Fumarate Hydratase-deficient Renal Cell Carcinoma Is Strongly Correlated With Fumarate Hydratase Mutation and Hereditary Leiomyomatosis and Renal Cell Carcinoma Syndrome

Am J Surg Pathol. 2016 Jul;40(7):865-75. doi: 10.1097/PAS.0000000000000617.

Abstract

Hereditary leiomyomatosis and renal cell carcinoma syndrome-associated renal cell carcinomas (RCC) are difficult to diagnose prospectively. We used immunohistochemistry (IHC) to identify fumarate hydratase (FH)-deficient tumors (defined as FH negative, 2-succinocysteine [2SC] positive) in cases diagnosed as "unclassified RCC, high grade or with papillary pattern," or "papillary RCC type 2," from multiple institutions. A total of 124 tumors (from 118 patients) were evaluated by IHC for FH and 2SC. An FH deficiency was found in 24/124 (19%) cases. An indeterminate result (only 1 marker abnormal) was found in 27/124 (22%) cases. In a tissue microarray of 776 RCCs of different types, only 2 (0.5%) tumors, initially considered papillary type 2, were FH deficient. FH mutations were found in 19/21 FH-deficient tumors (with confirmed germline mutations in 9 of 9 tumors in which germline status could be assessed) and in 1/26 FH-indeterminate tumors identified by IHC. No FH mutations were found in 2/21 FH-deficient RCCs, 25/26 FH-indeterminate RCCs, and 10/10 RCCs demonstrating FH expression by IHC. Patients with FH-deficient RCC had a median age of 44 years (range, 21 to 65 y). Average tumor size was 8.2 cm (range, 0.9 to 18 cm). FH-deficient RCCs were characterized by at least focal macronucleoli and demonstrated 2 or more growth patterns in 93% cases. Papillary was the most common (74%) and dominant (59%) pattern, whereas other common patterns included: solid (44%), tubulocystic (41%), cribriform (41%), and cystic (33%). At presentation, 57% were stage ≥pT3, 52% had positive nodes, and 19% had distant metastases. After a mean follow-up of 27 months (range, 1 to 114 mo), 39% of patients were dead of disease, and 26% had disease progression. We conclude that FH and 2SC are useful IHC ancillary tools, which allow recognition of FH-deficient RCC.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Carcinoma, Renal Cell / diagnosis
  • Carcinoma, Renal Cell / etiology
  • Cysteine / analogs & derivatives
  • Cysteine / analysis
  • Cysteine / biosynthesis
  • Female
  • Fumarate Hydratase / deficiency*
  • Fumarate Hydratase / genetics*
  • Germ-Line Mutation
  • Humans
  • Immunohistochemistry
  • Kidney Neoplasms / diagnosis
  • Kidney Neoplasms / etiology
  • Leiomyomatosis / diagnosis*
  • Leiomyomatosis / genetics
  • Leiomyomatosis / pathology*
  • Male
  • Metabolism, Inborn Errors / complications*
  • Middle Aged
  • Muscle Hypotonia / complications*
  • Neoplastic Syndromes, Hereditary / diagnosis*
  • Neoplastic Syndromes, Hereditary / genetics
  • Neoplastic Syndromes, Hereditary / pathology*
  • Psychomotor Disorders / complications*
  • Skin Neoplasms / diagnosis*
  • Skin Neoplasms / genetics
  • Skin Neoplasms / pathology*
  • Tissue Array Analysis
  • Uterine Neoplasms / diagnosis*
  • Uterine Neoplasms / genetics
  • Uterine Neoplasms / pathology*
  • Young Adult

Substances

  • S-(2-succinyl)cysteine
  • Fumarate Hydratase
  • Cysteine

Supplementary concepts

  • Fumaric aciduria
  • Hereditary leiomyomatosis and renal cell cancer