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Paediatric lung disease
Original research
Five-year follow-up of phase II trial of stromal cells for bronchopulmonary dysplasia
  1. So Yoon Ahn1,2,
  2. Yun Sil Chang1,2,3,
  3. Myung Hee Lee4,
  4. Sein Sung1,
  5. Ai-Rhan Kim5,
  6. Won Soon Park6
  1. 1Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea
  2. 2Cell and Gene Therapy Institute, Samsung Medical Center, Seoul, South Korea
  3. 3Department of Health Sciences and Technology, SAIHST, Sungkyunkwan University, Seoul, South Korea
  4. 4Social Information Research Institute, Seoul, South Korea
  5. 5Pediatrics, University of Ulsan, Ulsan, South Korea
  6. 6Pediatrics, Gangnam CHA Hospital, CHA University School of Medicine, Seoul, South Korea
  1. Correspondence to Dr Won Soon Park, Pediatrics, CHA University School of Medicine, Pocheon, Gyeonggi-do, Korea (the Republic of); wonspark{at}skku.edu

Abstract

Background We previously performed a phase II randomised double-blind clinical trial of mesenchymal stromal cell (MSCs) transplantation to prevent bronchopulmonary dysplasia in extremely premature infants. Subsequently, we followed the infants enrolled in this clinical trial to determine the safety and effectiveness of MSCs against bronchopulmonary dysplasia at 5-year follow-up.

Methods We evaluated infants at 5 years of age receiving placebo or MSCs in a prospective follow-up study.

Results In terms of the primary end point of composite respiratory morbidities, including respiratory problem-related readmission, emergency department visits or oxygen therapy, the MSC group had a rate of 60.7% for composite morbidities, while the control group showed a tendency of higher rate of 83.9% for the same outcomes without statistical significance. In terms of the secondary outcomes, the MSC group infants showed a tendency of being less likely to visit emergency department (control 67.7% vs MSC 35.7%) and to receive oxygen therapy (control 29.0% vs MSC 3.6%). No difference was observed in the incidence of respiratory problem-related hospital readmission or wheezing episodes between the groups.

Conclusion Intratracheally instilled MSCs showed the possibility of potential to decrease respiratory symptom-related emergency department visits and oxygen therapy episodes in infants born extremely preterm during the 5 years after a phase II randomised controlled, double-blind trial of MSCs transplantation for bronchopulmonary dysplasia. This small size study suggests preliminary insights that can be further tested using larger sample sizes.

Trial registration number NCT01897987.

  • critical care
  • paediatric lung disaese

Data availability statement

Data are available on reasonable request. The data supporting the findings of this study are available from the corresponding author, WSP, on reasonable request. Investigators whose proposed use of the data was approved by an independent review committee were identified for individual participant data meta-analysis. The data included de-identified participants that underlie the results reported in this article.

https://doi.org/10.1136/thorax-2022-219622

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    Data availability statement

    Data are available on reasonable request. The data supporting the findings of this study are available from the corresponding author, WSP, on reasonable request. Investigators whose proposed use of the data was approved by an independent review committee were identified for individual participant data meta-analysis. The data included de-identified participants that underlie the results reported in this article.

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    Footnotes

    • SYA and YSC are joint first authors.

    • SYA and YSC contributed equally.

    • A-RK and WSP contributed equally.

    • Contributors SYA, YSC, SS, MHL and WSP were responsible for the conceptualisation and design of the study. SYA, SS and MHL were responsible for data acquisition. SYA, YSC, MHL and WSP were responsible for the data analysis and interpretation. SYA, YSC, MHL and WSP prepared and drafted the manuscript. SYA, YSC, MHL and WSP were responsible for the statistical methods and data analysis. SYA, YSC, SS, MHL and WSP were responsible for manuscript critiques and reviews. All the authors have approved the manuscript. The corresponding authors had full access to all data in this study and take complete responsibility for the integrity of the data and accuracy of the data analysis.

    • Funding This study was supported by MEDIPOST (PHO0133531); the Future Medicine 2030 project of the Samsung Medical Center (#SMX1230441) and the Korea Drug Development Fund funded by the Ministry of Science and ICT, Ministry of Trade, Industry, and Energy and Ministry of Health and Welfare (HN22C0414, Republic of Korea). Data collection, analysis and interpretation of the results were primarily performed by the principal investigators. The principal investigators reviewed all datasets. The writing of this report and the decision to submit it were made by all the authors who take responsibility for the manuscript content.

    • Disclaimer The funding agencies did not play any role in this study.

    • Competing interests Samsung Medical Center and MEDIPOST have issued or filed patents for ‘Method of treating lung diseases using cells separated or proliferated from umbilical cord blood’ under the name of YSC and WSP. The relevant application number is PCT/KR2007/000535.

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.