Cancer Medicine (Jan 2024)

The efficacy of immunotherapy and chemoimmunotherapy in patients with advanced rare tumors: A Turkish oncology group (TOG) study

  • Deniz Can Guven,
  • Musa Baris Aykan,
  • Harun Muglu,
  • Ertugrul Bayram,
  • Kaan Helvaci,
  • Bengü Dursun,
  • Melisa Celayir,
  • Elvin Chelebiyev,
  • Erdinc Nayir,
  • Mustafa Erman,
  • Ahmet Sezer,
  • Yuksel Urun,
  • Umut Demirci,
  • Ozlem Er,
  • Umut Disel,
  • Ahmet Bilici,
  • Cagatay Arslan,
  • Nuri Karadurmus,
  • Saadettin Kilickap

DOI
https://doi.org/10.1002/cam4.6869
Journal volume & issue
Vol. 13, no. 1
pp. n/a – n/a

Abstract

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Abstract Introduction The advances in immune checkpoint inhibitors (ICIs) were relatively slow in rare tumors. Therefore, we conducted a multi‐center study evaluating the efficacy of ICI monotherapy and the combination of ICIs with chemotherapy (CT) in patients with advanced rare tumors. Methods In this retrospective cohort study, we included 93 patients treated with ICIs for NCI‐defined rare tumors from the 12 cancer centers in Turkey. The primary endpoints were the overall response (ORR) and disease control rate (DCR). Results The cohort's median age was 56, and 53.8% of the patients were male. The most frequent diagnosis was sarcoma (29%), and 81.7% of the patients were previously treated with at least one line of systemic therapy in the advanced stage. The ORR and DCR were 36.8% and 63.2%, respectively. The germ cell tumors had the lowest ORR (0%), while the Merkel cell carcinoma had the highest ORR to ICIs (57.1%). Patients treated with ICI + ICI or ICI plus chemotherapy combinations had higher ORR (55.2% vs. 27.6%, p = 0.012) and DCR (82.8% vs. 53.4%, p = 0.008). The median OS was 13.47 (95% CI: 7.79–19.15) months, and the six and 12‐month survival rates were 71% and 52%. The median duration of response was 16.59 months, and the 12‐month progression‐free survival rate was 66% in responders. The median time‐to‐treatment failure was 5.06 months (95% CI: 3.42–6.71). Three patients had high‐grade irAEs with ICIs (grade 3 colitis, grade 3 gastritis, and grade 3 encephalitis in one patient each). Conclusion We observed over 30% ORR and a 13‐month median OS in patients with rare cancers treated with ICI monotherapy or ICI plus CT combinations. The response rates to ICIs or ICIs plus CT significantly varied across different tumor types. Responding patients had over 2 years of survival, highlighting a need for further trials with ICIs for patients with rare tumors.

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