Two to five repeated measurements per patient reduced the required sample size considerably in a randomized clinical trial for patients with inflammatory rheumatic diseases

BMC Research Notes

Kåre Birger Hagen¹  Petter Mowinckel¹  Heidi Andersen Zangi¹  Geir Smedslund¹ 

[1] National Resource Centre for Rehabilitation in Rheumatology, Diakonhjemmet Hospital, P.O. Box 23 Vindern, Oslo, 0319, Norway

关键词: General health questionnaire;    Arthritis;    Clinical trial;    Statistical power;    Sample size;   

Others  :  1144868
DOI  :  10.1186/1756-0500-6-37

 received in 2012-09-07, accepted in 2013-01-29,  发布年份 2013

【 摘 要 】

Background

Patient reported outcomes are accepted as important outcome measures in rheumatology. The fluctuating symptoms in patients with rheumatic diseases have serious implications for sample size in clinical trials. We estimated the effects of measuring the outcome 1-5 times on the sample size required in a two-armed trial.

Findings

In a randomized controlled trial that evaluated the effects of a mindfulness-based group intervention for patients with inflammatory arthritis (n=71), the outcome variables Numerical Rating Scales (NRS) (pain, fatigue, disease activity, self-care ability, and emotional wellbeing) and General Health Questionnaire (GHQ-20) were measured five times before and after the intervention. For each variable we calculated the necessary sample sizes for obtaining 80% power (α=.05) for one up to five measurements.

Two, three, and four measures reduced the required sample sizes by 15%, 21%, and 24%, respectively. With three (and five) measures, the required sample size per group was reduced from 56 to 39 (32) for the GHQ-20, from 71 to 60 (55) for pain, 96 to 71 (73) for fatigue, 57 to 51 (48) for disease activity, 59 to 44 (45) for self-care, and 47 to 37 (33) for emotional wellbeing.

Conclusions

Measuring the outcomes five times rather than once reduced the necessary sample size by an average of 27%. When planning a study, researchers should carefully compare the advantages and disadvantages of increasing sample size versus employing three to five repeated measurements in order to obtain the required statistical power.

【 授权许可】

   
2013 Smedslund et al.; licensee BioMed Central Ltd.

【 图 表 】

Figure 1.

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