European Calciphylaxis Registry Network

Study Overview
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Brief Summary
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Observational, registry, prospective, non-interventional collection of CUA patient data. Patient treatment is carried out within clinical routine, at the discretion of the physicians and according to existing treatment guidelines. Participating physicians will not be subject to any instructions with regard to the diagnosis and therapy of their patients.

Detailed Description
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Calcific uremic arteriolopathy (CUA, calciphylaxis) is a rare disease (ORPHA280062) with dramatically high mortality characterized clinically by the occurrence of painful debilitating cutaneous lesions and ulcerations. Median survival time is about 1.5 years. Many uncertainties still exist about risk factors and optimal therapy. CUA mainly occurs in patients with severe renal insufficiency or ESRD. The aim of the present initiative is to establish an international (European) observational registry for CUA patients. This registry will help to reach a critical mass of CUA patients, will allow novel insights into pathophysiology, and will help establishing systematically an overview upon risk factors and long-term outcome of CUA patients. The design of the international registry is supposed to adopt the structure of the presently running nation-wide German calciphylaxis registry. The investigators plan to initiate a registry in which treating physicians can provide data upon demographics, clinical picture, comorbidities, medical treatments and laboratory data at the time of diagnosis and serial follow-up time points. In addition investigators will build-up a biobank for full blood, serum and plasma samples as well as tissue samples. The registry will allow investigations regarding potential subgroups of patients (proximal versus distal forms of CUA), geno-phenotype correlations, description of international state-of-the-art treatment, and identification of preventive tools, risk factors and biomarkers for estimation of prognosis. Furthermore, the registry will be the nidus for further scientific exchange between experts and clinicians on the field of uremic vascular calcification.

Official Title
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European Calciphylaxis Registry Network

Conditions
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Calcific Uremic Arteriolopathy (CUA), Calciphylaxis

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: diagnosed CUA according to the judgement of locally treating physicians, who have the option to ask for external diagnostic advice from the country-specific sub-investigators Male or female aged > 18 years Written informed consent prior to study participation The subject is not mentally or legally incapacitated Exclusion Criteria: • The subject is not able to give informed consent

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Prognosis of patients with calciphylaxis (survival time) | Survival time is defined as 1) time between diagnosis of calciphylaxis and death (or end of follow-up) or 2) - in case time of diagnosis is not recorded - time between registry recording and death (or end of follow-up). Analysis of survival time is done using the Kaplan-Meier survival analysis and Kaplan-Meier plot. With a Cox hazard model, we will try to identify additional factors influencing survival time. Hereby it is possible to take into consideration interaction between the parameters. | 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Diagnosis of calciphylaxis | An internet based multilingual registry in which treating physicians can provide patients' data (online questionnaire) upon: demographics, the clinical picture including photo documentation and pain scale (VAS) reporting, comorbidities, medical treatments including dialysis and operations, wound management and laboratory data (serum-creatinine, , BUN, ionized / total calcium, serum phosphorus, PTH, alkaline, phosphatase, calcidiol, albumin, CRP, HbA1c, cholesterol, ferritin, hemoglobin, leukocyte count). The international registry will allow a more rapid identification of patients with calciphylaxis than on a national basis alone, will help establish diagnostic algorithms, and will increase the awareness of the disease. | 5 years | 
| Risk factors | More detailed identification of risk factors: the registry will allow investigations regarding potential subgroups of patients (proximal versus distal forms of CUA), geno-phenotype correlations, description of international state-of-the-art treatment, and identification of preventive tools, risk factors and biomarkers for estimation of prognosis. | 5 years | 
| Factors influencing prognosis of patients with calciphylaxis | Evaluation of the collected data from the time point of diagnosis and serial follow-up time points and further scientific exchange between experts and clinicians on the field of uremic vascular calcification will allow identification of subgroups of patients with potentially distinct pattern of risk factors, clinical picture and prognosis, and establishing factors influencing prognosis of patients with calciphylaxis and to develop novel and/or individualized treatment strategies | 5 years | 
| Novel therapeutic options | By increasing the reported cases towards a critical threshold in Europe enabling the performance of RCTs comparing available or upcoming therapeutic options in CUA pts. | 5 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Calciphylaxis, Rare disease (ORPHA280062), Renal insufficiency, ESRD, Calcification, Chronic kidney disease, Dialysis, Vascular disease