Pharmacokinetics and Safety of Oral Pyrophosphate in Systemic Sclerosis Patients-A Pilot Study.

ABSTRACT:

Objective

Calcinosis is a manifestation of systemic sclerosis with a severe negative impact on quality of life. Efforts to find a treatment for calcinosis have been hindered by our limited understanding of the underlying pathomechanism. We propose that extracellular pyrophosphate deficiency may have a causal role in the formation of these mineralized deposits, because pyrophosphate inhibits the deposition of hydroxyapatite-the major constituent of calcinosis-and supplementing pyrophosphate may have therapeutic potential in the treatment of calcinosis.

Methods

Systemic sclerosis patients were treated with a single dose of 50 mg/kg disodium pyrophosphate (cohort 1, n = 10), or a daily dose of 25 mg/kg disodium pyrophosphate for seven consecutive days (cohort 2, n = 10). Safety

SUBMITTER: Bodor G 

PROVIDER: S-EPMC12037987 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature