Responses to systemic therapy in metastatic pheochromocytoma/paraganglioma: a retrospective multicenter cohort study
Authors' conclusion
We demonstrate in a real-life clinical setting that all current therapies show reasonable efficacy in preventing disease progression, and this is equally true for patients with germline SDHB mutations.
Study context
- Population
- Patients with metastatic pheochromocytoma/paraganglioma treated at 6 specialized national centers, selected from the ENSAT registry
- Participants
- 74 patients
- Exposure
- Systemic therapies in mPPGL
- CVDn = 4
- PPRTn = 21
- 131I-MIBGn = 23
- Sunitinibn = 7
- Somatostatin analogsn = 4
- Temozolomiden = 5
- Outcome
- Survival until detected progression (SDP) and disease-control rates (DCRs), based on imaging reports
- Follow-up
- DCR at 3 months
Methodological detailsmulticentre
- Multicentre
Main finding
As reported in the abstract
DCR in patients with progressive disease at baseline (83.8%) · 3 months
| Group | Value | SDP |
|---|---|---|
| CVD | 75.0% | 11 months |
| PPRT | 85.7% | 17 months |
| 131I-MIBG | 82.6% | 43 months |
| Sunitinib | 100% | 18 months |
| Somatostatin analogs | 100% | not reached |
| Temozolomide | 60.0% | 10 months |
Value
CVD75.0%
PPRT85.7%
131I-MIBG82.6%
Sunitinib100%
Somatostatin analogs100%
Temozolomide60.0%
SDP
CVD11 months
PPRT17 months
131I-MIBG43 months
Sunitinib18 months
Somatostatin analogsnot reached
Temozolomide10 months
Observational data: associations, not effects.
Further findings
| Outcome | Values |
|---|---|
| DCR in SDHB pathogenic variant subgroup · 3 months | CVD 100% · n = 1 · SDP 30 months · PPRT 100% · n = 10 · SDP 14 months · 131I-MIBG 100% · n = 4 · SDP 24 months · Sunitinib 100% · n = 3 · SDP 18 months · Temozolomide 75% · n = 4 · SDP 10 months |
Original publication
Responses to systemic therapy in metastatic pheochromocytoma/paraganglioma: a retrospective multicenter cohort study. Eur J Endocrinol. Published 9 November 2023. doi:10.
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