Wood Colleen, Hasan Sana, Darukhanavala Amy, Tangpricha Vin
Billings Clinic, Pediatric Subspecialties Department, Pediatric Diabetes and Endocrinology, 2800 Tenth Avenue North, P.O. Box 37000, Billings, MT 59107, United States.
Department of Endocrinology and Metabolism, Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH 44195, United States.
J Clin Transl Endocrinol. 2021 Nov 6;26:100273. doi: 10.1016/j.jcte.2021.100273. eCollection 2021 Dec.
Vitamin D deficiency is common in the general population, and even more so in patients with cystic fibrosis. Deficiency is exacerbated in cystic fibrosis patients because of a myriad of causes including malabsorption, decreased fat mass, reduced 25-hydroxylation of vitamin D, reduced exposure to sunlight, decreased vitamin D binding protein, and exposure to drugs that increase catabolism. In turn, vitamin D deficiency can contribute to poor bone health. Additionally, it may contribute to pulmonary decline in the form of worsening pulmonary function, increased colonization with pathogens, and increased pulmonary exacerbation. Because vitamin D deficiency is correlated with negative clinical effects in multiple organ systems of patients with cystic fibrosis, it is important to screen for and treat deficiency in these patients. The Cystic Fibrosis Foundation has issued guidelines for the treatment of vitamin D deficiency, targeting serum levels of 25-hydroxyvitamin D of at least 30 ng/ml. The guidelines offer age-specific escalating dose regimens depending on serum vitamin D levels, with monitoring at 12- week intervals after changing therapy. They address the literature on alternative vitamin D sources, such as UV lamps, ideal formulations (cholecalciferol in preference to ergocalciferol), and optimal vehicles of administration. Despite these detailed recommendations, most centers are still unable to achieve in-target serum vitamin D levels for many of their patients. Future research examining ideal treatment regimens to achieve serum targets and maximize clinical effects are needed. Moreover, it is unknown whether vitamin D sufficiency will be easier to achieve on new triple therapy cystic fibrosis drug combinations, and how these drugs will contribute to vitamin D-related clinical outcomes.
维生素D缺乏在普通人群中很常见,在囊性纤维化患者中更为普遍。由于多种原因,包括吸收不良、脂肪量减少、维生素D的25-羟化作用降低、阳光照射减少、维生素D结合蛋白减少以及接触增加分解代谢的药物,囊性纤维化患者的维生素D缺乏情况会加剧。反过来,维生素D缺乏会导致骨骼健康不佳。此外,它可能会以肺功能恶化、病原体定植增加和肺部病情加重的形式导致肺部功能下降。由于维生素D缺乏与囊性纤维化患者多器官系统的负面临床影响相关,因此对这些患者进行维生素D缺乏的筛查和治疗很重要。囊性纤维化基金会已发布维生素D缺乏的治疗指南,目标是血清25-羟基维生素D水平至少达到30 ng/ml。该指南根据血清维生素D水平提供了针对不同年龄段的递增剂量方案,并在改变治疗后每12周进行监测。指南还讨论了关于替代维生素D来源的文献,如紫外线灯、理想配方(优先选择胆钙化醇而非麦角钙化醇)以及最佳给药载体。尽管有这些详细建议,但大多数中心仍无法使许多患者的血清维生素D水平达到目标。需要开展进一步研究以探究实现血清目标并最大化临床效果的理想治疗方案。此外,尚不清楚在新的三联疗法囊性纤维化药物组合中是否更容易实现维生素D充足,以及这些药物将如何影响与维生素D相关的临床结果。