Interferon-alpha (rIFNα) is the only disease-modifying treatment for polycythemia vera (PV), but whether or not it prolongs survival is unknown. This large single center retrospective study of 470 PV patients compares the myelofibrosis-free survival (MFS) and overall survival (OS) with rIFNα to two other primary treatments, hydroxyurea (HU) and phlebotomy-only (PHL-O). The median age at diagnosis was 54 years (range 20–94) and the median follow-up was 10 years (range 0–45). Two hundred and twenty-nine patients were women (49%) and 208 were high-risk (44%). The primary treatment was rIFNα in 93 (20%), HU in 189 (40%), PHL-O in 133 (28%) and other cytoreductive drugs in 55 (12%). The treatment groups differed by ELN risk score (p < 0.001). In low-risk patients, 20-year MFS for rIFNα, HU, and PHL-O was 84%, 65% and 55% respectively (p < 0.001) and 20-year OS was 100%, 85% and 80% respectively (p = 0.44). In high-risk patients, 20-year MFS for rIFNα, HU, and PHL-O was 89%, 41% and 36% respectively (p = 0.19) and 20-year OS was 66%, 40%, 14% respectively (p = 0.016). In multivariable analysis, longer time on rIFNα was associated with a lower risk of myelofibrosis (HR: 0.91, p < 0.001) and lower mortality (HR: 0.94, p = 0.012). In conclusion, this study supports treatment of PV with rIFNα to prevent myelofibrosis and potentially prolong survival.
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We acknowledge Paul J Christos, PhD for his review of the statistical methodology and results.
This study was supported by grants from the David L. Johns Family of the Cancer Research & Treatment Fund (CR&T), the Myeloproliferative Neoplasms Research Foundation (MPN-RF), the Clinical & Translational Science Center (CTSC) of Weill Cornell and the National Center for Advancing Translational Sciences (NCATS) (grant # 1 UL1 TR002384-04).
Data sharing statement
Data will not be publicly available due to HIPAA regulations. Similarly, individual patient data will not be shared. However, a request for sharing de-identified data can be made to the corresponding author and approval will be sought from the IRB.
Conflict of interest
GAZ – Consultancy, PharmaEssentia. SK, TC, GH, DJ, NS, CS, EKR, and JMS – No conflicts of interest to disclose. RTS – Speaker bureau and consultancy, PharmaEssentia.
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Presented in part at the annual meetings of the American Society of Hematology in Atlanta, GA, 2018; Orlando, FL, 2019, and San Diego, CA, 2020.
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Abu-Zeinah, G., Krichevsky, S., Cruz, T. et al. Interferon-alpha for treating polycythemia vera yields improved myelofibrosis-free and overall survival. Leukemia 35, 2592–2601 (2021). https://doi.org/10.1038/s41375-021-01183-8
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