Table 2 Response rates by adjudicated mIWG–MRT–ECNM response criteria

From: Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial

Best confirmed response, n (%)

All AdvSM

(n = 32)

AdvSM subtype

Previous therapy

Previous midostaurin

ASM

(n = 2)

SM-AHN

(n = 26)

MCL

(n = 4)

Yes

(n = 23)

No

(n = 9)

Yes

(n = 17)

No

(n = 15)

ORR (CR + CRh + PR + Cl)

24 (75)

2 (100)

21 (81)

1 (25)

17 (74)

7 (78)

14 (82)

10 (67)

95% Confidence interval

57–89

16–100

61–93

1–81

52–90

40–97

57–96

38–88

Best response

 CR or CRh

6 (19)

1 (50)

5 (19)

0

3 (13)

3 (33)

3 (18)

3 (20)

 CR

0

0

0

0

0

0

0

0

 CRh

6 (19)

1 (50)

5 (19)

0

3 (13)

3 (33)

3 (18)

3 (20)

 PR

10 (31)

1 (50)

8 (31)

1 (25)

7 (30)

3 (33)

5 (29)

5 (33)

 CI

8 (25)

0

8 (31)

0

7 (30)

1 (11)

6 (35)

2 (13)

Stable disease

4 (13)

0

2 (8)

2 (50)

2 (9)

2 (22)

0

4 (27)

Progressive disease

1 (3)

0

0

1 (25)

1 (4)

0

0

1 (7)

Not evaluable

3 (9)a

0

3 (12)

0

3 (13)

0

3 (18)

0

  1. aThree (9%) patients were in the interim analysis efficacy population but were assessed as not evaluable for response due to coming off trial before a confirmed response could be determined (13 weeks).