Shalitin Shlomit, Davidovits Miriam, Lazar Liora, Weintrob Naomi
Institute for Endocrinology and Diabetes, National Center for Childhood Diabetes, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.
Horm Res. 2008;70(3):137-44. doi: 10.1159/000137658. Epub 2008 Jul 29.
Pseudohypoparathyroidism (PHP) is a rare inherited syndrome characterized by parathyroid hormone (PTH) resistance and is frequently associated with Albright's hereditary osteodystrophy and resistance to other cAMP-mediated hormones. The usual neonatal presentation is mild primary hypothyroidism secondary to resistance to thyroid-stimulating hormone; hypocalcemia usually develops after age 3-5 years. This work describes the diversity in the clinical expression and course of PHP, with emphasis on calcium levels by age and treatment, in 8 children under long-term follow-up at our pediatric tertiary center. The calcium levels at presentation ranged from transient neonatal hypocalcemia to infantile hypercalcemia to childhood/adolescence hypocalcemia. Interestingly, relative hypocalciuria at diagnosis and during therapy, in the presence of renal PTH resistance, was the rule. These findings indicate that transient neonatal hypocalcemia associated with other clinical features or a family history of PHP may be a flag for clinicians to screen for PTH resistance later in life. In addition, PTH resistance may be missed by surveying calcium levels only; thus the PTH levels have to be checked as well. In addition, the recommendation for patients with hypoparathyroidism that strict low-normal calcium levels be maintained during therapy in order to prevent hypercalciuria is probably not applicable in PHP.
假性甲状旁腺功能减退症(PHP)是一种罕见的遗传性综合征,其特征为甲状旁腺激素(PTH)抵抗,常与奥尔布赖特遗传性骨营养不良以及对其他环磷酸腺苷(cAMP)介导激素的抵抗相关。通常的新生儿表现为因对促甲状腺激素抵抗继发的轻度原发性甲状腺功能减退症;低钙血症通常在3至5岁后出现。这项研究描述了在我们儿科三级中心接受长期随访的8名儿童中PHP临床表型和病程的多样性,重点关注不同年龄段的血钙水平及治疗情况。就诊时的血钙水平范围从短暂性新生儿低钙血症到婴儿高钙血症再到儿童期/青春期低钙血症。有趣的是,在存在肾脏PTH抵抗的情况下,诊断时及治疗期间相对低尿钙是常见现象。这些发现表明,与其他临床特征或PHP家族史相关的短暂性新生儿低钙血症可能是临床医生在患者后期筛查PTH抵抗的一个标志。此外,仅通过检测血钙水平可能会漏诊PTH抵抗;因此还必须检查PTH水平。此外,对于甲状旁腺功能减退症患者,为预防高尿钙而在治疗期间维持严格的低正常血钙水平的建议可能不适用于PHP。