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

• Published in: Bone marrow transplantation (Basingstoke) Vol. 56; no. 9; pp. 2137 - 2143
• Main Authors: Bodea, Jessica, Beebe, Kristen, Campbell, Courtney, Salzberg, Dana, Miller, Holly, Adams, Roberta, Mirea, Lucia, Castillo, Paul, Horn, Biljana, Bansal, Sandhya, Mohanakumar, Thalachallour, Ngwube, Alexander
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01-09-2021; Nature Publishing Group
• Subjects: 25-Hydroxyvitamin D; 631/250/1904; 692/308/2779/777; Adolescent; Adult; Alfacalcidol; Calcifediol; Calciferol; Care and treatment; Cell Biology; Child; Child, Preschool; Dietary Supplements; Dosage and administration; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Humans; Infant; Internal Medicine; Medicine; Medicine & Public Health; Patients; Pediatric research; Pediatrics; Public Health; Risk factors; Stem cell transplantation; Stem Cells; Therapy; Toxicity; Transplantation; Treatment Outcome; Vitamin D; Vitamin D deficiency; Vitamin D Deficiency - drug therapy; Vitamin deficiency; Young Adult; United States
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/s41409-021-01294-x

Vitamin D deficiency remains common among pediatric patients undergoing hematopoietic stem cell transplant (HSCT) despite both aggressive and standard of care strategies. This study examined the safety and efficacy of single high-dose oral vitamin D therapy (Stoss therapy) for treatment of vitamin D deficiency in HSCT recipients. Patients ages 1–21 years presenting for HSCT were randomized to receive either Stoss regimen plus weekly/daily supplementation or standard of care, per US Endocrine Society guidelines. Among the total 48 subjects, 22 (46%) were randomized to Stoss and 26 (54%) to control arms. Baseline 25-hydroxyvitamin D (25-OHD) levels were insufficient/deficient in total of 34 (71%) patients, without difference between treatment groups. The Stoss regimen was well tolerated and no toxicity was observed. At Day +30, mean 25-OHD levels were significantly higher (
P
 = 0.04) with Stoss (42.3 ± 12 μg/l) compared to controls (35.6 ± 14.3 μg/l), and a higher proportion of Stoss patients had adequate vitamin D levels than controls (85% vs 65%). Stoss therapy is a safe and efficacious treatment option for vitamin D deficiency in children undergoing HSCT and may achieve sufficient levels more rapidly than standard of care. This trial was registered at
www.clinicaltrials.gov
as NCT03176849.