Baumann Matthias, Korenke G Christoph, Weddige-Diedrichs Almuth, Wilichowski Ekkehard, Hunneman Donald H, Wilken Bernd, Brockmann Knut, Klingebiel Thomas, Niethammer Dietrich, Kühl Jörn, Ebell Wolfram, Hanefeld Folker
Department of Paediatrics and Neuropaediatrics, Georg-August University Göttingen, Robert-Koch-Strasse 40, 37075 Göttingen, Germany.
Eur J Pediatr. 2003 Jan;162(1):6-14. doi: 10.1007/s00431-002-1097-3. Epub 2002 Nov 19.
In an attempt to elucidate prognostic factors, the data on 12 boys who underwent haematopoietic stem cell transplantation (HSCT) for cerebral X-linked adrenoleukodystrophy were evaluated. Two further patients received HSCT but died from transplantation-related complications. The data included neurological examination, neuropsychological testing and magnetic resonance imaging (MRI). Follow-up after HSCT was up to 5.5 years. Six patients showed a moderate to severe clinical deterioration after HSCT including two who died within 6 months. In this group, a MRI severity score of 10 or higher before HSCT was associated with severe impairment and a score of more than 12 was followed by rapid deterioration and death after HSCT. The presence of neurological symptoms before HSCT also affected prognosis. Six patients showed no deterioration in neurological or neuropsychological assessment after HSCT.
our data confirm that haematopoietic stem cell transplantation can stop the progress of demyelination when performed at a critical early stage of the disease. The prognosis in an individual patient for the clinical course after stem cell transplantation can in general be given based on the status before transplantation, although individual patients may show an unexpected course.
为了阐明预后因素，对12例因脑型X连锁肾上腺脑白质营养不良接受造血干细胞移植（HSCT）的男孩的数据进行了评估。另有2例患者接受了HSCT，但死于移植相关并发症。数据包括神经学检查、神经心理学测试和磁共振成像（MRI）。HSCT后的随访时间长达5.5年。6例患者在HSCT后出现中度至重度临床恶化，其中2例在6个月内死亡。在该组中，HSCT前MRI严重程度评分为10或更高与严重损伤相关，评分超过12则在HSCT后迅速恶化并死亡。HSCT前存在神经症状也会影响预后。6例患者在HSCT后神经学或神经心理学评估未出现恶化。
我们的数据证实，造血干细胞移植在疾病的关键早期进行时可以阻止脱髓鞘进程。尽管个别患者可能出现意外病程，但一般可以根据移植前的状况给出个体患者干细胞移植后临床病程的预后。
