Condition

Hermansky-Pudlak syndrome

Endocrine, Nutritional and Metabolic Diseases

Hermansky-Pudlak syndrome, often shortened to HPS, is an extremely rare inherited disorder passed on in an autosomal recessive pattern. It combines three features: oculocutaneous albinism, meaning reduced pigment in the skin, hair and eyes; bleeding problems caused by an abnormality of the platelets known as a storage pool defect; and the buildup of an abnormal fat and protein compound called ceroid lipofuscin inside lysosomes. Worldwide it is thought to affect about one person in 500,000, but it is far more common among people of Puerto Rican descent, where prevalence is cited at about one in 1,800, and much of the clinical research has been done in Puerto Rico. Clinicians recognize eight classic forms, distinguished by which gene carries the causative mutation. The condition is named for the Czech physicians František Heřmanský and Pavel Pudlák.

Facts
Classification
Body System
Integumentary System 1
ICD-10 Chapter
IV: Endocrine, Nutritional and Metabolic Diseases 2
Communicable or Non-communicable
Non-Communicable 1
Transmission Route
Non-Transmissible 3
Causative Agent
Causative Agent (category)
Genetic / Hereditary 1
Prevalence Band
Rare (Under 1 in 2000) 4
Sources
1. Hermansky-Pudlak syndrome (Wikipedia)
  • Lead paragraph
    results in oculocutaneous albinism (decreased pigmentation)
  • Lead paragraph [causative-agent-category]
    extremely rare autosomal recessive disorder
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2. Monarch Initiative: Hermansky-Pudlak syndrome - Monarch Initiative MONDO:0019312
Monarch InitiativeCross reference: ICD10CM
Quote, Cross reference: ICD10CM
ICD10CM:E70.331
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3. MedlinePlus Genetics (U.S. National Library of Medicine): Hermansky-Pudlak syndrome - MedlinePlus Genetics
MedlinePlus Genetics (U.S. National Library of Medicine)Description and Inheritance
Quote, Description and Inheritance
Mutations in the HPS1 gene cause approximately 75 percent of the Hermansky-Pudlak syndrome cases from Puerto Rico.
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4. Orphadata (Orphanet): Orphadata epidemiology for ORPHA:79430 Hermansky-Pudlak syndrome
Orphadata (Orphanet)Prevalence, Worldwide
Quote, Prevalence, Worldwide
"PrevalenceClass": "1-9 / 1 000 000", "PrevalenceGeographic": "Worldwide"
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