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| 1 | Δ4-3-oxosteroid-5β-reductase deficiency: Responses to oral bile acid therapy and long-term outcomes显示文摘BACKGROUND Disorders of primary bile acid synthesis may be life-threatening if undiagnosed,or not treated with primary bile acid replacement therapy. To date, there are few reports on the management and follow-up of patients with Δ4-3-oxosteroid 5β-reductase(AKR1 D1) deficiency. We hypothesized that a retrospective analysis of the responses to oral bile acid replacement therapy with chenodeoxycholic acid(CDCA) in patients with this bile acid synthesis disorder will increase our understanding of the disease progression and permit evaluation of this treatment regimen as an alternative to the Food and Drug Administration(FDA) approved drug cholic acid, which is currently unavailable in China.AIM To evaluate the therapeutic responses of patients with AKR1 D1 deficiency to oral bile acid therapy, specifically CDCA.METHODS Twelve patients with AKR1 D1 deficiency, confirmed by fast atom bombardment ionization-mass spectrometry analysis of urine and by gene sequencing for mutations in AKR1 D1, were treated with differing doses of CDCA or ursodeoxycholic acid(UDCA). The clinical and biochemical responses to therapy were monitored over a period ranging 0.5-6.4 years. Dose adjustment, to optimize the therapeutic dose, was based on changes in serum biochemistry parameters,notably liver function tests, and suppression of the urinary levels of atypical hepatotoxic 3-oxo-Δ4-bile acids measured by mass spectrometry.RESULTS Physical examination, serum biochemistry parameters, and sonographic findings improved in all 12 patients during bile acid therapy, except one who underwent liver transplantation. Urine bile acid analysis confirmed a significant reduction in atypical hepatotoxic 3-oxo-Δ4 bile acids concomitant with clinical and biochemical improvements in those patients treated with CDCA. UDCA was ineffective in down-regulating endogenous bile acid synthesis as evidenced from the inability to suppress the urinary excretion of atypical 3-oxo-Δ4-bile acids. The dose of CDCA required for optimal clinical and biochemical responses varied from 5.5-10 mg/kg per day among patients based on maximum suppression of the atypical bile acids and improvement in serum biochemistry parameters, and careful titration of the dose was necessary to avoid side effects from CDCA.CONCLUSION The primary bile acid CDCA is effective in treating AKR1 D1 deficiency but the therapeutic dose requires individualized optimization. UDCA is not recommended for long-term management. | Mei-Hong Zhang Kenneth DR Setchell Jing Zhao Jing-Yu Gong Yi Lu Jian-She Wang | 2019 | World Journal of Gastroenterology2019，25，7： | 7 |
| 2 | Disseminated malignant solitary fibrous tumor of the pleura 显示文摘 | Zhang HQ Lucas DR Pass HI | 2004 | Pathol int2004，54，2： | 1 |
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| 6 | Disseminated malignant solitary fibrous tumor of the pleura显示文摘 | Zhang H Lucas DR Pass HI | 2004 | Pathol Int2004，54，2： | 1 |
| 7 | Semaphorin 3B is a 1,25-Dihydroxyvitamin D3-induced gene in osteoblasts that promotes osteoclastogenesis and induces osteopenia in mice显示文摘 | Sutton AL Zhang X Dowd DR | 2008 | Mol Endocrinol2008，22，6： | 1 |
| 8 | Disseminated malignant solitary fibrous tumor of the pleura显示文摘 | Zhang H Lucas DR Pass HI | 2004 | Pathol Int2004，54，2： | 1 |
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| 13 | B lymphocytes from early vertebrates have potent phagocytic and microbicidal abilities显示文摘 | Li J Barreda DR Zhang YA | 2006 | Nature Immunology2006，7，10： | 1 |
| 14 | Progress in research into the mech- anism of radix salviae mihiorrhizae in treatment of acute pan- creatitis 显示文摘 | Zhang XP Li ZJ Liu DR | 2006 | Hepatobiliary Pancreat Dis Int2006，5，4： | 1 |
| 15 | Drug nanocrystals for the formulation of poorly soluble drugs and its application as a potential drug delivery system 显示文摘 | Gao L Zhang DR Chen MH | 2008 | J Nanoparticle Res2008，10，5： | 1 |
| 16 | Apoptosis in astrocytomas with different grades of malignancy显示文摘 | Yew DT Wang HH Zhang DR | 1998 | Acta Neurochir (wien)1998，140，3： | 1 |
| 17 | Indirect detection of protein-metal binding:interaction of serum transferrin with In3 + and Bi3+显示文摘 | Zhang MX Gumerov DR Kaltashov IA | 2004 | AM Soc Mass Spectrom2004，15，： | 1 |
| 18 | Defect characterization using an ultrasonic array to measure the scattering coefficient matrix 显示文摘 | ZHANG J DR/NKWATER B W WILCOX P D | 2008 | IEEE Transactions on Ultrasonics Ferroelectrics and Frequency Control2008，55，10： | 1 |
| 19 | Improved cathode materials for microbial electrosynthesis 显示文摘 | Zhang T Nie H Bain TS Lu H Cui M Snoeyenbos-West OL Franks AE Nevin KP Russell TP Lovley DR | 2012 | Energy Environ Sci2012，6，1： | 1 |
| 20 | Expression of eukaryotic proteins in soluble form in Escherichia coli显示文摘 | ZHANG Y OLSEN DR NGUYEN KB | 1998 | Pro- tein Expr Purif1998，12，2： | 1 |
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