Clinical course of neurogenic bladder dysfunction in human T-cell leukemia virus type-1-associated myelopathy/tropical spastic paraparesis: a nationwide registry study in Japan

Orphanet Journal of Rare Diseases

Ayako Takata¹  Naoko Yagishita²  Kenichiro Tanabe²  Natsumi Araya²  Satoko Aratani³  Naoki Iijima⁴  Tomoo Sato⁵  Yoshihisa Yamano⁵  Junji Yamauchi⁵ 

[1] Department of Preventive Medicine, St. Marianna University School of Medicine, Kawasaki, Japan;Department of Rare Diseases Research, Institute of Medical Science, St. Marianna University School of Medicine, Kawasaki, Japan;Department of Rare Diseases Research, Institute of Medical Science, St. Marianna University School of Medicine, Kawasaki, Japan;LSI Medience Co., Tokyo, Japan;Division of Neurology, Department of Internal Medicine, St. Marianna University School of Medicine, 2168511, Kawasaki, Japan;Division of Neurology, Department of Internal Medicine, St. Marianna University School of Medicine, 2168511, Kawasaki, Japan;Department of Rare Diseases Research, Institute of Medical Science, St. Marianna University School of Medicine, Kawasaki, Japan;

关键词: Human T-cell leukemia virus type 1;    Human T-cell leukemia virus type 1-associated myelopathy/tropical spastic paraparesis;    Neurogenic bladder;    Urinary symptom score;    Mirabegron;   

DOI  :  10.1186/s13023-021-01990-3

来源: Springer

【 摘 要 】

BackgroundMost patients with human T-cell leukemia virus type 1-associated myelopathy/tropical spastic paraparesis (HAM/TSP) develop neurogenic bladder dysfunction. However, longitudinal changes and treatment effects remain poorly understood. This study aimed to characterize the clinical course of urinary dysfunction in this population.MethodsThis prospective observational study included 547 patients enrolled in HAM-net, a nationwide registry for HAM/TSP in Japan. Urinary dysfunction severity was evaluated using the HAM/TSP-bladder dysfunction symptom score (HAM-BDSS) and the HAM/TSP-bladder dysfunction severity grade (HAM-BDSG). These specific measures were recently developed for assessing urinary dysfunction in HAM/TSP. We analyzed longitudinal changes over a 6-year follow-up period, associations between urinary and gait dysfunction, and treatment efficacy of urinary catheterization and mirabegron (a β3-adrenergic agonist for overactive bladder symptoms).ResultsThe mean (standard deviation [SD]) age and disease duration at enrollment were 61.9 (10.7) years and 16.6 (11.6) years, respectively, and 74.6% of patients were women. Only 8.0% were free from urinary symptoms (HAM-BDSG 0), 65.4% had urinary symptoms or were on medication (HAM-BDSG I), and 23.2% and 3.3% used intermittent and indwelling catheters (HAM-BDSG II and III), respectively. HAM-BDSG and BDSS were worse in patients with greater gait dysfunction (p < 0.001 for both). During the 6-year follow-up, 66.7% of patients with HAM-BDSG 0 developed new urinary symptoms. Of those with HAM-BDSG I at enrollment, 10.8% started using urinary catheters. Importantly, HAM-BDSS significantly improved after initiating catheterization (mean [SD] change, − 8.93 [10.78], p < 0.001). The number of patients receiving mirabegron increased in the fourth year. Multivariable linear regression analysis significantly associated mirabegron with improvement in HAM-BDSS (− 5.82, 95% confidence interval − 9.13 to − 2.51, p = 0.001).ConclusionsUrinary dysfunction affected 92% of patients and progressed over the 6-year follow-up. Urinary symptoms were more severe in patients with poorer gait function. Urinary catheterization and mirabegron were effective in relieving symptoms. Effective utilization of real-world data is key to establishing evidence for rare diseases, such as HAM/TSP.

【 授权许可】

CC BY