背景:在X连锁低磷酸盐血症(XLH)患者中,口服磷酸盐和活性维生素D的常规治疗与肾钙化相关。然而,XLH之间关系的性质,其治疗,肾钙化病,和肾功能仍然知之甚少。
方法:在参与Burosumab临床试验(NCT02181764,NCT02526160,NCT02537431,NCT02163577,NCT02750705,NCT029365LH监测项目)或NCT0LH监测项目中,在基线时进行肾脏超声检查并估计肾小球滤过率(eGFR)。在这个横截面分析中,病人,疾病,描述了有和没有肾钙质沉着症的患者的治疗特征。
结果:分析包括196名儿童(平均[SD]年龄7.6[4.0]岁)和318名成人(40.3[13.1]岁)。儿童的平均(SD)身高z评分为-1.9(1.2),成人为-2.3(1.7)。几乎所有儿童(97%)和成人(94%)以前都接受过常规治疗。在22%的儿童和38%的成年人中检测到肾钙化病。在儿童中,降低的eGFR<90毫升/分钟/1.73平方米是更普遍的肾钙化(25%)比那些没有(11%),这一发现在成年人中没有观察到。肾钙化症患儿的TmP/GFR平均值较低(P<0.05),血清1,25(OH)2D(P<0.05),eGFR(P<.001)和平均血清钙浓度(P<.05)高于无肾钙化症的患者。患有肾钙质沉着症的成年人的平均血清磷(P<.01)和1,25(OH)2D(P<.05)浓度低于没有肾钙质沉着症的成年人。探索性逻辑回归分析显示,肾钙化病的存在与其他描述的患者或疾病特征之间没有显着关联。
结论:在近四分之一的XLH儿童和超过三分之一的成人中观察到肾钙质沉着。需要进一步研究以更好地了解肾钙质沉着症的预测因素和长期后果,肾钙质沉着的监测在XLH的管理中仍然很重要。
背景:通常,X连锁低磷血症(XLH)患者接受磷酸盐和维生素D口服治疗.然而,这种疗法可能会导致肾脏中的钙积聚,叫做肾钙质沉着症。这里,我们试图更好地理解XLH,常规治疗,肾钙化病,和肾功能有关.
方法:用肾脏超声检测肾钙质沉着。肾功能,称为估计肾小球滤过率(eGFR),使用血肌酐水平测定。患者是burosumab临床试验的一部分或XLH疾病监测计划的一部分。数据是从患者接受burosumab之前收集的。
结果:该研究包括196名儿童和318名成人。几乎所有儿童和成人都接受过常规治疗。22%的儿童患有肾钙质沉着症,38%的成年人患有肾钙质沉着症。在儿童中,较低的eGFR在有肾钙质沉着症的患者(25%)中比在无肾钙质沉着症的患者(11%)中更常见.在成年人中,有和没有肾钙质沉着的患者的eGFR水平相似.某些实验室值在患有肾钙质沉着症的患者与没有肾钙质沉着症的患者之间有所不同。患有肾钙质沉着症的儿童肾脏的磷酸盐丢失明显更大,降低维生素D活性形式(1,25(OH)2D)的血液水平,较低的eGFR,血钙水平高于没有肾钙质沉着症的人。患有肾钙质沉着症的成年人的血磷水平和1,25(OH)2D浓度明显低于没有肾钙质沉着症的成年人。
结论:近四分之一的儿童和超过三分之一的成年人患有XLH,大多数人接受过常规治疗,有肾钙化病.需要进一步的研究来更好地了解哪些因素可以预测谁会患上肾钙质沉着症,并了解肾钙质沉着症的长期后果。监测XLH患者的肾钙质沉着仍然很重要。
BACKGROUND: In patients with X-linked hypophosphatemia (XLH), conventional therapy with oral phosphate salts and active vitamin D has been associated with nephrocalcinosis. However, the nature of the relationships among XLH, its treatment, nephrocalcinosis, and kidney function remain poorly understood.
METHODS: Renal ultrasounds were performed and glomerular filtration rates were estimated (eGFR) at baseline in burosumab-naïve patients with XLH who participated in burosumab clinical trials (NCT02181764, NCT02526160, NCT02537431, NCT02163577, NCT02750618, NCT02915705) or enrolled in the XLH Disease Monitoring Program (XLH-DMP; NCT03651505). In this cross-sectional analysis, patient, disease, and treatment characteristics were described among patients with and without nephrocalcinosis.
RESULTS: The analysis included 196 children (mean [SD] age 7.6 [4.0] years) and 318 adults (40.3 [13.1] years). Mean (SD) height z-score was -1.9 (1.2) for children and -2.3 (1.7) for adults. Nearly all children (97%) and adults (94%) had previously received conventional therapy. Nephrocalcinosis was detected in 22% of children and 38% of adults. In children, reduced eGFR <90 ml/min/1.73 m2 was more prevalent in those with nephrocalcinosis (25%) than in those without (11%), a finding that was not observed in adults. Children with nephrocalcinosis had lower mean values of TmP/GFR (P<.05), serum 1,25(OH)2D (P<.05), and eGFR (P<.001) and higher mean serum calcium concentrations (P<.05) than did those without nephrocalcinosis. Adults with nephrocalcinosis had lower mean serum phosphorus (P<.01) and 1,25(OH)2D (P<.05) concentrations than those without. Exploratory logistic regression analyses revealed no significant associations between the presence of nephrocalcinosis and other described patient or disease characteristics.
CONCLUSIONS: Nephrocalcinosis was observed in nearly one quarter of children and more than one-third of adults with XLH. Further study is needed to better understand the predictors and long-term consequences of nephrocalcinosis, with surveillance for nephrocalcinosis remaining important in the management of XLH.
BACKGROUND: Conventionally, patients with X-linked hypophosphatemia (XLH) were treated with phosphate and vitamin D taken by mouth. However, this therapy might lead to a buildup of calcium in the kidney, called nephrocalcinosis. Here, we tried to better understand how XLH, conventional therapy, nephrocalcinosis, and kidney function are related.
METHODS: Nephrocalcinosis was detected with kidney ultrasounds. Kidney function, called the estimated glomerular filtration rate (eGFR), was determined using the blood level of creatinine. Patients had been part of burosumab clinical trials or part of the XLH Disease Monitoring Program. Data were collected from patients before they received burosumab.
RESULTS: The study included 196 children and 318 adults. Almost all children and adults had received conventional therapy. 22% of children had nephrocalcinosis and 38% of adults had nephrocalcinosis. In children, low eGFR was more common in those with nephrocalcinosis (25%) than in those without (11%). In adults, levels of eGFR were similar among those with and without nephrocalcinosis.Some lab values were different among patients with versus those without nephrocalcinosis. Children with nephrocalcinosis had significantly greater loss of phosphate by the kidneys, lower blood levels of the active form of vitamin D (1,25(OH)2D), lower eGFR, and higher blood levels of calcium than those without nephrocalcinosis. Adults with nephrocalcinosis had significantly lower blood levels of phosphorus and 1,25(OH)2D concentrations than those without.
CONCLUSIONS: Nearly one quarter of children and more than one-third of adults with XLH, most of whom had received conventional therapy, had nephrocalcinosis. Further study is needed to better understand what factors can predict who will get nephrocalcinosis and to understand the long-term consequences of nephrocalcinosis. It remains important to monitor patients with XLH for nephrocalcinosis.