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

Bone Marrow Transplantation volume 56, pages 2137–2143 (2021)Cite this article

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Abstract

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.

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Fig. 1: Design and recruitment of pediatric patients presenting for HSCT in the randomized controlled study of Stoss regimen.
Fig. 2: Vitamin D levels in the both arms.
Fig. 3: Toxicity related to hypervitaminosis D.

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Acknowledgements

The authors thank research coordinators Kayla Burgett, Kara Kronemeyer, and Jessica Cline, who collected the data for the trial.

Funding

Grants from the Learners Research Fund, Phoenix Children’s Hospital and Valley Research Partnership Grant (P1201707), University of Arizona, provided support for the trial.

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Authors and Affiliations

  1. St Jude Children’s Research Hospital, Phoenix, AZ, USA

    Jessica Bodea

  2. Phoenix Children’s Hospital, Phoenix, AZ, USA

    Kristen Beebe, Courtney Campbell, Dana Salzberg, Holly Miller, Roberta Adams, Lucia Mirea & Alexander Ngwube

  3. Mayo Clinic Arizona, Phoenix, AZ, USA

    Kristen Beebe, Roberta Adams & Alexander Ngwube

  4. UF Health Shands Children’s Hospital, Gainesville, FL, USA

    Paul Castillo & Biljana Horn

  5. St. Joseph’s Hospital & Medical Center, Phoenix, AZ, USA

    Sandhya Bansal & Thalachallour Mohanakumar

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Correspondence to Alexander Ngwube.

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Bodea, J., Beebe, K., Campbell, C. et al. Stoss therapy is safe for treatment of vitamin D deficiency in pediatric patients undergoing HSCT. Bone Marrow Transplant 56, 2137–2143 (2021). https://doi.org/10.1038/s41409-021-01294-x

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