Two-Year Follow-Up Outcomes of Premature Infants Enrolled in the Phase I Trial of Mesenchymal Stem Cells Transplantation for Bronchopulmonary Dysplasia.
To determine the long-term safety and outcomes of mesenchymal stem cells (MSCs) for bronchopulmonary dysplasia in premature infants enrolled in a previous phase I clinical trial up to 2 years of corrected age (CA).



We assessed serious adverse events, somatic growth, and respiratory and neurodevelopmental outcomes at visit 1 (4-6 months of CA), visit 2 (8-12 months of CA), and visit 3 (18-24 months of CA) in a prospective longitudinal follow-up study up to 2 years' CA of infants who received MSCs (MSC group). We compared these data with those from a historical case-matched comparison group.



One of 9 infants in the MSC group died of Enterobacter cloacae sepsis at 6 months of CA, the remaining 8 infants survived without any transplantation-related adverse outcomes, including tumorigenicity. No infant in the MSC group was discharged with home supplemental oxygen compared with 22% in the comparison group. The average rehospitalization rate in the MSC group was 1.4/patient because of respiratory infections during 2 years of follow-up. The mean body weight of the MSC group at visit 3 was significantly higher compared with that of the comparison group. No infant in the MSC group was diagnosed with cerebral palsy, blindness, or developmental delay; in the comparison group, 1 infant was diagnosed with cerebral palsy and 1 with developmental delay.



Intratracheal transplantation of MSCs in preterm infants appears to be safe, with no adverse respiratory, growth, and neurodevelopmental effects at 2 years' CA.



ClinicalTrials.gov: NCT01632475.
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
So Yoon
SY
Ahn
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea; Department of Health Sciences and Technology, Samsung Advanced Institute for Health Sciences & Technology, Sungkyunkwan University, Seoul, Republic of Korea; Stem Cell and Regenerative Medicine Institute, Samsung Medical Center, Seoul, Republic of Korea.
Yun Sil
YS
Chang
Department of Radiology and Center for Imaging Science, Samsung Medical Center, Sungkyunkwan University School of Medicine, Republic of Korea.
Ji Hye
JH
Kim
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Se In
SI
Sung
Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea; Department of Health Sciences and Technology, Samsung Advanced Institute for Health Sciences & Technology, Sungkyunkwan University, Seoul, Republic of Korea; Stem Cell and Regenerative Medicine Institute, Samsung Medical Center, Seoul, Republic of Korea. Electronic address: wonspark@skku.edu.
Won Soon
WS
Park
D001997:Bronchopulmonary Dysplasia / Q000517:prevention & control*; D002657:Child Development; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D016972:Enterobacter cloacae; D004756:Enterobacteriaceae Infections / Q000401:mortality; D005500:Follow-Up Studies; D065386:Historically Controlled Study; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007234:Infant, Premature; D045164:Mesenchymal Stem Cell Transplantation; D010359:Patient Readmission / Q000706:statistics & numerical data; D011446:Prospective Studies; D056910:Republic of Korea / Q000453:epidemiology; D018805:Sepsis / Q000382:microbiology / Q000401:mortality; D015430:Weight Gain
bronchopulmonary dysplasia; cell transplantation; cord blood stem cell transplantation; infant, premature; mesenchymal stem cells