Cystinuria in children and young adults: success of monitoring free-cystine urine levels
Cystinuria in children and young adults: success of monitoring free-cystine urine levels
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DOI：
10.1007/s00467-007-0575-2
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发表时间：
2007-11-01
影响因子：
3
通讯作者：
Pastore, Anna
中科院分区：
文献类型：
--
作者：
Dello Strologo, Luca;Laurenzi, Chiara;Pastore, Anna
胱氨酸尿症的药物治疗往往令人失望。患者经常接受手术，这往往是早期复发。本研究的目的是评估药物治疗胱氨酸尿症的疗效，以防止肾结石的形成或减少肾结石的数量和尺寸。20例胱氨酸尿症患者采用联合方法治疗，包括胱氨酸结合药物。每4个月测量一次游离和结合尿胱氨酸水平。调整药物剂量以维持游离尿胱氨酸水平低于100 μ mol/mmol肌酐。18名患者完成了研究;新结石的检出率从每年0.28例减少到每年0.03例，6名患者的既存肾结石数量和尺寸减少。1例受试者需要手术，12个月后未观察到复发。达到目标水平所需的剂量与患者体重密切相关：年龄较大的儿童需要较低的剂量。胱氨酸尿症的药物治疗是可行的。儿童的治疗必须个性化，因为所需的药物量严格取决于身体大小。
Medical treatment of cystinuria is often disappointing. Patients undergo frequent surgery, which is often followed by early relapse. The aim of our study was to evaluate the efficacy of medical treatment of cystinuria, to prevent formation or to reduce the numbers and dimensions of renal stones. Twenty cystinuric patients were treated with a combined approach, including cystine-binding drugs. Free and bound urine cystine levels were measured every 4 months. Drug dosage was adjusted to maintain free urine cystine level below 100 mu mol/mmol creatinine. Eighteen patients completed the study; detection of new stones was reduced from 0.28 per year to 0.03 per year, and, in six patients, the numbers and dimensions of pre-existing renal stones were reduced. Surgery was required in one subject, and no relapse was observed 12 months afterwards. The dosage required to achieve target levels was closely correlated with patient body weight: older children required a lower dose. Medical management of cystinuria is feasible. The treatment must be personalised in children, as the amount of drug required is strictly dependent on body size.