Table 5.

A proposal for a protocol synopsis in advanced systemic mastocytosis with objectives reflecting tiered response criteria.

Primary Objective To determine the SM pathologic response rate (CR+PR) in patients with advSM

Secondary Objectives To determine the KIT D816V molecular response rate To determine the AHN pathologic response rate To determine the clinical response rate, measured by IWG clinical improvement (CI) To evaluate the EFS, PFS, LFS, and OS based on pre-treatment variables (advSM subtype; S/A/R status; prognostic score, e.g., IPSM, MARS, GPSM) and on-treatment variables such as SM pathologic response, molecular response, AHN pathologic response, and clinical response To evaluate time to initial SM pathologic response and duration of response To evaluate changes in the mutational profile with NGS compared to baseline and correlate with SM pathologic response, AHN pathologic response, EFS, PFS, LFS, and OS To evaluate changes in patient-reported symptoms using the AdvSM-SAF

SM: systemic mastocytosis; CR: complete remission; PR: partial remission; advSM: advanced systemic mastocytosis; AHN: associated hematologic neoplasm; IWG: International Working Group; EFS: event-free survival; PFS: progression-free survival; LFS: leukemia-free survival; OS: overall survival; S/A/R: SRSF2/ASXL1/RUNX1; IPSM: International Prognostic Score in Mastocytosis; MARS: Mutation-Adjusted Risk Score; GPSM: Global Prognostic Score in Mastocytosis; NGS: next generation sequencing; AdvSM-SAF: advanced systemic mastocytosis-symptom assessment form.