Table 2.

Trial outcomes

Trial cohort	Coprimary outcomes	Secondary outcomes
Untreated sAA (FIRST)	Primary efficacy endpoint is rate of CR at 6 months Primary safety endpoint is ACE at 6 months	Time to first haematological response (CR or PR), described by cumulative incidence curve Time to best haematological response, described by a cumulative incidence curve Time to CR, described by cumulative incidence curve Rates of haematological response (OR, CR and PR) at 6, 12, 18 and 24 months OS probability; OS is defined as time from day 1 of trial treatment to death, or last follow-up for patients alive EFS probability; EFS is defined as time from day 1 of trial treatment to either relapse, death, treatment failure or ACE (whichever occurs first), or last follow-up for patients alive in response QOL as measured by the EORTC QLQ-C30 questionaries at 6, 12, 18 and 24 months Cumulative incidence of PNH population occurrence and clinical haemolytic PNH occurrence Need for and number of transfusions (RBC and platelet units) Need for supportive care, including number and length of hospitalisations and ICU admissions Rate of ACE at 12, 18 and 24 months Rate of acquired somatic mutations detected on genomic testing at 6, 12, 18 and 24 months Safety and tolerability of the avatrombopag, including serious adverse events
Relapsed or refractory (NEXT)	Primary efficacy endpoint is rate of OR at 6 months. Primary safety endpoint is ACE at 6 months	Time to first haematological response (CR or PR), described by cumulative incidence curve Time to best haematological response, described by cumulative incidence curve Time to CR, described by cumulative incidence curve Rates of haematological response (OR, PR and CR) at 6, 12, 18 and 24 months OS probability; OS is defined as time from day 1 of trial treatment to death, or last follow-up for patients alive EFS probability; EFS is defined as time from day 1 of trial treatment to either relapse, death, treatment failure or ACE (whichever occurs first), or last follow-up for patients alive in response QOL as measured by the EORTC QLQ-C30 questionaries at 6, 12, 18 and 24 months Rate of occurrence of PNH clones and clinical PNH haemolysis Need for and number of transfusions (RBC and platelet units) Need for supportive care, including hospitalisation and ICU admission Safety and tolerability of the avatrombopag, including serious adverse events Rate of ACE (defined in section 24.4) at 6, 12, 18 and 24 months Rate of acquired somatic mutations detected on genomic testing at 6, 12, 18 and 24 months

ACE, acquired clonal evolution; CR, complete response; EFS, event free survival; ICU, intensive care unit; OR, overall response; OS, overall survival; PNH, paroxysmal nocturnal haemoglobinuria; PR, partial response; QOL, quality of life; RBC, red blood cell; sAA, severe aplastic anaemia.