Long-term weekly ACTH therapy for relapsed West syndrome in tuberous sclerosis complex: A case report.
BACKGROUND
In Japan, adrenocorticotropic hormone (ACTH) therapy has been the mainstay of treatment of West syndrome. Conventional ACTH therapy is administered short-term with efficacy, yet the relapse rate is high. Relapse after initial ACTH therapy is a poor prognostic factor for long-term seizure control and outcome of cognitive function. Here, we report successful long-term weekly ACTH therapy for relapsed WS in a tuberous sclerosis complex (TSC) child after conventional ACTH therapy.


METHODS
The patient had a series of epileptic spasms (ES) and hypsarrhythmia at age 3 months. She was diagnosed with WS associated with TSC, and was treated with conventional ACTH therapy at age 4 months, and a second course of ACTH therapy at age 8 months. Both courses of therapy were transiently effective. A third course of ACTH therapy was started at age 1 year and 2 months, and long-term weekly ACTH therapy was continued thereafter. During this therapy, both ES and hypsarrhythmia remained completely resolved. Therapy was continued, and dose reduction was started when the patient was 2 years and 10 months old. No serious adverse events had occurred during this therapy.


CONCLUSIONS
This case demonstrated that long-term weekly ACTH may be safe and effective. Although at present, this therapy may only be considered for relapsed symptomatic WS patients, it may be a good alternative therapy when frequent relapses occur after favorable response to conventional ACTH therapy.
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Masatoshi
M
Nakata
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan. Electronic address: takeo819@kuhp.kyoto-u.ac.jp.
Takeo
T
Kato
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Minako
M
Ide
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Keiko
K
Saito
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Takeshi
T
Yoshida
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Tomonari
T
Awaya
Department of Pediatric Neurology, Shiga Medical Center for Children, Shiga, Japan.
Minoru
M
Shibata
Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Toshio
T
Heike
D000324:Adrenocorticotropic Hormone / Q000627:therapeutic use*; D001921:Brain / Q000187:drug effects / Q000503:physiopathology; D002675:Child, Preschool; D004569:Electroencephalography; D005260:Female; D006801:Humans; D007223:Infant; D007564:Japan; D012008:Recurrence; D013036:Spasms, Infantile / Q000150:complications / Q000188:drug therapy*; D016896:Treatment Outcome; D014402:Tuberous Sclerosis / Q000150:complications*
D000324:Adrenocorticotropic Hormone
Epileptic spasms; Hypsarrhythmia; Long-term weekly ACTH therapy; Relapse; Tuberous sclerosis complex; West syndrome