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. Author manuscript; available in PMC: 2022 Dec 12.
Published in final edited form as: Ann Oncol. 2019 Dec 9;31(3):412–421. doi: 10.1016/j.annonc.2019.11.018

Table 2.

Clinical outcomes by patient

Pt Alteration Time on treatment, months Best response Duration of response, months Duration of SD, months Best change in target lesion size from baseline, % PFS, months OS, months

HER2 amplification and/or overexpression: treated with pertuzumab + trastuzumab
 1 HER2 amplification 16.5+ CR 15.2+ −91.7a 16.5+ 16.5+
 2 HER2 overexpression 26.1+ PR 19.7+ −33.3 25.2+ 26.1+
 3 HER2 amplification and overexpression 12.6 PR 9.2 −62.5 13.4 20.4
 4 HER2 amplification and overexpression 8.3 PR 7.3 −100.0b 8.6 14.9+
 5 HER2 amplification and overexpression 10.6+ PR 7.2+ −66.7 8.5+ 10.6+
 6 HER2 amplification, overexpression, and mutation (L755F and D769H) 19.8 PR 4.2 −85.7 5.6 21.2
 7 HER2 amplification and overexpression 4.1+ PR 2.8+ −73.0 4.0+ 4.1+
 8 HER2 amplification and overexpression 4.1c PR 2.7 −68.2 9.1 9.1
 9 HER2 amplification and mutation (G776V) 3.5+ PR 1.4+ −55.7 2.8+ 3.5+
 10 HER2 amplification 11.2 SD 11.7 −27.9 11.7 14.0+
 11 HER2 amplification 3.5 SD 3.9 −25.6 3.9 10.4
 12 HER2 overexpression 2.9+ SD 2.9+ −24.3 2.9+ 2.9+
 13 HER2 amplification and overexpression 2.1 SD 2.3 1.4 2.3 8.2
 14 HER2 amplification and overexpression 0.7 PD 3.6 1.5 8.3
 15 HER2 amplification 0.7 PD 22.5 1.4 3.1
HER2 mutation: treated with pertuzumab + trastuzumab
 16 HER2 mutation (S310F) 10.4 SD 11.0 −12.8 11.0 13.7+
Hh alteration: treated with vismodegib
 17 Hh alteration (PTCH-1 Q400*) 14.4 PR 10.7 −55.6 14.3 17.3+
BRAF V600 mutation: treated with vemurafenib
 18 BRAF mutation (V600E) 16.8 PR 15.1 −43.4 18.5 20.1
High TMB: treated with atezolizumab
 19 High TMB (31 mutations/Mb) 5.7+ PR 0.03+ −56.0 5.5+ 5.7+

CR, complete response; HER2, human epidermal growth factor receptor 2; Hh, Hedgehog; OS, overall survival; PD, progressive disease; PFS, progression-free survival; PR, partial response; Pt, patient; PTCH-1, patched homolog-1; SD, stable disease; TMB, tumor mutational burden.

+

indicates measure was ongoing at the most recent assessment.

a

Patient’s target lesions were both lymph nodes with a measurement of <10 mm and absent/normal status of non-target lesions following treatment cycle 12, considered a CR per RECIST v1.1.

b

Patient had a 100% reduction in the target lesion but had a remaining non-target lesion and therefore did not qualify for a CR per RECIST v1.1.

c

Patient had an ongoing tumor response but withdrew from treatment and later died.