Date of Award

2020

Degree Name

Master of Medicine / Surgery (Thesis)

Schools and Centres

Medicine

First Supervisor

Professor Craig Munns

Abstract

Objectives: Paediatric vitamin D (25-hydroxyvitamin D - 25OHD) deficiency can lead to nutritional rickets and extra-skeletal complications. Compliance with daily therapy can be difficult, making high dose, short-term vitamin D (stoss) therapy attractive to correct vitamin D deficiency. We compared the effectiveness and safety of standard versus stoss therapy in treating childhood 25OHD deficiency.

Study Design: Children aged 2 - 16 years with 25OHD <50nmol/L were randomized to either standard (5,000IU daily for 80 days) or stoss (100,000 IU weekly for 4 weeks) cholecalciferol. Participants underwent evaluation of effectiveness and safety. 25OHD, random spot calcium: creatinine ratio (Ca:Cr) and compliance were measured at 12 weeks.

Results: 151 children were enrolled in the study (68 standard and 83 stoss), median age 9 years (IQR: 6 - 12 years). Baseline 25OHD levels were 26 nmol/L (IQR: 19 - 35 nmol/L) and 32 nmol/L (IQR: 24 - 39 nmol/L) in the standard and stoss groups respectively. At 12 weeks, the median 25OHD level was significantly greater in the standard vs. stoss group (81 vs. 67 nmol/L; p=0.005), however, >80% of participants in both groups achieved sufficiency (25OHD>50nmol/L) and had normal urinary Ca:Cr, with no significant difference seen between groups. Compliance was similar in the two groups.

Conclusion: Compared to stoss, standard therapy achieved higher 25OHD levels at 12 weeks; however, in both groups there were a similar proportion of participants who achieved 25OHD sufficiency, with no evidence of toxicity. Unlike other studies, simplifying the treatment regimen did not improve compliance. These results support stoss therapy as an effective and safe alternative therapy for the treatment of paediatric vitamin D deficiency.

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