BTK (Bruton's Tyrosine Kinase)

Key signaling kinase downstream of MYD88

Expression
Constitutively activated
Evidence level
established
Targeted by
Ibrutinib, Zanubrutinib, Pirtobrutinib

Role in pathogenesis

BTK is a non-receptor tyrosine kinase recruited to the Myddosome complex by mutated MYD88. It activates NF-kB pro-survival signaling independently of IRAK4/IRAK1. BTK also participates in B-cell receptor signaling. It is the direct target of ibrutinib and zanubrutinib, the most effective WM therapies.

Targeting drugs (3)

DrugMechanismResponseLine
ZanubrutinibSecond-generation selective BTK inhibitor~95% ORR; 36% VGPR+CR1st
IbrutinibFirst-generation BTK inhibitor90% ORR; 73% MRR (previously treated)1st
PirtobrutinibNon-covalent (reversible) BTK inhibitor~68% ORR (after covalent BTKi); 56% VGPR with venetoclax combo2nd

Sources (4)

DetailsTreon SP et al. (2015) Ibrutinib in Previously Treated Waldenström's Macroglobulinemia · N Engl J MedDOI
DetailsTam CS et al. (2020) A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic WM: the ASPEN study · BloodDOI
DetailsBibas M et al. (2024) WM: State-of-the-Art Review Part 1 - Epidemiology, Pathogenesis, Clinical · Mediterr J Hematol Infect DisDOI
DetailsTreon SP et al. (2024) How I use genomics and BTK inhibitors in the treatment of WM · BloodDOI