Sunitinib for metastatic progressive phaeochromocytomas and paragangliomas: results from FIRSTMAPPP, an academic, multicentre, international, randomised, placebo-controlled, double-blind, phase 2 trial
Authors' conclusion
This first randomised trial supports the use of sunitinib as the medical option with the highest level of evidence for anti-tumour efficacy in progressive metastatic phaeochromocytomas and paragangliomas.
Study context
- Population
- Adults (≥18 years) with sporadic or inherited progressive metastatic phaeochromocytomas and paragangliomas, 14 academic centres in four European countries; 32% germline SDHx variants, 69% previous therapiesrandomised 1:1
- Participants
- 78 adults
- Comparison
- Oral sunitinib vs placebo
- Sunitinibn = 39
- Placebon = 39reference
- Primary endpoint
- Rate of progression-free survival by real-time central review (RECIST 1.1)
- Follow-up
- 12 months
Methodological detailsmulticentre, international, placebo-controlled, double-blind, phase 2, stratified by SDHB status and number of previous systemic therapies, crossover from placebo allowed
- Multicentre
- International
- Placebo-controlled
- Double-blind
- Phase 2
- Stratified by SDHB status and number of previous systemic therapies
- Crossover from placebo allowed
Primary result
As reported in the abstract
Progression-free survival
Further findings
Events
| Outcome | Sunitinib | Placebo |
|---|---|---|
| Deaths | 3 | 2 |
Deaths
- Sunitinib deaths due to respiratory insufficiency, amyotrophic lateral sclerosis and rectal bleeding; only rectal bleeding considered drug related
- Placebo deaths due to aspiration pneumonia and septic shock
Safety
Grade 3 or 4 adverse events
- Asthenia: Sunitinib vs Placebo
- 7 [18%] vs 1 [3%]
- Hypertension: Sunitinib vs Placebo
- 5 [13%] vs 4 [10%]
- Back or bone pain: Sunitinib vs Placebo
- 1 [3%] vs 3 [8%]
Original publication
Sunitinib for metastatic progressive phaeochromocytomas and paragangliomas: results from FIRSTMAPPP, an academic, multicentre, international, randomised, placebo-controlled, double-blind, phase 2 trial. Lancet. Published 22 February 2024. doi:10.
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