Pediatric Medullary Thyroid Carcinoma: Clinical Presentations and Long-Term Outcomes in 144 Patients Over 6 Decades
Authors' conclusion
sMTC is primarily a RET-driven disease that represents 14% of childhood-onset MTC in this cohort. Pediatric sMTC patients are older, present with clinical disease at a more advanced TNM classification, and have more persistent disease at last follow-up compared with hMTC, but these differences disappear when comparing those presenting clinically. Somatic molecular testing should be considered in sMTC patients who would benefit from systemic therapy.
Study context
- Population
- Patients diagnosed with MTC at age ≤ 21 years between 1961 and 2019, evaluated at a tertiary referral center
- Participants
- 144 patients
- Exposure
- Sporadic MTC vs hereditary MTC
- Sporadic medullary thyroid carcinoma (sMTC)n = 20
- Hereditary MTC (hMTC)n = 124reference
- Outcome
- Clinical presentation and long-term outcomes, sMTC vs hMTC
Methodological detailssingle tertiary referral centre
- Single tertiary referral centre
Main finding
As reported in the abstract
sMTC vs hMTC: P < 0.0001 · sMTC vs hMTC: P < 0.0001 · sMTC vs hMTC: P = 0.001 · sMTC vs hMTC: P = 0.0045 · sMTC vs hMTC: P = 0.00084 · sMTC vs hMTC: P = 0.42
Observational data: associations, not effects.
Further findings
| Outcome | P value |
|---|---|
| Patients diagnosed clinically, not by family history (20/20 sMTC, 57/124 hMTC; n=77)sMTC vs hMTC · Initial stage: no difference | P = 0.27 |
| Patients diagnosed clinically, not by family history (20/20 sMTC, 57/124 hMTC; n=77)sMTC vs hMTC · DM at diagnosis: no difference | P = 1.0 |
| Patients diagnosed clinically, not by family history (20/20 sMTC, 57/124 hMTC; n=77)sMTC vs hMTC · Disease status at last follow-up: no difference | P = 0.13 |
| Patients diagnosed clinically, not by family history (20/20 sMTC, 57/124 hMTC; n=77)sMTC vs hMTC · Overall survival: no difference | P = 0.57 |
| Patients diagnosed clinically, not by family history (20/20 sMTC, 57/124 hMTC; n=77)sMTC vs hMTC · Disease-specific survival: no difference | P = 0.87 |
Somatic alterations in sMTC tumours tested (n=12)
- Identifiable alteration
- 11 (91%)
- RET gene alterations
- 10
- ALK fusion
- 1
Original publication
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