Heubi J E, Tsang R C, Steichen J J, Chan G M, Chen I W, DeLuca H F
J Pediatr. 1979 Jun;94(6):977-82. doi: 10.1016/s0022-3476(79)80243-7.
Osteodystrophy frequently accompanies severe childhood hepatobiliary disease. Proposed causes include malabsorption of vitamin D and calcium, and diminished 25-hydroxylation of vitamin D. Two children, ages 23 and 35 months, with radiographic and biochemical evidence of rickets with extrahepatic biliary atresia, were treated with 1,25-dihydroxyvitamin D3. The minimal effective therapeutic dose and efficacy of 1,25-(OH)2D3 in the treatment of rickets associated with severe childhood hepatic disease were determined. Oral 1,25-(OH)2D3 was ineffective at doses of 0.10 microgram/kg/day. Parenteral doses of 0.20 microgram/kg/day effectively produced radiographic, bone mineral (photon absorptiometric), and biochemical evidence of healing. The need for four times the physiologic dose of 1,25-(OH)2D3 by the parenteral route suggested enhanced catabolism of, or end-organ resistance to, 1,25-(OH)2D3 in our patients with severe cholestatic liver disease treated with phenobarbital.
骨营养不良常伴随儿童严重肝胆疾病。推测的病因包括维生素D和钙吸收不良，以及维生素D的25 - 羟化作用减弱。两名年龄分别为23个月和35个月的儿童，有肝外胆道闭锁伴佝偻病的影像学和生化证据，接受了1,25 - 二羟维生素D3治疗。确定了1,25 - (OH)2D3治疗与儿童严重肝脏疾病相关佝偻病的最小有效治疗剂量和疗效。口服1,25 - (OH)2D3剂量为0.10微克/千克/天无效。胃肠外给药剂量为0.20微克/千克/天能有效产生影像学、骨矿物质（光子吸收法）及生化方面的愈合证据。对于我们用苯巴比妥治疗的严重胆汁淤积性肝病患者，胃肠外途径需要四倍生理剂量的1,25 - (OH)2D3，这表明1,25 - (OH)2D3在体内分解代谢增强或存在终末器官抵抗。
