TY - JOUR
T1 - Phase 2 study of ruxolitinib and decitabine in patients with myeloproliferative neoplasm in accelerated and blast phase
AU - Mascarenhas, John O.
AU - Rampal, Raajit K.
AU - Kosiorek, Heidi E.
AU - Bhave, Rupali
AU - Hexner, Elizabeth
AU - Wang, Eunice S.
AU - Gerds, Aaron
AU - Abboud, Camille N.
AU - Kremyanskaya, Marina
AU - Berenzon, Dimitry
AU - Odenike, Olatoyosi
AU - Farnoud, Noushin
AU - Krishnan, Aishwarya
AU - Weinberg, Rona Singer
AU - McGovern, Erin
AU - Salama, Mohamed E.
AU - Najfeld, Vesna
AU - Medina-Martinez, Juan S.
AU - Arango Ossa, Juan E.
AU - Levine, Max F.
AU - Zhou, Yangyu
AU - Sandy, Lonette
AU - Heaney, Mark L.
AU - Levine, Ross L.
AU - Mesa, Ruben A.
AU - Dueck, Amylou C.
AU - Hoffman, Ronald
N1 - Publisher Copyright:
© 2020 by The American Society of Hematology
PY - 2020/10/26
Y1 - 2020/10/26
N2 - Myeloproliferative neoplasms (MPN) that have evolved into accelerated or blast phase disease (MPN-AP/BP) have poor outcomes with limited treatment options and therefore represent an urgent unmet need. We have previously demonstrated in a multicenter, phase 1 trial conducted through the Myeloproliferative Neoplasms Research Consortium that the combination of ruxolitinib and decitabine is safe and tolerable and is associated with a favorable overall survival (OS). In this phase 2 trial, 25 patients with MPN-AP/BP were treated at the recommended phase 2 dose of ruxolitinib 25 mg twice daily for the induction cycle followed by 10 mg twice daily for subsequent cycles in combination with decitabine 20 mg/m2 for 5 consecutive days in a 28-day cycle. Nineteen patients died during the study follow-up. The median OS for all patients on study was 9.5 months (95% confidence interval, 4.3-12.0). Overall response rate (complete remission 1 incomplete platelet recovery 1 partial remission) was 11/25 (44%) and response was not associated with improved survival. We conclude that the combination of decitabine and ruxolitinib was well tolerated, demonstrated favorable OS, and represents a therapeutic option for this high-risk patient population. This trial was registered at www.clinicaltrials.gov as #NCT02076191.
AB - Myeloproliferative neoplasms (MPN) that have evolved into accelerated or blast phase disease (MPN-AP/BP) have poor outcomes with limited treatment options and therefore represent an urgent unmet need. We have previously demonstrated in a multicenter, phase 1 trial conducted through the Myeloproliferative Neoplasms Research Consortium that the combination of ruxolitinib and decitabine is safe and tolerable and is associated with a favorable overall survival (OS). In this phase 2 trial, 25 patients with MPN-AP/BP were treated at the recommended phase 2 dose of ruxolitinib 25 mg twice daily for the induction cycle followed by 10 mg twice daily for subsequent cycles in combination with decitabine 20 mg/m2 for 5 consecutive days in a 28-day cycle. Nineteen patients died during the study follow-up. The median OS for all patients on study was 9.5 months (95% confidence interval, 4.3-12.0). Overall response rate (complete remission 1 incomplete platelet recovery 1 partial remission) was 11/25 (44%) and response was not associated with improved survival. We conclude that the combination of decitabine and ruxolitinib was well tolerated, demonstrated favorable OS, and represents a therapeutic option for this high-risk patient population. This trial was registered at www.clinicaltrials.gov as #NCT02076191.
UR - http://www.scopus.com/inward/record.url?scp=85096514426&partnerID=8YFLogxK
U2 - 10.1182/BLOODADVANCES.2020002119
DO - 10.1182/BLOODADVANCES.2020002119
M3 - Article
C2 - 33104796
AN - SCOPUS:85096514426
SN - 2473-9529
VL - 4
SP - 5246
EP - 5256
JO - Blood Advances
JF - Blood Advances
IS - 20
ER -