Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome

Chediak-Higashi syndrome (CHS) is a rare haematological and immunodeficiency disorder that occurs in childhood leading to recurrent infections, bleeding tendencies and progressive neurological dysfunction. Partial oculocutaneous albinism occurs in almost all cases. The exact prevalence is unknown, a...

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Main Authors: Abu Bakar, Asrar, Shunmugarajoo, Haema, Nachiappan, Jeyaseelan P., Ismail, Intan Hakimah
Format: Article
Language:English
Published: The Korean Society of Pediatric Infectious Diseases 2024
Online Access:http://psasir.upm.edu.my/id/eprint/113395/
http://psasir.upm.edu.my/id/eprint/113395/1/113395.pdf
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author Abu Bakar, Asrar
Shunmugarajoo, Haema
Nachiappan, Jeyaseelan P.
Ismail, Intan Hakimah
author_facet Abu Bakar, Asrar
Shunmugarajoo, Haema
Nachiappan, Jeyaseelan P.
Ismail, Intan Hakimah
author_sort Abu Bakar, Asrar
building UPM Institutional Repository
collection Online Access
description Chediak-Higashi syndrome (CHS) is a rare haematological and immunodeficiency disorder that occurs in childhood leading to recurrent infections, bleeding tendencies and progressive neurological dysfunction. Partial oculocutaneous albinism occurs in almost all cases. The exact prevalence is unknown, and the disease is caused by over 70 identified mutations in the lysosomal trafficking regulator gene. The presence of a bright polychromatic appearance from hair shaft and abnormally large intracytoplasmic granules, especially within neutrophils and platelets in the bone marrow is highly suggestive. Treatment is largely supportive, and the only curative treatment is through an allogeneic hematopoietic stem cell transplant. Without transplant, most patients will enter an accelerated phase of hemophagocytic lymphohistiocytosis (HLH) which carries a high mortality rate. We present a young male with CHS who we had followed through and eventually developed a fulminant accelerated phase. We believe this is only the second reported case of CHS in Malaysia.
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publisher The Korean Society of Pediatric Infectious Diseases
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spelling upm-1133952024-11-22T06:21:33Z http://psasir.upm.edu.my/id/eprint/113395/ Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome Abu Bakar, Asrar Shunmugarajoo, Haema Nachiappan, Jeyaseelan P. Ismail, Intan Hakimah Chediak-Higashi syndrome (CHS) is a rare haematological and immunodeficiency disorder that occurs in childhood leading to recurrent infections, bleeding tendencies and progressive neurological dysfunction. Partial oculocutaneous albinism occurs in almost all cases. The exact prevalence is unknown, and the disease is caused by over 70 identified mutations in the lysosomal trafficking regulator gene. The presence of a bright polychromatic appearance from hair shaft and abnormally large intracytoplasmic granules, especially within neutrophils and platelets in the bone marrow is highly suggestive. Treatment is largely supportive, and the only curative treatment is through an allogeneic hematopoietic stem cell transplant. Without transplant, most patients will enter an accelerated phase of hemophagocytic lymphohistiocytosis (HLH) which carries a high mortality rate. We present a young male with CHS who we had followed through and eventually developed a fulminant accelerated phase. We believe this is only the second reported case of CHS in Malaysia. The Korean Society of Pediatric Infectious Diseases 2024 Article PeerReviewed text en http://psasir.upm.edu.my/id/eprint/113395/1/113395.pdf Abu Bakar, Asrar and Shunmugarajoo, Haema and Nachiappan, Jeyaseelan P. and Ismail, Intan Hakimah (2024) Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome. Pediatric Infection and Vaccine, 31 (1). pp. 122-129. ISSN 2384-1079; eISSN: 2384-1087 https://piv.or.kr/DOIx.php?id=10.14776/piv.2024.31.e14 10.14776/piv.2024.31.e14
spellingShingle Abu Bakar, Asrar
Shunmugarajoo, Haema
Nachiappan, Jeyaseelan P.
Ismail, Intan Hakimah
Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome
title Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome
title_full Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome
title_fullStr Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome
title_full_unstemmed Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome
title_short Variants of LYST and novel STK4 gene mutation in a child with accelerated Chediak Higashi syndrome
title_sort variants of lyst and novel stk4 gene mutation in a child with accelerated chediak higashi syndrome
url http://psasir.upm.edu.my/id/eprint/113395/
http://psasir.upm.edu.my/id/eprint/113395/
http://psasir.upm.edu.my/id/eprint/113395/
http://psasir.upm.edu.my/id/eprint/113395/1/113395.pdf