CAJ | 학술논문

目的观察阿仑膦酸钠治疗成骨不全患儿的临床疗效和安全性.方法对2008年8月至2011年4月上海交通大学医学院附属新华医院11例成骨不全的患儿给予口服阿仑膦酸钠治疗,平均治疗时间为(1.7±0.3)年,评估治疗后患儿的生长情况、骨折频率、运动及生活自理能力以及药物的安全性.结果经口服阿仑膦酸钠治疗后,成骨不全患儿临床症状明显改善.治疗后的年骨折次数(0～1.2次/年,中位数0次/年)较治疗前(0.5 ～5.0次/年,中位数1.40次/年)明显降低(P=0.003),治疗后的运动功能等级及自理生活能力评分(中位数为3级及73分)均较治疗前明显改善(中位数为4级及43分,P =0.004、0.003).骨X线影像显示治疗后腰椎、长骨及干骺端密度增加,骨皮质增厚.治疗对身高及骨代谢标志物指标无显著影响,患儿未见药物不良反应.结论口服阿仑膦酸钠可明显降低成骨不全患儿骨折频率,提高患儿运动功能及生活质量,患儿对药物耐受性良好,是一种方便且有效的治疗成骨不全患儿的药物.
목적 관찰아륜련산납치료성골불전환인적림상료효화안전성.방법 대2008년8월지2011년4월상해교통대학의학원부속신화의원11례성골불전적환인급여구복아륜련산납치료,평균치료시간위(1.7±0.3)년,평고치료후환인적생장정황、골절빈솔、운동급생활자리능력이급약물적안전성.결과 경구복아륜련산납치료후,성골불전환인림상증상명현개선.치료후적년골절차수(0～1.2차/년,중위수0차/년)교치료전(0.5 ～5.0차/년,중위수1.40차/년)명현강저(P=0.003),치료후적운동공능등급급자리생활능력평분(중위수위3급급73분)균교치료전명현개선(중위수위4급급43분,P =0.004、0.003).골X선영상현시치료후요추、장골급간후단밀도증가,골피질증후.치료대신고급골대사표지물지표무현저영향,환인미견약물불량반응.결론 구복아륜련산납가명현강저성골불전환인골절빈솔,제고환인운동공능급생활질량,환인대약물내수성량호,시일충방편차유효적치료성골불전환인적약물.
Objective To explore the clinical efficacy and safety of oral alendronate in children with osteogenesis imperfecta(OI).Methods Eleven OI children were recruited from August 2008 to April 2011 at Shanghai Institute of Pediatric Research to receive alendronate for a duration of( 1.7 ± 0.3 ) years.The growth,fracture incidence,physical activity,the quality of daily life and safety parameters were evaluated.Results All patients obtained marked improvement.The rates of bone fractures decreased more remarkably than that at pre-treatment (0 - 1.2 fractures per year vs 0.5 - 5.0 fractures per year,medium 0 vs 1.40 fractures per year) ( P =0.003).Their levels of physical activities improved significantly ( median level from 4 to 3,P =0.004).There was significant post-treatment improvement in the self-care activity scores (median score from 43 to 73,P =0.003 ). The bone density of lumbar vertebrae,long bones and metaphysis improved at post-treatment.The radiographic examinations revealed the thickness of bone cortex.The change in height did not show any significant difference.No change was found in the serum levels of calcium,phosphorus,parathyroid hormone,alkaline phosphate or other biochemical markers. No adverse reaction occurred throughout treatment. Conclusion Oral alendronate treatment reduces the incidence of bone fracture and improves physical activity and life quality in OI children,and as a well-tolerated regimen,it is both safe and effective in clinical practice.