Crigler-Najjar syndrome type I (CN-I, MIM #218800) is a rare and severe autosomal disorder. It is caused by deficiency of the liver enzyme responsible for bilirubin elimination, the uridine diphosphate glucuronosyltransferase 1A1 (UGT1A1; EC 2.4.1.17). Biologically, the disease manifests itself with severe and persistent unconjugated hyperbilirubinemia. Kernicterus is a well-known complication of severe unconjugated hyperbilirubinemia in infants and young children, especially in patients with CN-I.Few articles have shown the efficiency of plasmapheresis for extreme hyperbilirubinemia.In this report, we describe the efficiency of plasmapheresis for a rapid control of acute and severe unconjugated hyperbilirubinemia in a 6-year-old CN-I patient who had previously developed kernicterus in the neonatal period. In spite of intensification of phototherapy, the patient developed severe hyperbilirubinemia (up to 830 μmol/l, with bilirubin/albumin ratio at 1.2). With two plasmapheresis procedures, bilirubin serum concentration decreased to 420 μmol/ and bilirubin/albumin ratio to 0.55. Following the acute episode of very severe unconjugated hyperbilirubinemia, the child recovered and neurological examination was unchanged, thus suggesting that plasmapheresis possibly prevented further worsening of kernicterus.
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http://dx.doi.org/10.1007/8904_2011_40 | DOI Listing |
Gynecol Endocrinol
December 2024
Department of Gynecological Endocrinology, Beijing Obstetrics and Gynecology Hospital, Capital Medical University, Beijing Maternal and Child Health Care Hospital, Beijing, China.
Objective: To present a young girl with pyruvate kinase deficiency (PKD) and concurrent severe hemolytic anemia who underwent fertility preservation and cryopreservation. Clinical symptoms, diagnosis, treatment, and new strategies for fertility protection and preservation in PKD patients who require allogeneic hematopoietic stem cell therapy are explored.
Case Presentation: Six-year-old girl with persistent unconjugated hyperbilirubinemia and severe hemolytic anemia since birth, continuous elevation of bilirubin levels and severe splenomegaly.
Introduction: Jaundice is a state of disease in liver function often along with gastrointestinal (GI) complications primarily characterized by hyperbilirubinemia. Other tests regarding liver function are often used besides bilirubin as during any liver complications biomarkers such as alanine aminotransferase (ALT), serum glutamic pyruvic transaminase (SGPT), aspartate aminotransferase (AST), serum glutamic-oxaloacetic transaminase (SGOT), alkaline phosphatase (ALP), and many other biomarkers even total protein metabolism might get disrupted and get released in the blood. However, no comparison or association among those biomarkers was determined in a cohort.
View Article and Find Full Text PDFMol Ther Methods Clin Dev
December 2024
International Centre for Genetic Engineering and Biotechnology (ICGEB), Padriciano 99, 34149 Trieste, Italy.
Crigler-Najjar syndrome is an ultra-rare monogenic recessive liver disease caused by gene mutations. Complete UGT1A1 deficiency results in severe unconjugated hyperbilirubinemia in newborns that, if not treated, may lead to brain damage and death. Treatment is based on intensive phototherapy, but its efficacy decreases with age, rendering liver transplantation the only curative option.
View Article and Find Full Text PDFACS Chem Neurosci
December 2024
Department of Radiology, The Second Affiliated Hospital, Medical College of Shantou University, Shantou 515041, China.
Unconjugated bilirubin (UCB) visualization is valuable for early bilirubin encephalopathy (BE) diagnosis and management. UCB neurotoxicity is a challenge, necessitating improved imaging modalities for precise localization and characterization. This study developed a noninvasive method for UCB imaging in the brain using chemical exchange saturation transfer (CEST) magnetic resonance imaging, which visualizes UCB distribution through amide-bulk water proton exchange, a process termed bilirubin CEST (Bil-CEST) imaging.
View Article and Find Full Text PDFEur J Pediatr
November 2024
Área de Pediatria, Unidade de Pediatria Médica, Hospital Dona Estefânia, Unidade Local de Saúde São José, Centro Académico de Lisboa, 5.1, Lisbon, Portugal.
Neonatal hyperbilirubinaemia is more common in Asian-descendent populations, but differences in disease severity are poorly reported. Our study aimed to compare neonatal hyperbilirubinaemia severity between European and Indian subcontinent descendent newborns. We conducted a single-centre retrospective cohort study including newborns admitted with unconjugated hyperbilirubinaemia (January 2016 to December 2021).
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