StudyRareStudyRare

Hermansky-Pudlak syndrome

Log in to star

Last updated 2mo ago

Log in to add personal notes on this page.

A Puerto Rican patient with oculocutaneous albinism has prolonged bleeding after dental extraction. Pulmonary function tests show early restrictive lung disease.

AR; multiple genes (HPS1-HPS10)

  • HPS1 and HPS3 common in Puerto Rico (founder effect)
  • Oculocutaneous albinism (variable)
  • Bleeding diathesis (platelet storage pool deficiency: absent dense granules)
  • Pulmonary fibrosis (HPS1, HPS4 types: leading cause of death)
  • Granulomatous colitis (some types)
  • Whole-mount transmission electron microscopy of platelets showing absent dense bodies (the defining finding)
  • Platelet aggregation studies with a normal platelet count but impaired secondary aggregation
  • Molecular testing of the HPS genes (HPS1-HPS10) confirms the type and predicts pulmonary fibrosis risk
  • Bleeding precautions: avoid aspirin and other antiplatelet agents; desmopressin or platelet transfusion for procedures and bleeding
  • Ophthalmologic care and photoprotection for the oculocutaneous albinism, with skin cancer surveillance
  • Pulmonary monitoring in fibrosis-prone types (HPS1, HPS4); refer for transplant evaluation as disease progresses
  • Gastroenterology evaluation and treatment for granulomatous colitis when present

HPS = HypoPigmented Skin. Think "Hermansky-Pudlak = Platelet" defect.

Put your "Potluck" (Pudlak) on a plate, a reminder of the platelet aggregation defect (absent dense granules).

For the Puerto Rico founder effect, think "Mi Hermano de Puerto Rico" (Hermansky-Pudlak).

Reference Links