Radboud Institute for Health Sciences, Department of Medical Psychology, Radboud Center for Mitochondrial Medicine, Amalia Children's Hospital, Radboud University Medical Center, Geert Grooteplein Zuid 10, P.O. Box 9101, 6500HB, Nijmegen, The Netherlands.
Radboud Institute for Health Sciences, Department of Medical Psychology, Radboud Center for Mitochondrial Medicine, Radboud University Medical Center, Nijmegen, The Netherlands.
Orphanet J Rare Dis. 2020 Mar 24;15(1):76. doi: 10.1186/s13023-020-1342-8.
Mitochondrial diseases (MD) are generally serious and progressive, inherited metabolic diseases. There is a high comorbidity of anxiety and depression and limitations in daily functioning. The complexity and duration of the diagnostic process and lack of knowledge about prognosis leads to uncertainty. In this study, we investigated the psychological well-being of children who are suspected for MD and their parents.
In total 122 children suspected for MD and their parents, received questionnaires as part of standard clinical investigation.
Parent proxy report revealed a lower quality of life (QoL) compared to norms and even more physical problems compared to chronically ill patients. They also reported more behavioral problems in general and more internalizing problems compared to the norms. Most frequent reported somatic complaints were tiredness and pain. Parents did not report enhanced levels of stress regarding parenting and experienced sufficient social support. At the end of the diagnostic process, 5.7% of the children received the genetically confirmed diagnosis of MD, 26% showed non-conclusive abnormalities in the muscle biopsy, 54% did not receive any diagnosis, and the remaining received other diagnoses. Strikingly, children without a diagnosis showed equally QoL and behavioral problems as children with a diagnosis, and even more internalizing problems.
This study highlights the psychological concerns of children with a suspicion of MD. It is important to realize that as well as children with a confirmed diagnosis, children without a diagnosis are vulnerable since explanation for their complaints is still lacking.
线粒体疾病(MD)通常是严重且进行性的遗传性代谢疾病。焦虑和抑郁共病率高,日常功能受限。诊断过程复杂且耗时,且对预后缺乏了解,导致存在不确定性。在这项研究中,我们调查了疑似 MD 的儿童及其父母的心理健康状况。
共有 122 名疑似 MD 的儿童及其父母作为标准临床检查的一部分接受了问卷调查。
父母代表报告显示,生活质量(QoL)较正常值低,甚至比慢性病患者的身体问题更多。与正常值相比,他们还报告了更多的一般行为问题和更多的内化问题。最常报告的躯体抱怨是疲劳和疼痛。父母没有报告在育儿方面的压力增加,并且有足够的社会支持。在诊断过程结束时,5.7%的儿童被基因确诊为 MD,26%的儿童肌肉活检显示非结论性异常,54%的儿童未得到任何诊断,其余儿童得到其他诊断。引人注目的是,没有诊断的儿童与确诊儿童一样存在 QoL 和行为问题,甚至更多的内化问题。
本研究强调了疑似 MD 儿童的心理问题。重要的是要意识到,正如确诊儿童一样,没有诊断的儿童也很脆弱,因为他们的症状仍缺乏解释。