Assessment of documentation and reporting methods for annual bleeding rate in hemophilia clinical trials: communication from the ISTH SSC Subcommittee on Factor VIII, Factor IX, and Rare Coagulation Disorders.

What was studied

This paper looked at how hemophilia trials report bleed rates. The yearly bleed rate is a key outcome for judging hemophilia drugs. Earlier work found big gaps in bleed definitions and in follow-up time. A society group had set shared definitions over a decade ago. This project asked if recent main trials now use them. The team reviewed trial papers on clotting factor drugs, non-factor drugs and gene therapies.

What they found

27 trials were assessed. Most did not define a bleed event. Only 17 trials used the yearly bleed rate in their main outcome. Trials differed in which bleeds they counted. Some reported all bleeds, some only treated bleeds, and some both. In most trials, planned follow-up was under 52 weeks.

What it means for patients

The authors said uniform bleed definitions would help. So would clear follow-up time and analysis methods. This matters most for main trials. They said it would ease comparison of drugs and shared choices about care. They said wide use would also improve pooled analyses.

Why it is not certain yet

The review covered main trial papers only. It did not assess registries or observational studies. A review of reports shows how studies are written, not how well drugs work.

Source

Assessment of documentation and reporting methods for annual bleeding rate in hemophilia clinical trials: communication from the ISTH SSC Subcommittee on Factor VIII, Factor IX, and Rare Coagulation Disorders.

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