[Clinical and genetic characteristics of congenital isolated adrenocorticotropic hormone deficiency]. 2022

M Du, and Z Q Liu, and F Y Song, and K Qian, and X B Chen
Department of Endocrinology, Children's Hospital, Capital Institute of Pediatrics, Beijing 100020, China.

Objective: To investigate the clinical and genetic characteristics of congenital isolated adrenocorticotropic hormone (ACTH) deficiency. Methods: The clinical and laboratory characteristics of 5 cases with congenital isolated ACTH deficiency diagnosed in the Department of Endocrinology of the Children's Hospital, Capital Institute of Pediatrics from January 2019 to March 2021 were retrospectively analyzed. The general conditions, clinical manifestations, laboratory examinations, genetic charcteistics, treatment and follow-up (up to October 2021) were analyzed. Results: Of the 5 cases, 1 was female and 4 were males, aged from 13 months to 6 years at the time of consultation. The symptoms of hypoglycemia and convulsion were presented in infancy, and 4 cases had infantile cholestasis. Glucose level of 5 cases ranged from 0.79-2.20 mmol/L, ACTH ranged from <1.00-4.17 ng/L, and cortisol ranged from 0.2-3.8 μg/L. Whole exon sequencing revealed that 3 cases carried homozygous TBX19 variations, and 2 cases had compound heterozygous TBX19 variations, including 3 variants that had been reported before and 3 novel variants were found. After the diagnosis was confirmed, all the cases were treated with hydrocortisone. Hypoglycemia was all corrected during the follow-up, and 4 cases no longer had convulsions. Conclusion: Congenital isolated ACTH deficiency should be considered in neonates and infants with cholestasis and hypoglycemia, and the diagnosis can be confirmed by genetic testing.

UI MeSH Term Description Entries
D007003 Hypoglycemia A syndrome of abnormally low BLOOD GLUCOSE level. Clinical hypoglycemia has diverse etiologies. Severe hypoglycemia eventually lead to glucose deprivation of the CENTRAL NERVOUS SYSTEM resulting in HUNGER; SWEATING; PARESTHESIA; impaired mental function; SEIZURES; COMA; and even DEATH. Fasting Hypoglycemia,Postabsorptive Hypoglycemia,Postprandial Hypoglycemia,Reactive Hypoglycemia,Hypoglycemia, Fasting,Hypoglycemia, Postabsorptive,Hypoglycemia, Postprandial,Hypoglycemia, Reactive
D007223 Infant A child between 1 and 23 months of age. Infants
D007231 Infant, Newborn An infant during the first 28 days after birth. Neonate,Newborns,Infants, Newborn,Neonates,Newborn,Newborn Infant,Newborn Infants
D008297 Male Males
D002648 Child A person 6 to 12 years of age. An individual 2 to 5 years old is CHILD, PRESCHOOL. Children
D002779 Cholestasis Impairment of bile flow due to obstruction in small bile ducts (INTRAHEPATIC CHOLESTASIS) or obstruction in large bile ducts (EXTRAHEPATIC CHOLESTASIS). Bile Duct Obstruction,Biliary Stasis,Bile Duct Obstructions,Biliary Stases,Cholestases,Duct Obstruction, Bile,Duct Obstructions, Bile,Obstruction, Bile Duct,Obstructions, Bile Duct,Stases, Biliary,Stasis, Biliary
D005260 Female Females
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D006854 Hydrocortisone The main glucocorticoid secreted by the ADRENAL CORTEX. Its synthetic counterpart is used, either as an injection or topically, in the treatment of inflammation, allergy, collagen diseases, asthma, adrenocortical deficiency, shock, and some neoplastic conditions. Cortef,Cortisol,Pregn-4-ene-3,20-dione, 11,17,21-trihydroxy-, (11beta)-,11-Epicortisol,Cortifair,Cortril,Epicortisol,Hydrocortisone, (11 alpha)-Isomer,Hydrocortisone, (9 beta,10 alpha,11 alpha)-Isomer,11 Epicortisol
D000309 Adrenal Insufficiency Conditions in which the production of adrenal CORTICOSTEROIDS falls below the requirement of the body. Adrenal insufficiency can be caused by defects in the ADRENAL GLANDS, the PITUITARY GLAND, or the HYPOTHALAMUS. Adrenal Gland Hypofunction,Hypoadrenalism,Adrenal Insufficiencies,Hypofunction, Adrenal Gland

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