TY - JOUR
T1 - A first-in-human clinical study of a new SP-B and SP-C enriched synthetic surfactant (CHF5633) in preterm babies with respiratory distress syndrome: two-year outcomes
AU - Sweet, David G.
AU - Turner, Mark
AU - Straňák, Zbynek
AU - Plavka, Richard
AU - Clarke, Paul
AU - Stenson, Ben
AU - Singer, Dominique
AU - Goelz, Rangmar
AU - Fabbri, Laura
AU - Varoli, Guido
AU - Piccinno, Annalisa
AU - Santoro, Debora
AU - Del Buono, Dorothea
AU - Speer, Christian P.
PY - 2022/9
Y1 - 2022/9
N2 - Objective: To assess at 24 months corrected age (CA) the neurological, respiratory, and general health status of children born prematurely from 27 +0 to 33 +6 weeks’ gestation who were treated in a first-in-human study with a new fully synthetic surfactant (CHF5633) enriched with SP-B and SP-C proteins. Outcome measures: Children were assessed using Bayley Scales of Infant Development (BSID), with a score below normal defined as BSID-II Mental Development Index score <70, or BSID-III cognitive composite score <85. In addition, a health status questionnaire was used to check for functional disability including respiratory problems and related treatments, sensory and neurodevelopment assessments, communication skills as well as the number of hospitalizations. Results: 35 of 39 survivors had a neurodevelopmental assessment, 24 infants being evaluated by Bayley’s Scales and 11 by health status questionnaires only. 23 children had scores within normal limits and one had BSID-III <85. The remaining 11 were judged clinically to have normal development. Health status questionnaires detected only issues that would normally be expected in preterm-born children. Conclusions: This assessment offers reassurance that treatment with CHF5633 surfactant was not associated with adverse neurodevelopmental, respiratory, or health outcomes by two years corrected age.
AB - Objective: To assess at 24 months corrected age (CA) the neurological, respiratory, and general health status of children born prematurely from 27 +0 to 33 +6 weeks’ gestation who were treated in a first-in-human study with a new fully synthetic surfactant (CHF5633) enriched with SP-B and SP-C proteins. Outcome measures: Children were assessed using Bayley Scales of Infant Development (BSID), with a score below normal defined as BSID-II Mental Development Index score <70, or BSID-III cognitive composite score <85. In addition, a health status questionnaire was used to check for functional disability including respiratory problems and related treatments, sensory and neurodevelopment assessments, communication skills as well as the number of hospitalizations. Results: 35 of 39 survivors had a neurodevelopmental assessment, 24 infants being evaluated by Bayley’s Scales and 11 by health status questionnaires only. 23 children had scores within normal limits and one had BSID-III <85. The remaining 11 were judged clinically to have normal development. Health status questionnaires detected only issues that would normally be expected in preterm-born children. Conclusions: This assessment offers reassurance that treatment with CHF5633 surfactant was not associated with adverse neurodevelopmental, respiratory, or health outcomes by two years corrected age.
KW - 24-month developmental assessment
KW - Synthetic surfactant
KW - first-in-human study
KW - respiratory distress syndrome
UR - http://www.scopus.com/inward/record.url?scp=85097831503&partnerID=8YFLogxK
U2 - 10.1080/14767058.2020.1863363
DO - 10.1080/14767058.2020.1863363
M3 - Article
SN - 1476-4954
VL - 35
SP - 4739
EP - 4742
JO - Journal of Maternal-Fetal & Neonatal Medicine
JF - Journal of Maternal-Fetal & Neonatal Medicine
IS - 24
ER -
