TY - JOUR
T1 - Oral sodium phenylbutyrate for hyperammonemia associated with congenital portosystemic shunt
T2 - A case report
AU - Hasegawa, Tomonobu
AU - Sato, Takeshi
AU - Ishii, Tomohiro
AU - Oyanagi, Takayuki
AU - Kato, Mototoshi
AU - Kuroda, Tatsuo
AU - Nakatsuka, Seishi
N1 - Funding Information:
We thank the patients' families for participating in this study. We also thank Drs. Yosuke Ichihashi and Takeshi Munenaga for fruitful discussions.
Publisher Copyright:
Open Access. © 2020 Takeshi Sato et al., published by De Gruyter.
PY - 2021/3/1
Y1 - 2021/3/1
N2 - Objectives: The efficacy of sodium phenylbutyrate (SPB) for hyperammonemia associated with congenital portosystemic shunt (CPSS) remains unknown. We show the effectiveness of oral SPB. Case presentation: Our patient had CPSS with severe hypoplasia of extrahepatic portal veins. At 9 months of age, to assess the efficacy of oral SPB, we evaluated the 24 h fluctuations of venous ammonia levels. In the first two days without SPB, ammonia levels were above 80 μmol/L for half a day. On the third and fourth days, administration of oral SPB three times a day decreased ammonia to acceptable levels, except at midnight. On the fifth day, another oral SPB administration at 8 pm decreased ammonia at midnight. Low levels of branched-chain amino acids, as well as coagulation disturbances, were observed without apparent symptoms. At 12 months of age, he showed normal psychomotor development. Conclusions: Oral SPB may be effective for hyperammonemia associated with CPSS.
AB - Objectives: The efficacy of sodium phenylbutyrate (SPB) for hyperammonemia associated with congenital portosystemic shunt (CPSS) remains unknown. We show the effectiveness of oral SPB. Case presentation: Our patient had CPSS with severe hypoplasia of extrahepatic portal veins. At 9 months of age, to assess the efficacy of oral SPB, we evaluated the 24 h fluctuations of venous ammonia levels. In the first two days without SPB, ammonia levels were above 80 μmol/L for half a day. On the third and fourth days, administration of oral SPB three times a day decreased ammonia to acceptable levels, except at midnight. On the fifth day, another oral SPB administration at 8 pm decreased ammonia at midnight. Low levels of branched-chain amino acids, as well as coagulation disturbances, were observed without apparent symptoms. At 12 months of age, he showed normal psychomotor development. Conclusions: Oral SPB may be effective for hyperammonemia associated with CPSS.
KW - Congenital portosystemic shunt
KW - Hyperammonemia
KW - Sodium phenylbutyrate
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U2 - 10.1515/jpem-2020-0603
DO - 10.1515/jpem-2020-0603
M3 - Article
C2 - 33675205
AN - SCOPUS:85098850376
SN - 0334-018X
VL - 34
SP - 407
EP - 410
JO - Journal of Pediatric Endocrinology and Metabolism
JF - Journal of Pediatric Endocrinology and Metabolism
IS - 3
ER -
