Different diseases, different needs: Patient preferences for gene therapy in lysosomal storage disorders, a probabilistic threshold technique survey
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View abstract on PubMed
Summary
This summary is machine-generated.Patient risk tolerance for gene therapy varies with lysosomal storage disease severity. Those with poorer prognoses and fewer treatment options show higher risk tolerance for novel gene therapies.
Area Of Science
- Genetics and Genomics
- Medical Genetics
- Rare Diseases
Background
- Gene therapy is a developing treatment for monogenetic diseases, including lysosomal storage disorders (LSDs).
- Limited data exists on patient preferences and risk tolerance for gene therapy in LSD populations.
- This study investigates risk tolerance across different LSDs with varying prognoses and treatment availabilities.
Purpose Of The Study
- To compare gene therapy-related risk tolerance among patients with Gaucher disease, Fabry disease, and mucopolysaccharidosis type III (MPS III).
- To understand how disease severity and current treatment options influence patient preferences for gene therapy.
Main Methods
- A survey utilizing the probabilistic threshold technique was administered to assess risk tolerance.
- Participants chose between gene therapy and standard care, considering attributes like side effect risks, need for medication, and long-term effectiveness.
- Survey respondents included patients with Gaucher disease (Type 1), Fabry disease, and parents of MPS III (A/B) patients.
Main Results
- Higher disease severity correlated with increased risk tolerance; MPS III representatives were more risk-tolerant than Gaucher disease respondents.
- Fabry disease patients exhibited heterogeneous risk tolerance, with males being more tolerant than females.
- Long-term effectiveness was the least tolerated attribute across all participant groups.
Conclusions
- Patient risk tolerance for gene therapy is influenced by disease prognosis and the efficacy of current treatments.
- Incorporating patient preferences early in gene therapy development is crucial for rare diseases to ensure alignment with patient needs and expectations.
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