Investigational Approaches in Treatment of Nonadvanced Mastocytosis.

PURPOSE OF REVIEW: Recent strategies to treat nonadvanced SM have focused on targeted therapies to inhibit mast cell activation and/or proliferation. Several investigational approaches are being explored to treat nonadvanced SM in this targeted manner. RECENT FINDINGS: Investigational approaches to treat nonadvanced SM have included targeting KIT D816V mutant receptor, siglec-8, mTOR, interleukin-6, and Bruton tyrosine kinase. Many of these different approaches have demonstrated potential efficacy and therapeutic benefit for diminishing symptoms and mast cell burden. Clinical trials involving investigational treatments targeting KIT D816V mutant receptor are ongoing and have demonstrated the ability to improve symptoms, decrease mast cell burden, and improve patient quality of life. Other investigational approaches targeting siglec-8 and mTOR have demonstrated the potential to reduce symptoms in small patient cohorts. Results from targeted therapy with interleukin-6 did not support this approach being further pursued. Finally, a clinical trial targeting Bruton tyrosine kinase is also ongoing with no published data regarding efficacy or safety yet available.