Stoss therapy is safe for treatment of vitamin D deficiency in pediatric patients undergoing HSCT

Area of Science:

• Pediatric Hematology
• Endocrinology
• Transplant Medicine

Background:

• Vitamin D deficiency is prevalent in pediatric hematopoietic stem cell transplant (HSCT) recipients.
• Standard and aggressive treatments often fail to resolve vitamin D deficiency in these patients.

Purpose of the Study:

• To evaluate the safety and efficacy of single high-dose oral vitamin D (Stoss therapy) for treating vitamin D deficiency in pediatric HSCT recipients.
• To compare Stoss therapy with standard of care in achieving adequate vitamin D levels.

Main Methods:

• A randomized trial involving 48 pediatric patients (ages 1-21) undergoing HSCT.
• Patients received either Stoss regimen plus supplementation or standard of care based on US Endocrine Society guidelines.
• Baseline and Day +30 serum 25-hydroxyvitamin D (25-OHD) levels were measured.

Main Results:

• 71% of patients had insufficient or deficient vitamin D at baseline.
• Stoss therapy was well-tolerated with no observed toxicity.
• At Day +30, Stoss therapy resulted in significantly higher mean 25-OHD levels (42.3 μg/l vs. 35.6 μg/l) and a greater proportion of patients achieving adequate levels (85% vs. 65%).

Conclusions:

• Single high-dose vitamin D (Stoss therapy) is a safe and effective treatment for vitamin D deficiency in children undergoing HSCT.
• Stoss therapy may achieve sufficient vitamin D levels more rapidly than standard care.
• ClinicalTrials.gov registration: NCT03176849.