ArticleJournal for immunotherapy of cancer2018
Durable response to anti-PD-1 immunotherapy in epithelioid angiomyolipoma: a report on the successful treatment of a rare malignancy.
Article in Journal for immunotherapy of cancer, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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Who cites it
26 citing papers in PubMed, 28 citations in OpenAlex.
- Complete response to dual immune checkpoint blockade in a patient with Birt-Hogg-Dubé syndrome and epithelioid angiomyolipoma.Journal for immunotherapy of cancer · 2026Article
- TFE3-rearranged PEComa with hepatic and pulmonary involvement: diagnostic challenges and resistance to mTOR-targeted therapy.Journal of cancer research and clinical oncology · 2026Article
- PEComas: current concepts in diagnosis, molecular pathways, and emerging treatments.The oncologist · 2026Review
- Epithelioid angiomyolipoma: a rare renal tumor with histopathological complexity.Journal of surgical case reports · 2026Article
- A rare case of malignant epithelioid angiomyolipoma: involvement of the lumbar vein pathway with downward extension to the iliac veins.International journal of surgery case reports · 2026Article
- Durable Disease Control With Nivolumab in Malignant Epithelioid Angiomyolipoma: A Case Report.IJU case reports · 2026Article
- Case Report: Acquired CDKN2A/B deletion and mTOR inhibitor resistance in TSC2-mutant metastatic epithelioid angiomyolipoma.Frontiers in medicine · 2026Article
- Metastatic perirectal PEComa treated by checkpoint inhibitor immunotherapy and multimodal treatment: case report and review of the literature.Therapeutic advances in medical oncology · 2024Article
- Perivascular epithelioid cell tumor of the uterus and pelvic cavity.Frontiers in oncology · 2024Article
- Systemic Treatments and Molecular Biomarkers for Perivascular Epithelioid Cell Tumors: A Single-institution Retrospective Analysis.Cancer research communications · 2023Article
- Management Strategies for Adults with Locally Advanced, Unresectable or Metastatic Malignant Perivascular Epithelioid Cell Tumor (PEComa): Challenges and Solutions.Cancer management and research · 2023Review
- Anti-PD-1 immunotherapy combined with stereotactic body radiation therapy and GM-CSF for the treatment of advanced malignant PEComa: A case report.Frontiers in oncology · 2023Article
- Primary renal malignant epithelioid angiomyolipoma with distant metastasis: a case report and literature review.Frontiers in oncology · 2023Article
- Advancements in the diagnosis and treatment of renal epithelioid angiomyolipoma: A narrative review.The Kaohsiung journal of medical sciences · 2022Review
- [Analysis of the diagnosis and treatment of 24 cases of hepatic perivascular epithelioid cell tumor].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2022Article
- Angiomyolipoma of the kidney-Clinicopathological analysis of 52 cases.Pathology oncology research : POR · 2022Article
- Spectrum of Presentations and Management Strategies in Renal Angiomyolipoma.Journal of kidney cancer and VHL · 2022Article
- Review
- Sustained response to pembrolizumab in recurrent perivascular epithelioid cell tumor with elevated expression of programmed death ligand: a case report.Journal of medical case reports · 2021Article
- Primary perivascular epithelioid cell tumor (PEComa) in bone: A review of the literature and a case arising in the humerus with multiple metastases.Journal of bone oncology · 2021Article
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Authors and funding
10 authors at 4 institutions in 2 countries.
Funding
Abstract
backgroundMalignant angiomyolipoma is an uncommon tumor of the class of perivasciular epithelioid cell neoplasms (PEComas). These tumors are characteristically driven by deleterious mutations in the tumor suppressors TSC1 and TSC2, whose gene products typically act to inhibit mTOR. There are several cases of malignant angiomyolipoma which exhibit transient responses to mTOR inhibitors, forming the basis of current practice guidelines in malignant PEComa. However the tumors ultimately acquire resistance, and there is no well-established second-line option. Despite the increasing prevalence of immunotherapy across a wide range of solid tumors, little is known about the immune infiltrate and PD-L1 expression of angiomyolipoma. Furthermore, there is no reported case on the treatment of malignant angiomyolipoma with an immune checkpoint inhibitor. CASE PRESENTATION: A 38 year-old man presented with gross hematuria and was diagnosed with renal epithelioid angiomyolipoma. Despite surgical resection, the tumor recurred and metastasized. Targeted genomic sequencing revealed a deleterious mutation in TSC2, and the patient was treated with the mTOR inihbitor everolimus. The patient went on to have a partial response but ultimately progressed. He was then treated with the anti-PD-1 immune checkpoint inhibitor nivolumab, and achieved a durable near-complete response which is ongoing after two years of treatment. Immunohistochemical staining of tumor tissue revealed strong PD-L1 expression and a brisk T-cell infiltrate.
conclusionsWe report on the first durable systemic treatment of malignant epithelioid angiomyolipoima with the use of PD-1 antibody nivolumab. Given the absence of prospective clinical trials in this exceedingly rare disease, particularly in the second-line setting, immune checkpoint inhibitors like nivolumab should be considered.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.