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地中海贫血性骨病:一种新的骨质沉积标志物。

Thalassemic osteopathy: a new marker of bone deposition.

机构信息

U.O. Medicina Interna 1A, Fondazione IRCCS "Ca' Granda" Ospedale Maggiore Policlinico, Milano, Italy; Dipartimento di Medicina Interna, Università degli Studi di Milano, Milano, Italy.

U.O. Audiologia, Fondazione IRCCS "Ca' Granda" Ospedale Maggiore Policlinico, Milano, Italy.

出版信息

Blood Cells Mol Dis. 2014 Feb-Mar;52(2-3):91-4. doi: 10.1016/j.bcmd.2013.09.008. Epub 2013 Oct 3.

DOI:10.1016/j.bcmd.2013.09.008
PMID:24091145
Abstract

Osteopathy represents a prominent cause of morbidity in patients with beta-thalassemia major (TM) and manifests as osteopenia/osteoporosis. Biochemical turnover markers (BTMs) are considered a useful, non-invasive tool for the clinical follow-up of osteoporotic patients; they can provide a dynamic view of the remodeling process and give information on the metabolic activity of bone tissue as well as on the pathogenesis of bone loss. The amino-terminal pro-peptide of type I procollagen (P1NP) is a recently introduced marker that is considered the most sensitive index of bone formation. Although demonstrated in several categories of patients with bone disease, there is little information on the clinical usefulness of this bone formation index in thalassemic patients. We evaluated the P1NP levels of 53 adult patients with b-thalassemia major (21 males and 32 females, mean age 34.5 ± 5.7, range 22-46 years) and associated osteopathy. We investigated the correlation between P1NP and bone condition as examined by dual X-ray photon absorptiometry and with BTMs expressing bone resorption and bone mineralization (carboxyterminal collagen cross-linked (CTX) terminal regions of type I collagen and osteocalcin, respectively). P1NP serum levels were correlated with CTX levels (r=0.545, p<0.001); the results were unchanged when males and females, as well as osteoporotic and osteopenic subgroups, were considered separately. No correlation was demonstrated neither between OC and CTX (r=0.17, p=ns), nor between P1NP and OC levels (r=0.11, p=ns). No correlation was demonstrated among the P1NP/CTX ratio and age, OC or densitometric values and no difference was found in the same ratio between osteopenic (0.19 ± 0.16) and osteoporotic (0.15 ± 0.14) patients. Similar results were obtained for the OC/CTX ratio, as it was not correlated with age, P1NP or densitometric values. This is the first report of circulating P1NP in patients with TM-associated osteoporosis. P1NP and CTX assays show good precision and low analytical CV, and, compared to other markers, they can acceptably reflect bone metabolic processes and promptly respond to antiosteoporotic treatments. We trust that this sensitive marker can be useful in the assessment of treatment efficacy and can overcome the pitfalls due to wide variability in the normal values of most BTMs that create difficulty in pinpointing the individual patient's response.

摘要

骨病是重型β地中海贫血(TM)患者发病和致残的主要原因,表现为骨质疏松/骨量减少。生化转换标志物(BTMs)被认为是骨质疏松症患者临床随访的一种有用的、非侵入性工具;它们可以提供骨重塑过程的动态视图,并提供有关骨组织代谢活性以及骨丢失发病机制的信息。I 型前胶原氨基端前肽(P1NP)是一种新引入的标志物,被认为是骨形成最敏感的指标。尽管在几类骨疾病患者中得到了证实,但关于该骨形成指标在地中海贫血患者中的临床应用的信息很少。我们评估了 53 例重型β地中海贫血成年患者(21 名男性和 32 名女性,平均年龄 34.5 ± 5.7 岁,范围 22-46 岁)及其相关的骨病的 P1NP 水平。我们研究了 P1NP 与双能 X 线吸收仪检查的骨状况之间的相关性,并与表示骨吸收和骨矿化的 BTMs(I 型胶原羧基末端交联(CTX)末端区域和骨钙素)进行了相关性分析。P1NP 血清水平与 CTX 水平相关(r=0.545,p<0.001);当分别考虑男性和女性以及骨质疏松和骨量减少亚组时,结果保持不变。OC 和 CTX 之间(r=0.17,p=ns)以及 P1NP 和 OC 水平之间(r=0.11,p=ns)均未显示相关性。P1NP/CTX 比值与年龄、OC 或密度测定值之间无相关性,在骨量减少(0.19 ± 0.16)和骨质疏松(0.15 ± 0.14)患者之间,该比值无差异。OC/CTX 比值也得到了相似的结果,因为它与年龄、P1NP 或密度测定值均无相关性。这是首次报道 TM 相关骨质疏松症患者的循环 P1NP。P1NP 和 CTX 检测具有良好的精密度和低分析变异系数,与其他标志物相比,它们可以较好地反映骨代谢过程,并能迅速对抗骨质疏松治疗做出反应。我们相信,这种敏感的标志物可用于评估治疗效果,并可克服大多数 BTMs 正常值的广泛变异性所带来的缺陷,从而难以确定个体患者的反应。

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