Skip to main content
construction release_alert
Scholars@Duke will be undergoing maintenance April 11-15. Some features may be unavailable during this time.
cancel

A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia.

Publication ,  Journal Article
Mascarenhas, J; Kosiorek, HE; Prchal, JT; Rambaldi, A; Berenzon, D; Yacoub, A; Harrison, CN; McMullin, MF; Vannucchi, AM; Ewing, J; Mead, AJ ...
Published in: Blood
May 12, 2022

The goal of therapy for patients with essential thrombocythemia (ET) and polycythemia vera (PV) is to reduce thrombotic events by normalizing blood counts. Hydroxyurea (HU) and interferon-α (IFN-α) are the most frequently used cytoreductive options for patients with ET and PV at high risk for vascular complications. Myeloproliferative Disorders Research Consortium 112 was an investigator-initiated, phase 3 trial comparing HU to pegylated IFN-α (PEG) in treatment-naïve, high-risk patients with ET/PV. The primary endpoint was complete response (CR) rate at 12 months. A total of 168 patients were treated for a median of 81.0 weeks. CR for HU was 37% and 35% for PEG (P = .80) at 12 months. At 24 to 36 months, CR was 20% to 17% for HU and 29% to 33% for PEG. PEG led to a greater reduction in JAK2V617F at 24 months, but histopathologic responses were more frequent with HU. Thrombotic events and disease progression were infrequent in both arms, whereas grade 3/4 adverse events were more frequent with PEG (46% vs 28%). At 12 months of treatment, there was no significant difference in CR rates between HU and PEG. This study indicates that PEG and HU are both effective treatments for PV and ET. With longer treatment, PEG was more effective in normalizing blood counts and reducing driver mutation burden, whereas HU produced more histopathologic responses. Despite these differences, both agents did not differ in limiting thrombotic events and disease progression in high-risk patients with ET/PV. This trial was registered at www.clinicaltrials.gov as #NCT01259856.

Duke Scholars

Altmetric Attention Stats
Dimensions Citation Stats

Published In

Blood

DOI

EISSN

1528-0020

Publication Date

May 12, 2022

Volume

139

Issue

19

Start / End Page

2931 / 2941

Location

United States

Related Subject Headings

  • Thrombosis
  • Thrombocythemia, Essential
  • Polycythemia Vera
  • Interferon-alpha
  • Immunology
  • Hydroxyurea
  • Humans
  • Disease Progression
  • 3213 Paediatrics
  • 3201 Cardiovascular medicine and haematology
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Mascarenhas, J., Kosiorek, H. E., Prchal, J. T., Rambaldi, A., Berenzon, D., Yacoub, A., … Hoffman, R. (2022). A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia. Blood, 139(19), 2931–2941. https://doi.org/10.1182/blood.2021012743
Mascarenhas, John, Heidi E. Kosiorek, Josef T. Prchal, Alessandro Rambaldi, Dmitriy Berenzon, Abdulraheem Yacoub, Claire N. Harrison, et al. “A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia.Blood 139, no. 19 (May 12, 2022): 2931–41. https://doi.org/10.1182/blood.2021012743.
Mascarenhas J, Kosiorek HE, Prchal JT, Rambaldi A, Berenzon D, Yacoub A, et al. A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia. Blood. 2022 May 12;139(19):2931–41.
Mascarenhas, John, et al. “A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia.Blood, vol. 139, no. 19, May 2022, pp. 2931–41. Pubmed, doi:10.1182/blood.2021012743.
Mascarenhas J, Kosiorek HE, Prchal JT, Rambaldi A, Berenzon D, Yacoub A, Harrison CN, McMullin MF, Vannucchi AM, Ewing J, O’Connell CL, Kiladjian J-J, Mead AJ, Winton EF, Leibowitz DS, De Stefano V, Arcasoy MO, Kessler CM, Catchatourian R, Rondelli D, Silver RT, Bacigalupo A, Nagler A, Kremyanskaya M, Levine MF, Arango Ossa JE, McGovern E, Sandy L, Salama ME, Najfeld V, Tripodi J, Farnoud N, Penson AV, Weinberg RS, Price L, Goldberg JD, Barbui T, Marchioli R, Tognoni G, Rampal RK, Mesa RA, Dueck AC, Hoffman R. A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia. Blood. 2022 May 12;139(19):2931–2941.

Published In

Blood

DOI

EISSN

1528-0020

Publication Date

May 12, 2022

Volume

139

Issue

19

Start / End Page

2931 / 2941

Location

United States

Related Subject Headings

  • Thrombosis
  • Thrombocythemia, Essential
  • Polycythemia Vera
  • Interferon-alpha
  • Immunology
  • Hydroxyurea
  • Humans
  • Disease Progression
  • 3213 Paediatrics
  • 3201 Cardiovascular medicine and haematology