Adrenal disease

Efficacy and Safety of Tyrosine Kinase Inhibitors in Patients with Metastatic Pheochromocytomas/Paragangliomas

J Clin Endocrinol MetabPublished Systematic review and meta-analysis

Authors' conclusion

This meta-analysis suggests that patients with metastatic PPGLs can benefit from TKI therapy with PR and DCR up to more than 30% and 80%. However, because of restricted studies, larger clinical trials should be performed in the future.

Study context

Population
Patients with metastatic PPGLs
Participants
160 patients7 studies
Outcome
Tumor response or survival data and incidence of AEs after treatment
Methodological detailsfixed- or random-effect models, weighted median of medians method, Searched PubMed, Cochrane Library, Scopus, Clinical Trial and Embase to August 2022; risk of bias by MIONRS scale and JBI tools
  • Fixed- or random-effect models
  • Weighted median of medians method
  • Searched PubMed, Cochrane Library, Scopus, Clinical Trial and Embase to August 2022; risk of bias by MIONRS scale and JBI tools

Pooled result

As reported in the abstract

Tumour response (pooled proportions; 5 studies)

Pooled proportion of partial response (PR)

0.320

95% CI 0.155–0.486

Pooled proportion of stable disease

0.520

95% CI 0.409–0.630

Pooled disease control rate (DCR)

0.856

95% CI 0.734–0.979

Further pooled results

Progression-free survival (combined median; 6 studies)

Combined median progressive-free survival
8.9 months (95% CI 4.1-13.5)

Safety

Discontinuation due to adverse events (AEs) (5 studies)

Proportion discontinued due to AEs
0.143 (95% CI 0.077-0.209)

Original publication

Efficacy and Safety of Tyrosine Kinase Inhibitors in Patients with Metastatic Pheochromocytomas/Paragangliomas. J Clin Endocrinol Metab. Published 16 November 2022. doi:10.1210/clinem/dgac657

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