Calcific Uremic Arteriolopathy, Calciphylaxis, Chronic Kidney Diseases, End Stage Renal Disease
Conditions
Keywords
Calciphylaxis, Warfarin, Dialysis
Brief summary
Calciphylaxis, a vascular calcification disorder, is a rare and serious disorder characterized by calcification of dermal arterioles. There are significant gaps in the understanding of the pathophysiology and risk factors for calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare incidence and lack of collaborative approach have been some of the major limiting factors towards treating calciphylaxis. The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within calciphylaxis research by utilizing existing and, when necessary, developing new infrastructure to support the consent of patients and the collection of dedicated samples for a calciphylaxis repository.
Detailed description
Calciphylaxis, a vascular calcification disorder, is a rare and serious disorder characterized by calcification of dermal arterioles. There are significant gaps in the understanding of the pathophysiology and risk factors for calciphylaxis. At present, there is no effective treatment. Uncertain pathobiology, rare incidence and lack of collaborative approach have been some of the major limiting factors towards treating calciphylaxis. The Partners Calciphylaxis Biorepository (PCB) aims to address these gaps within calciphylaxis research by utilizing existing and, when necessary, developing new infrastructure to support the consent of patients and the collection of dedicated samples for a calciphylaxis repository. This repository is working in collaboration with the Partners Biobank as they will still be receiving samples from the PCB and storing them and distributing them according to their own procedures.
Interventions
None listed
Sponsors
Massachusetts General Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male or female ≥18 years old 2. Skin lesions consistent with calciphylaxis diagnosis as determined by the treating clinician 3. Informed of the investigational nature of the study and sign written Informed consent OR are eligible for surrogate consent process based on impaired decision making
Exclusion criteria
1. Patients \<18 years of age 2. Prisoners
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with improvement in calciphylaxis skin lesions | 6 months | Skin lesions will be assessed by investigators at baseline (entry into study) and at 6 months to determine whether there is improvement. The investigators will determine clinical, laboratory and genetic predictors of this outcome in calciphylaxis patients. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with >30% improvement in pain severity | 4weeks | Pain severity will be assessed by investigators at baseline (entry into study) and at 4 weeks to determine whether there is improvement. Pain is measured using Wong-Baker Faces pain rating scale. The investigators will examine clinical, laboratory and genetic predictors of this outcome in calciphylaxis patients. |
| Time to death in patients with calciphylaxis | Up to 5 years | Time to death is defined as time from the diagnosis of calciphylaxis to death (or to the end of follow up). The investigators will examine clinical, laboratory and genetic predictors of this outcome in calciphylaxis patients. |
Countries
United States
Contacts
CONTACTSagar U Nigwekar, MD, MMSc
PRINCIPAL_INVESTIGATORSagar U Nigwekar, MD, MMSc
Massachusetts General Hospital
Outcome results
None listed