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Clinical Trial
. 2020 Dec;191(5):764-774.
doi: 10.1111/bjh.16729. Epub 2020 Jun 24.

Real-world non-interventional long-term post-authorisation safety study of ruxolitinib in myelofibrosis

Fiorenza Barraco  1 , Richard Greil  2 , Raoul Herbrecht  3 , Burkhard Schmidt  4 , Andreas Reiter  5 , Wolfgang Willenbacher  6   7 , Reinier Raymakers  8 , Rüdiger Liersch  9 , Monika Wroclawska  10 , Robert Pack  10 , Karin Burock  10 , Divyadeep Karumanchi  11 , Heinz Gisslinger  12
Affiliations
Clinical Trial

Real-world non-interventional long-term post-authorisation safety study of ruxolitinib in myelofibrosis

Fiorenza Barraco et al. Br J Haematol. 2020 Dec.

Abstract

Primary objective of this non-interventional, post-authorisation safety study was to provide real-world safety data [incidence of adverse drug reactions (ADRs)/serious adverse events (SAEs)] on adult patients with myelofibrosis exposed/or not exposed to ruxolitinib. Key secondary objectives included the incidence/outcome of events of special interest (bleeding events, serious/opportunistic infections, second primary malignancies, and deaths). Overall, 462 patients were included [prevalent users = 260, new users = 32, non-exposed = 170 (inclusive of ruxolitinib-switch, n = 57)]. The exposure-adjusted incidence rates (per 100 patient-years) of ADRs (19·3 vs. 19·6) and SAEs (25·2 vs. 25·0) were comparable amongst new-users versus prevalent-users cohorts, respectively; most frequent ADRs across all cohorts included thrombocytopenia, anaemia, epistaxis, urinary tract infection, and herpes zoster. Anaemia, pneumonia, general physical health deterioration, sepsis, and death were the most frequent SAEs across all cohorts. Incidence rates of bleeding events (21·6) and serious/opportunistic infections (34·5) were higher in ruxolitinib-switch cohort versus other cohorts. The incidence rate of second primary malignancies was higher in the prevalent-users cohort (10·1) versus other cohorts. The observed safety profile of ruxolitinib in the present study along with the safety findings from the COMFORT/JUMP/EXPAND studies support the use of ruxolitinib for long-term treatment of patients with myelofibrosis.

Keywords: adverse drug reaction; adverse event; myelofibrosis; ruxolitinib; safety.

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