Table 5. Characteristics and outcomes of phase III clinical trials using BTK inhibitors in first-line settings in WM patients. ORR, objective response rate; MRR, major response rate; VGPR, very good partial response; CR, complete response; PFS, progression-free survival; OS, overall response; R/R, relapsed/refractory; TN, treatment naïve; WT, wildtype; WM, Waldenström’s macroglobulinemia. ^aGenotype for this study was for the entire cohort (TN + R/R). Adapted from Abneh et al. 2022.[52]

Study ID	Intervention	Participants, n (%)	Mutational Status	Outcomes
ASPEN (Tam 2020)[51]	Ibrutinib	18 (18.2)	MYD88L265P/CXCR4WT: 17 MYD88L265P/CXCR4UNK: 1	ORR: 89% MRR: 67% VGPR/CR: 17% 18-mo PFS: 94%
ASPEN (Tam 2020)[51]	Zanubrutinib	19 (18.6)	MYD88L265P/CXCR4WT: 18 MYD88L265P/CXCR4WHIM: 1	ORR: 95% MRR: 74% VGPR/CR: 26% 18-mo PFS: 78%
iNNOVATE (Buske 2022)[47]	Ibrutinib + Rituximab	34 (45.3)	^aMYD88L265P/CXCR4WT: 32 MYD88L265P/CXCR4WHIM: 26 MYD88WT/CXCR4WT: 11	ORR: 91% MRR: 76% VGPE/CR: 27%
ASPEN substudy (Dimopoulos 2020)[53]	Zanubrutinib	(17.9)	MYD88WT/CXCR4WT: 5	ORR: 80%

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