Substituting a randomised placebo control group with a historical placebo control in an endometriosis pain trial: a case study re-evaluating trial data using historical control data from another trial

Marius Sieverding, Christoph Gerlinger, Christian Seitz
BMJ open · 2023 · vol. 13(7) , pp. e063188 · doi:10.1136/bmjopen-2022-063188 · PMID:37479520 · PMC10364147 · W4385064449
article OA: gold CC0 ⤵ 1 in-corpus citation
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Substituting a randomized placebo control group with a historical control group and propensity score matching reproduced similar endometriosis pain trial results as the original study.

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Abstract

OBJECTIVE: The substitution of an in-study control population with a historical control (HC) population is considered a viable option for reducing the necessary recruitment of control patients. However, it is necessary to evaluate whether this method is applicable to studies on indications targeting endometriosis-associated pelvic pain (EAPP). This study aims to evaluate the potential bias in the results of an EAPP study with an HC arm. METHODS: For this case study, we re-evaluated data from a randomised, placebo-controlled trial using dienogest daily to treat EAPP with an HC arm based on data from a second randomised, placebo-controlled trial in the same indication. Propensity Score (PS) matching was used to match between the treatment and HC arm on all baseline variables. To evaluate the effect of matching on the introduced bias, we evaluated efficacy parameters with the full treatment and control group, as well as the matched group. RESULTS: The difference between means (placebo minus treatment) in change in pain, as measured on the Visual Analogue Scale from baseline to end of treatment, deviates in the comparison treatment/pool of HC (7.15 (0.22 to 14.08)) from the overall in-study group (reference: 11.89 (6.06 to 17.73)). After PS matching on the baseline variables, the difference between means (11.79 (4.09 to 19.5)) is close to the reference. CONCLUSIONS: Using HC with PS matching has proven to be useful in the setting of treating EAPP, while emphasis must be given to the selection mechanism and the underlying assumptions. This case study has shown that even for studies which are very similar in design, heterogeneity and between-study variations are present. With the use of an HC arm, it was possible to reproduce similar results than in the original study, while the PS matching improved the comparability considerably. For the main endpoint, PS matching could reproduce the original study results. TRIAL REGISTRATION NUMBER: NCT00225199, NCT00185341.

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Condition tags

endometriosischronic_pelvic_pain

MeSH descriptors

Endometriosis Endometriosis Endometriosis Endometriosis Endometriosis Control Groups Control Groups Control Groups Female Female Female Humans Humans Humans Pain Measurement Pain Measurement Pain Measurement Pelvic Pain Pelvic Pain Pelvic Pain

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References (25)

Cited by (1)

SciLite annotations

chemicals 7
dienogest dienogest ibuprofen ibuprofen dienogest dienogest dienogest

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