REAL-WORLD SAFETY DATA FROM A NONINTERVENTIONAL LONG-TERM POSTAUTHORIZATION SAFETY STUDY OF RUXOLITINIB IN MYELOFIBROSIS
Author(s): ,
Fiorenza Barraco
Affiliations:
Centre Hospitalier Lyon Sud,Pierre-Benite,France
,
Richard Greil
Affiliations:
Paracelsus Medical University Salzburg; Salzburg Cancer Research Institute; Cancer Cluster Salzburg,Salzburg,Austria
,
Raoul Herbrecht
Affiliations:
Hôpital de Hautepierre,Strasbourg,France
,
Helmut Burkhard Schmidt
Affiliations:
Haemato-Onkol. Gemeinschaftspraxis,Munich,Germany
,
Andreas Reiter
Affiliations:
Universitaets-klinikum Mannheim,Mannheim,Germany
,
Wolfgang Willenbacher
Affiliations:
Universitaetsklinik Innsbruck; Oncotyrol, Center for Personalized Cancer Medicine,Innsbruck,Austria
,
Reinier Raymakers
Affiliations:
University Medical Center Utrecht,Utrecht,Netherlands
,
Rüdiger Liersch
Affiliations:
Internal Medicine Hematology and Oncology,Studienzentrale GEHO,Muenster,Germany
,
Monika Wroclawska
Affiliations:
Novartis AG,Basel,Switzerland
,
Robert Pack
Affiliations:
Novartis AG,Basel,Switzerland
,
Karin Burock
Affiliations:
Novartis AG,Basel,Switzerland
,
Michael Levine
Affiliations:
Novartis Pharmaceuticals Corporation,East Hanover, New Jersey,United States
Heinz Gisslinger
Affiliations:
Department of Hematology and Blood Coagulation,Medical University of Vienna,Vienna,Austria
EHA Library. BARRACO F. Jun 14, 2019; 266478; PF679
Dr. Fiorenza BARRACO
Dr. Fiorenza BARRACO
Contributions
Abstract

Abstract: PF679

Type: Poster Presentation

Presentation during EHA24: On Friday, June 14, 2019 from 17:30 - 19:00

Location: Poster area

Background
Ruxolitinib (RUX, oral JAK inhibitor) was approved for treatment (Tx) of patients (pts) with myelofibrosis (MF) based on the data from COMFORT I/II (phase 3 registrational studies). At the time of submission of the Marketing Authorization Application in the European Union (EU), a significant number of pts (589) had been exposed to RUX in clinical studies. However, there was a lack of postmarketing exposure data leading to an agreement with the European Medicines Agency to conduct this postauthorization safety study (PASS [ENCePP No. 3296]: per EU Vol. 9a [Rules for Medicinal Products]).

Aims
To provide real-world safety data on pts with MF exposed/nonexposed to RUX.

Methods
This was a prospective, multicenter, noninterventional PASS study for adult pts with MF (primary [per WHO criteria]/secondary [per IWG-MRT criteria]). The primary objective was to study long-term safety (incidence of adverse drug reactions [ADRs]/serious adverse events [SAEs]) in pts with MF treated with RUX as per prescribing information. The key secondary objectives included the incidence/outcome of events of special interest (EoSI [bleeding events, serious/opportunistic infections, second primary malignancies, and deaths]). The statistical analysis was done after last pt completed 3 years on the study or discontinued early (final database lock, July 5, 2018).

Results
Overall, 462 pts were included (prevalent users=260, new users=32, nonexposed=170 [inclusive of RUX-switch {N=57}]). The demographic/baseline pt characteristics (representative of pts with MF and well balanced across all types of users) and pt disposition will be described in the presentation. The study duration, RUX-exposure, summary of dose reductions/interruptions (primary reasons: medical decisions/ADRs across all cohorts), Tx-emergent ADRs, and Tx-emergent SAEs are summarized in Table A. The exposure-adjusted incidence rates of ADRs and SAEs were comparable among the new users’ vs the prevalent users’ cohorts (ADR: 19.3 vs 19.6; SAE: 25.2 vs 25.0; Table A). The most frequently reported ADRs across all types of cohorts included thrombocytopenia, anemia, epistaxis, and herpes zoster. The incidence rates of thrombocytopenia and anemia were higher in the RUX-switch vs prevalent users’/new users’ cohorts (shorter duration of exposure in the RUX-switch cohort). Anemia, pneumonia, and general physical health deterioration were the most frequently reported SAEs across all cohorts. The incidence rate of Tx-emergent SAE (any SAE) was slightly higher in RUX-switch cohort vs the prevalent users’/new users’ cohorts. The Tx-emergent EoSIs are presented in Table B. Bleeding events were reported in a larger proportion of pts in the prevalent users’ cohort; while serious/opportunistic infections were reported in a larger proportion of pts in the new users’ cohort (vs other cohorts). The second primary malignancies were reported in a larger proportion of pts in the prevalent users’ cohort (Table B). The incidence rate of on-Tx deaths (due to any cause [mostly attributed to progression of underlying MF, sepsis, and pneumonia]) is listed in Table B.

Conclusion
The long-term safety of RUX as assessed in this real-world PASS study was consistent with the previous findings (COMFORT I/II). Of note, the study comprised of a broader population including pts at lower risk/elderly pts vs the COMFORT I/II study populations. No new or unexpected safety signals were identified with the long-term Tx. Overall, the observed safety profile of RUX in this PASS study along with the safety findings from COMFORT I/II support the long-term Tx with RUX in pts with MF.

Session topic: 16. Myeloproliferative neoplasms - Clinical

Keyword(s): Myelofibrosis, Ruxolitinib, Safety

By continuing to browse or by clicking “Accept Terms & all Cookies”, you agree to the storing of third-party cookies on your device to enhance your user experience and agree to the user terms and conditions of this learning management system (LMS).

Cookie Settings
Accept Terms & all Cookies