Ikeda Azusa, Tsuji Megumi, Goto Tomohide, Iai Mizue
Institution for Children with Profound Multiple Disabilities, Kanagawa Children's Medical Center, Japan; Department of Neurology, Kanagawa Children's Medical Center, Japan.
Institution for Children with Profound Multiple Disabilities, Kanagawa Children's Medical Center, Japan; Department of Neurology, Kanagawa Children's Medical Center, Japan.
Brain Dev. 2018 Aug;40(7):558-565. doi: 10.1016/j.braindev.2018.03.006. Epub 2018 Apr 7.
Non-invasive positive pressure ventilation (NPPV) in children has recently increased worldwide and is used not only for neuromuscular diseases but for various other diseases. However, there have been few observational studies on long-term NPPV in children in Japan.
Based on medical records, we retrospectively evaluated patients aged ≤20 years who were initiated long-term NPPV at our hospital from January 2001 to December 2015.
A total of 53 patients on long-term NPPV were identified; 38 (72%) had severe motor and intellectual disabilities (SMID). Compared to those with non-neuromuscular diseases, those with neuromuscular diseases had significantly more planned initiations and less frequent use of oxygen. Regarding patient outcome, 34 patients continued NPPV (64%), and there were three discontinues (6%), seven tracheostomies (13%), and nine deaths (17%). The continuation rate was high among those with neuromuscular disorders (15/19 cases, 79%) and that of tracheotomy was high in those with metabolic/degenerative diseases (3/9 cases, 33%). Ten patients transitioned to adult care, accounting for 29% of the 34 continuing patients.
This is the first observational study on long-term NPPV use in children in Japan that examined outcomes in patients with a range of disorders. The initiation situation, management, and outcomes differed between patients with neuromuscular and non-neuronal muscular diseases. Long-term use of NPPV is possible in many cases, including children with SMID, but can be challenging to continue in patients with progressive diseases such as metabolic/degenerative diseases. Careful discussions regarding the management of each patient are necessary.
近年来，无创正压通气（NPPV）在全球范围内应用于儿童的情况有所增加，不仅用于神经肌肉疾病，还用于其他多种疾病。然而，在日本，针对儿童长期使用NPPV的观察性研究较少。
基于病历，我们回顾性评估了2001年1月至2015年12月在我院开始长期使用NPPV的20岁及以下患者。
共确定53例长期使用NPPV的患者；其中38例（72%）有严重运动和智力残疾（SMID）。与非神经肌肉疾病患者相比，神经肌肉疾病患者的计划启动次数明显更多，吸氧频率更低。关于患者预后，34例患者继续使用NPPV（64%），3例停用（6%），7例行气管切开术（13%），9例死亡（17%）。神经肌肉疾病患者的继续使用率较高（19例中的15例，79%），代谢/退行性疾病患者的气管切开率较高（9例中的3例，33%）。10例患者转至成人护理，占34例继续使用患者的29%。
这是日本第一项关于儿童长期使用NPPV的观察性研究，该研究调查了一系列疾病患者的预后情况。神经肌肉疾病和非神经肌肉疾病患者在启动情况、管理和预后方面存在差异。在许多情况下，包括患有SMID的儿童，长期使用NPPV是可行的，但对于代谢/退行性疾病等进展性疾病患者，继续使用可能具有挑战性。有必要针对每位患者的管理进行仔细讨论。
