Using Steroids to Improve Rhabdomyolysis Outcomes in Pediatric Patients

​Principal Investigator: Dr. Natasha Shur

Condition: Rhabdomyolysis

Research Description: Rhabdomyolysis is a rare disease characterized by muscle breakdown, resulting in damaged muscle tissues releasing proteins and electrolytes into the blood. The severity of illness ranges from asymptomatic to hospitalization, kidney failure and life-threatening disease. In addition, rhabdomyolysis affects minority populations at significantly higher rates, and research has shown an increased risk in patients with the sickle cell trait. Current rhabdomyolysis treatments remain largely limited to fluids and supportive therapy. The research team found that off-label treatment with a generic, readily available steroid, dexamethasone, may be effective in potentially decreasing hospitalization. This prospective, controlled, open label clinical trial will formally test these results seen in the clinic with off-label use. Pediatric rhabdomyolysis patients will be recruited during in-patient episodes and be offered steroids plus standard of care or standard of care alone. The research team expects that the addition of steroids to standard of care will improve patient outcomes and decrease hospitalization rates.

Funding Partners: Cures Within Reach

CWR funding role: Primary funder