Ringuier B, Leboucher B, Leblanc M, Troussier F, Duveau E, Audran M, Giniès J L
Centre de ressources et de compétence pour la mucoviscidose, département de pédiatrie, CHU d'Angers, 49033 Angers, France.
Arch Pediatr. 2004 Dec;11(12):1445-9. doi: 10.1016/j.arcped.2004.07.028.
Disturbances in bone mineralization are frequent in cystic fibrosis but few studies have focused on the use of biphosphonates in this indication, and none on the use of oral etidronate. We report our experience using this latter treatment.
The study was retrospective and included five children and three adults with cystic fibrosis (six males and two females) aged seven to 30 years with Z-scores for lumbar bone density lower than -2 SD after one year of calcium (1 g/day) and vitamin D (900 UI/day and 300,000 UI/6 months) supplementation. All were treated during one year with etidronate: four courses of 15 days (one course per trimester) with doses ranging from 4 to 8 mg/kg per day. Calcium and vitamin D supplementation was continued between the etidronate treatment course. Total body and lumbar bone mineral density (BMD) were measured three times: at the beginning and the end of the year of calcium and vitamin D supplementation and at the end of the year of supplementation plus the four courses of etidronate treatment.
The increase in BMD in absolute value (g/cm2) and in Z-score was significantly higher (P <0.05) after the year of combined supplementation and etidronate treatment (total body g/cm2: 3+/-1%, Z-score: 2+/-1% and lumbar spine g/cm2: 6+/-5%, Z-score: 3+/-4%) than after supplementation alone (total body g/cm2: -1+/-3%, Z-score: -4+/-3% and lumbar spine g/cm2: -1+/-3%, Z-score: -4+/-4%). Supplementation alone improved the total BMD in only one patient and the lumbar BMD in three, whereas after etidronate treatment the total and lumbar BMD were improved in the eight patients. None of the patients presented with side effects that could be attributed to the treatment.
Oral etidronate treatment is well-tolerated and capable of improving bone mineralization in patients with cystic fibrosis. Further work will be necessary to determine the optimal dosage and the optimal frequency for the treatment series.
骨矿化紊乱在囊性纤维化中很常见,但很少有研究关注双膦酸盐在该适应症中的应用,且没有关于口服依替膦酸使用的研究。我们报告了使用后一种治疗方法的经验。
该研究为回顾性研究,纳入了5名儿童和3名成人囊性纤维化患者(6名男性和2名女性),年龄在7至30岁之间,在补充钙(1克/天)和维生素D(900国际单位/天和30万国际单位/6个月)一年后,腰椎骨密度Z值低于-2标准差。所有患者均接受了为期一年的依替膦酸治疗:四个疗程,每个疗程15天(每三个月一个疗程),剂量范围为每天4至8毫克/千克。在依替膦酸治疗疗程之间继续补充钙和维生素D。全身和腰椎骨密度(BMD)测量了三次:在补充钙和维生素D的一年开始时和结束时,以及在补充一年加四个依替膦酸治疗疗程结束时。
联合补充和依替膦酸治疗一年后,骨密度绝对值(克/平方厘米)和Z值的增加显著更高(P<0.05)(全身克/平方厘米:3±1%,Z值:2±1%;腰椎克/平方厘米:6±5%,Z值:3±4%),高于单独补充后(全身克/平方厘米:-1±3%,Z值:-4±3%;腰椎克/平方厘米:-1±3%,Z值:-4±4%)。单独补充仅使1名患者的全身骨密度和3名患者的腰椎骨密度得到改善,而依替膦酸治疗后,8名患者的全身和腰椎骨密度均得到改善。没有患者出现可归因于治疗的副作用。
口服依替膦酸治疗耐受性良好,能够改善囊性纤维化患者的骨矿化。需要进一步的研究来确定治疗系列的最佳剂量和最佳频率。