Jonsson Comprehensive Cancer Center
This phase I/II trial studies the side effects and best dose of mercaptopurine, and to see how well it works in treating patients with hereditary leiomyomatosis and renal cell carcinoma (HLRCC). HLRCC is a rare inherited disorder that increases the risk of developing benign (not cancer) tumors of the skin and the uterus (leiomyomas) and malignant (cancer) tumors of the uterus (leiomyosarcoma) and the kidney. Mercaptopurine is in a class of medications called purine antagonists. It works by stopping the growth of cancer cells.
PRIMARY OBJECTIVE: I. To identify the safety, side effects, and best dose of mercaptopurine (6-MP). SECONDARY OBJECTIVES: I. To assess clinical activity in treating metastatic fumarate hydratase (FH) Deficient Kidney Cancer. II. To assess how treatment impacts uterine fibroid clinical symptoms. III. To determine the change in menstrual bleeding. IV. To evaluate changes in pain due to leiomyomas. V. To evaluate the overall improvement in leiomyoma symptoms. EXPLORATORY OBJECTIVES: I. Associate changes in serum thiopurine metabolites (6-methylmercaptopurine \[6-MMP\] and 6-thioguanine \[6-…
Inclusion Criteria: * Age ≥ 18 * Disease-causing, germline FH mutation including variants considered either: * a) Pathogenic/likely pathogenic OR * b) variants of unknown significance (VUS) with immunohistochemical staining showing loss of FH or high 2-SC expression in tumor tissue * Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 1 * Thiopurine S-methyltransferase (TPMT) and NUDT15 homozygous, wild-type genotype * TPMT\*1/TPMT\*1 and NUDT15\*1/ NUDT15\*1 * Calculated creatinine clearance ≥ 30 milliliters per minute (mL/min) per the Cockcroft and Gault formula OR s…
Undergo tumor biopsy
Undergo collection of blood
Undergo CT
Undergo MRI
Given PO
Ancillary studies
Undergo skin biopsy
Arctuva estimate
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