Citations 12
Ixazomib significantly prolongs progression-free survival in high-risk relapsed/refractory myeloma patients
Avet‐Loiseau, Hervé , Masszi, Tamás , Viterbo, Luísa, Pour, Luděk, Palumbo, Antonio , Cavo, Michèle , Langer, Christian , Pluta, Andrzej, Nagler, Arnon , Kumar, Shaji , Ben‐Yehuda, Dina, Miguel, Jesús F. San , Berg, Deborah, Lin, Jianchang , Velde, Helgi van de , Esseltine, Dixie‐Lee, Bacco, Alessandra Di , Moreau, Philippe , Richardson, Paul G. , Moreau, Philippe , San-Miguel, Jesus , Masszi, Tamas , Bahlis, Nizar Jacques , Chng, Wee Joo , Ganly, Peter S. , Rajkumar, S. Vincent , Esseltine, Dixie‐Lee, Richardson, Paul Gerard Guy
Blood | October 20, 2017 | Volume: 130 | Issue: 24 | Pages: 2610-2618
Key Points IRd was associated with a consistent PFS benefit vs placebo-Rd in RRMM patients with high-risk and standard-risk cytogenetics. The addition of ixazomib to Rd overcomes the poor PFS associated with high-risk cytogenetics in patients with RRMM.
Results of the PERSIST-1 phase III study of pacritinib (PAC) versus best available therapy (BAT) in primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia-myelofibrosis (PET-MF).
Egyed, Miklós , Szőke, Anita , Suvorov, Aleksandr , Perkins, Andrew C. , Mayer, Jiřı́ , Jourdan, Éric , Schouten, Harry C. , Tosi, Patrizia , Cernohous, Paul, Nangalia, Jyoti , Harrison, Claire , Mesa, Ruben A. , Ganly, Peter S. , Farber, Charles Michael , Zachée, Pierre , Scheid, Christof , Dean, James P. , Kiladjian, Jean‐Jacques , Vannucchi, Alessandro M. , Mead, Adam J. , Perkins, Andrew C
Journal of Clinical Oncology | February 24, 2017 | Volume: 33 | Issue: 18_suppl | Pages: LBA7006-LBA7006
10.1200/jco.2015.33.18_suppl.lba7006
LBA7006 Background: PAC is a potent JAK2 inhibitor without significant JAK1 inhibition with minimal myelosuppression in early-phase studies in MF. Methods: The efficacy and safety of daily oral PAC was compared to BAT (2:1 randomization stratified for risk and platelet count). The 10 endpoint was the proportion of ITT patients (pts) achieving ≥ 35% spleen volume reduction (SVR) at week (wk) 24 by centrally reviewed MRI or CT. Secondary endpoints included the proportion achieving ≥ 50% reduction in total symptom score (TSS) at wk 24 using the MPN Symptom Assessment Form. Results: Patients:327 were enrolled (PAC:220, BAT:107), 62% with 10 MF. Median time from diagnosis was 1.12 years (PAC 0.99, BAT 1.60): 32% and 15% had a platelet counts < 100,000/µL or <50,000/ µL; 75% were JAK2V617F positive. Efficacy: The median duration of treatment was 16.2 months PAC and 5.9 months BAT. Sixty-two percent of BAT patients received active disease directed therapy. The SVR rates at week 24 were 19.1% for PAC vs. 4.7% for BAT (p=0.0003) in ITT and 25% vs. 5.9% (p=0.0001) in the evaluable population. 79% of BAT patients crossed over to PAC; 21% had achieved a >35% reduction in spleen volume at data cutoff. TSS composite V1 + V2 response rates were 24.5%for PAC vs. 6.5% for BAT (p<0.0001) by ITT, and were 40.9% vs. 9.9% in evaluable pts (p<0.0001). Efficacy with baseline cytopenias: In pts with <100,000 and <50,000 platelets/μ/L, the SVR rates were 16.7% for PAC vs. 0% for BAT (p=0.009), and 22.9% vs. 0% (p=0.045) by ITT and 23.5% vs. 0% (p=0.007) and 33.3% vs. 0% (p=0.037) in evaluable pts. In RBC transfusion dependent pts, 25.7% of PAC pts became RBC independent vs. 0% of BAT pts (p=0.043). Safety: The most common adverse events (AE) for PAC were diarrhea, nausea, and vomiting; (grade 3 were <5%, <1%, <1% respectively). Hematologic AEs were similar between PAC and BAT. Conclusions: This study demonstrated PAC was well tolerated and induced significant and sustained SVR and symptom control even in patients with severe thrombocytopenia. PAC therapy resulted in RBC transfusion independence in a significant proportion of pts. Clinical trial information: NCT01773187.