Department of Neurology, Faculdade de Medicina da Universidade de São Paulo (FMUSP), São Paulo, SP, Brazil.
Pediatr Neurol. 2023 Jun;143:1-5. doi: 10.1016/j.pediatrneurol.2023.01.017. Epub 2023 Feb 7.
Hypoglycemia has been reported in patients with LAMA2-CMD, but the frequency, risk factors, and correlation to genotype/phenotype have not been systematically assessed to date.
A retrospective cohort study was performed on 48 patients with LAMA2-CMD. Patients were divided into two groups: a hypoglycemic group, with at least one episode of hypoglycemia, and a nonhypoglycemic group. The groups were compared according to gait function, epilepsy, intellectual disability, constipation, gastroesophageal reflux, gastrostomy, weight percentile, scoliosis, the use of a ventilator device, the use of a feeding device, neuromuscular disease swallowing status scale, and type of mutation.
Fifteen patients (31.2%) presented with at least one episode of symptomatic hypoglycemia and eight (16.6% of the cohort) had two or more episodes. All patients who had hypoglycemia were in the nonambulant group. We observed a correlation between gait, the use of ventilator and feeding devices, and swallow function with hypoglycemia. Patients with extremely low weight were five times more likely to have recurrent episodes of hypoglycemia. The presence of at least one missense variant appears to be associated with a lower risk of hypoglycemia.
Patients with LAMA2-CMD are at risk of hypoglycemia. The risk is more relevant in patients with severe phenotype and patients with loss-of-function variants. For patients with extremely low weight, the risk is higher. Blood glucose should be actively measured in patients who are fasting or have infections, and health care providers should be prepared to identify and treat these patients.
已报道 LAMA2-CMD 患者出现低血糖症,但迄今为止尚未系统评估其发生频率、风险因素及其与基因型/表型的相关性。
对 48 例 LAMA2-CMD 患者进行了回顾性队列研究。患者分为低血糖组和非低血糖组,低血糖组至少有一次低血糖发作。根据步态功能、癫痫、智力障碍、便秘、胃食管反流、胃造口术、体重百分位、脊柱侧凸、呼吸机设备使用、喂养设备使用、神经肌肉疾病吞咽状态量表和突变类型对两组进行比较。
15 例(31.2%)患者至少出现一次有症状低血糖发作,8 例(占队列的 16.6%)患者出现两次或更多次低血糖发作。所有出现低血糖的患者均未行走。我们观察到步态、呼吸机和喂养设备的使用以及吞咽功能与低血糖之间存在相关性。体重极低的患者发生反复低血糖发作的可能性增加了五倍。至少存在一个错义变异似乎与低血糖风险降低相关。
LAMA2-CMD 患者存在低血糖风险。严重表型和功能丧失变异患者的风险更高。对于体重极低的患者,风险更高。对于禁食或感染的患者,应积极测量血糖,医护人员应做好识别和治疗这些患者的准备。