NICE Recommends Ruxolitinib for Hydroxycaramide/Hydroxyurea Resistant or Intolerant Polycythemia Vera


The National Institute for Health and Care Excellence has published its Final Draft Guidance recommending the use of ruxolitinib as treatment for eligible patients in England and Wales with polycythemia vera that is resistant or intolerant to hydroxycarbamide/hydroxyurea.

Claire Harrison, MD, FRCP, FRCPath

Claire Harrison, MD, FRCP, FRCPath

The National Institute for Health and Care Excellence (NICE) has published its Final Draft Guidance recommending the use of ruxolitinib (Jakafi) as treatment for eligible patients in England and Wales with polycythemia vera (PV) that is resistant or intolerant to hydroxycarbamide/hydroxyurea (HC/HU).1

“There is a significant unmet need for people with PV in England and Wales, who live with a large symptom burden as a result of their condition,’’ Claire Harrison, MD, FRCP, consultant hematologist, Guy’s and St Thomas’ NHS Foundation Trust in London, said in a news release. “Today’s decision is a step in the right direction for providing additional treatment options that reduce the burden of these symptoms and improve disease progression, in this under-represented patient population.”

In 2017, findings from the MPN LANDMARK survey demonstrated that 72% of patients with symptomatic PV experienced reduced quality of life. Moreover, approximately one-third of patients in the study experienced disease-related anxiety.2

Although venesection and HC/HU are commonly used treatments for patients with PV, approximately one-quarter of patients who receive HC/HU will develop resistance or intolerance, leading to subpar disease control and increased risk of progression.1

Ruxolitinib has been approved for use in patients with PV who have had an inadequate response to or are intolerant of hydroxyurea in the United States since 2014.3

The approval in this population was based on findings from the pivotal phase 3 RESPONSE trial (NCT01243944), which was conducted under a Special Protocol Assessment from the FDA.

Results from the trial demonstrated improved hematocrit control and reductions in spleen volume with ruxolitinib compared with best available therapy (BAT). In addition, more patients experienced hematologic remission—defined as achieving hematocrit control and lowering platelet and white blood cell counts—with ruxolitinib vs BAT.

Specifically, the study met its primary end point of the study of hematocrit control through week 32 and at least a 35% reduction in spleen volume at week 32 (21% vs 1% with ruxolitinib and BAT, respectively; P < .001). Hematocrit control was achieved in 60% of patients who received ruxolitinib and 20% of those who received BAT; 38% and 1% of patients in the two arms, respectively, had at least a 35% reduction in spleen volume.4

In terms of safety, the most frequent hematologic adverse effects (AEs) that occurred in more than 20% of patients were thrombocytopenia and anemia. The most common non-hematologic AEs that occurred in over 10% of patients were headache, abdominal pain, diarrhea, dizziness, fatigue, pruritus, dyspnea, and muscle spasms.4

“We welcome this recommendation from NICE, as PV can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms. It affects not only patients but also their families and careers and turns many everyday tasks into major hurdles. Ruxolitinib addresses a significant unmet need in patients who cannot tolerate or no longer respond to HC/HU,” Jon Mathias, co-chair of MPN Voice, said.1

“Today’s NICE recommendation in PV is an example of how we are reimagining medicine to transform the treatment of people with various blood cancers. The availability of ruxolitinib for eligible patients with PV in England and Wales will give them and their healthcare professionals more options in the management of this debilitating condition,” Marie-Andree Gamache, president & managing director of Novartis UK and Ireland, stated.


  1. NICE recommends Novartis Jakavi (ruxolitinib) for patients living with polycythaemia vera (PV). News release. Novartis. September 14, 2023. Accessed September 14, 2023.
  2. Harrison CN, Koschmieder S, Foltz L, et al. The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey. Ann Hematol. 2017;96(10):1653-1665. doi:10.1007/s00277-017-3082-y
  3. FDA approves Jakafi (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera. News release. Incyte Corporation. December 4, 2014. Accessed September 14, 2023.
  4. Vannucchi AM, Kiladjian JJ, Griesshammer M, et al. Ruxolitinib versus standard therapy for the treatment of polycythemia vera. N Engl J Med. 2015;29(372):426-435. doi:10.1056/NEJMoa1409002
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