臨床研究等提出・公開システム

Treatment of Neuromyelitis Optica with Mycophenolate Mofetil in Japanese Patients (An open treatment trial of NMO with MMF)

Treatment of Neuromyelitis Optica with Mycophenolate Mofetil in Japanese Patients (An open treatment trial of NMO with MMF)

9

The mean age of 9 patients was 56.4 years old (standard deviation(SD), 8.92 years), all of the patients were women, median disease duration was 68 months (9-84), and the mean number of recurrences over the last 2 years was 1.33 (SD, 1.22), the average number of recurrences in the past is 3.56 (SD, 2.27). The amount of oral prednisolone was 5-10 mg / day for 3 patients and 11-20 mg / day for other 6 patients. The average EDSS score was 2.9 (SD 2.2).

Ten patients were screened. One was excluded by the age limiation, and finally 9 patients were enrolled.

A total of 10 adverse reactions, such as upper respiratory tract infection (2 cases), were reported during the study period, but all were mild (CTCAE Grade 1).

During the study period, 2 patients showed clinical recurrence and dropped out from the study, but 7 (77.8%, 95% CI (0.46%, 97.3%)) achieved the primary endpoint. The average number of recurrences per year during the study period decreased from 0.67 to 0.22 (p < 0.05, paired t test). On the other hand, the amount of oral prednisolone was reduced from an average of 13.3 mg / day to 5.00 mg / day. In addition, EDSS did not show a significant change from 2.43 to 2.35 on average (p = 0.36, paired t test).

MMF was effective for preventing relapse of neuromyelitis optica spectrum disorders with concomitant half dose oral corticosteroid reduction in 7/9 (77.8%, 95%CI (0.46%, 97.3%)) patients. MMF induction ameliorated cognitive tests scores without exacerbating EDSS scores. There were no cases of discontinuation of MMF due to adverse effects.

Feb. 18, 2022

Yes

We have a plan to share IPD after publication of the results.

https://jrct.niph.go.jp/latest-detail/jRCTs051180080

Sekiguchi Kenji

Kobe University Hospital

7-5-2, Kusunoki-cho, Chuo-ku, Kobe City

neuro@med.kobe-u.ac.jp

Chihara Norio

Kobe University Hospital

7-5-2, Kusunoki-cho, Chuo-ku, Kobe City

+81-78-382-5885

neuro@med.kobe-u.ac.jp

Complete

Mar. 29, 2018

Interventional

single arm study

open(masking not used)

no treatment control/standard of care control

single assignment

treatment purpose

Stable Neuromyelitis Optica patients with EDSS score less than 7.0 who wish to participate in this study

1. Patients with infection
2. Patients with pregnancy or breast-feeding
3. Patients who take any immunosuppressant other than steroid
4. patients with severe liver or kidney dysfunction
5. patients with a history of cancer within 5 years (including carcinoma in situ)
6. In addition, patients judged inappropriate by the investigators

Both

Neuromyelitis optica

Mycophenolate mofetil treatment for 1 year

Whether the steroid dose is halved according to the treatment protocol 1 year after starting Mycophenolate mofetil and there is no clinical relapse with this combination therapy.

Decreased steroid dose
The number of radiological relapse
Changes in EDSS score
Neuro-ophthalmologic examination
Safety assessment

+81-78-382-6669

none