A study of thyrotropin-releasing hormone for the treatment of spinal muscular atrophy: a preliminary report.
OBJECTIVE
To determine whether thyrotropin-releasing hormone (TRH) can increase muscle strength in children with spinal muscular atrophy types 2 and 3.


METHODS
A randomized, double-blinded, controlled, 5-wk drug trial of six subjects and three controls. Subjects and controls ranged from 4 to 8 yr of age and were randomly assigned to treatment and placebo groups in a ratio of 2:1. TRH (protirelin) or placebo was delivered intravenously through percutaneous intravenous catheters at a dose of 0.1 mg/kg (in 50 ml of normal saline) for a total of 29 days. Patients were evaluated using electromyography and handheld dynamometry of the deltoids, biceps, triceps, wrist extensors, hip flexors, quadriceps, hamstrings, and grip strength before and immediately after 5 wk of treatment. A unidirectional t test was used to compare mean values.


RESULTS
Dynamometry improved significantly only for the six treated subjects (P < 0.02). Peroneal nerve conduction velocities were significantly faster in the treatment group (paired t test, P = 0.036). The parents of the treated children also provided anecdotal evidence of improvements in function. Improvements lasted 6-12 mo.


CONCLUSIONS
TRH may be a useful treatment for spinal muscular atrophy. A larger, crossover design group comparison study is warranted.
Tzeng
A C
AC
University Hospital, University of Medicine and Dentistry of New Jersey-The New Jersey Medical School, Newark, USA.
Cheng
J
J
Fryczynski
H
H
Niranjan
V
V
Stitik
T
T
Sial
A
A
Takeuchi
Y
Y
Foye
P
P
DePrince
M
M
Bach
J R
JR
D000200:Action Potentials; D000870:Anterior Horn Cells / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D004576:Electromyography; D005260:Female; D005500:Follow-Up Studies; D018737:Hand Strength; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D018908:Muscle Weakness / Q000209:etiology; D009134:Muscular Atrophy, Spinal / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D009431:Neural Conduction; D013973:Thyrotropin-Releasing Hormone / Q000494:pharmacology / Q000627:therapeutic use*; D016896:Treatment Outcome
D013973:Thyrotropin-Releasing Hormone