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DICER1 germline pathogenic variants cause DICER1 tumor predisposition syndrome — broad sp...

Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.

IDBMA-DICER1-GERMLINE-PLEUROPULMONARY-BLASTOMA
TypeActionability
Statusreviewed 2026-05-18 | pending_clinical_signoff | actionability review required
DiseasesDIS-OVARIAN
SourcesSRC-NCCN-PEDIATRIC-SARCOMA

Actionability Facts

BiomarkerBIO-DICER1-GERMLINE
VariantDICER1 germline pathogenic (RNase IIIb domain hotspot or LoF)
DiseaseDIS-OVARIAN
ESCAT tierIIA
Recommended combinationsInternational PPB Registry consultation on first PPB suspicion (resection + adjuvant chemotherapy protocol), fertility-sparing oophorectomy for unilateral Sertoli-Leydig cell tumor in young women, thyroidectomy decision-making per multinodular goiter / nodule risk; total thyroidectomy with risk-reducing intent debated, lifestyle: smoking avoidance critical (synergistic lung-tumor risk in DICER1 carriers), DICER1 surveillance per International Consensus (Stewart et al., Clin Cancer Res 2018; AACR Pediatric Working Group 2017)
Evidence summaryDICER1 germline pathogenic variants cause DICER1 tumor predisposition syndrome — broad spectrum centered on pleuropulmonary blastoma (PPB) in early childhood, ovarian Sertoli-Leydig cell tumor in adolescence / young-adult females, multinodular goiter + differentiated thyroid cancer, cystic nephroma, embryonal rhabdomyosarcoma (cervix / bladder / kidney), pineoblastoma, and pituitary blastoma. Confirmed-carrier surveillance protocol (per International PPB Registry Consensus / Stewart et al. 2019 / DICER1 Cancer Predisposition Surveillance Statement): chest CT (low-dose) at age 3-6 months + 2.5 years + 8 years (PPB peak incidence < 6 years), chest X-ray q6mo to age 8 then annually to age 12, abdominal/pelvic US q6-12mo from infancy through age 8 then annual through adolescence, thyroid US q3y starting age 8, gynecologic exam + transabdominal pelvic US annual from age 8-10 (Sertoli-Leydig). Symptom-directed brain MRI for pineoblastoma / pituitary blastoma concern. Smoking avoidance is critical (synergistic lung-tumor risk). ESCAT IIA.

Notes

STUB — Wave A+B germline expansion. Linked Indication: none (no DICER1- specific surveillance Indication exists yet). Two-Clinical-Co-Lead signoff queued. Disease anchor uses DIS-OVARIAN (Sertoli-Leydig); PPB lacks a Disease entity. Variant biology: pathognomonic dual-hit pattern with germline LoF (truncating/splice) + somatic missense in RNase IIIb hotspot (codons E1705, D1709, G1809, D1810, E1813) inside tumor — tumor sequencing alone misses the germline allele.

Used By

No reverse references found in the YAML corpus.