Nabbout Lara Al-Khoury, Salti Ibrahim S, Choucair Mahmoud K
Department of Medicine, Division of Endocrinology and Metabolism, American University of Beirut Medical Center, Beirut, Lebanon.
Am J Med Sci. 2009 Feb;337(2):134-7. doi: 10.1097/MAJ.0b013e31816ecb00.
We are reporting an unusual patient who presented to our medical center at 18 years of age for evaluation of disabling bilateral lower extremity deformity and delayed puberty. Extensive clinical, laboratory, and radiologic evaluation confirmed the coexistence of 2 X-linked inherited disorders, X-linked hypophosphatemic rickets (XLH) and Kallmann syndrome (KS). Treatment with oral phosphate and calcitriol along with intramuscular testosterone injections was initiated. Despite a dramatic response, the course of treatment was complicated by secondary hyperparathyroidism and, 13 years later, by the development of an autonomous parathyroid adenoma that was surgically resected. Furthermore, the coexistence of XLH and KS has not been reported before. We believe that the proximity of the KAL-1 gene (Xp 22.3), involved in the pathogenesis of KS, to the phosphate regulating endopeptidase on the X chromosome gene (Xp 22.1-22.2), involved in XLH, might be responsible for this association.
我们报告了一名特殊患者,该患者18岁时前往我们的医疗中心,因双侧下肢残疾性畸形和青春期延迟前来评估。广泛的临床、实验室和放射学评估证实,该患者同时患有两种X连锁遗传性疾病,即X连锁低磷性佝偻病(XLH)和卡尔曼综合征(KS)。随后开始采用口服磷酸盐和骨化三醇以及肌肉注射睾酮进行治疗。尽管治疗反应显著,但治疗过程中出现了继发性甲状旁腺功能亢进,13年后又出现了自主性甲状旁腺腺瘤,并接受了手术切除。此外,此前尚未有XLH和KS并存的报道。我们认为,参与KS发病机制的KAL-1基因(Xp 22.3)与参与XLH的X染色体上的磷酸盐调节内肽酶基因(Xp 22.1 - 22.2)位置相近,可能是导致这种关联的原因。