Supplementary Material for: Sustained clinical response to ivosidenib in previously treated patients with advanced intrahepatic cholangiocarcinoma harboring an IDH1 R132 mutation: two case reports

Introduction. Patients with progressing intrahepatic cholangiocarcinoma (iCCA) harboring an isocitrate dehydrogenase 1 (IDH1) mutation who received ivosidenib showed a median progression-free survival benefit of 1.3 months compared to placebo in the Phase 3 ClarIDHy trial.
Case Presentations. We describe two consecutive patients with previously treated unresectable and metastatic iCCA harboring an IDH1 R132 mutation who achieved durable clinical responses with ivosidenib 500 mg once daily for >12 months until disease progression. In 1 case with a mixed response, a single progressive liver metastasis was additionally treated locally with interstitial brachytherapy while ivosidenib was continued until further progression. Ivosidenib therapy resulted in long-term disease control with progression-free survival of 20 and 13 months, respectively. The duration of treatment with ivosidenib was 26 and 13 months, respectively, with no relevant side effects.
Conclusion. Patients with unresectable or metastatic IDH1 mutated iCCA can achieve sustained clinical responses for >12 months with ivosidenib. No new safety signals were observed during long-term treatment with ivosidenib.