The role of aggressive corticosteroid therapy in patients with juvenile dermatomyositis

A propensity score analysis

Roopa Seshadri, Brian M. Feldman, Norman Todd Ilowite, Gail Cawkwell, Lauren M. Pachman

Research output: Contribution to journalArticle

43 Citations (Scopus)

Abstract

Objective. To compare outcomes at 36 months in patients newly diagnosed with juvenile dermatomyositis (DM) treated with aggressive versus standard therapy. Methods. At diagnosis, 139 untreated juvenile DM patients were given aggressive therapy (intravenous methylprednisolone or oral prednisone 5-30 mg/kg/day; n = 76) or standard therapy (1-2 mg/kg/day; n = 63) by the treating physician. Aggressive therapy patients were more ill at diagnosis. Matching was based on the propensity for aggressive therapy because propensity scoring can reduce confounding by indication. Logistic regression of the matched data determined predictors of outcomes, controlling for clinical confounders and propensity score. Outcomes comprised Disease Activity Score (DAS) for skin and muscle, range of motion (ROM), and calcification. Results. Sex, race, and age were similar between groups, and initial DAS weakness and ROM significantly predicted the therapy chosen. Based on propensity scores, 42 patients from each group were well matched. In the matched pairs, there were no significant differences in outcomes. Methotrexate use (odds ratio [OR] 3.6, 95% confidence interval [95% CI] 1.15-11.5) and duration of untreated disease (OR 1.2, 95% CI 1-1.38) were associated with ROM loss, hydroxychloroquine use (OR 11.2, 95% CI 3.7-33) and calcification (OR 6.8, 95% CI 1.8-25.4) with persistent rash, abnormal baseline lactate dehydrogenase (OR 11.2, 95% CI 1.4-92) and age at onset (OR 1.3, 95% CI 1-1.4) with weakness, and duration of untreated disease (OR 1.2, 95% CI 1-1.39) with calcification. Conclusion. Using a retrospective, nonrandomized design with propensity score matching, there was little difference in efficacy outcomes between aggressive and standard therapy; however, the sickest patients were treated with aggressive therapy and were not included in the matched analysis. Comprehensive clinical studies are needed to determine therapeutic pathways to the best outcome.

Original languageEnglish (US)
Pages (from-to)989-995
Number of pages7
JournalArthritis Care and Research
Volume59
Issue number7
DOIs
StatePublished - Jul 15 2008

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Propensity Score
Adrenal Cortex Hormones
Odds Ratio
Confidence Intervals
Articular Range of Motion
Therapeutics
Hydroxychloroquine
Juvenile dermatomyositis
Methylprednisolone
Prednisone
Exanthema
Age of Onset
L-Lactate Dehydrogenase
Methotrexate
Logistic Models
Physicians
Muscles
Skin

ASJC Scopus subject areas

  • Rheumatology

Cite this

The role of aggressive corticosteroid therapy in patients with juvenile dermatomyositis : A propensity score analysis. / Seshadri, Roopa; Feldman, Brian M.; Ilowite, Norman Todd; Cawkwell, Gail; Pachman, Lauren M.

In: Arthritis Care and Research, Vol. 59, No. 7, 15.07.2008, p. 989-995.

