Evaluation of teriparatide treatment in adults with osteogenesis imperfecta

被引:162
作者
Orwoll, Eric S. [1 ]
Shapiro, Jay [2 ]
Veith, Sandra [1 ]
Wang, Ying [1 ]
Lapidus, Jodi [1 ]
Vanek, Chaim [1 ]
Reeder, Jan L. [1 ]
Keaveny, Tony M. [3 ,4 ,5 ]
Lee, David C. [3 ]
Mullins, Mary A. [6 ]
Nagamani, Sandesh C. S. [6 ]
Lee, Brendan [6 ,7 ]
机构
[1] Oregon Hlth & Sci Univ, Portland, OR 97239 USA
[2] Kennedy Krieger Inst, Baltimore, MD USA
[3] ON Diagnost, Berkeley, CA USA
[4] Univ Calif Berkeley, Dept Mech Engn, Berkeley, CA USA
[5] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA USA
[6] Baylor Coll Med, Houston, TX 77030 USA
[7] Howard Hughes Med Inst, Houston, TX 77030 USA
关键词
BONE-MINERAL DENSITY; PLACEBO-CONTROLLED TRIAL; PARATHYROID-HORMONE; 1-34; POSTMENOPAUSAL WOMEN; INTRAVENOUS NERIDRONATE; FRACTURE RATE; CHILDREN; OSTEOPOROSIS; ALENDRONATE; STRENGTH;
D O I
10.1172/JCI71101
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
100103 [病原生物学]; 100218 [急诊医学];
摘要
Background. Adults with osteogenesis imperfecta (OI) have a high risk of fracture. Currently, few treatment options are available, and bone anabolic therapies have not been tested in clinical trials for OI treatment. Methods. 79 adults with OI were randomized to receive 20 mu g recombinant human parathyroid hormone (teriparatide) or placebo for 18 months in a double-blind, placebo-controlled trial. The primary endpoint was the percent change in areal bone mineral density (aBMD) of the lumbar spine (LS), as determined by dual-energy X-ray absorptiometry. Secondary endpoints included percent change in bone remodeling markers and vertebral volumetric BMD (vBMD) by quantitative computed tomography, estimated vertebral strength by finite element analysis, and self-reported fractures. Results. Compared with the placebo group, the teriparatide group showed increased LS aBMD (6.1% +/- 1.0% vs. 2.8% +/- 1.0% change from baseline; P < 0.05) and total hip aBMD (2.6% +/- 1.0% vs. -2.4% +/- 1.0% change; P < 0.001). Vertebral vBMD and strength improved with teriparatide therapy (18% +/- 6% and 15% +/- 3% change, respectively), but declined with placebo (-5.0% +/- 6% and -2.0% +/- 3% change; P < 0.05 for both comparisons). Serum procollagen type 1 N-terminal propeptide (P1NP) and urine collagen N-telopeptide (NTx) levels increased with teriparatid.e therapy (135% +/- 14% and 64% +/- 10% change, respectively). Teriparatid.e-induced elevation of P1NP levels was less pronounced in severe forms of OI (type III/IV) compared with the milder form (type I). Type I OI patients exhibited robust BMD increases with teriparatide; however, there was no observed benefit for those with type III/IV OI. There was no difference in self-reported fractures between the 2 groups. Conclusions. Adults with OI, particularly those with less severe disease (type I), displayed a teriparatid.e-induced anabolic response, as well as increased hip and spine aBMD, vertebral vBMD, and estimated vertebral strength.
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收藏
页码:491 / 498
页数:8
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