135 - Five-year follow-up of phase 2 trial of stem cells for bronchopulmonary dysplasia

We followed up infant enrolled in this clinical trial to determine the long-term safety and effectiveness of MSCs for BPD.

Design/Methods:

In a prospective longitudinal follow-up study up to 5 years of infants received placebo and MSCs, we evaluated respiratory morbidity and neurodevelopmental outcomes.

Primary outcome was composite respiratory morbidities including emergency department (ED) visit, readmission related to respiratory problem, or oxygen therapy.

Results:

MSC group infants had decreased incidence of composite respiratory morbidity during the follow-up period compared to control group infants without statistical significance. However, they were less likely to visit emergency department (ED) (odds ratio, 0.21; 95% credible interval 0.06–0.69) and receive oxygen therapy (odds ratio, 0.11; 95% credible interval 0.01–0. 94). No difference was observed between groups in the incidence of respiratory problem related hospital readmission and wheezing episode. Only in the subgroup of infants born at 25-28 gestational weeks, wheezing episode was decreased (odds ratio, 0.09; 95% credible interval 0.01–0.94) in MSC group than control group. No difference was observed between groups in the incidence of developmental delay at 5 years old assessed with Korean Developmental Screening Test. Only in the subgroup of infants born at 25-28 gestational weeks, MSC group showed lower incidence of motor function delay (odds ratio, 0.10; 95% credible interval 0.01–0.97) than control group.

Conclusion(s):

 Intratracheally instilled MSCs significantly reduced respiratory symptom related ED visits and oxygen therapy episodes in infants born extremely preterm during 5 years follow-up period after phase 2 randomized, controlled, double-blind trial of MSCs transplantation for BPD.