# Perivascular epithelioid cell tumour (PEComa)

Source: https://onco.cc/cancers/pecoma/  
OnCo record `pecoma` (Cancer). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

PEComa is a rare tumour, grouped with the sarcomas, of cells that sit around blood vessels and share features of muscle and pigment cells. Most are benign, but malignant ones spread and resist chemotherapy. They usually have lost the TSC1 or TSC2 brake on the growth signal mTOR, and in 2021 the mTOR blocker nab-sirolimus became the first approved treatment.

## Summary

Perivascular epithelioid cell tumours express both smooth muscle and melanocytic markers (HMB-45, Melan-A) and include renal angiomyolipoma, pulmonary lymphangioleiomyomatosis and clear cell sugar tumour of the lung as well as PEComa not otherwise specified of the uterus, retroperitoneum, gastrointestinal tract and soft tissue. Most carry biallelic loss of TSC1 or TSC2, with or without tuberous sclerosis complex, which unleashes mTOR signalling; a minority instead carry TFE3 fusions and do not respond to mTOR inhibition. Malignancy is predicted by size over five centimetres, infiltrative growth, high grade, necrosis, mitotic count and vascular invasion.

Complete surgical resection is the treatment for localised tumours, with no established role for adjuvant therapy, and surveillance for those with high-risk features. Conventional chemotherapy has little activity in malignant PEComa. Case series of sirolimus, everolimus and temsirolimus showed responses in TSC-altered tumours, establishing mTOR inhibition as the rational systemic therapy.

The single-arm phase 2 AMPECT trial tested albumin-bound sirolimus (nab-sirolimus) in advanced malignant PEComa and reported objective responses in around four in ten patients, with responses lasting years in some and higher response rates in TSC2-mutant tumours, leading to FDA approval in November 2021, the first drug approved for PEComa. The PRECISION 1 basket trial extends nab-sirolimus to any solid tumour with inactivating TSC1 or TSC2 alterations.

## Fields

- Kind: Cancer
- Last checked: 2026-09-17
- Also known as: PEComa; Malignant PEComa; Angiomyolipoma and lymphangioleiomyomatosis (PEComa family)
- Tags: subtype-page
- Group: sarcoma
- Burden: A very rare family of tumours, a few hundred malignant cases reported worldwide, arising in the uterus, retroperitoneum, gut and soft tissue of adults, more often women; most are benign, and malignant PEComa did not respond to chemotherapy until mTOR inhibitors.
- Subtypes: Uterine PEComa (commonest site of malignant PEComa); Retroperitoneal and abdominopelvic PEComa; Gastrointestinal PEComa; Soft tissue and cutaneous PEComa; TFE3-rearranged PEComa (younger patients; not TSC-driven); Angiomyolipoma and lymphangioleiomyomatosis (related, mostly benign)
- Biomarkers: TSC1 or TSC2 inactivation (mTOR inhibitor response); TFE3 fusion (excludes TSC pathway; poor mTOR response); HMB-45, Melan-A and smooth muscle actin co-expression; Size over 5 cm, mitoses, necrosis and infiltration (malignancy criteria)

## Sections of this record

The page is a hub with ten sections in reading order; large sections have their own page. The same plan as JSON: https://onco.cc/api/v1/cancers/pecoma/sections.json

- Overview (on the hub): The TL;DR, the family this cancer belongs to, the organ, who gets it and what the state of the art is. https://onco.cc/cancers/pecoma/#overview [3 state-of-the-art points]
- Types and stages (on the hub): Anatomy, the subtypes and how they differ, how it is staged, and where advanced disease spreads. https://onco.cc/cancers/pecoma/#what-it-is [6 subtypes]
- Symptoms and diagnosis (on the hub): How this cancer shows itself, how the diagnosis is confirmed, and the biomarkers clinicians test for. https://onco.cc/cancers/pecoma/#finding-it [4 biomarkers]
- Treatment (on the hub): The standard of care by setting, the medicines, surgery and radiotherapy named in it, and the regimens behind them. https://onco.cc/cancers/pecoma/#treating-it [3 settings, 3 decisions with options]
- Trials and papers (on the hub): Trials recruiting now, the landmark trials, the trials held by this cancer's subtypes, the key papers and what they mean, the latest literature, and the milestones year by year. https://onco.cc/cancers/pecoma/#evidence [2 trials, 3 key papers, 4 milestones]
- Biology and targets (on the hub): The molecular landscape: the targets and how often each appears, the pathways, the mechanics stages and the preclinical models. https://onco.cc/cancers/pecoma/#science [3 targets]
- Countries and centres (own page): Cases by country, the UK and NHS pathway and other country lenses, the expert centres with trials on record, and the centres named on this cancer's subtypes. https://onco.cc/cancers/pecoma/where-you-are/
- Decisions and support (on the hub): The decisions you may face, the aids that walk through them, the warnings on record, the first sixty days and the questions to ask. https://onco.cc/cancers/pecoma/#living-with-it [16 questions, 6 red cards]
- Pipeline and open problems (own page): Everything in development, the medicines held by this cancer's subtypes, the open problems and what is being done about them, the roadmaps, and what changed on this record. https://onco.cc/cancers/pecoma/coming/ [5 medicines, 2 trials, 3 open problems]
- Data (own page): Every connected record, the notes, the JSON, Markdown and RDF twins, and where the record came from and when it was checked. https://onco.cc/cancers/pecoma/data/ [25 connected records]

