Hermansky-Pudlak syndrome in a Swiss population

Dermatology. 1993;187(4):248-56. doi: 10.1159/000247258.

Abstract

Tyrosinase-positive albinism, previously diagnosed as Hermansky-Pudlak Syndrome (HPS), has been examined in four generations from a village of the canton Valais, Switzerland. Homozygotes, obligate heterozygotes and putative heterozygotes in this geneology yielded lower than normal membrane-associated thioredoxin reductase (TR) activities compared with normal family members and controls. All of the homozygotes and 50% of each the obligate and putative heterozygotes showed an increase in bleeding time associated with storage-pool-deficient platelets lacking dense bodies. The TR activity profile and the platelet-dense body deficiency in the Swiss albinos was the same as that in the HPS population from Puerto Rico. However, in albinos from Puerto Rico, there is an accumulation of ceroid/lipofuscin-like pigment in lysosomal structures causing tissue damage, and, upon kidney involvement, this leads to increased urinary dolichol excretion. Approximately half of the Puerto Rican HPS cases had clinical evidence of storage disease with restrictive lung disease, granulomatous colitis, kidney failure and cardiomyopathy. By comparison, the Swiss HPS geneology had a normal life expectancy with no significant evidence for ceroid accumulation or urinary dolichol excretion. An examination of antioxidant enzymes, catalase, TR and glutathione reductase in epidermal suction blisters from Swiss HPS homozygotes showed a similar result for catalase and TR levels to the depigmented epidermis of patients with vitiligo, except that intracellular TR was found to be calcium free in HPS compared with vitiligo. Intracellular glutathione reductase levels were highest in HPS. Both the Swiss and Puerto Rican HPS homozygotes and heterozygotes have giant melanosomes in skin melanocytes.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Albinism, Oculocutaneous / enzymology
  • Albinism, Oculocutaneous / genetics
  • Albinism, Oculocutaneous / metabolism*
  • Albinism, Oculocutaneous / pathology*
  • Albinism, Oculocutaneous / urine
  • Blister / enzymology
  • Catalase / analysis
  • Cytosol / enzymology
  • Dolichols / urine
  • Female
  • Genetic Carrier Screening
  • Glutathione Reductase / analysis
  • Humans
  • Male
  • Membrane Proteins / analysis
  • Middle Aged
  • Pedigree
  • Puerto Rico
  • Skin / enzymology
  • Skin / pathology
  • Switzerland
  • Thioredoxin-Disulfide Reductase / analysis
  • Thioredoxin-Disulfide Reductase / metabolism

Substances

  • Dolichols
  • Membrane Proteins
  • Catalase
  • Glutathione Reductase
  • Thioredoxin-Disulfide Reductase