Stalvey Michael S, Havasi Viktoria, Tuggle Katherine L, Wang Dezhi, Birket Susan, Rowe Steve M, Sorscher Eric J
Department of Pediatrics, University of Alabama at Birmingham, Birmingham, AL, United States of America.
Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, AL, United States of America.
PLoS One. 2017 Nov 30;12(11):e0188497. doi: 10.1371/journal.pone.0188497. eCollection 2017.
Reduced growth and osteopenia are common in individuals with cystic fibrosis (CF). Additionally, improved weight and height are associated with better lung function and overall health in the disease. Mechanisms for this reduction in growth are not understood. We utilized a new CFTR knockout rat to evaluate growth in young CF animals, via femur length, microarchitecture of bone and growth plate, as well as serum IGF-I concentrations.
Femur length was measured in wild-type (WT) and SD-CFTRtm1sage (Cftr-/-) rats, as a surrogate marker for growth. Quantitative bone parameters in Cftr-/- and WT rats were measured by micro computed tomography (micro-CT). Bone histomorphometry and cartilaginous growth plates were analyzed. Serum IGF-I concentrations were also compared.
Femur length was reduced in both Cftr-/- male and female rats compared to WT. Multiple parameters of bone microarchitecture (of both trabecular and cortical bone) were adversely affected in Cftr-/- rats. There was a reduction in overall growth plate thichkness in both male and female Cftr-/- rats, as well as hypertrophic zone thickness and mean hypertrophic cell volume in male rats, indicating abnormal growth characteristics at the plate. Serum IGF-I concentrations were severely reduced in Cftr-/- rats compared to WT littermates.
Despite absence of overt lung or pancreatic disease, reduced growth and bone content were readily detected in young Cftr-/- rats. Reduced size of the growth plate and decreased IGF-I concentrations suggest the mechanistic basis for this phenotype. These findings appear to be intrinsic to the CFTR deficient state and independent of significant clinical confounders, providing substantive evidence for the importance of CFTR on maintinaing normal bone growth.
生长发育迟缓及骨质减少在囊性纤维化(CF)患者中很常见。此外,体重和身高的改善与该疾病中更好的肺功能及整体健康状况相关。生长发育迟缓的机制尚不清楚。我们利用一种新的CFTR基因敲除大鼠,通过股骨长度、骨微结构和生长板以及血清IGF-I浓度来评估幼年CF动物的生长情况。
测量野生型(WT)和SD-CFTRtm1sage(Cftr-/-)大鼠的股骨长度,作为生长的替代指标。通过微型计算机断层扫描(micro-CT)测量Cftr-/-和WT大鼠的定量骨参数。分析骨组织形态计量学和软骨生长板。还比较了血清IGF-I浓度。
与WT相比,Cftr-/-雄性和雌性大鼠的股骨长度均缩短。Cftr-/-大鼠的骨微结构(小梁骨和皮质骨)的多个参数受到不利影响。雄性和雌性Cftr-/-大鼠的整体生长板厚度均降低,雄性大鼠的肥大带厚度和平均肥大细胞体积也降低,表明生长板处存在异常生长特征。与WT同窝仔鼠相比,Cftr-/-大鼠的血清IGF-I浓度严重降低。
尽管没有明显的肺部或胰腺疾病,但在幼年Cftr-/-大鼠中很容易检测到生长发育迟缓及骨量减少。生长板尺寸减小和IGF-I浓度降低提示了该表型的机制基础。这些发现似乎是CFTR缺陷状态所固有的,且独立于显著的临床混杂因素,为CFTR在维持正常骨骼生长中的重要性提供了实质性证据。