Introduction:
Calcinosis is a highly debilitating condition in Scleroderma causing pain and functional limitation often leading to ulceration with risk of infection and amputation. The pathogenesis of calcinosis remains unknown limiting the treatments available. With such high debilitation, patients highlight calcinosis as a priority area for research and clinical management.
This study explores the literature, describes the clinical characteristics and patient-reported impact of calcinosis, and initiates a framework of appropriate outcome measures to evaluate targeted treatments in future clinical trials.
Methodology:
Phase One: Postal survey of patient-reported prevalence and impact of calcinosis across six centres.
Phase Two: Expert consensus Delphi Agreement to define the characteristics of calcinosis using ultrasound.
Phase Three: Case/control observational study describing the clinical characteristics of calcinosis and patient-reported impact. Proof-of-concept study comparing the presence of US defined calcinosis at the hands with patient, physician and x-ray reported presence.
Results:
Patient-reported presence and hand function/disability of calcinosis is significantly higher in Scleroderma compared to SLE and DM.
A consensus-driven definition of calcinosis using US was agreed and identified important features which may impact severity.
Reduced QoL is associated with calcinosis but negatively correlated with x-ray scores. Health and hand function/disability is not significantly different between matched case/control groups. US can detect calcinosis with minimal differences between x-ray reported regions. Imaging and patient reports confirm subclinical calcinosis. Calcinosis is less prevalent outside of the hands but more painful.
Discussion:
Psychosocial factors are impacted more with calcinosis and early identification of subclinical calcinosis may be predictive of severe disease. Negative correlation of x-ray severity scores with patient-reported impact prioritises the need for alternative imaging, identified via ultrasound.
Summary:
No effective therapeutics exist to prevent or treat calcinosis and validated outcome measures are key for clinical trials exploring novel therapeutics. Ultrasound offers a valuable outcome measure for early diagnosis, severity, and impact to support future clinical trials in calcinosis.
