Ruxolitinib In The Treatment Of Polycythemia Vera: An Update On Health-Related Quality Of Life And Patient-Reported Outcomes

Abstract

Polycythemia vera (PV) is a rare myeloproliferative neoplasm (MPN) associated with significant impairment in quality of life (QoL) due to disease-related symptoms and complications. Assessment of disease burden constitutes standard monitoring of symptoms and response. Conventional treatments for MPN, such as hydroxyurea, phlebotomy, or interferon, have not shown a significant impact in QoL or patient-reported outcomes (PRO). Ruxolitinib (RUX) is a JAK2 inhibitor approved for patients intolerant or resistant to hydroxyurea (HA). We conducted a systematic review of clinical trials of RUX in patients with PV that incorporated PRO measures to evaluate the effects on PRO and QoL. Three randomized Phase 3 studies reported in four publications were relevant for analysis. Although the small number of trials and potential for treatment bias in the review, treatment with RUX was associated with improved QoL and PRO in PV patients intolerant or resistant to hydroxyurea.

Keywords:

• polycythemia vera
• ruxolitinib
• quality of life
• patient-reported outcomes

Disclosure

CAY reports involvement in advisory boards with JAZZ in 2018/2019 and with Pfizer in 2017. The authors report no other conflicts of interest in this work.