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德斯布瓦氏发育不良的长期预后:4例成年患者的随访

Long-term outcome in Desbuquois dysplasia: a follow-up in four adult patients.

作者信息

Faivre Laurence, Cormier-Daire Valérie, Young Ian, Bracq Henri, Finidori Georges, Padovani Jean Paul, Odent Sylvie, Lachman Ralph, Munnich Arnold, Maroteaux Pierre, Le Merrer Martine

机构信息

Département de Génétique et INSERM U393, Hôpital Necker-Enfants Malades, 149 rue de Sèvres, 75015 Paris, France.

出版信息

Am J Med Genet A. 2004 Jan 1;124A(1):54-9. doi: 10.1002/ajmg.a.20441.

Abstract

Desbuquois dysplasia is a rare chondrodysplasia characterized by short stature, joint laxity, and specific radiographic findings. We report the natural history of four patients (three boys and one girl) with Desbuquois dysplasia ages 16-22 years. The mean height in adulthood was 114 cm (-8.5 SD) with progressive deceleration of the growth curve from birth (-4 SD) to adulthood. Obesity was noted consistently and facial abnormalities were still present but less obvious than in childhood. Three of four patients had mental retardation of varying degree. Hyperlaxity was persistent but limited motion of various joints was also noted. Orthopedic complications included coxa vara or valga (3/4), scoliosis (3/4), marked lordosis (3/4), and ambulatory difficulties (3/4). Surgical treatment was necessary for all four patients, involving large joints, spine and hands. Other complications included acute open-angle glaucoma secondary to a congenital malformation of the angle in one case. In addition to consistent radiological findings, elevated greater trochanter, generalized osteoporosis especially of the spine, scoliosis and/or lordosis, wide metaphyses, flat epiphyses, and coxa vara or valga were part of the natural history of the disorder. Our study emphasizes the care of older patients with Desbuquois dysplasia.

摘要

德布凯发育不良是一种罕见的软骨发育不良,其特征为身材矮小、关节松弛以及特定的影像学表现。我们报告了4例年龄在16至22岁之间的德布凯发育不良患者(3名男孩和1名女孩)的自然病史。成年后的平均身高为114厘米(-8.5标准差),生长曲线从出生时(-4标准差)到成年期逐渐减速。持续存在肥胖现象,面部异常仍然存在,但比儿童期时不太明显。4名患者中有3名存在不同程度的智力障碍。关节过度松弛持续存在,但也注意到各个关节活动受限。骨科并发症包括髋内翻或髋外翻(4例中的3例)、脊柱侧弯(4例中的3例)、明显的脊柱前凸(4例中的3例)以及行走困难(4例中的3例)。所有4例患者均需要手术治疗,涉及大关节、脊柱和手部。其他并发症包括1例因先天性房角畸形继发的急性开角型青光眼。除了一致的影像学表现外,大转子升高、全身性骨质疏松尤其是脊柱骨质疏松、脊柱侧弯和/或脊柱前凸、干骺端增宽、骨骺扁平以及髋内翻或髋外翻都是该疾病自然病史的一部分。我们的研究强调了对老年德布凯发育不良患者的护理。

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