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. 2019 Aug 27;10:291–300. doi: 10.2147/JBM.S183997

Table 1.

Overview of clinical studies evaluating ibrutinib-based regimens among patients with WM

Study Phase Number of patients Design ORR Major RR PFS OS Toxicities
Advani (2013) I 4

  • Prospective

  • Relapsed/refractory patients received PO ibrutinib

 3 of 4 patients achieved a response Not reported Not reported Not reported Grades 3–4: neutropenia 12.5%, thrombocytopenia 7.2%, anemia 7.1%
Treon (2015) II 63

  • Prospective

  • Previously treated patients received PO ibrutinib

 90.5% 73% 2-year PFS rate: 69.1% 2-year OS rate: 95.2% Grades 2–4: neutropenia 22%, thrombocytopenia 14%, anemia 6%, AF 5%, pneumonia 8%, hemorrhage 2%
Dimopoulos (2016) III 31

  • Open-label

  • Rituximab-refractory patients received per os (PO) ibrutinib

 90% 71% 18-month PFS rate: 86% 18-month OS rate: 97% Grade 3 or worse: neutropenia 13%, thrombocytopenia 6%, anemia 6%, hypertension 10%, infections 16%
Dimopoulos (2018) III 150

  • Randomized-controlled trial

  • 1:1 randomized to receive rituximab+ibrutinib OR rituximab+placebo

 Significantly higher in the ibrutinib arm compared to the placebo arm (92% vs 47%) Significantly higher in the ibrutinib arm compared to the placebo arm (72% vs 32%) 30-month PFS rate: 82% (ibrutinib–rituximab) vs 28% (placebo–rituximab) 30-month OS rate: 94% (ibrutinib–rituximab) vs 92% (placebo–rituximab) Grade 3 or 4 in the ibrutinib vs placebo arm: hypertension 13% vs 4%, AF 12% vs 1%, anemia 11% vs 17%, neutropenia9 % vs 3%, pneumonia 9% vs 3%, IRR 1% vs 16%
Treon (2018) 2 30

  • Prospective

  • Untreated patients received PO ibrutinib

  • The effect of CXCR4 status on outcome was investigated

  • 47% carried CXCR4 mutation

 100% for all patients 83% for all patients/94% in WT CXCR4/71% in CXCR4 mutated 18-month PFS rate: 92% 18-month OS rate: 100% Grades 2–3 (No grade 4 toxicities): hypertension 13%, AF 10%, neutropenia 7%, upper respiratory tract infection 7%, urinary tract infection 7%

Abbreviations: ORR, overall response rate; Major RR, major response rate; PFS, progression-free survival; OS, overall survival; AF, atrial fibrillation; WM, Waldenstrom’s macroglobulinemia.