Treatment Patterns and Blood Counts in Patients With Polycythemia Vera Treated With Hydroxyurea in the United States: An Analysis From the REVEAL Study

Abstract

National guidelines support the use of hydroxyurea (HU) in high-risk patients with polycythemia vera (PV). In this study, we investigated HU treatment patterns in patients with PV. Of patients who received HU for ‡ 3 months, 32.3% had dose adjustments, 23.7% had dose interruptions, and 18.6% discontinued HU. These results emphasize the need for active management of patients with PV. Background: Polycythemia vera (PV) is associated with increased blood cell counts, risk of thrombosis, and symptoms including fatigue and pruritus. National guidelines support the use of hydroxyurea (HU) in high-risk patients or those with some other clinical indication for cytoreduction. Patients and Methods: REVEAL is a prospective, observational study designed to collect data pertaining to demographics, disease burden, clinical management, patient-reported outcomes, and health care resource utilization of patients with PV in the United States. In this analysis, HU treatment patterns and outcomes were assessed from 6 months prior to enrollment to the time of discontinuation, death, or data cutoff. Results: Of the 1381 patients who received HU for ! 3 months, the median HU exposure was 23.6 months (range, 3.1-38.5 months). The most common maximum daily HU doses were 1000 mg (30.6%) and 500 mg (30.1%); only 6.4% received ! 2 g/d HU. Approximately one-third (32.3%) of patients had dose adjustments, 23.8% had dose interruptions, and 257 (18.6%) discontinued HU. The most common reasons for HU discontinuations and interruptions were adverse events/intolerance (37.1% and 54.5%, respectively) and lack of efficacy (35.5% and 22.1%, respectively). Of those who received HU for ! 3 months, 57.1% had hematocrit values > 45% on ! 1 occasion, 33.1% continued to receive phlebotomies, and 27.4% had uncontrolled myeloproliferation. Conclusion:The results of this analysis emphasize the need for active management of patients with PV with appropriate HU dose titration to maintain blood count control while monitoring for signs and symptoms of HU intolerance.

Absence of hydroxyurea‐induced mutational effects supports higher utilisation for the treatment of sickle cell anaemia

“…There are also non-haematological side-effects that affect 5-10% of patients receiving hydroxyurea including mucocutaneous ulcers (oral, genital, leg), pneumonitis, leg ulcers, fever and various gastrointestinal symptoms. 59,60 Table II. Selected published in vitro hydroxyurea genotoxicity data, derived from Liebelt et al, 2007 with updates.…”

Real-World Dosing Patterns of Ruxolitinib in Patients With Polycythemia Vera Who Are Resistant to or Intolerant of Hydroxyurea

“…Reasons for lack of guidelineconcordant dosing cannot be determined from this chart review, but this observation is similar to findings from a prospective observational study (REVEAL), in which only 89 of 1381 patients with PV (6.4%) treated with hydroxyurea for ≥3 months received a maximum dose of 2 g/day. 15 All patients in the current study of community hematology/oncology practices received ruxolitinib after discontinuing hydroxyurea, roughly half of whom (53%) initiated ruxolitinib at the US PI-recommended dose of 10 mg BID. 12 Although lower doses are recommended under certain circumstances (ie, for patients with renal or hepatic impairment), only 3% of patients initiated ruxolitinib at a dose lower than 10 mg BID, whereas 44% of patients received an initial ruxolitinib dose higher than 10 mg BID, which is not supported by the US PI.…”

Is there a gender effect in polycythemia vera?

“…Reduction of cardiovascular events was higher in the former cohort and independent from sex, as well as age, previous thrombosis, platelet or leukocyte counts, splenomegaly, past cytoreductive treatment, or antiplatelet/anticoagulant drugs [45]. The observational study REVEAL prospectively followed 2510 PV patients in the USA [87]. The reported proportion of females receiving HU was comparable with that of males [87].…”

“…The observational study REVEAL prospectively followed 2510 PV patients in the USA [87]. The reported proportion of females receiving HU was comparable with that of males [87].…”