NF1 plexiform neurofibroma growth rate by volumetric MRI - Relationship to age and body weight
NF1 plexiform neurofibroma growth rate by volumetric MRI - Relationship to age and body weight
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DOI：
10.1212/01.wnl.0000250332.89420.e6
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发表时间：
2007-02-27
期刊：
影响因子：
9.9
通讯作者：
Widemann, B. C.
中科院分区：
文献类型：
--
作者：
Dombi, E.;Solomon, J.;Widemann, B. C.
目的：利用一种灵敏、自动化的体积MRI分析方法，纵向分析1型神经纤维瘤病和不能手术、有症状或进展性神经纤维瘤的儿童和年轻人的丛状神经纤维瘤（PN）体积变化与年龄和身体生长的关系。方法：我们纳入了年龄在25岁及以下的PNs患者，这些患者进入了自然史研究或治疗试验，并且在bb0 = 16个月以上进行了体积MRI检查。结果：我们研究了49例患者（中位年龄8.3岁），61例PNs，中位评估期为34个月（18至70个月）。不同患者的PN生长速率不同，但患者体内的PN生长速率不变。34例患者（69%）在观察期内PN体积增加>= 20%。随着时间的推移，PN体积比体重增加得更快（p = 0.026）。年轻患者的PN生长速度最快。结论：丛状神经纤维瘤体积增大是一个现实而有意义的试验终点。在大多数患者中，丛状神经纤维瘤的生长速度超过了身体的生长速度。最年轻的患者具有最快的丛状神经纤维瘤生长速度，临床药物开发应针对这一人群。丛状神经纤维瘤的临床试验应考虑年龄分层。
Objective: To longitudinally analyze changes in plexiform neurofibroma (PN) volume in relation to age and body growth in children and young adults with neurofibromatosis type 1 and inoperable, symptomatic, or progressive PNs, using a sensitive, automated method of volumetric MRI analysis. Methods: We included patients 25 years of age and younger with PNs entered in a natural history study or in treatment trials who had volumetric MRI over >= 16 months. Results: We studied 49 patients (median age 8.3 years) with 61 PNs and a median evaluation period of 34 months (range 18 to 70). The PN growth rates varied among patients, but were constant within patients. Thirty-four patients (69%) experienced >= 20% increase in PN volume during the observation period. PN volume increased more rapidly than body weight over time (p = 0.026). Younger patients had the most rapid PN growth rate. Conclusions: Volume increase of plexiform neurofibromas is a realistic and meaningful trial endpoint. In most patients plexiform neurofibroma growth rate exceeded body growth rate. The youngest patients had the fastest plexiform neurofibroma growth rate, and clinical drug development should be directed toward this population. Age stratification for clinical trials for plexiform neurofibromas should be considered.