Bockenmeyer J, Chamboredon E, Missud F, Benkerrou M, Holvoët L, Ithier G, Lescoeur B, Yakouben K, Ouachée-Chardin M, Rohrlich P-S, Duval M, Baruchel A, Dalle J-H
Service d'hématologie et immunologie pédiatrique, hôpital Robert-Debré, Assistance publique-Hôpitaux de Paris, 48, boulevard Sérurier, 75935 Paris cedex 19, France.
Arch Pediatr. 2013 Jul;20(7):723-30. doi: 10.1016/j.arcped.2013.04.012. Epub 2013 Jun 12.
Allogeneic hematopoietic stem-cell transplantation (HSCT) is the only curative treatment for sickle cell disease (SCD). Cerebral vasculopathy was the principal indication for transplantation. These children could present impaired neuropsychological development related to different causes, hence the value of exploring their intellectual capacities before and after transplantation.
Prospective longitudinal study from 1992 to 2006 in all transplanted SCD patients. The patients were assessed using Wechsler scales with four different indices: verbal comprehension, perceptual reasoning, working memory, and processing speed (PSI), providing a full-scale intellectual quotient (IQ).
Fifteen SCD patients (8 females and 7 males; mean age, 8.9 years) were evaluated before and 36 and 60 months after transplantation. All were from Africa and lived in France. All patients except 2 had experienced ischemic stroke before HSCT. The median full-scale IQ was 87, 94, and 94 before transplantation and 36 months and 60 months after HSCT, respectively.
At pre-HSCT evaluation, full-scale IQ was considered as "low average". This relatively poor result could be related to impairment of PSI, which reflects frequent graphic and motor abnormalities related to the previous stroke experienced by almost all patients. At 3 years after HSCT, all indices including IQ had increased. Only the PSI had decreased, this observation being potentially related to previous stroke and to the depression frequently experienced by the transplant recipient patient after the acute phase, when the disease is cured. At 5 years after HSCT, the median full-scale IQ was stable and the PSI had increased.
At the end of follow-up, the patients improved their physical and psychological well-being. This allowed them to build projects for the future and to manifest the desire of becoming an adult. Bone marrow transplantation in this cohort of children with SCD and severe cerebral vasculopathy is associated with improved performance as measured by the Wechsler scale.
异基因造血干细胞移植（HSCT）是镰状细胞病（SCD）的唯一治愈性治疗方法。脑血管病变是移植的主要指征。这些儿童可能因不同原因出现神经心理发育受损，因此探索他们移植前后的智力能力具有重要意义。
1992年至2006年对所有接受移植的SCD患者进行前瞻性纵向研究。使用韦氏智力量表对患者进行评估，该量表有四个不同指标：言语理解、知觉推理、工作记忆和处理速度（PSI），可得出全量表智商（IQ）。
对15例SCD患者（8名女性和7名男性；平均年龄8.9岁）在移植前以及移植后36个月和60个月进行了评估。所有患者均来自非洲，居住在法国。除2例患者外，所有患者在HSCT前均经历过缺血性中风。移植前、移植后36个月和60个月的全量表智商中位数分别为87、94和94。
在HSCT前评估时，全量表智商被认为是“低平均水平”。这一相对较差的结果可能与PSI受损有关，PSI反映了几乎所有患者之前中风所导致的频繁的图形和运动异常。HSCT后3年，包括智商在内的所有指标均有所提高。只有PSI有所下降，这一观察结果可能与之前的中风以及移植受者在急性期后疾病治愈时经常经历的抑郁有关。HSCT后5年，全量表智商中位数稳定，PSI有所提高。
随访结束时，患者的身心健康状况有所改善。这使他们能够为未来制定计划，并表现出成为成年人的愿望。在这组患有SCD和严重脑血管病变的儿童中，骨髓移植与韦氏智力量表测量的表现改善相关。
