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Patients with polycythemia vera often experience severe symptom burden
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Patients with polycythemia vera and a history of prior hydroxyurea use, phlebotomy requirements or palpable splenomegaly tended to have a significant disease-associated symptom burden, according to prospective study results published in Journal of Clinical Oncology.
Polycythemia vera — a myeloproliferative neoplasm that evolves from dysregulated clonal erythropoiesis — is associated with a high symptom burden and potential life-threatening complications.
Ruben A. Mesa, MD, FACP
“It has been our clinical impression that patients with polycythemia vera have a very heterogeneous burden of disease and that this could have real implications in terms of their management,” Ruben A. Mesa, MD, FACP, professor of medicine and chair of hematology and medical oncology at Mayo Clinic in Scottsdale, Arizona, as well as a HemOnc Today Editorial Board member, told HemOnc Today. “We conducted a very significant analysis in our aggregated data of patients with polycythemia vera to better understand the contributions of each problematic clinical feature on symptom burden.”
Recent research has suggested that JAK inhibitor therapy may serve as an effective management tool for patients with polycythemia vera who have previously used hydroxyurea (including patients who developed resistance or intolerance), have phlebotomy requirements or a palpably enlarged spleen.
Thus, Mesa and colleagues sought to determine how these features contributed to the polycythemia vera symptom burden, alone and in aggregate.
They accessed prospective data from 1,334 patients enrolled in two trials. All patients had known hydroxyurea use (n = 499), known phlebotomy (n = 646), palpable splenomegaly (n = 369) or all three features (n = 148). Researchers created control groups for each polycythemia vera clinical feature cohort composed of the remaining total cohort without that specific feature.
Disease duration varied among these subgroups (range, 6.5 years-11.5 years) and appeared significantly longer among patients with known hydroxyurea use (median, 8.8 years vs. 6.6 years; P = .008) and spleen enlargement (median, 8.6 years vs. 6.5 years; P = .005) compared with controls.
Intermediate to high symptom burdens occurred in all subgroups, with the highest symptom burden among patients harboring all three features (total symptom score [TSS], 32.5; range, 27.7-32.5).
Symptom burden persisted independent of polycythemia vera risk category, and increased incrementally in severity with the addition of multiple features.
Although patients harboring all features had the highest total score, they had similar individual symptom scores — such as those for abdominal discomfort, inactivity, night sweats, bone pain and weight loss — as patients with known hydroxyurea use, phlebotomy or splenomegaly.
“These findings were surprising, as many treating physicians are observing that treating polycythemia vera symptoms are problematic,” Mesa said in an interview. “Polycythemia vera is a heterogeneous disease with over 150,000 U.S. patients at the moment, and the key outcome of the study is for physicians to be mindful of the spectrum of difficulties and for those patients with the greatest difficulties to consider they may need further interventions.”
The researchers acknowledged limitations of their study, including the inability to determine whether hydroxyurea use or phlebotomy were being used currently or had been used remotely. Further, although the researchers judged potential selection bias to be minimal, they did not directly investigate it in their study.
“Our study helps to define the significant burden of polycythemia vera, specifically in a variety of subgroups with more problematic clinical features,” Mesa said. “There are certainly implications currently on clinical practice helping to highlight the unmet needs in these patients, and particularly may have relevance regarding selection of JAK inhibition, of which there is an indication for ruxolitinib [Jakafi, Novartis Oncology], as well as potentially second- or third-line therapy with pegylated interferon alpha or even other experimental medications.” – by Cameron Kelsall
For more information:
Ruben A. Mesa, MD, can be reached at mesa.ruben@mayo.edu.
Disclosure: Mesa reports honoraria from and a consultant role with Novartis, as well as institutional research funding from CTI, Gilead Sciences, Incyte and Promedior. Please see the full study for a list of all other researchers’ relevant financial disclosures.
Perspective
Back to TopBart Lee Scott, MD, and Bethany Samuelson, MD
Polycythemia vera (PV) is a myeloproliferative neoplasm with an incidence of one person in 50,000 per year. Historically, PV management was focused on reducing thrombosis risk, a common complication in these patients. The decision to initiate treatment in PV is usually based on risk for thrombotic complications — based largely on age, history of thrombosis and blood counts — rather than symptoms. The mainstay of therapy for PV has been phlebotomy and antiplatelet therapy with aspirin. Many therapeutic interventions to date have not given due diligence to the importance of symptom control in PV.
In their recent study, Geyer and colleagues sought to describe the symptom burden of PV in a large cohort of patients. They quantified the symptom burden of 1,334 patients using the validated myeloproliferative neoplasm symptom assessment form. Patients were grouped according to presence or absence of three features: phlebotomy requirement, hydroxyurea use or splenomegaly. A significant symptom burden was noted in all patients, with burden quantified as intermediate to high in all subgroups, increasing with each additional feature. The greatest symptom burden was reported in patients with all three features. Fatigue and microvascular-related symptoms, such as poor concentration and pruritus, were the most commonly reported symptoms.
This article suggests that symptom burden in PV patients has been historically underrecognized and undertreated. It emphasizes the need for better options for symptom control and the importance of including assessments of impact on symptom burden in future studies of treatment response and in the ongoing management of patients with PV.
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