Five-year follow-up of phase II trial of autologous cord blood mononuclear cells for bronchopulmonary dysplasia prevention in very preterm infants.
We previously performed a phase II clinical trial involving the intravenous infusion of autologous cord blood mononuclear cells (ACBMNCs) to prevent bronchopulmonary dysplasia in premature infants. We reported the outcomes before discharge and at 2-year follow-up of the enrolled infants. We have reported the developmental outcomes at the 5-year follow-up visit.
We evaluated the 5-year long-term outcomes of ACBMNCs infusion, including growth, respiratory, and neurodevelopmental outcomes, by interviewing the parents. Children's development was assessed using scores on the Chinese version of the Ages & Stages Questionnaires (ASQ), third edition (ASQ-3), completed by the parents or guardians. The primary outcome was the neurodevelopmental outcome. The secondary outcomes were growth and respiratory outcomes.
In terms of the primary endpoint, intravenously infused ACBMNCs significantly improved the scores of the fine motor and personal-social domains in the ASQ (P = .044 and P = .048, respectively). This study also showed high consistency between the motor index in the Bayley score at a 2-year follow-up with both gross and fine motor domain scores, as well as the language index with communication, problem-solving, and personal-social domain scores. Although pneumonia-related hospitalizations were similar between the two groups, the intervention group showed a lower possibility of nocturnal cough.
Infants who received intravenous ACBMNC infusion had better fine motor and personal-social abilities at 5 years than those in the control group. In addition to providing reassuring safety data, the study suggested longer-term neurodevelopmental benefits of ACBMNC intervention early in the NICU course. Further randomized placebo-controlled trials are essential to confirm these findings.
The trial was prospectively registered at ClinicalTrials.gov (NCT02999373), registered on December 21, 2016.
We evaluated the 5-year long-term outcomes of ACBMNCs infusion, including growth, respiratory, and neurodevelopmental outcomes, by interviewing the parents. Children's development was assessed using scores on the Chinese version of the Ages & Stages Questionnaires (ASQ), third edition (ASQ-3), completed by the parents or guardians. The primary outcome was the neurodevelopmental outcome. The secondary outcomes were growth and respiratory outcomes.
In terms of the primary endpoint, intravenously infused ACBMNCs significantly improved the scores of the fine motor and personal-social domains in the ASQ (P = .044 and P = .048, respectively). This study also showed high consistency between the motor index in the Bayley score at a 2-year follow-up with both gross and fine motor domain scores, as well as the language index with communication, problem-solving, and personal-social domain scores. Although pneumonia-related hospitalizations were similar between the two groups, the intervention group showed a lower possibility of nocturnal cough.
Infants who received intravenous ACBMNC infusion had better fine motor and personal-social abilities at 5 years than those in the control group. In addition to providing reassuring safety data, the study suggested longer-term neurodevelopmental benefits of ACBMNC intervention early in the NICU course. Further randomized placebo-controlled trials are essential to confirm these findings.
The trial was prospectively registered at ClinicalTrials.gov (NCT02999373), registered on December 21, 2016.