Polycythemia Vera Grand Rounds References and Footnotes

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1. Arber DA, Orazi A, Hasserjian R, et al. The 2016 revisions to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127:2391-2405.

2. Rumi E, Cazzola M. Diagnosis, risk stratification, and response evaluation in classical myeloproliferative neoplasms. Blood. 2017;129(6):680-692.

3. Parasuraman S, DiBonaventura M, Reith K, et al. Patterns of hydroxyurea use and clinical outcomes among patients with polycythemia vera in real-world clinical practice: a chart review. Experimental Hematology Oncology. 2016;5:3.

4. Mascarenhas J. A concise update on risk factors, therapy, and outcome of leukemic transformation of myeloproliferative neoplasms. Clinical Lymphoma, Myeloma & Leukemia. 2016;16 suppl:S124-S129.

5. Spivak JL, Considine M, Williams DM, et al. Two clinical phenotypes in polycythemia vera. The New England Journal of Medicine. 2014;371(9):808-817.

6. Barosi G, Birgegard G, Finazzi G, et al. A unified definition of clinical resistance and intolerance to hydroxycarbamide in polycythaemia vera and primary myelofibrosis: results of a European LeukemiaNet (ELN) consensus process. The British Journal of Haematology. 2010;148(6):961-963.

7. Marchioli R, Finazzi G, Specchia G, et al. Cardiovascular events and intensity of treatment in polycythemia vera. The New England Journal of Medicine. 2013;368(1):22-33.

8. Barbui T, Masciulli A, Marfisi MR, et al. White blood cell counts and thrombosis in polycythemia vera: a subanalysis of the CYTO-PV study. Blood. 2015;126(4):560-561.

9. Emanuel RM, Dueck AC, Geyer HL, et al. Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs. Journal of Clinical Oncology. 2012;30(33):4098-4103.

10. Spivak JL. Narrative review: thrombocytosis, polycythemia vera, and jak2 mutations: the phenotypic mimicry of chronic myeloproliferation. Annals of Internal Medicine. 2010;152:300-306.

11. Passamonti F. How I treat polycythemia vera. Blood. 2012;120:275-284.

12. Stuart BJ, Viera AJ. Polycythemia vera. American Family Physician. 2004;69:2139-2144.

13. Data on file. Incyte Corporation. Polycthemia vera market sizing. Wilmington, DE. 2012.

14. Tefferi A, Rumi E, Finazzi G, et al. Survival and prognosis among 1545 patients with contemporary polycythemia vera: an international study. Leukemia. 2013;27:1874-81.

15. Barbui T, Barosi G, Birgegard G, et al. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. Journal of Clinical Oncology. 2011;29:761-770.

16. Falanga A, Marchetti M. Thrombotic disease in myeloproliferative neoplasms. Hematology American Society Hematology Education Program. 2012;2012:571-581.

17. Gangat N, Strand J, Li C-Y, et al. Leucocytosis in polycythaemia vera predicts both inferior survival and leukaemic transformation. British Journal of Haematology. 2007;138:354-358.

18. Landolfi R, Di Gennaro L, Barbui T, et al. Leukocytosis as a major risk factor in patients with polycythemia vera. Blood. 2007;109:2446-52.

19. Mesa R, Miller CB, Thyne M, et al. Myeloproliferative neoplasms (MPNs) have a significant impact on patients’ overall health and productivity: the MPN Landmark survey. BMC Cancer. 2016;16:167.

20. Geyer H, Scherber R, Kosiorek H, et al. Symptomatic profiles of patients with polycythemia vera: implications of inadequately controlled disease. Journal of Clinical Oncology. 2016;34:151-159.


ᵃ Patients reported whether they strongly agreed, somewhat agreed, somewhat disagreed, or strongly disagreed with the following statement: PV symptoms reduce my quality of life.¹⁹

ᵇ Symptoms were evaluated using an adapted version of the MPN Symptom Assessment Form (MPN-SAF) and were rated on a scale of 0 (absent) to 10 (worst imaginable). The incidence is the percentage of those respondents who reported experiencing the symptom (ie, score ≥1) within the 12 months preceding the survey.¹⁹

ᶜ Patients reported impact on their activities of daily living on a scale that ranged from 1 (not at all) to 5 (a great deal). The patient was included as having interference with daily activities if they had ever experienced the issue and reported a score >1.¹⁹

* Bone marrow biopsy may not be required in cases with sustained absolute erythrocytosis: hemoglobin levels >18.5 g/dL in men (hematocrit 55.5%) or >16.5 g/dL in women (hematocrit 49.5%) if mutation criterion and the minor criterion are present. However, initial myelofibrosis (MF) (present in up to 20% of patients) can only be detected by performing a bone marrow biopsy; hypercellularity may predict a more rapid progression to overt MF (post-PV MF).¹

† In the Cytoreductive Therapy in Polycythemia Vera (CYTO-PV) study of 365 adult patients with PV treated with phlebotomy, hydroxyurea, or both, patients were randomized to 1 of 2 groups—either the low-Hct group (n=182; with more intensive therapy to maintain a target Hct level <45%) or the high-Hct group (n=183; with less intensive therapy to maintain a target Hct level of 45% to 50%). Baseline characteristics were balanced between the groups. Approximately 50% of patients had received an initial diagnosis of PV within 2 years prior to randomization. 67.1% of patients (n=245) were at high risk because of age ≥65 years or previous thrombosis. The composite primary end point was the time until cardiovascular death or major thrombosis. ⁷

‡ Adjusted for age, gender, cardiovascular risk factors, previous thrombosis, and Hct levels.⁸

§ The Myeloproliferative Neoplasm (MPN) Landmark Survey, funded by Incyte Corporation, was a web-based questionnaire composed of 65 multiple-choice questions intended to help evaluate the patient disease burden in the MPN disease setting. A total of 813 patients in the US with a previous diagnosis of MF, PV, or essential thrombocythemia (ET) completed the survey (MF, n=207; PV, n=380; ET, n=226).¹⁹

‖ A prospective study of 1334 patients with PV was conducted to assess baseline symptoms with certain disease features (ie, known hydroxyurea use, known phlebotomy requirements, and splenomegaly). The patients had the following characteristics: known hydroxyurea use (n=499), known phlebotomy (n=646), palpable splenomegaly (n=369), or all three features (n=148). Assessment of myeloproliferative neoplasm (MPN) symptoms was performed by using the MPN-Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; MPN-10). All items were evaluated on a 0 (absent) to 10 (worst imaginable) scale. The MPN-10 TSS has a possible range of 0 to 100 with 100 representing the highest level of symptom severity. The TSS for each patient was analyzed to place the patient into the quartiles of low symptom burden (TSS, 0 to 7), intermediate symptom burden (TSS, 8 to 17), moderately high symptom burden (TSS, 18 to 31), or high symptom burden (TSS, ≥32).²⁰