Page last updated: 2024-10-23

azathioprine and Muscular Dystrophies

azathioprine has been researched along with Muscular Dystrophies in 8 studies

Azathioprine: An immunosuppressive agent used in combination with cyclophosphamide and hydroxychloroquine in the treatment of rheumatoid arthritis. According to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985), this substance has been listed as a known carcinogen. (Merck Index, 11th ed)
azathioprine : A thiopurine that is 6-mercaptopurine in which the mercapto hydrogen is replaced by a 1-methyl-4-nitroimidazol-5-yl group. It is a prodrug for mercaptopurine and is used as an immunosuppressant, prescribed for the treatment of inflammatory conditions and after organ transplantation and also for treatment of Crohn's didease and MS.

Muscular Dystrophies: A heterogeneous group of inherited MYOPATHIES, characterized by wasting and weakness of the SKELETAL MUSCLE. They are categorized by the sites of MUSCLE WEAKNESS; AGE OF ONSET; and INHERITANCE PATTERNS.

Research Excerpts

ExcerptRelevanceReference
"Prednisone has been shown to improve strength in Duchenne dystrophy."2.67Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months) ( Brooke, MH; Cwik, VA; Fenichel, GM; Griggs, RC; Mendell, JR; Miller, JP; Moxley, RT; Pandya, S; Pestronk, A; Robison, J, 1993)

Research

Studies (8)

TimeframeStudies, this research(%)All Research%
pre-19906 (75.00)18.7374
1990's2 (25.00)18.2507
2000's0 (0.00)29.6817
2010's0 (0.00)24.3611
2020's0 (0.00)2.80

Authors

AuthorsStudies
Foidart, M1
Liem, HH1
Adornato, BT1
Engel, WK1
Muller-Eberhard, U1
Griggs, RC2
Moxley, RT3
Mendell, JR2
Fenichel, GM1
Brooke, MH2
Pestronk, A1
Miller, JP1
Cwik, VA2
Pandya, S1
Robison, J1
Kissel, JT1
Lynn, DJ1
Rammohan, KW1
Klein, JP1
Jerusalem, F1
Kingston, WJ1
Kinoshita, M1
Nishina, M1
Koya, N1
Haas, DC1
Arnason, BG1
Miehlke, K1
Kohlhardt, J1
Wirth, B1
Kafarnik, U1

Clinical Trials (1)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen[NCT01603407]Phase 3196 participants (Actual)Interventional2013-01-31Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

6 Minute Walk Test

Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionmeters (Least Squares Mean)
Daily Prednisone384.95
Daily Deflazacort384.17
Intermittent Prednisone346.81

Forced Vital Capacity

Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionliters (Least Squares Mean)
Daily Prednisone1.4
Daily Deflazacort1.4
Intermittent Prednisone1.5

Fractional Shortening Percent

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionpercentage of fractional shortening (Least Squares Mean)
Daily Prednisone33.74
Daily Deflazacort34.01
Intermittent Prednisone34.33

Heart Rate

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionbpm (Least Squares Mean)
Daily Prednisone94.10
Daily Deflazacort93.52
Intermittent Prednisone91.65

Left Ventricular Ejection Fraction Percent

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionpercentage of ejection fraction (Least Squares Mean)
Daily Prednisone61.88
Daily Deflazacort62.65
Intermittent Prednisone62.45

North Star Ambulatory Assessment (NSAA) Score

"The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects.~The activities are graded as follows:~2 - Normal - no obvious modification of activity~1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function." (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone23.7
Daily Deflazacort24.0
Intermittent Prednisone20.7

Number of Participants Who Tolerated the Regimen

The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication. (NCT01603407)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Daily Prednisone36
Daily Deflazacort36
Intermittent Prednisone37

Participant Body Mass Index

(NCT01603407)
Timeframe: 36 months

Interventionkilograms/square meter (Least Squares Mean)
Daily Prednisone18.9
Daily Deflazacort18.3
Intermittent Prednisone18.1

Participant Height

(NCT01603407)
Timeframe: 36 months

Interventioncentimeters (Least Squares Mean)
Daily Prednisone116.8
Daily Deflazacort115.3
Intermittent Prednisone119.9

Participant Weight

(NCT01603407)
Timeframe: 36 months

Interventionkilograms (Least Squares Mean)
Daily Prednisone26.3
Daily Deflazacort24.9
Intermittent Prednisone26.3

