Importance of neonatal screening: A case study of sickle cell disease and cystic fibrosis coexistence.

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Tác giả: Nathalia Noyma Sampaio Magalhães, Lucas Barra Mathiasi, Daniela de Oliveira Werneck Rodrigues

Ngôn ngữ: eng

Ký hiệu phân loại: 628.16841 Water supply

Thông tin xuất bản: United States : World journal of clinical pediatrics , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 177347

BACKGROUND: Neonatal screening (NS) is a public health policy to identify genetic pathologies such as cystic fibrosis (CF), sickle cell disease, and other diseases. Sickle cell disease is the comprehensive term for a group of hemoglobinopathies characterized by the presence of hemoglobin S. CF is an autosomal recessive multisystemic disease with pathophysiology involving deleterious mutations in the transmembrane regulatory gene that encodes a protein that regulates the activity of chloride and sodium channels in the cell surface epithelium. NS is crucial for early diagnosis and management, which ensures a better quality of life. AIM: To report a case of the coexistence of sickle cell anemia (SCA) and CF and perform an integrative literature review. METHODS: This is an observational study and a review of the literature focusing on two rare genetic pathologies identified simultaneously in NS from the perspective of a clinical case. The authors identified only 5 cases of SCA associated with CF. No clinical trials or review articles were identified considering the rarity of the coexistence of these two pathologies. RESULTS: Herein, the authors reported the case of a girl who after undergoing NS on day 8 of life was diagnosed with SCA with an alteration in the dosage of immunoreactive trypsin. The diagnosis of CF was confirmed by the Coulometry Sweat Test. The rarity of the co-occurrence of these two severe genetic pathologies (CF and SCA) is a challenge for medical science. CONCLUSION: This study adds to the few case reports present in the literature that highlight the identification of two severe diseases
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