Research output: Contribution to journalArticle

Seshadri, Roopa ; Feldman, Brian M. ; Ilowite, Norman Todd ; Cawkwell, Gail ; Pachman, Lauren M. / The role of aggressive corticosteroid therapy in patients with juvenile dermatomyositis : A propensity score analysis. In: Arthritis Care and Research. 2008 ; Vol. 59, No. 7. pp. 989-995.
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abstract = "Objective. To compare outcomes at 36 months in patients newly diagnosed with juvenile dermatomyositis (DM) treated with aggressive versus standard therapy. Methods. At diagnosis, 139 untreated juvenile DM patients were given aggressive therapy (intravenous methylprednisolone or oral prednisone 5-30 mg/kg/day; n = 76) or standard therapy (1-2 mg/kg/day; n = 63) by the treating physician. Aggressive therapy patients were more ill at diagnosis. Matching was based on the propensity for aggressive therapy because propensity scoring can reduce confounding by indication. Logistic regression of the matched data determined predictors of outcomes, controlling for clinical confounders and propensity score. Outcomes comprised Disease Activity Score (DAS) for skin and muscle, range of motion (ROM), and calcification. Results. Sex, race, and age were similar between groups, and initial DAS weakness and ROM significantly predicted the therapy chosen. Based on propensity scores, 42 patients from each group were well matched. In the matched pairs, there were no significant differences in outcomes. Methotrexate use (odds ratio [OR] 3.6, 95{\%} confidence interval [95{\%} CI] 1.15-11.5) and duration of untreated disease (OR 1.2, 95{\%} CI 1-1.38) were associated with ROM loss, hydroxychloroquine use (OR 11.2, 95{\%} CI 3.7-33) and calcification (OR 6.8, 95{\%} CI 1.8-25.4) with persistent rash, abnormal baseline lactate dehydrogenase (OR 11.2, 95{\%} CI 1.4-92) and age at onset (OR 1.3, 95{\%} CI 1-1.4) with weakness, and duration of untreated disease (OR 1.2, 95{\%} CI 1-1.39) with calcification. Conclusion. Using a retrospective, nonrandomized design with propensity score matching, there was little difference in efficacy outcomes between aggressive and standard therapy; however, the sickest patients were treated with aggressive therapy and were not included in the matched analysis. Comprehensive clinical studies are needed to determine therapeutic pathways to the best outcome.",
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N2 - Objective. To compare outcomes at 36 months in patients newly diagnosed with juvenile dermatomyositis (DM) treated with aggressive versus standard therapy. Methods. At diagnosis, 139 untreated juvenile DM patients were given aggressive therapy (intravenous methylprednisolone or oral prednisone 5-30 mg/kg/day; n = 76) or standard therapy (1-2 mg/kg/day; n = 63) by the treating physician. Aggressive therapy patients were more ill at diagnosis. Matching was based on the propensity for aggressive therapy because propensity scoring can reduce confounding by indication. Logistic regression of the matched data determined predictors of outcomes, controlling for clinical confounders and propensity score. Outcomes comprised Disease Activity Score (DAS) for skin and muscle, range of motion (ROM), and calcification. Results. Sex, race, and age were similar between groups, and initial DAS weakness and ROM significantly predicted the therapy chosen. Based on propensity scores, 42 patients from each group were well matched. In the matched pairs, there were no significant differences in outcomes. Methotrexate use (odds ratio [OR] 3.6, 95% confidence interval [95% CI] 1.15-11.5) and duration of untreated disease (OR 1.2, 95% CI 1-1.38) were associated with ROM loss, hydroxychloroquine use (OR 11.2, 95% CI 3.7-33) and calcification (OR 6.8, 95% CI 1.8-25.4) with persistent rash, abnormal baseline lactate dehydrogenase (OR 11.2, 95% CI 1.4-92) and age at onset (OR 1.3, 95% CI 1-1.4) with weakness, and duration of untreated disease (OR 1.2, 95% CI 1-1.39) with calcification. Conclusion. Using a retrospective, nonrandomized design with propensity score matching, there was little difference in efficacy outcomes between aggressive and standard therapy; however, the sickest patients were treated with aggressive therapy and were not included in the matched analysis. Comprehensive clinical studies are needed to determine therapeutic pathways to the best outcome.

AB - Objective. To compare outcomes at 36 months in patients newly diagnosed with juvenile dermatomyositis (DM) treated with aggressive versus standard therapy. Methods. At diagnosis, 139 untreated juvenile DM patients were given aggressive therapy (intravenous methylprednisolone or oral prednisone 5-30 mg/kg/day; n = 76) or standard therapy (1-2 mg/kg/day; n = 63) by the treating physician. Aggressive therapy patients were more ill at diagnosis. Matching was based on the propensity for aggressive therapy because propensity scoring can reduce confounding by indication. Logistic regression of the matched data determined predictors of outcomes, controlling for clinical confounders and propensity score. Outcomes comprised Disease Activity Score (DAS) for skin and muscle, range of motion (ROM), and calcification. Results. Sex, race, and age were similar between groups, and initial DAS weakness and ROM significantly predicted the therapy chosen. Based on propensity scores, 42 patients from each group were well matched. In the matched pairs, there were no significant differences in outcomes. Methotrexate use (odds ratio [OR] 3.6, 95% confidence interval [95% CI] 1.15-11.5) and duration of untreated disease (OR 1.2, 95% CI 1-1.38) were associated with ROM loss, hydroxychloroquine use (OR 11.2, 95% CI 3.7-33) and calcification (OR 6.8, 95% CI 1.8-25.4) with persistent rash, abnormal baseline lactate dehydrogenase (OR 11.2, 95% CI 1.4-92) and age at onset (OR 1.3, 95% CI 1-1.4) with weakness, and duration of untreated disease (OR 1.2, 95% CI 1-1.39) with calcification. Conclusion. Using a retrospective, nonrandomized design with propensity score matching, there was little difference in efficacy outcomes between aggressive and standard therapy; however, the sickest patients were treated with aggressive therapy and were not included in the matched analysis. Comprehensive clinical studies are needed to determine therapeutic pathways to the best outcome.

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