Language：English   Publishing type：Research paper (scientific journal)  

DOI： 10.1097/WNR.0000000000000068

Language：English  

Language：English  

Language：English   Publishing type：Research paper (scientific journal)   Publisher：Japanese Journal of Ophthalmology  

PURPOSE: The Retinitis Pigmentosa Progression and Inflammatory Marker Registry (RP-PRIMARY) is intended as a prospective observational study aimed at establishing sensitive outcome measures to detect the efficacy of anti-inflammatory agents in future clinical trials. The following is the RP-PRIMARY study protocol. STUDY DESIGN: Prospective, multicenter study. METHODS: We will recruit 100 patients with typical RP (any genetic mutation) and the following characteristics: age 20-70 years; mean retinal sensitivity ≥ 10 dB at 12 central points on Humphrey 10-2 visual field tests; central foveal thickness ≤ 250 μm on optical coherence tomography (OCT); and no ocular complications unrelated to RP or serious systemic complications. Early Treatment Diabetic Retinopathy Study (ETDRS). visual acuity, Humphrey 10-2 visual field tests, OCT, and fundus autofluorescence imaging will be performed every 3 months for 2 years. Inflammatory indices such as aqueous flare values, high-sensitivity C-reactive protein (CRP), serum IL-8, and CD14/16 inflammatory monocyte proportion will be measured every year. The primary endpoint will be the progression rate of retinal sensitivity loss on the Humphrey 10-2 visual field tests. The secondary endpoints will be the rate of decline of each parameter and its association with inflammatory indices. Standard-operation-procedure documents were prepared for all study procedures, and consultations with the regulatory agency were conducted to ensure the data reliability for future use in clinical trials. CONCLUSIONS: Detailed registry data on the natural history and inflammatory profile of RP will be useful in designing study protocols for anti-inflammatory therapy for RP and as natural history data for drug applications.

DOI： 10.1007/s10384-025-01179-2

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PubMed

Language：English   Publishing type：Research paper (scientific journal)   Publisher：Elsevier BV  

The epidemiology and etiology of facial onset sensory and motor neuronopathy (FOSMN), a rare syndrome that initiates with facial sensory disturbances followed by bulbar symptoms, remain unknown. To estimate the prevalence of FOSMN in Japan and establish the characteristics of this disease, we conducted a nationwide epidemiological survey. In the primary survey, we received answers from 604 facilities (49.8%), leading to an estimated number of 35.8 (95% confidential interval: 21.5–50.2) FOSMN cases in Japan. The secondary survey collected detailed clinical and laboratory data from 21 cases. Decreased or absent corneal and pharyngeal reflexes were present in over 85% of the cases. Electrophysiological analyses detected blink reflex test abnormalities in 94.1% of the examined cases. Immunotherapy was administered in 81% of cases and all patients received intravenous immunoglobulin. Among them, 35.3% were judged to have temporary beneficial effects evaluated by the physicians in charge. Immunotherapy tended to be effective in the early stage of disease. The spreading pattern of motor and sensory symptoms differed between cases and the characteristics of the motor-dominant and sensory-dominant cases were distinct. Cases with motor-dominant progression appeared to mimic amyotrophic lateral sclerosis. This is the first nationwide epidemiological survey of FOSMN in Japan. The clinical course of FOSMN is highly variable and motor-dominant cases developed a more severe condition than other types of cases. Because clinical interventions tend to be effective in the early phase of the disease, an early diagnosis is desirable.

DOI： 10.1016/j.jns.2024.122957

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Language：Japanese   Publisher：(株)日本プランニングセンター  

神経筋疾患を対象とした臨床試験における患者報告アウトカム(Patient-Reported Outcome:PRO)の利用状況を明らかにすることを目的に、グローバルな臨床試験登録データベースであるClinicalTrials.govを用いて、筋萎縮性側索硬化症など神経筋疾患の中でも運動機能障害をきたす代表的な9疾患を対象に検索を行い、評価項目にPROを含む臨床試験(PRO関連臨床試験)の割合について、疾患および期間別に比較した。その結果、最もPRO関連臨床試験数の割合が高い疾患は重症筋無力症で、2017～2021年に開始された臨床試験の約8割で評価項目にPROが使用されていた。一方、PRO関連臨床試験の割合が低い疾患は、筋ジストロフィーおよびハンチントン病で、2017～2021年に開始された臨床試験におけるPRO使用の割合は、いずれも3割以下であった。また、筋萎縮性側索硬化症に対するPRO関連臨床試験について、期間別に検討した結果、2007年～2011年の5年間に比べ2017～2021年の5年間では、PRO関連臨床試験は1.7倍に増加しており、前者の期間ではほとんどの臨床試験で包括的(疾患特異的ではない)PRO尺度が使用されていたが、2012年以降に開始された臨床試験では、約7割で疾患特異的PRO尺度が使用されていることが分かった。

Language：English   Publishing type：Research paper (scientific journal)  

DOI： https://doi.org/10.3390/ijms232416046

Language：English   Publishing type：Research paper (scientific journal)  

