A randomized phase 3 trial of interferon-α vs hydroxyurea in polycythemia vera and essential thrombocythemia.
Author
Mascarenhas, JohnKosiorek, Heidi E
Prchal, Josef T
Rambaldi, Alessandro
Berenzon, Dmitriy
Yacoub, Abdulraheem
Harrison, Claire N
McMullin, Mary Frances
Vannucchi, Alessandro M
Ewing, Joanne
O'Connell, Casey L
Kiladjian, Jean-Jacques
Mead, Adam J
Winton, Elliott F
Leibowitz, David S
De Stefano, Valerio
Arcasoy, Murat O
Kessler, Craig M
Catchatourian, Rosalind
Rondelli, Damiano
Silver, Richard T
Bacigalupo, Andrea
Nagler, Arnon
Kremyanskaya, Marina
Levine, Max F
Arango Ossa, Juan E
McGovern, Erin
Sandy, Lonette
Salama, Mohamad E
Najfeld, Vesna
Tripodi, Joseph
Farnoud, Noushin
Penson, Alexander V
Weinberg, Rona Singer
Price, Leah
Goldberg, Judith D
Barbui, Tiziano
Marchioli, Roberto
Tognoni, Gianni
Rampal, Raajit K
Mesa, Ruben A
Dueck, Amylou C
Hoffman, Ronald
Publication date
2022-05-12Subject
Oncology
Metadata
Show full item recordAbstract
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.Citation
Mascarenhas J, Kosiorek HE, Prchal JT, Rambaldi A, Berenzon D, Yacoub A, Harrison CN, McMullin MF, Vannucchi AM, Ewing J, O'Connell CL, Kiladjian JJ, 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. doi: 10.1182/blood.2021012743Type
ArticleAdditional Links
https://ashpublications.org/bloodPMID
35007321Journal
BloodPublisher
Elsevierae974a485f413a2113503eed53cd6c53
10.1182/blood.2021012743