Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy.
Muscle Nerve
; 44(2): 170-3, 2011 Aug.
Article
em En
| MEDLINE
| ID: mdl-21674534
ABSTRACT
INTRODUCTION:
In this study we performed an open-label, pilot study of an orally administered liquid formulation of immediate-release pentoxifylline (PTX) on patients with Duchenne muscular dystrophy (DMD). Treatment efficacy, safety, and tolerability were assessed.METHODS:
The tolerability and safety of PTX and measures of muscle strength and function were evaluated during 12 months of treatment.RESULTS:
Seventeen boys with DMD, between 4 and 8 years of age, were enrolled at one of five Cooperative International Neuromuscular Research Group (CINRG) centers. Only 9 were able to complete the 12-month PTX treatment phase; the primary reason for discontinuation was adverse events. Intolerable gastrointestinal side effects were experienced by 65% of participants. Two participants had severe leukopenia that resolved with medication withdrawal.CONCLUSIONS:
Open-label treatment with a liquid formulation of immediate-release PTX resulted in a high incidence of adverse events in boys with DMD. Poor tolerability of this PTX formulation precluded adequate assessment of efficacy.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Pentoxifilina
/
Distrofia Muscular de Duchenne
Limite:
Child
/
Child, preschool
/
Humans
/
Male
Idioma:
En
Revista:
Muscle Nerve
Ano de publicação:
2011
Tipo de documento:
Article
País de afiliação:
Estados Unidos