Antonini Ursula, Soldini Emiliano Alessandro, D'Apuzzo Vincenzo, Brunner Rainhald, Ramelli Gian Paolo
Institute Provvida Madre, Balerna, Switzerland.
Brain Dev. 2013 Jun;35(6):548-54. doi: 10.1016/j.braindev.2012.07.019. Epub 2012 Sep 1.
The aim of this study was to evaluate the longitudinal neurodevelopmental evolution in children with severe non-progressive encephalopathy.
Between 1984 and 2005, 17 patients diagnosed with severe non-progressive encephalopathy under the care of the Institute Provvida Madre underwent neurodevelopmental evaluation on an annual basis for at least five consecutive years using the Munich Functional Developmental Diagnostics (MFDD). The severity of each patient's encephalopathy was assessed using the Capacity Profile (CAP). Longitudinal development trends were assessed by means of linear regression analysis, while the degree of discontinuity of the development trajectories was quantified using the Mean Absolute Deviation from Perfect Linear Development (MADPLD). Spearman's rank correlation coefficient and the Mann-Whitney test have been used to investigate the statistical significance of the relationships among the various parameters.
We found that patients with severe non-progressive encephalopathy showed, on average, a linear maturation of 1.5-2.5months per year, irrespective of the neurodevelopmental area considered. Nevertheless, we also discovered that the development trajectories could be discontinuous. Indeed, a given child can show no development sign at all for many years and then suddenly encounter a "development jump", especially in the active language and autonomy areas. However, the long-term development linearity hypothesis seemed to hold true in our study. We also found evidences suggesting that faster development in a given domain could be linked to faster development in other domains, that higher discontinuity in a given area could be associated with higher discontinuity in other areas and that higher degrees of discontinuity could be related to lower developmental evolutions.
The main findings of this study are important for physicians to form prognoses, counsel effectively and appropriately target therapeutic interventions. In this perspective, there is a strong need to collect long-term repeated follow-up data concerning this group of infants in order to reinforce the findings presented. In fact, these results should be considered as a starting point for further research because they are based on a limited number of patients and more data are needed to confirm the findings.
本研究旨在评估重度非进行性脑病患儿的纵向神经发育演变情况。
1984年至2005年间，在Provvida Madre研究所接受治疗的17例被诊断为重度非进行性脑病的患者，每年至少连续五年使用慕尼黑功能发育诊断法（MFDD）进行神经发育评估。使用能力概况（CAP）评估每位患者脑病的严重程度。通过线性回归分析评估纵向发育趋势，同时使用离完美线性发育的平均绝对偏差（MADPLD）对发育轨迹的不连续程度进行量化。使用Spearman等级相关系数和Mann-Whitney检验来研究各种参数之间关系的统计学意义。
我们发现，无论考虑哪个神经发育领域，重度非进行性脑病患者平均每年有1.5 - 2.5个月的线性成熟。然而，我们也发现发育轨迹可能是不连续的。事实上，某个特定儿童可能多年都没有任何发育迹象，然后突然出现“发育跳跃”，尤其是在主动语言和自主领域。然而，长期发育线性假设在我们的研究中似乎成立。我们还发现有证据表明，特定领域的更快发育可能与其他领域的更快发育相关，特定区域的更高不连续性可能与其他区域的更高不连续性相关，并且更高程度的不连续性可能与更低的发育进展相关。
本研究的主要发现对于医生进行预后判断、有效咨询以及合理确定治疗干预目标具有重要意义。从这个角度来看，非常有必要收集关于这组婴儿的长期重复随访数据，以加强所呈现的研究结果。实际上，这些结果应被视为进一步研究的起点，因为它们是基于有限数量的患者得出的，需要更多数据来证实这些发现。
