A national survey on current use of mycophenolate mofetil for childhood-onset systemic lupus erythematosus in Japan

Abstract

Purpose. To conduct a national survey of systemic lupus erythematosus (SLE) patients treated with mycophenolate mofetil (MMF). Based on current information on the use of MMF, we aimed to evaluate its efficacy and safety for childhood-onset (c-) SLE.

Target. We evaluated 115 patients by questionnaire on MMF use for c-SLE in medical facilities specializing in pediatric rheumatic and renal diseases.

Results. Average age at SLE onset was 10.6 (range, 2–15) years; average age at the time of starting MMF was 12.3 (range, 2–15) years. Average dose per body surface area was 1,059.3 mg/m²/day. Corticosteroid dosing was 20.9 mg/day before treatment but 7.7 mg/day after treatment. Laboratory values before and after MMF treatment were as follows: C3 increased from 67.0 to 84.9 mg/dl (p < 0.001), C4 increased from 10.2 to 15.1 mg/dl (p < 0.001), and anti-DNA antibody decreased from 154.2 to 18.4 IU/ml (p < 0.001). 24 adverse events in 21 cases were reported, but MMF was not discontinued in any.

Conclusions. The amount of MMF for c-SLE in Japan is similar to the standard dose in other countries. Reduction of corticosteroid dose and improvement of laboratory values represent efficacy of MMF. The side effects recorded here indicated tolerability of the drug.

Keywords:

• Childhood-onset systemic lupus erythematosus
• Effectiveness
• Lupus nephritis
• Mycophenolate mofetil
• Tolerability

Acknowledgments

We express our gratitude to the following collaborators for their efforts to facilitate our study (the facilities are in alphabetical order): Naomi Iwata (Aichi Children's Health and Medical Center), Minako Tomiita, Akiko Yamaide, Sachiko Misumi (Chiba Children's hospital), Ryojiro Tanaka (Hyogo Prefectural Kobe Children's Hospital), Akira Yoshida, Mihoko Inoue, Daisuke Fukao (Japanese Red Cross Wakayama Medical Center), Tomohiro Kubota (Kagoshima University Graduate School of Medical and Dental Sciences), Shuichi Ito, Koichi Kamei, Masao Ogura, Mai Sato, Masaki Takahashi, Zentaro Kiuchi, Masaki Fuyama (National Center for Child Health and Development), Yasuhiko Ito (Nippon Medical School), Hiroshi Yoshimura, Tomoo Kise (Okinawa Prefectural Nanbu Medical Center and Children's Medical Center), Shuichiro Fujinaga, Akifumi Yamada, Yasuko Urushibara, Taichi Hara (Saitama Children's Medical Center), Tadashi Matsubayashi, Naoya Fujita (Seirei Hamamatsu General Hospital), Hiroshi Hataya, and Riku Hamada (Tokyo Metropolitan Children's Medical Center).

Conflict of interest

R. H., H. M., K. N., T. M., T. N., M. K., N. S., T. K., S. S., K. S., S. T., Y. K.: none.

S. Y. has received research grant and/or speaking fee from Bristol Myers Squibb, Astellas Pharma Inc., and Chugai Pharmaceutical Co.

K. H. has received grant and/or speaking fee from Astellas Pharma Inc. and Chugai Pharmaceutical Co.

N. T. has received research grant and/or speaking fee from Chugai Pharmaceutical Co and Astellas Pharma Inc.

Y. T. has received research grant and/or speaking fee from Santen Pharmaceutical Co., Ltd., Daiichi Sankyo Company Limited, Mitsubishi Tanabe Pharma Corporation, Bristol-Myers Squibb, AstraZeneca plc, Astellas Pharma Inc., MSD K.K.,, Chugai Pharmaceutical Co., Asahi　Kasei　Pharma　Corporation, Eisai Co., and Janssen Pharmaceutical K.K.

T. A. has received research grant and/or speaking fees from Astellas Pharma Inc., Bristol Myers Squibb Co., Chugai Pharmaceutical Co. Ltd., Daiichi Sankyo Co. Ltd., Eisai Co. Ltd., and Mitsubishi-Tanabe Pharma Co.

M. M. has received lecture fees from MSD, Sumitomo Dainippon Pharma, and Pfizer Japan Inc., and has served as a consultant adviser to Bristol-Myers Squibb and Astellas Pharma.