What type of study is this?

This is a Human Clinical Trial study (also classified as: Pre-Registered Trial).

What question did this study set out to answer?

To examine trial-level predictors for health-related quality-of-life (HRQOL) data collection in hematology gene therapy trials.

December 8, 2025BloodOpen Access

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Analysis reveals low collection rates of HRQOL data in hematology gene therapy trials, suggesting gaps despite FDA guidance.

To examine trial-level predictors for health-related quality-of-life (HRQOL) data collection in hematology gene therapy trials.
Analyzed 207 trials from ClinicalTrials.gov fitting inclusion criteria of interventional hematology gene therapies.
Collected data on HRQOL as an endpoint based on standard definitions and trial characteristics.
Used multivariable logistic regression to assess associations between HRQOL collection and factors like trial phase and sponsor type.
Only 35 out of 207 trials (16.9%) collected HRQOL data related to endpoints.
Fewer trials registered after 2020 collected HRQOL data compared to earlier periods.
Key factors linked to lower HRQOL data collection included trial type and registration year.

Abel et al. (2025) studied this question.

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