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chediak_higashi_syndrome [2025/02/25 04:09] – created Scott Larsonchediak_higashi_syndrome [2025/06/04 18:33] (current) Scott Larson
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 ====== Chediak-Higashi syndrome ====== ====== Chediak-Higashi syndrome ======
 +====Main Features====
 +  * Oculocutaneous albinism
 +    * varying degrees of pigmentation
 +  * **Congenital Immunodeficiency**
 +    * Recurrent pyogenic infections of skin and respiratory tract
 +  * Bleeding Diathesis
 +    * mucosal bleeding
 +    * increased bruising
 +  * Progressive Neurodegeneration
 +====Eye Findings====
 +  * Reduced Pigment in Iris and Retina
 +====Other Findings====
 +  * Hemophagocytic lymphohistiocytosis (HLH)
 +    * potentially fatal inflammatory disorder characterized by:
 +    * Fever
 +    * cytopenia 
 +    * hepatosplenomegaly
 +    * lymphadenopathy 
 +  * Learning disability
 +  * Sensory motor neuropathy in 2nd or 3rd decade 
 +  * Cerebellar ataxia
 +  * Parkinsonism
 +  * Cerebellar and cerebral atrophy
 +  * Spastic paraplegia
 +====Etiology====
 +  * Autosomal Recessive 
 +  * Biallelic mutations in LYST gene- 1q42.3
 +    * lysosomal trafficking function 
 +====Pathology====
 +  * Giant inclusions in the cytoplasm of leukocytes ⇒ pathognomonic 
 +    * reduced leukocyte function
 +    * neutropenia possible
 +    * decreased natural killer cells
 +  * Platelet number usually normal but absent platelet dense granules 
 +====Treatment====
 +  * Steroids and chemotherapy
 +  * Hematopoietic stem cell transplantation (HSCT)
 +====Reference====
 +  - [[https://pubmed.ncbi.nlm.nih.gov/37254856/|Talbert ML, et al. Chediak-Higashi Syndrome. Curr Opin Hematol. 2023;30(4):144-151]][[https://1drv.ms/b/c/31d83ae8e55e0542/EYptoZtZJIhJrGl2zO40G2UB2rqB8uiFgGs5zp26NSK2Ig?e=mbwUxZ |-]]
  
 +{{tag>syndrome albinism}}