Vera A. Tsetlina, MD
Medical Director of Pediatric Physiatry
Westchester Medical Center, New York Medical College
Ossining, New York, United States
Binni Kunvarjee, PharmD
Clinical Pharmacy Specialist, Pediatric Transplant & Cellular Therapy
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Deborah M. Cassidy, DO
Assistant Attending
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Theresa A. Gillis, MD
Chief, Rehabilitation Service and Associate Professor
Memorial Sloan Kettering Cancer Center
Montclair, New Jersey, United States
Andrew C. Harris, MD
Associate Attending, Pediatric Transplantation and Cellular Therapy
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Katarzyna Ibanez, MD
Associate Attending Physiatrist
Memorial Sloan Kettering
New York, New York, United States
Steroid myopathy (SM) is a common complication of glucocorticoid administration. Anabolic steroids have been studied in adult patients to help combat SM, however to our knowledge, there is no published data on the use of anabolic steroids for SM in children. At Memorial Sloan Kettering Cancer Center (MSKCC) a small cohort of children with severe SM secondary to prolonged glucocorticoid exposure for the treatment of Graft versus Host Disease (GVHD) have been treated with oxandrolone, an anabolic steroid that had an approval by the Food and Drug Administration (FDA) as “adjunctive therapy to offset the protein catabolism associated with prolonged administration of corticosteroids” since 1964. Effective June 28, 2023 the FDA has withdrawn approval of oxandrolone and this medication is no longer available. The primary objective of this retrospective study was to assess the safety and preliminary efficacy of oxandrolone when used for severe SM in children with GVHD.
A retrospective medical records review of the eight pediatric and young adult patients (age 8-22 years, median age 14.5). MSKCC REDCap database and descriptive statistics was utilized for data collection and analysis.
Oxandrolone daily dose varied from 1.25 to 10 mg (median 5 mg). The duration of the treatment varied from 6 to 803 days (median 45 days). We report no confirmed adverse effects of oxandrolone use and no data to suggest it having a negative impact on steroid effectiveness in treating GVHD. We also saw no data to suggest any interference of oxandrolone with immune reconstitution.
Longitudinal functional outcomes were recorded and analyzed, however given the retrospective nature of this study, very selective use of oxandrolone (limited to the most debilitated and commonly the sickest population with a poor prognosis) as well as the small patient sample, definitive conclusions regarding efficacy of oxandrolone could not be established.
