A randomized phase 2 placebo-controlled trial of clobetasol solution for oral chronic graft-versus-host disease therapy.
A topical treatment achieved a 91% response rate for mouth problems in graft-versus-host disease, potentially improving quality of life.
Chronic graft-versus-host disease (cGVHD) affects multiple organ systems and frequently the mouth. Of the 35 of 40 randomized subjects who reached the day 28, 19% achieved a complete response (CR), 72% a partial response (PR), and 9% had stable disease (SD), resulting in an overall response rate (ORR) of 91%.
What the study was
- Study design
- Randomized study
- Population
- patients
- Sample size
- 14
- Category
- Other
- Maturity
- Exploratory
- Journal
- Blood advances
Why it surfaced
Score 7/10 [PROMISING_PRELIMINARY]: Randomized study (Journal Article) matched 'Rare diseases with high unmet need'. Components — novelty:1/3, relevance:2/3, design:2/2, population:2/2. Confidence: high. Conservative scoring applied per v1.3 rubric.
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