Measurement in Scleroderma Clinical Trials

Pope, Janet

doi:10.1007/978-1-4615-1199-1_16

Cited by 1 publication

(2 citation statements)

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“…Scleroderma is a chronic disease, and long‐term information would be important to obtain on all therapies that are considered to be effective based on short‐term successes. Long‐term followup would allow the assessment of hard clinical outcomes (2, 3) and would give a better picture of the long‐term toxicity and tolerability of each intervention, which are equally important for deciding on its optimal use (10). The available trials showed alarmingly high rates of withdrawals, with rates averaging 36% in trials with followup of longer than 1 year.…”

Section: Discussionmentioning

confidence: 99%

“…Although some insight may be gained from observational studies, the development and evaluation of new therapeutic interventions typically requires the performance of randomized controlled trials (RCTs). The heterogeneity of disease manifestations, the relatively low prevalence of each of them in the general population, the long‐term nature of the major outcomes (2, 3), and logistic considerations make the performance of RCTs in persons with SSc a challenge. Nevertheless, several trials have been performed already in this field.…”

Section: Introductionmentioning

confidence: 99%

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Design and quality considerations for randomized controlled trials in systemic sclerosis

Kyriakidi¹,

Ioannidis²

2002

Arthritis & Rheumatism

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Objective. To appraise systematically randomized controlled trials (RCTs) on systemic sclerosis (SSc) in order to determine whether the parameter of study design and its quality may influence the reporting of efficacy for tested interventions. Methods. Seventy RCTs were analyzed in terms of design, patient characteristics, outcomes, and reported results. Results. Median sample size was 28 patients. Fifty-nine trials were double blind, but only 16 mentioned the randomization mode and only 7 described allocation concealment. There was sufficient information on withdrawals in 37 trials. Larger trials with longer followup scored higher on quality characteristics, but had higher withdrawal rates. Only 8 trials had a followup of more than 1 year. Significant efficacy was less likely to be reported in double-blind studies (P ‫؍‬ 0.029) and in studies with larger rates of withdrawal (P ‫؍‬ 0.032). Specification of the following parameters improved over time: power calculations (P ‫؍‬ 0.0003), outcomes (P ‫؍‬ 0.001), and sample size per arm (P ‫؍‬ 0.011). Conclusions. Several aspects of the quality of design and conduct of SSc RCTs can be improved. Adequately powered trials with longer followup and clear outcomes are needed.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%