Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute (NCI), National Institutes of Health (NIH), Bethesda, Maryland.
Center for Cancer Research, NCI, NIH, Bethesda, Maryland.
Neuro Oncol. 2020 Sep 29;22(9):1368-1378. doi: 10.1093/neuonc/noaa053.
Understanding the natural history of non-malignant peripheral nerve sheath tumors (PNSTs) in neurofibromatosis type 1 (NF1) is critical to optimal clinical care and the development of meaningful clinical trials.
We longitudinally analyzed growth of plexiform neurofibromas (PNs) and of PNSTs with distinct nodular appearance (distinct nodular lesions [DNLs]) using volumetric MRI analysis in patients enrolled on a natural history study (NCT00924196).
DNLs were observed in 58/122 (45.6%) patients (median 2 DNLs/patient). In DNLs that developed during follow-up, median age of development was 17 years. A moderate negative correlation was observed between the estimated PN growth rate and patients' age at initial MRI (Spearman's r [95% CI]: -0.60 [-0.73, -0.43], n = 70), whereas only a weak correlation was observed for DNLs (Spearman's r [95% CI]: -0.25 [-0.47, 0.004]; n = 61). We observed a moderate negative correlation between tumor growth rate and baseline tumor volume for PNs and DNLs (Spearman's r [95% CI]: -0.52 [-0.67, -0.32] and -0.61 [-0.75, -0.42], respectively). Spontaneous tumor volume reduction was observed in 10 PNs and 7 DNLs (median decrease per year, 3.6% and 7.3%, respectively).
We corroborate previously described findings that most rapidly growing PNs are observed in young children. DNLs tend to develop later in life and their growth is minimally age related. Distinct growth characteristics of PNs and DNLs suggest that these lesions have a different biology and may require different clinical management and clinical trial design. In a subset of PNs and DNLs, slow spontaneous regression in tumor volume was seen.
了解神经纤维瘤病 1 型（NF1）中非恶性周围神经鞘瘤（PNST）的自然史对于优化临床护理和开展有意义的临床试验至关重要。
我们使用容积 MRI 分析，对参加自然史研究（NCT00924196）的患者进行了丛状神经纤维瘤（PNs）和具有明显结节外观的 PNST（明显结节性病变[DNL]）的生长纵向分析。
在 122 例患者中观察到 58 例（45.6%）有 DNL（中位数为每例患者 2 个 DNL）。在随访中发生的 DNL 中，中位发病年龄为 17 岁。在初始 MRI 时，PN 生长率与患者年龄之间观察到中度负相关（Spearman r [95%CI]：-0.60 [-0.73，-0.43]，n=70），而 DNL 仅观察到弱相关（Spearman r [95%CI]：-0.25 [-0.47，0.004]；n=61）。我们观察到 PN 和 DNL 的肿瘤生长率与基线肿瘤体积之间存在中度负相关（Spearman r [95%CI]：-0.52 [-0.67，-0.32]和-0.61 [-0.75，-0.42]）。在 10 个 PNs 和 7 个 DNL 中观察到肿瘤体积自发减少（每年减少的中位数，分别为 3.6%和 7.3%）。
我们证实了以前描述的发现，即大多数生长最快的 PNs 见于幼儿。DNL 往往在生命后期发展，其生长与年龄的关系最小。PN 和 DNL 的不同生长特征表明这些病变具有不同的生物学特性，可能需要不同的临床管理和临床试验设计。在 PNs 和 DNL 的亚组中，观察到肿瘤体积的自发缓慢消退。
