Dassen Amber R, van Schaik Jiska, van den Munckhof Pepijn, Schuurman P R, Hoving Eelco W, van Santen Hanneke M
Department of Pediatric Endocrinology, Wilhelmina Children's Hospital, Utrecht, the Netherlands.
Department of Pediatric Oncology, Princess Máxima Center, Utrecht, the Netherlands.
Heliyon. 2023 Mar 9;9(3):e14411. doi: 10.1016/j.heliyon.2023.e14411. eCollection 2023 Mar.
Hypothalamic dysfunction may result in morbid obesity as a consequence of decreased energy expenditure, decreased feelings of satiety, and increased fat storage. In patients with hypothalamic dysfunction, neurobehavioral dysfunction is also often present. Currently, no effective treatment has been found for hypothalamic obesity (HO). We hypothesize that deep brain stimulation (DBS) may be an effective treatment for patients with hypothalamic dysfunction, aiming to treat HO as well as the neurobehavioral dysfunction.
A systematic search was conducted in the PubMed, EMBASE and Cochrane Library databases for studies published until May 2022 reporting on DBS for the treatment of HO.
Three studies met the predetermined inclusion criteria, with in total six patients treated with DBS for HO, of which five patients with Prader-Willi syndrome (PWS) and one patient with HO after treatment for craniopharyngioma (CP). Targets of DBS included the lateral hypothalamic area (LHA) and the nucleus accumbens (NAcc). In patients with PWS, LHA-DBS was associated with a mean increase of Body Mass Index (BMI) (+5.8%), with no change in hormonal levels, results of blood workup, sleep, or neuropsychological evaluation. In the patient with CP, NAcc-DBS was associated with a decrease in BMI (-8.7%) and a subjective increase in mental health, energy and willingness to act, and no feeling of increased appetite. No objective measurements on neurobehavioral function were reported. No severe adverse events were reported in these cases. Mild to moderate adverse events included hypomanic symptoms and infection. All patients with a described follow-up period ( = 5) were able to sustain the treatment for at least 6 months with few interruptions.
There is limited research reporting on DBS for HO. The effectiveness differed across studies and the evidence is limited. Although there may be potential for DBS treatment in the severe-refractory condition of HO in patients with CP, more research is needed for target selection and evaluation of effectiveness.
下丘脑功能障碍可能由于能量消耗减少、饱腹感降低和脂肪储存增加而导致病态肥胖。在下丘脑功能障碍患者中，神经行为功能障碍也经常存在。目前，尚未发现针对下丘脑性肥胖（HO）的有效治疗方法。我们假设，深部脑刺激（DBS）可能是治疗下丘脑功能障碍患者的有效方法，旨在治疗HO以及神经行为功能障碍。
在PubMed、EMBASE和Cochrane图书馆数据库中进行了系统检索，以查找截至2022年5月发表的关于DBS治疗HO的研究。
三项研究符合预定的纳入标准，共有6例患者接受了DBS治疗HO，其中5例患有普拉德-威利综合征（PWS），1例在颅咽管瘤（CP）治疗后患有HO。DBS的靶点包括下丘脑外侧区（LHA）和伏隔核（NAcc）。在PWS患者中，LHA-DBS与体重指数（BMI）平均增加（+5.8%）相关，激素水平、血液检查结果、睡眠或神经心理学评估均无变化。在CP患者中，NAcc-DBS与BMI降低（-8.7%）以及心理健康、能量和行动意愿主观增加相关，且无食欲增加的感觉。未报告神经行为功能的客观测量结果。这些病例中未报告严重不良事件。轻度至中度不良事件包括轻躁狂症状和感染。所有有随访期描述（n = 5）的患者都能够持续治疗至少6个月，很少中断。
关于DBS治疗HO的研究报告有限。不同研究的有效性存在差异，证据有限。尽管DBS治疗CP患者HO的严重难治性情况可能有潜力，但在靶点选择和有效性评估方面还需要更多研究。
