments included Kaplan-Meier analysis of event-free survival, phlebotomy need and JAK2V617F allele burden. Analyses were conducted on the CONTINUATION-PV full analysis set over 6 years of treatment.

Results
The full analysis set comprised 95 patients in the ropeginterferon alfa-2b arm and 74 in the control arm.

Patient-reported symptoms defined in the MPN-SAF TSS were present in a small minority (9.5%) of patients per arm at baseline (up to Week 4 of treatment) in this early-stage PV population. Occurrence of the defined symptoms remained low over long-term treatment, reported in 15.7% of patients in the ropeginterferon alfa-2b arm and 20.7% in the control arm during the 6th year of treatment.

No phlebotomies were required to maintain hematocrit <45% in the 6th year of treatment in 81.4% of patients receiving ropeginterferon alfa-2b compared with 60.0% of patients in the control arm (p=0.005).

Depletion of the JAK2V617F alle burden, which may lower the risk of progression to myelofibrosis, was observed in ropeginterferon alfa-2b treated patients; JAK2V617F allele burden <1% at 6 years was achieved in 19/92 (20.7%) patients in the ropeginterferon alfa-2b arm with baseline allele burden >10%. One patient met this threshold in the control arm (1/70 [1.4%]; p=0.0001).

Event-free survival (risk events: disease progression, death and thromboembolic events) over ≥6 years of treatment was significantly higher among ropeginterferon alfa-2b treated patients than the control group (risk events reported in 5/95 vs. 12/74 patients, respectively; p=0.04 [Log-Rank]; Fig 1).

Conclusion
Long-term ropeginterferon alfa-2b therapy fulfils treatment goals important to patients with PV: a good quality of life as indicated by a low symptom burden and phlebotomy requirement, the potential to influence myelofibrosis risk, and better event-free survival versus BAT.

Keyword(s): Clinical trial, Interferon alpha, Pegylated Interferon, Polycythemia vera

Presentation during EHA2022: All Oral presentations will be presented between Friday, June 10 and Sunday, June 12 and will be accessible for on-demand viewing from Monday, June 20 until Monday, August 15, 2022 on the Congress platform.

Abstract: S196

Type: Oral Presentation

Session title: Treatments and complications in MPN

Background
Treatment of polycythemia vera (PV) aims to prevent thromboembolic complications, reduce the risk of progression to acute leukemia or myelofibrosis - of particular concern to patients - and ameliorate the symptom burden; specifically, to improve quality of life, therapy should address the most clinically important symptoms while reducing phlebotomies to avoid iron-deficiency symptoms. Long-term efficacy and safety of ropeginterferon alfa-2b have been demonstrated in PROUD-PV/CONTINUATION-PV; the final analysis applied a patient-focused approach.

Aims
To analyze the patient-relevant benefit of ropeginterferon alfa-2b versus hydroxyurea (HU)/best available treatment (BAT) over 6 years.

Methods

Patients diagnosed with PV according to WHO 2008 criteria who were cytoreduction-naïve or hydroxyurea pre-treated and gave written informed consent were randomized 1:1 to ropeginterferon alpha-2b or control treatment (HU) for one year in PROUD-PV. In CONTINUATION-PV, control arm patients could switch from HU to BAT. Patient-reported PV symptom burden was assessed based on adverse events documented in the patient diary and recorded at each visit; items defined in the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; fatigue, concentration problems, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers) and medical synonyms were evaluated post-hoc. Efficacy assessments included Kaplan-Meier analysis of event-free survival, phlebotomy need and JAK2V617F allele burden. Analyses were conducted on the CONTINUATION-PV full analysis set over 6 years of treatment.

Results
The full analysis set comprised 95 patients in the ropeginterferon alfa-2b arm and 74 in the control arm.

Patient-reported symptoms defined in the MPN-SAF TSS were present in a small minority (9.5%) of patients per arm at baseline (up to Week 4 of treatment) in this early-stage PV population. Occurrence of the defined symptoms remained low over long-term treatment, reported in 15.7% of patients in the ropeginterferon alfa-2b arm and 20.7% in the control arm during the 6th year of treatment.

No phlebotomies were required to maintain hematocrit <45% in the 6th year of treatment in 81.4% of patients receiving ropeginterferon alfa-2b compared with 60.0% of patients in the control arm (p=0.005).

Depletion of the JAK2V617F alle burden, which may lower the risk of progression to myelofibrosis, was observed in ropeginterferon alfa-2b treated patients; JAK2V617F allele burden <1% at 6 years was achieved in 19/92 (20.7%) patients in the ropeginterferon alfa-2b arm with baseline allele burden >10%. One patient met this threshold in the control arm (1/70 [1.4%]; p=0.0001).

Event-free survival (risk events: disease progression, death and thromboembolic events) over ≥6 years of treatment was significantly higher among ropeginterferon alfa-2b treated patients than the control group (risk events reported in 5/95 vs. 12/74 patients, respectively; p=0.04 [Log-Rank]; Fig 1).

Conclusion
Long-term ropeginterferon alfa-2b therapy fulfils treatment goals important to patients with PV: a good quality of life as indicated by a low symptom burden and phlebotomy requirement, the potential to influence myelofibrosis risk, and better event-free survival versus BAT.

Keyword(s): Clinical trial, Interferon alpha, Pegylated Interferon, Polycythemia vera

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