Experience with stem cell therapy in neonates: Hope or disappointment?

Background: Mesenchymal stem cell (MSC) therapy offers a novel treatment option for neonatal diseases. This study aimed to evaluate the clinical characteristics, efficacy, and early prognosis of neonates treated with MSC therapy in Turkiye. Methods: This retrospective, cross-sectional study included neonates treated with MSC therapy at multiple centers from 2015 to 2022. Data included antenatal, natal, and postnatal characteristics, MSC therapy indications, timing, dosage, treatment response (such as reduction in respiratory support or extubation within 72 h) and pre/post-discharge evaluations. Results: MSC therapy indications in 29 cases included bronchopulmonary dysplasia (BPD, n = 21), intraventricular hemorrhage (IVH, n = 9), and necrotizing enterocolitis (NEC, n = 6), with seven treated for two diagnoses. For those treated for BPD, mean ± SD gestational age was 26^2/7 ± 1^4/7weeks (23^3/7–28^1/7) and birth weight was 784 ± 235 g (420–1470). Five received simultaneous treatment for Grade 3 IVH, and one for Stage 3 NEC. Median MSC therapy start was 33 days (7–181), with routes including intratracheal+intravenous (n = 15), intratracheal (n = 4), and intravenous (n = 2). Median MSC dose was 1x10⁷cells/kg, with 43 % receiving repeated treatments. Seventeen (81 %) responded to treatment at varying levels, with higher antenatal steroid use in responders (p = 0.021). All treated >30 days had severe BPD, while 5/9 treated ≤30 days had severe BPD (p = 0.021). Treatment ≤30 days was associated with discharge, whereas 5/12 treated >30 days died (p = 0.045). At a median of 9 months, 31 % of survivors had neurodevelopmental delay. Conclusion: Early MSC treatment in BPD is promising, but larger randomized controlled trials are required to better define optimal timing, dosage, and long-term outcomes.