Pentoxifylline as a rescue treatment for DMD

A randomized double-blind clinical trial

D. M. Escolar, A. Zimmerman, Tulio Bertorini, P. R. Clemens, A. M. Connolly, L. Mesa, K. Gorni, A. Kornberg, H. Kolski, N. Kuntz, Y. Nevo, C. Tesi-Rocha, K. Nagaraju, S. Rayavarapu, L. P. Hache, J. E. Mayhew, J. Florence, F. Hu, A. Arrieta, E. Henricson & 2 others R. T. Leshner, J. K. Mah

Research output: Contribution to journalArticle

18 Citations (Scopus)

Abstract

Objective: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD). Methods: This was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation (̃20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test. Results: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p > 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs. Conclusion: The addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. Classification of evidence: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.

Original languageEnglish (US)
Pages (from-to)904-913
Number of pages10
JournalNeurology
Volume78
Issue number12
DOIs
StatePublished - Mar 20 2012

Fingerprint

Pentoxifylline
Duchenne Muscular Dystrophy
Clinical Trials
Muscle Strength
Muscles
Adrenal Cortex Hormones
Placebos
Therapeutics
Students
Lung

All Science Journal Classification (ASJC) codes

  • Clinical Neurology

Cite this

Escolar, D. M., Zimmerman, A., Bertorini, T., Clemens, P. R., Connolly, A. M., Mesa, L., ... Mah, J. K. (2012). Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial. Neurology, 78(12), 904-913. https://doi.org/10.1212/WNL.0b013e31824c46be

Pentoxifylline as a rescue treatment for DMD : A randomized double-blind clinical trial. / Escolar, D. M.; Zimmerman, A.; Bertorini, Tulio; Clemens, P. R.; Connolly, A. M.; Mesa, L.; Gorni, K.; Kornberg, A.; Kolski, H.; Kuntz, N.; Nevo, Y.; Tesi-Rocha, C.; Nagaraju, K.; Rayavarapu, S.; Hache, L. P.; Mayhew, J. E.; Florence, J.; Hu, F.; Arrieta, A.; Henricson, E.; Leshner, R. T.; Mah, J. K.

In: Neurology, Vol. 78, No. 12, 20.03.2012, p. 904-913.

Research output: Contribution to journalArticle

Escolar, DM, Zimmerman, A, Bertorini, T, Clemens, PR, Connolly, AM, Mesa, L, Gorni, K, Kornberg, A, Kolski, H, Kuntz, N, Nevo, Y, Tesi-Rocha, C, Nagaraju, K, Rayavarapu, S, Hache, LP, Mayhew, JE, Florence, J, Hu, F, Arrieta, A, Henricson, E, Leshner, RT & Mah, JK 2012, 'Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial', Neurology, vol. 78, no. 12, pp. 904-913. https://doi.org/10.1212/WNL.0b013e31824c46be
Escolar, D. M. ; Zimmerman, A. ; Bertorini, Tulio ; Clemens, P. R. ; Connolly, A. M. ; Mesa, L. ; Gorni, K. ; Kornberg, A. ; Kolski, H. ; Kuntz, N. ; Nevo, Y. ; Tesi-Rocha, C. ; Nagaraju, K. ; Rayavarapu, S. ; Hache, L. P. ; Mayhew, J. E. ; Florence, J. ; Hu, F. ; Arrieta, A. ; Henricson, E. ; Leshner, R. T. ; Mah, J. K. / Pentoxifylline as a rescue treatment for DMD : A randomized double-blind clinical trial. In: Neurology. 2012 ; Vol. 78, No. 12. pp. 904-913.
@article{25e48f16a62d45e9b744d0f1046ffd3b,
title = "Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial",
abstract = "Objective: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD). Methods: This was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation (̃20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test. Results: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p > 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs. Conclusion: The addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. Classification of evidence: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.",
author = "Escolar, {D. M.} and A. Zimmerman and Tulio Bertorini and Clemens, {P. R.} and Connolly, {A. M.} and L. Mesa and K. Gorni and A. Kornberg and H. Kolski and N. Kuntz and Y. Nevo and C. Tesi-Rocha and K. Nagaraju and S. Rayavarapu and Hache, {L. P.} and Mayhew, {J. E.} and J. Florence and F. Hu and A. Arrieta and E. Henricson and Leshner, {R. T.} and Mah, {J. K.}",
year = "2012",
month = "3",
day = "20",
doi = "10.1212/WNL.0b013e31824c46be",
language = "English (US)",
volume = "78",
pages = "904--913",
journal = "Neurology",
issn = "0028-3878",
publisher = "Lippincott Williams and Wilkins",
number = "12",

}

TY - JOUR

T1 - Pentoxifylline as a rescue treatment for DMD

T2 - A randomized double-blind clinical trial

AU - Escolar, D. M.

AU - Zimmerman, A.

AU - Bertorini, Tulio

AU - Clemens, P. R.

AU - Connolly, A. M.

AU - Mesa, L.

AU - Gorni, K.

AU - Kornberg, A.

AU - Kolski, H.

AU - Kuntz, N.

AU - Nevo, Y.

AU - Tesi-Rocha, C.

AU - Nagaraju, K.

AU - Rayavarapu, S.

AU - Hache, L. P.

AU - Mayhew, J. E.

AU - Florence, J.

AU - Hu, F.

AU - Arrieta, A.

AU - Henricson, E.

AU - Leshner, R. T.

AU - Mah, J. K.

PY - 2012/3/20

Y1 - 2012/3/20

N2 - Objective: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD). Methods: This was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation (̃20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test. Results: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p > 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs. Conclusion: The addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. Classification of evidence: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.

AB - Objective: To determine whether pentoxifylline (PTX) slows the decline of muscle strength and function in ambulatory boys with Duchenne muscular dystrophy (DMD). Methods: This was a multicenter, randomized, double-blinded, controlled trial comparing 12 months of daily treatment with PTX or placebo in corticosteroid-treated boys with DMD using a slow-release PTX formulation (̃20 mg/kg/day). The primary outcome was the change in mean total quantitative muscle testing (QMT) score. Secondary outcomes included changes in QMT subscales, manual muscle strength, pulmonary function, and timed function tests. Outcomes were compared using Student t tests and a linear mixed-effects model. Adverse events (AEs) were compared using the Fisher exact test. Results: A total of 64 boys with DMD with a mean age of 9.9 ± 2.9 years were randomly assigned to PTX or placebo in 11 participating Cooperative International Neuromuscular Research Group centers. There was no significant difference between PTX and the placebo group in total QMT scores (p > 0.14) or in most of the secondary outcomes after a 12-month treatment. The use of PTX was associated with mild to moderate gastrointestinal or hematologic AEs. Conclusion: The addition of PTX to corticosteroid-treated boys with DMD at a moderate to late ambulatory stage of disease did not improve or halt the deterioration of muscle strength and function over a 12-month study period. Classification of evidence: This study provides Class I evidence that treatment with PTX does not prevent deterioration in muscle function or strength in corticosteroid-treated boys with DMD.

UR - http://www.scopus.com/inward/record.url?scp=84860327196&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84860327196&partnerID=8YFLogxK

U2 - 10.1212/WNL.0b013e31824c46be

DO - 10.1212/WNL.0b013e31824c46be

M3 - Article

VL - 78

SP - 904

EP - 913

JO - Neurology

JF - Neurology

SN - 0028-3878

IS - 12

ER -