BackgroundEpidemiological studies have suggested an association between maternal vitamin D dietary intake during pregnancy and risk of asthma and allergy in the offspring. However, prospective clinical studies on vitamin D measured in cord blood and development of clinical end-points are sparse.ObjectiveTo investigate the interdependence of cord blood 25-hydroxyvitamin D (25(OH)-Vitamin D) level and investigator-diagnosed asthma- and allergy-related conditions during preschool-age.MethodsCord blood 25(OH)-Vitamin D level was measured in 257 children from the Copenhagen Prospective Studies on Asthma in Childhood (COPSAC2000) at-risk mother-child cohort. Troublesome lung symptoms (TROLS), asthma, respiratory infections, allergic rhinitis, and eczema, at age 0–7 yrs were diagnosed exclusively by the COPSAC pediatricians strictly adhering to predefined algorithms. Objective assessments of lung function and sensitization were performed repeatedly from birth.ResultsAfter adjusting for season of birth, deficient cord blood 25(OH)-Vitamin D level (<50 nmol/L) was associated with a 2.7-fold increased risk of recurrent TROLS (HR = 2.65; 95% CI = 1.02–6.86), but showed no association with respiratory infections or asthma. We saw no association between cord blood 25(OH)-Vitamin D level and lung function, sensitization, rhinitis or eczema. The effects were unaffected from adjusting for multiple lifestyle factors.ConclusionCord blood 25(OH)-Vitamin D deficiency associated with increased risk of recurrent TROLS till age 7 years. Randomized controlled trials of vitamin D supplementation during pregnancy are needed to prove causality.

The interobserver agreement was moderate to almost perfect, but the intraobserver agreement was only fair to moderate. A simpler classification system or a centralization of assessments would probably increase kappa values.

Neuromuscular Disorders 31 (2021) S47-S162 treatment was ongoing in 7/13 patients. As of November 12, 2020, 31 patients (IV cohort, n = 23; IT cohort, n = 8) were enrolled in LT-002. One patient in each cohort reported SAEs, and one AESI was reported in the IV cohort (treatment-unrelated). None required permanent ventilation and 9 patients reported baseline respiratory support. Among patients for whom motor milestone data are available from baseline visits as of the data cutoff, 11 new milestones that were not achieved in the parent study were achieved for 4 patients, all in the IV cohort. The highest new milestone achieved in the 4 IV cohort patients was sitting without support. Enrollment is ongoing in LT-002. A single IV administration of onasemnogene abeparvovec at the therapeutic dose provided a sustained, durable response for up to 5.6 years since dosing for patients up to 6.1 years of age. There have been no reported treatment-related SAEs or AESIs, supporting a favorable benefit-risk profile.