Thakur Mrinali, Pande Vineeta, Mane Shailaja V
Pediatrics, Dr. D. Y. Patil Medical College, Hospital and Research Center, Dr. D. Y. Patil Vidyapeeth (Deemed to Be University), Pune, IND.
Cureus. 2024 Aug 17;16(8):e67050. doi: 10.7759/cureus.67050. eCollection 2024 Aug.
Vanishing white matter disease (VWMD), also known as childhood ataxia with central hypoventilation, is a rare leukodystrophy that is inherited in an autosomal recessive manner. It is triggered by either traumatic brain injury or a febrile episode. The patient was a three-year-old male child who presented with complaints of fever and diarrhea for three days, along with a paucity of movements of both upper and lower limbs, with decreased tone and diminished reflexes. Previously the child had normal developmental milestones. MRI done showed T2 hyperintensities involving bilateral peri-ventricular white matter, deep white matter, and bilateral sub-cortical U-fibres in bilateral fronto-parietal region and bilateral cerebellar hemispheres. The bilateral external capsule and posterior limb of the internal capsule were also involved. All these findings were likely suggestive of leukodystrophy. Whole exome sequencing was done and a homozygous mutation of the eIF2B5 was noted, which confirmed the diagnosis of VWMD. The physician must keep in mind this diagnosis in cases of sudden motor abnormalities following any event and proceed for early management such as controlling febrile episodes with liberal use of antibiotics and antipyretics, along with prevention of traumatic brain injury or any stressful event. There is no definitive treatment. Management of these patients includes symptomatic and supportive care. Patients with this disease (VMND) have a poor quality of life as the disease progresses and eventually, death occurs.
消失性白质病(VWMD),也称为伴有中枢性低通气的儿童共济失调,是一种罕见的脑白质营养不良,以常染色体隐性方式遗传。它由创伤性脑损伤或发热发作引发。该患者是一名三岁男童,主诉发热和腹泻三天,同时伴有双上肢和双下肢活动减少、肌张力降低和反射减弱。该儿童之前发育里程碑正常。MRI显示双侧额顶叶区域和双侧小脑半球的双侧脑室周围白质、深部白质以及双侧皮质下U形纤维T2高信号。双侧外囊和内囊后肢也受累。所有这些发现可能提示脑白质营养不良。进行了全外显子组测序,发现eIF2B5存在纯合突变,这证实了VWMD的诊断。医生在任何事件后出现突然运动异常的情况下必须牢记这一诊断,并进行早期管理,如大量使用抗生素和退烧药控制发热发作,同时预防创伤性脑损伤或任何应激事件。目前尚无确切的治疗方法。这些患者的管理包括对症和支持治疗。随着疾病进展,患有这种疾病(VMND)的患者生活质量较差,最终会死亡。