The first 60 days: Perivascular epithelioid cell tumour (PEComa)
PEComa is a rare tumour 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. Below, week by week, is what OnCo's record of Perivascular epithelioid cell tumour (PEComa) says about the first two months: the order is typical, the timing is yours to ask about. Sections appear only where the record has something to say. Orientation, not medical advice.
What happens now
Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.
Ask which of these were tested and what the results were; see the report reader for what each value means.
These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.
- PathologistNamed in the standard of care for: Localised.
- SurgeonNamed in the standard of care for: Localised.
- Medical oncologistNamed in the standard of care for: Localised, Advanced malignant PEComa, After mTOR inhibitor.
- Palliative and supportive care teamNamed in the standard of care for: After mTOR inhibitor.
A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.
Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.
Complete resection; surveillance for tumours with malignant features; no proven adjuvant therapy.
Nab-sirolimus (AMPECT; FDA approved 2021); oral sirolimus, everolimus or temsirolimus as alternatives; check TSC status.
Anthracycline- or gemcitabine-based chemotherapy has modest activity; trials of mTOR-based combinations; PRECISION 1 for TSC-altered tumours.
Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example TSC1 or TSC2 inactivation, TFE3 fusion, HMB-45, Melan-A and smooth muscle actin co-expression, Size over 5 cm, mitoses, necrosis and infiltration), and what were the results?These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Recognised subtypes for this cancer include Uterine PEComa, Retroperitoneal and abdominopelvic PEComa, Gastrointestinal PEComa.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Localised
- For my situation (localised), which of the standard options do you recommend and why?Guideline options include: Complete resection; surveillance for tumours with malignant features; no proven adjuvant therapy.
Advanced malignant PEComa
- For my situation (advanced malignant pecoma), which of the standard options do you recommend and why?Guideline options include: Nab-sirolimus (AMPECT; FDA approved 2021); oral sirolimus, everolimus or temsirolimus as alternatives; check TSC status.
- Am I a candidate for Sirolimus protein-bound particles, Everolimus, Temsirolimus, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of AMPECT apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
After mTOR inhibitor
- For my situation (after mtor inhibitor), which of the standard options do you recommend and why?Guideline options include: Anthracycline- or gemcitabine-based chemotherapy has modest activity; trials of mTOR-based combinations; PRECISION 1 for TSC-altered tumours.
- Am I a candidate for Doxorubicin, Gemcitabine, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
- How do the results of Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1) apply to someone like me?Trial populations differ from individual patients; ask how closely you match.
Any stage
- Are there clinical trials I could join, for example of Sirolimus protein-bound particles, Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1), AMPECT?Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Supportive care improves quality of life and helps patients complete treatment.
- I read that “Malignancy cannot always be predicted from histology”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “Resistance to mTOR inhibition eventually develops”. How does that affect my plan?Open problems are where trials and second opinions matter most.
Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- Perivascular epithelioid cell tumour (PEComa): the full pagePEComa is a rare tumour 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.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.