## Standard of care

- Localised: Complete resection; surveillance for tumours with malignant features; no proven adjuvant therapy. ([Limb-salvage surgery and endoprosthetic reconstruction](https://onco.cc/technologies/limb-salvage-surgery/), [Active surveillance](https://onco.cc/technologies/active-surveillance/))
- Advanced malignant PEComa: Nab-sirolimus (AMPECT; FDA approved 2021); oral sirolimus, everolimus or temsirolimus as alternatives; check TSC status. ([Sirolimus protein-bound particles](https://onco.cc/drugs/sirolimus-albumin-bound/), [AMPECT](https://onco.cc/trials/ampect/), [Everolimus](https://onco.cc/drugs/everolimus/), [Temsirolimus](https://onco.cc/drugs/temsirolimus/), [mTOR](https://onco.cc/targets/mtor/))
- After mTOR inhibitor: Anthracycline- or gemcitabine-based chemotherapy has modest activity; trials of mTOR-based combinations; PRECISION 1 for TSC-altered tumours. ([Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Gemcitabine](https://onco.cc/drugs/gemcitabine/), [Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)](https://onco.cc/trials/nct05103358/))

## State of the art

- Nab-sirolimus is the first approved drug for PEComa, with durable responses in TSC2-mutant tumours.
- TSC1/TSC2 loss defines a targetable pathway across the PEComa family and beyond.
- TFE3-rearranged tumours form a distinct group that needs different treatment.

## Open problems

- Malignancy cannot always be predicted from histology.
- Resistance to mTOR inhibition eventually develops.
- TFE3-rearranged PEComa lacks an effective drug.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Perivascular_epithelioid_cell_tumour
- Wikipedia: https://en.wikipedia.org/wiki/Perivascular_epithelioid_cell_tumour

## Connected records

- cancers: [Alveolar soft part sarcoma](https://onco.cc/cancers/alveolar-soft-part-sarcoma/), [Epithelioid sarcoma](https://onco.cc/cancers/epithelioid-sarcoma/), [Inflammatory myofibroblastic tumour (IMT)](https://onco.cc/cancers/inflammatory-myofibroblastic-tumour/), [Retroperitoneal sarcoma](https://onco.cc/cancers/retroperitoneal-sarcoma/), [Sarcomas (soft tissue, bone, GIST)](https://onco.cc/cancers/sarcoma/), [Tenosynovial giant cell tumour (TGCT)](https://onco.cc/cancers/tenosynovial-giant-cell-tumour/), [Uterine sarcoma](https://onco.cc/cancers/uterine-sarcoma/)
- key papers: [AMPECT: nab-sirolimus for malignant perivascular epithelioid cell tumours](https://onco.cc/key-papers/paper-ampect-nab-sirolimus-pecoma-wagner-jco-2021/), [Clinical activity of mTOR inhibition with sirolimus in malignant perivascular epithelioid cell tumours](https://onco.cc/key-papers/paper-wagner-sirolimus-pecoma-jco-2010/), [Soft tissue and visceral sarcomas: ESMO-EURACAN-GENTURIS clinical practice guideline](https://onco.cc/key-papers/paper-esmo-sts-guideline-gronchi-ann-oncol-2021/)
- targets: [mTOR](https://onco.cc/targets/mtor/)
- trials: [AMPECT](https://onco.cc/trials/ampect/), [Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)](https://onco.cc/trials/nct05103358/)
- drugs: [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Everolimus](https://onco.cc/drugs/everolimus/), [Gemcitabine](https://onco.cc/drugs/gemcitabine/), [Sirolimus protein-bound particles](https://onco.cc/drugs/sirolimus-albumin-bound/), [Temsirolimus](https://onco.cc/drugs/temsirolimus/)
- technologies: [Active surveillance](https://onco.cc/technologies/active-surveillance/), [Limb-salvage surgery and endoprosthetic reconstruction](https://onco.cc/technologies/limb-salvage-surgery/)
- people: [Andrew J. Wagner](https://onco.cc/people/andrew-wagner/)

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JSON: https://onco.cc/api/v1/entities/pecoma.json