Endocrine oncology

Outcome of immunotherapy in adrenocortical carcinoma: a retrospective cohort study

Eur J EndocrinolPublished Retrospective cohort study

Authors' conclusion

In the real-life setting, we observe a response comparable to other second-line therapies and an acceptable safety profile in ACC patients receiving different ICI. The relevance of PD-L1 as a marker of response and the potentially more favourable outcome in nivolumab-treated patients require confirmation.

Study context

Population
Patients with advanced adrenocortical carcinoma (ACC) receiving immune checkpoint inhibitors (ICI) as compassionate use at 6 German reference centres, 2016–202252 survived ≥4 weeks
Participants
54 patients
Outcome
Treatment response and survival with ICI
Methodological detailsmulticentre, real-life setting, compassionate use
  • Multicentre
  • Real-life setting
  • Compassionate use

Main finding

As reported in the abstract

Objective response rate (ORR) (patients surviving ≥4 weeks)

13.5%

6–26

Disease control rate (DCR) (patients surviving ≥4 weeks)

24%

16–41

Progression-free survival (PFS)

3.0 months

95% CI 2.3–3.7

Median overall survival (OS) (all patients)

10.4 months

3.8–17

Observational data: associations, not effects.

Further findings

OutcomeValuesEstimateP value
PFS by occurrence of TRAETRAEvs No TRAETRAE 5.5 (interval 1.9–9.2) months · No TRAE 2.5 (interval 2.0–3.0) monthsHR 0.29 (95% CI 0.13–0.66)P = 0.001
OS by occurrence of TRAETRAEvs No TRAETRAE 28.2 (interval 9.5–46.8) months · No TRAE 7.0 (interval 4.1–10.2) monthsHR 0.34 (95% CI 0.12–0.93)
Progression and death: nivolumab vs pembrolizumabNivolumabvs PembrolizumabAdjusted for concomitant mitotane useHR (progression) 0.36 (CI 0.15–0.90)
Progression and death: nivolumab vs pembrolizumabNivolumabvs PembrolizumabAdjusted for concomitant mitotane useHR (death) 0.20 (CI 0.06–0.72)
OutcomeValuesP value
PFS by PD-L1 tissue stainingPD-L1 positive vs PD-L1 negative · longer PFS with positive stainingPD-L1 positive 3.2 (interval 2.6–3.8) months · PD-L1 negative 2.3 (interval 1.6–3.0) monthsP < 0.05

Safety

Treatment-related adverse events (TRAE) occurred
17 TRAE in 15 patients

Original publication

Outcome of immunotherapy in adrenocortical carcinoma: a retrospective cohort study. Eur J Endocrinol. Published 1 June 2023. doi:10.1093/ejendo/lvad054

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