Pentoxifylline as a rescue treatment for DMD: A randomized double-blind clinical trial

Neurology

Volumn 78, Issue 12, 2012, Pages 904-913

  Escolar, D M ^a   Zimmerman, A ^a   Bertorini, T ^b   Clemens, P R ^c   Connolly, A M ^d   Mesa, L ^e   Gorni, K ^f   Kornberg, A ^g   Kolski, H ^h   Kuntz, N ⁱ   Nevo, Y ^j   Tesi Rocha, C ^a   Nagaraju, K ^a   Rayavarapu, S ^a   Hache, L P ^a   Mayhew, J E ^a   Florence, J ^a   Hu, F ^a   Arrieta, A ^a   Henricson, E ^a   Leshner, R T ^a   Mah, J K ^k   more..

^a CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^b UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

^c UNIVERSITY OF PITTSBURGH   (United States)

^d WASHINGTON UNIVERSITY   (United States)

^e HOSPITAL UNIVERSITARIO FUNDACIÓN FAVALORO   (Argentina)

^f C MONDINO NATIONAL NEUROLOGICAL INSTITUTE   (Italy)

^g ROYAL CHILDREN'S HOSPITAL   (Australia)

^h UNIVERSITY OF ALBERTA   (Canada)

ⁱ MAYO CLINIC   (United States)

^j HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

^k UNIVERSITY OF CALGARY   (Canada)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CAPSUGEL; CORTICOSTEROID; PENTOXIFYLLINE; PLACEBO; UNCLASSIFIED DRUG; PHOSPHODIESTERASE INHIBITOR;

ABNORMAL LABORATORY RESULT; ALLERGIC DISEASE; ARTICLE; BLEEDING; BLOOD CLOTTING DISORDER; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; CORTICOSTEROID THERAPY; DOUBLE BLIND PROCEDURE; DUCHENNE MUSCULAR DYSTROPHY; ENDOCRINE DISEASE; EYE DISEASE; GASTROINTESTINAL DISEASE; HEART DISEASE; HUMAN; IMMUNOPATHOLOGY; INFECTION; KIDNEY DISEASE; LUNG DISEASE; LUNG FUNCTION; MALE; METABOLIC DISORDER; MULTICENTER STUDY; MUSCLE FUNCTION; MUSCLE STRENGTH; MUSCULOSKELETAL DISEASE; NEUROLOGIC DISEASE; PAIN; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RESPIRATORY TRACT DISEASE; SCORING SYSTEM; SKIN DISEASE; SLOW RELEASE FORMULATION; SOFT TISSUE DISEASE; STATISTICAL ANALYSIS; TREATMENT DURATION; TREATMENT OUTCOME; UROGENITAL TRACT DISEASE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DELAYED RELEASE FORMULATION; DISEASE COURSE; DRUG COMBINATION; LUNG FUNCTION TEST; NEUROLOGIC EXAMINATION; PATHOPHYSIOLOGY; PHYSIOLOGY; PSYCHOLOGICAL ASPECT; QUALITY OF LIFE; SAMPLE SIZE;

ADRENAL CORTEX HORMONES; CHILD; DELAYED-ACTION PREPARATIONS; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; DRUG THERAPY, COMBINATION; HUMANS; MALE; MUSCLE STRENGTH; MUSCULAR DYSTROPHY, DUCHENNE; NEUROLOGIC EXAMINATION; PENTOXIFYLLINE; PHOSPHODIESTERASE INHIBITORS; QUALITY OF LIFE; RESPIRATORY FUNCTION TESTS; SAMPLE SIZE; TREATMENT OUTCOME;

EID: 84860327196     PISSN: 00283878     EISSN: 1526632X     Source Type: Journal    
DOI: 10.1212/WNL.0b013e31824c46be     Document Type: Article

References (33)

