Clinical and pathological features of pachyonychia congenita

J Investig Dermatol Symp Proc. 2005 Oct;10(1):3-17. doi: 10.1111/j.1087-0024.2005.10202.x.

Abstract

Pachyonychia congenita (PC) is a rare genodermatosis affecting the nails, skin, oral mucosae, larynx, hair, and teeth. Pathogenic mutations in keratins K6a or K16 are associated with the PC-1 phenotype whereas K6b and K17 mutations are associated with the PC-2 phenotype. Analysis of clinical, pathological, and genetic data from the literature and two research registries reveal that >97% of PC cases exhibit fingernail and toenail thickening, and painful plantar keratoderma. Prospective evaluation of 57 PC patients from 41 families revealed variable clinical findings: hyperhidrosis (79%), oral leukokeratosis (75%), follicular keratosis (65%), palmar keratoderma (60%), cutaneous cysts (35%), hoarseness or laryngeal involvement (16%), coarse or twisted hair (26%), early primary tooth loss (14%), and presence of natal or prenatal teeth (2%). Stratification of these data by keratin mutation confirmed the increased incidence of cyst formation and natal teeth among PC-2 patients, although cysts were more commonly seen in PC-1 than previously reported (25%-33%). Previously unreported clinical features of PC include development of painful oral and nipple lesions during breastfeeding, copious production of waxy material in ears, and inability to walk without an ambulatory aid (50%). Possible pathogenic mechanisms are discussed with respect to the clinicopathologic and genetic correlations observed.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Darier Disease / congenital
  • Darier Disease / genetics
  • Darier Disease / pathology
  • Ectodermal Dysplasia / genetics
  • Ectodermal Dysplasia / pathology*
  • Female
  • Genes, Dominant
  • Genotype
  • Humans
  • Keratins / chemistry
  • Keratins / genetics
  • Keratoderma, Palmoplantar / congenital
  • Keratoderma, Palmoplantar / genetics
  • Keratoderma, Palmoplantar / pathology*
  • Male
  • Mutation
  • Nails, Malformed / congenital
  • Nails, Malformed / genetics
  • Nails, Malformed / pathology*
  • Phenotype

Substances

  • Keratins