Intravenous Sodium Thiosulfate Demonstrates Efficacy for Calcinosis Associated with Adult and Juvenile Idiopathic Inflammatory Myopathy

Authors

• A Schiffenbauer
• J Agafonova
• A Derkyi
• K sarkar
• J Wilkerson
• A Ehrlich
• G Joe
• J Shrader
• M Jain
• LG Rider

Abstract

Background: The idiopathic inflammatory myopathies (IIM) are systemic autoimmune disorders that impact multiple organs, including muscle and skin. Calcinosis is a complication of IIM as well as a range of other disorders. There are no FDA approved or generally agreed upon highly efficacious treatments for calcinosis. Sodium thiosulfate (NaTS) is a calcium chelator and has been reported to potentially improve calcinosis.
Methods: Fourteen patients with moderate to severe widespread calcinosis were enrolled into the treatment phase of an investigational drug study to assess for the efficacy of IV NaTS for calcinosis associated with IIM. Patients were dosed three times a week for 10 weeks. Patient and physician measures were captured on 10 cm visual analogue scales. SkinDex, a QoL measure, was scored using its published scoring systems.
Results: Thirteen of 14 patients completed treatment. By physician assessment the patients had a statistically significant improvement in calcinosis activity, damage and severity over 10 weeks receiving sodium thiosulfate compared to 10 weeks on standard-of-care therapy (p=0.002 for all). Comparing the same time periods, patients also improved in overall IIM global damage and activity (p-0.004, 0.011 respectively). Using the same comparison adult patients and parents of minor patients rated their calcinosis as significantly improved (p=0.026). The SkinDex also demonstrated improvement (p-0.013). The drug was well tolerated, with only one serious adverse event leading to delay of a drug dose.
Conclusions: Sodium thiosulfate demonstrated efficacy for calcinosis associated with IIM over a 10-week period by physician and patient assessment.

Scientific Focus Area: Clinical Research

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