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

David G. Sweet, Mark Turner, Zbynek Straňák, Richard Plavka, Paul Clarke, Ben Stenson, Dominique Singer, Rangmar Goelz, Laura Fabbri, Guido Varoli, Annalisa Piccinno, Debora Santoro, Dorothea Del Buono, Christian P. Speer

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Abstract

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.

Original languageEnglish
JournalJournal of Maternal-Fetal & Neonatal Medicine
Early online date20 Dec 2020
DOIs
Publication statusE-pub ahead of print - 20 Dec 2020
Externally publishedYes

Keywords

  • 24-month developmental assessment
  • Synthetic surfactant
  • first-in-human study
  • respiratory distress syndrome

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