PR Interval

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Interventionmilliseconds (Least Squares Mean)
Daily Prednisone115.59
Daily Deflazacort116.87
Intermittent Prednisone117.90

Quality of Life - Parent

Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child. (NCT01603407)
Timeframe: Average of Months 12, 24, and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone64.88
Daily Deflazacort63.71
Intermittent Prednisone61.33

Quality of Life- Child

Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life. (NCT01603407)
Timeframe: Average of Months 12, 24, and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone67.39
Daily Deflazacort64.96
Intermittent Prednisone65.07

Range of Motion (Goniometry) of Left Ankle

Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventiondegrees (Mean)
Daily Prednisone4.39
Daily Deflazacort3.29
Intermittent Prednisone2.67

Range of Motion (Goniometry) of Right Ankle

Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventiondegrees (Mean)
Daily Prednisone4.05
Daily Deflazacort2.81
Intermittent Prednisone2.29

Rise From the Floor Velocity

Reciprocal of time to rise from the floor (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionrise/sec (Least Squares Mean)
Daily Prednisone0.24
Daily Deflazacort0.24
Intermittent Prednisone0.18

Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score

The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Interventionscore on a scale (Least Squares Mean)
Daily Prednisone71.2
Daily Deflazacort67.8
Intermittent Prednisone65.1

Reviews

2 reviews available for azathioprine and Muscular Dystrophies

ArticleYear
[Treatment of myopathies and spinal muscular atrophies (author's transl)].
    Schweizerische Rundschau fur Medizin Praxis = Revue suisse de medecine Praxis, 1978, Jan-24, Volume: 67, Issue:4

    Topics: Amyotrophic Lateral Sclerosis; Azathioprine; Collagen Diseases; Dermatomyositis; Glycogen Storage Di

1978
Treatment of muscular dystrophies and inflammatory myopathies.
    Clinical neuropharmacology, 1986, Volume: 9, Issue:4

    Topics: Azathioprine; Clinical Trials as Topic; Dermatomyositis; Humans; Muscular Dystrophies; Myositis; Myo

1986

Trials

3 trials available for azathioprine and Muscular Dystrophies

ArticleYear
Duchenne dystrophy: randomized, controlled trial of prednisone (18 months) and azathioprine (12 months)
    Neurology, 1993, Volume: 43, Issue:3 Pt 1

    Topics: Adolescent; Azathioprine; Body Height; Child; Child, Preschool; Double-Blind Method; Drug Therapy, C

1993
Mononuclear cell analysis of muscle biopsies in prednisone- and azathioprine-treated Duchenne muscular dystrophy.
    Neurology, 1993, Volume: 43, Issue:3 Pt 1

    Topics: Adolescent; Antigens, CD; Azathioprine; B-Lymphocytes; Child; Child, Preschool; Drug Therapy, Combin

1993
Treatment of muscular dystrophies and inflammatory myopathies.
    Clinical neuropharmacology, 1986, Volume: 9, Issue:4

    Topics: Azathioprine; Clinical Trials as Topic; Dermatomyositis; Humans; Muscular Dystrophies; Myositis; Myo

1986

Other Studies

4 other studies available for azathioprine and Muscular Dystrophies

ArticleYear
Hemopexin metabolism in patients with altered serum levels.
    The Journal of laboratory and clinical medicine, 1983, Volume: 102, Issue:5

    Topics: Adolescent; Adult; Aged; Azathioprine; Creatine Kinase; Female; Hemopexin; Humans; Male; Middle Aged

1983
Ten years follow up study of steroid therapy for congenital encephalomyopathy.
    Brain & development, 1986, Volume: 8, Issue:3

    Topics: Azathioprine; Brain Diseases; Child; Child, Preschool; Creatine Kinase; Follow-Up Studies; Humans; I

1986
Cell-mediated immunity in polymyositis. Creatine phosphokinase release from muscle cultures.
    Archives of neurology, 1974, Volume: 31, Issue:3

    Topics: Adolescent; Adult; Aged; Autoimmune Diseases; Azathioprine; Child; Creatine Kinase; Culture Techniqu

1974
[Basic therapy of progressive chronic arthritis. 2].
    Therapie der Gegenwart, 1970, Volume: 109, Issue:11

    Topics: Alkylating Agents; Antimetabolites; Antineoplastic Agents; Arthritis; Azathioprine; Chronic Disease;

1970