What type of study is this?

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

December 8, 2025BloodOpen Access

Trends in collecting health-related quality-of-life measures among hematology gene therapy clinical trials

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Authors

GAGregory A. AbelHarvard University AHAndrew HantelHarvard University

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Implication

Analysis reveals low health-related quality-of-life data collection in gene therapy trials for hematologic conditions, suggesting gaps in patient experience measures.

Key Points

Only 16.9% of gene therapy trials collected health-related quality-of-life data as a trial endpoint, highlighting a significant gap.
Multivariable logistic regression showed that odds of data collection decrease with later trial registration years and in trials for malignant conditions.
The majority of trials were phase I/II, with most conducted outside the US, indicating a geographical disparity in trial designs.
Despite FDA guidance to collect patient experience data, fewer trials registered after 2020 achieved health-related quality-of-life data collection.

Cite This Study

Abel et al. (2025) studied this question.

synapsesocial.com/papers/69362f7f4fa91c937236e5ab https://doi.org/10.1182/blood-2025-6157

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Implication

Analysis reveals low health-related quality-of-life data collection in gene therapy trials for hematologic conditions, suggesting gaps in patient experience measures.

Trends in collecting health-related quality-of-life measures among hematology gene therapy clinical trials

Discussion

Member takes

Implication

Key Points

Cite This Study

Trends in collecting health-related quality-of-life measures among hematology gene therapy clinical trials

Discussion

Member takes

Implication

Key Points

Cite This Study

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Also Consider