Acceptance of Cell and Gene Therapy Among Patients with Hematological Diseases in China: A Cross-Sectional Study.

BACKGROUND

Cell and gene therapy (CGT) offers breakthrough solutions for hematological diseases. Patient acceptance is a key determinant of its successful clinical translation. This study investigates CGT acceptance among hematological patients and explores its primary determinants.

METHODS

A cross-sectional study was conducted (June-July 2025) with 385 hematological patients at a single specialized hematology center (Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences). Each participant completed a sociodemographic and clinical questionnaire, the Risk Perception Assessment Scale for Patients with Hematological Diseases, and the EuroQol 5-Dimensional 5-Level (EQ-5D-5L) questionnaire. Correlation and multivariable ordinal logistic regression analyses were performed using SPSS 26.0.

RESULTS

Most patients (76.6%) were cautious toward CGT. Risk perception (OR = 0.944; 95% CI: 0.921-0.967), gender (male vs female: OR = 1.754; 95% CI: 1.061-2.901), medical insurance status (insured vs uninsured: OR = 5.485; 95% CI: 2.020-14.895), disease types (acute leukemia vs other hematological diseases: OR = 2.936; 95% CI: 1.169-7.374), and prior experience with CGT-related clinical trials (with experience vs without: OR = 6.278; 95% CI: 2.798-14.083) were five key predictors. Physical risk and economic risk constituted the two primary risk barriers hindering CGT acceptance. Subgroup analysis further showed that household income and treatment modality were significant factors for patients with acute leukemia and those with myelodysplastic syndrome, respectively. All P < 0.05.

CONCLUSION

Enhancing CGT acceptance among patients with hematological diseases may be facilitated through tailored educational interventions and expanded financial support. However, further multicenter and prospective studies are required to validate these cross-sectional associations and evaluate the actual effectiveness of specific interventions in clinical practice.