We developed the Forced Vital Capacity Decline Pattern scale (FVC-DiP) using a large pooled cohort of ALS patients

DOI： https://doi.org/10.1016/j.jns.2022.120389

Language：English   Publishing type：Research paper (scientific journal)  

DOI： https://doi.org/10.1136/bmjopen-2022-061225

Language：English   Publisher：BMJ Open  

Introduction Chronic thromboembolic pulmonary hypertension (CTEPH) is a complication of prior pulmonary thromboembolism (PE), caused by incomplete clot dissolution after PE. In patients with CTEPH, lifelong anticoagulation is mandatory to prevent recurrence of PE and secondary in situ thrombus formation. Warfarin, a vitamin K antagonist, is commonly used for anticoagulation in CTEPH based on historical experience and evidence. The anticoagulant activity of warfarin is affected by food and drug interactions, requiring regular monitoring of prothrombin time. The lability of anticoagulant effect often results in haemorrhagic and thromboembolic complications. Thus, lifelong warfarin is a handicap in terms of safety and convenience. Currently, the use of direct oral anticoagulants (DOACs) in CTEPH has increased with the advent of four DOACs. The safety of DOACs is superior to warfarin, with less intracranial bleeding in patients with non-valvular atrial fibrillation and venous thromboembolism. Edoxaban, the latest DOAC, also has proven efficacy and safety for those diseases in two large clinical trials; the ENGAGE-AF trial and HOKUSAI-VTE trial. The present trial seeks to evaluate whether edoxaban is non-inferior to warfarin in preventing worsening of CTEPH. Methods and analysis The KABUKI trial (is an investigator-initiated, multicentre, phase 3, randomised, single-blind, parallel-group, warfarin-controlled, non-inferiority trial to evaluate the efficacy and safety of edoxaban versus warfarin (vitamin K Antagonist) in subjects with chronic thromBoembolic pUlmonary hypertension taking warfarin (vitamin K antagonIst) at baseline) is designed to prove the non-inferiority of edoxaban to warfarin in terms of efficacy and safety in patients with CTEPH. Ethics and dissemination This study is approved by the Institutional Review Board of each participating institution. The findings will be published in a peer-reviewed journal, including positive, negative and inconclusive results. Trial registration number NCT04730037. Protocol version This paper was written per the study protocol V.4.0, dated 29 January 2021.

DOI： 10.1136/bmjopen-2022-061225

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Language：English   Publishing type：Research paper (scientific journal)  

DOI： 10.1038/s41598-021-96064-6

Language：Japanese   Publishing type：Research paper (scientific journal)  

Event date： 2022.5

Language：English  

Venue：東京   Country：Japan  

Event date： 2021.12

Language：Japanese  

Country：Other  

Event date： 2021.5

Language：Japanese  

Country：Japan  

Event date： 2026.3

Language：English   Presentation type：Poster presentation  

Country：United States  

Event date： 2026.2

Language：English   Presentation type：Poster presentation  

Event date： 2026.2

Language：Japanese   Presentation type：Public lecture, seminar, tutorial, course, or other speech  

Country：Japan  

Event date： 2026.1

Language：Japanese   Presentation type：Public lecture, seminar, tutorial, course, or other speech  

Country：Japan  

Event date： 2025.10

Language：Japanese   Presentation type：Public lecture, seminar, tutorial, course, or other speech  

Venue：東京   Country：Japan  

Event date： 2025.10

Language：English   Presentation type：Poster presentation  

Country：Korea, Republic of  

Event date： 2025.9

Language：Japanese   Presentation type：Poster presentation  

Event date： 2025.9

Language：Japanese   Presentation type：Public lecture, seminar, tutorial, course, or other speech  

Event date： 2025.8

Language：Japanese  

Event date： 2025.7

Language：Japanese   Presentation type：Symposium, workshop panel (nominated)  

Venue：佐賀   Country：Japan  

Event date： 2025.5

Language：English  

Venue：Osaka   Country：Japan  

Event date： 2024.11

Language：Japanese   Presentation type：Oral presentation (invited, special)  

Event date： 2024.10

Language：Japanese  

Event date： 2024.5

Language：English  

Event date： 2024.5

Language：English   Presentation type：Oral presentation (general)  

Event date： 2024.3

Language：Japanese  

Venue：日本（大阪）   Country：Japan  

Event date： 2023.12

Language：English  

Country：Switzerland  

Event date： 2023.11

Language：Japanese  

Venue：日本（名古屋）   Country：Japan  

Event date： 2023.11

Language：English  

Country：United States  

Event date： 2023.9

Language：Japanese  

Country：Japan  

Event date： 2023.9

Language：Japanese  

Country：Japan  

Event date： 2022.9

Language：Japanese  

Venue：千葉   Country：Japan  

Event date： 2022.5

Language：Japanese  

Venue：東京   Country：Japan  

Event date： 2022.5

Language：English  

Country：Other  

Event date： 2021.9

Language：English  

Country：Japan