1. Spencer MJ, Tidball JG. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord 2001;11:556-564. (Pubitemid 32761799)
2. Evans NP, Misyak SA, Robertson JL, Bassaganya-Riera J, Grange RW. Immune-mediated mechanisms potentially regulate the disease time-course of Duchenne muscular dystrophy and provide targets for therapeutic intervention. PM R 2009;1:755-768.
3. Moxley RT, Ashwal S, Pandya S, et al. Practice parameter: Corticosteroid treatment of Duchenne dystrophy: Report of the quality standards subcommittee of the American Academy of Neurology and the practice committee of the Child Neurology Society. Neurology 2005;64:13-20. (Pubitemid 40082930)
4. Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: Diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010;9:77-93.
5. McMillan HJ, Campbell C, Mah JK, Canadian Paediatric Neuromuscular Group. Duchenne muscular dystrophy: Canadian paediatric neuromuscular physicians survey. Can J Neurol Sci 2010;37:195-205.
6. Wagner KR. Approaching a new age in Duchenne muscular dystrophy treatment. Neurotherapeutics 2008;5:583-591.
7. Guglieri M, Bushby K. Molecular treatments in Duchenne muscular dystrophy. Curr Opin Pharmacol 2010;10:331-337.
8. Dorchies OM, Wagner S, Vuadens O, et al. Green tea extract and its major polyphenol (-)-epigallocatechin gallate improve muscle function in a mouse model for Duchenne muscular dystrophy. Am J Physiol Cell Physiol 2006; 290:C616-C625.
9. Burdi R, Rolland JF, Fraysse B, et al. Multiple pathological events in exercised dystrophic mdx mice are targeted by pentoxifylline: Outcome of a large array of in vivo and ex vivo tests. J Appl Physiol 2009;106:1311-1324.
10. Granchelli JA, Pollina C, Hudecki MS. Pre-clinical screening of drugs using the mdx mouse. Neuromuscul Disord 2000;10:235-239. (Pubitemid 30320912)
11. Zabel P, Schade FU, Schlaak M. Inhibition of endogenous TNF formation by pentoxifylline. Immunobiology 1993; 187:447-463. (Pubitemid 23150784)
12. Mandell GL. Cytokines, phagocytes, and pentoxifylline. J Cardiovasc Pharmacol 1995;25(suppl 2):S20-S22.
13. Witkamp R, Monshouwer M. Signal transduction in inflammatory processes, current and future therapeutic targets: A mini review. Vet Q 2000;22:11-16. (Pubitemid 30064703)
14. Romanelli RG, Caligiuri A, Carloni V, et al. Effect of pentoxifylline on the degradation of procollagen type I produced by human hepatic stellate cells in response to transforming growth factor-β1. Br J Pharmacol 1997;122: 1047-1054. (Pubitemid 27494217)
15. Ishitobi M, Haginoya K, Zhao Y, et al. Elevated plasma levels of transforming growth factor beta1 in patients with muscular dystrophy. Neuroreport 2000;11:4033-4035. (Pubitemid 32038799)
16. Chen YW, Nagaraju K, Bakay M, McIntyre O, Rawat R, Shi R, Hoffman EP. Early onset of inflammation and later involvement of TGF-β in Duchenne muscular dystrophy. Neurology 2005;65:826-834. (Pubitemid 41362124)
17. Ward A, Clissold SP. Pentoxifylline: A review of its pharmacodynamic and pharmacokinetic properties, and its therapeutic efficacy. Drugs 1987;34:50-97. (Pubitemid 17092322)
18. Funk JO, Ernst M, Schönharting MM, Zabel P. Pentoxifylline exerts synergistic immunomodulatory effects in combination with dexamethasone or cyclosporin A. Int J Immunopharmacol 1995;17:1007-1016. (Pubitemid 26097583)
19. Zimmerman A, Clemens P, Tesi-Rocha C, et al. Liquid formulation of pentoxifylline is a poorly tolerated treatment for Duchenne dystrophy. Muscle Nerve 2011;44: 170-173.
20. Furukawa S, Matsubara T, Umezawa Y, Motohashi T, Ino T, Yabuta K. Pentoxifylline and intravenous gamma globulin combination therapy for acute Kawasaki disease. Eur J Pediatr 1994;153:663-667. (Pubitemid 24257129)
21. Haque K, Mohan P. Pentoxifylline for neonatal sepsis. Cochrane Database Syst Rev 2003:CD004205.
22. Akhondzadeh S, Fallah J, Mohammadi MR, et al. Doubleblind placebo-controlled trial of pentoxifylline added to risperidone: Effects on aberrant behavior in children with autism. Prog Neuropsychopharmacol Biol Psychiatry 2010;34:32-36.
23. Harris E, Schulzke SM, Patole SK. Pentoxifylline in preterm neonates: A systematic review. Paediatr Drugs 2010; 12:301-311.
24. Escolar DM, Henricson EK, Mayhew J, et al. Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children. Muscle Nerve 2001;24: 787-793. (Pubitemid 32476525)
25. Mayhew JE, Florence JM, Mayhew TP, et al. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle Nerve 2007;35: 36-42. (Pubitemid 46052646)
26. Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 1981; 4:186-197. (Pubitemid 11098499)
27. Vignos PJ, Spencer GE, Archibald KC. Management of progressive muscular dystrophy in childhood. JAMA 1963;184:89-96.
28. ATS. Standardization of spirometry, 1994 update: American Thoracic Society. Am J Respir Crit Care Med 1995; 152:1107-1136.
29. Varni JW, Seid M, Kurtin PS. PedsQL 4.0: Reliability and validity of the pediatric quality of life inventory version 4.0 generic core scales in healthy and patient populations. Med Care 2001;39:800-812. (Pubitemid 33686263)
30. Varni JW, Burwinkle TM, Seid M, Skarr D. The PedsQL 4.0 as a pediatric population health measure: Feasibility, reliability, and validity. Ambul Pediatr 2003; 3:329-341. (Pubitemid 37527280)
31. Verbeke G, Molenberghs G. Linear Mixed Models for Longitudinal Data. New York: Springer; 2000.
32. Sun G, Haginoya K, Chiba Y, et al. Elevated plasma levels of tissue inhibitors of metalloproteinase-1 and their overexpression in muscle in human and mouse muscular dystrophy. J Neurol Sci 2010;297:19-28.
33. Krakauer T, Stephens J, Buckley M, Tate M. Superantigeninduced cytokine release from whole-blood cell culture as a functional measure of drug efficacy after oral dosing in nonhuman primates. Res Vet Sci 2007;83:182-187. (Pubitemid 46971708)