Efficacy and Safety of Mitoxantrone in Patients With Refractory Neuromyelitis Optica and Spectrum Disorders
The treatment protocol consisted of 12 mg/m2 MITO intravenous infusions every 3 months for 2 years. Dosage was adjusted according to side effects. Neurological assessment including the determination of the Expanded Disability Status Scale (EDSS) score and ophthalmologic evaluations were performed every 3 months and during relapses. Flow cytometric analysis, brain and spinal cord MRI was performed at baseline, 6, 12, 18, and 24 months.
Neuromyelitis Optica Spectrum Disorders
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Efficacy and Safety of Mitoxantrone in Patients With Refractory Neuromyelitis Optica and Spectrum Disorders|
- annual relapse rate (ARR) [ Time Frame: one year ] [ Designated as safety issue: No ]ARR is defined as the number of confirmed relapses in a year. The number of annual relapse rate was used as parameters of effectiveness and was compared between premitoxantrone and postmitoxantrone treatment during the follow-up period.
- EDSS [ Time Frame: six months ] [ Designated as safety issue: No ]Expanded Disability Status Scale (EDSS) scores was used as parameters of effectiveness and was compared between premitoxantrone and postmitoxantrone treatment during the follow-up period.
- Changes in LVEF [ Time Frame: six months ] [ Designated as safety issue: Yes ]- Assessment of cardiac function: Changes in LVEF by transthoracic echocardiography and determination of cardiac side effects by ECG and by measurement of CK-MB, Troponin and BNP.
- blood cell count [ Time Frame: three months ] [ Designated as safety issue: Yes ]- Assessment of hematological system: Monitoring blood cell count regularly. Considering marrow puncture if necessary.
- Flow cytometric analysis [ Time Frame: six months ] [ Designated as safety issue: Yes ]Immunofluorescent staining of wholeblood samples were performed of blood drawing using antibodies against CD3/CD4/CD8/CD19/CD20/CD56 with isotype controls, followed by lysis of red blood cells and immediate acquisition and analysis by flow cytometry.
|Study Start Date:||March 2009|
|Estimated Study Completion Date:||December 2015|
|Estimated Primary Completion Date:||December 2014 (Final data collection date for primary outcome measure)|
MITO, annual relapse rate, safety
For refractory NMO patients aged 18-55, the initial dose 12 mg/m2 mitoxantrone was administered over a five day course every 3 months for 2 years (a total of eight courses). The initial dose was reduced to 9 mg/m2 if the preinfusion white-blood-cell count was 3.0-3.99 ×109/L,and to 6 mg/m2 if the white-blood-cell count was 2.0-2.99 ×109/L. No infusion if the white-blood-cell count was less than 2.0×109/L. The initial dose was reduced to 10 mg/m2 for nonhaematological toxic effects of WHO grade 2-3. Subsequent dose after 3 month was reduced to 10 mg/m2 for infections that occurred within 3 weeks of a previous infusion accompanied by a white-blood-cell count below 2×109/L, or to 8 mg/m2 for infections accompanied by white-blood-cell count of less than 1×109/L.
The treatment protocol consisted of 12 mg/m2 MITO intravenous infusions every 3 months for 2 years. Dosage was adjusted according to side effects.
Other Name: Novantrone
Please refer to this study by its ClinicalTrials.gov identifier: NCT02021825
|Contact: Huiqing Dong, Doctorfirstname.lastname@example.org|
|Contact: Zheng Liu, Doctoremail@example.com|
|Department of Neurology, Xuanwu Hospital, Capital Medical University||Recruiting|
|Beijing, Beijing, China, 100053|
|Contact: Zheng Liu, Doctor 0086-13910320552 firstname.lastname@example.org|
|Principal Investigator: Huiqing Dong, Doctor|
|Principal Investigator:||Huiqing Dong, Doctor||Department of Neurology, Xuanwu Hospital, Capital Medical University|