Latest database descriptions

Based on the collection of capillary blood samples taken by the subjects, SAPRIS-SERO will help to:
• Estimate the cumulative incidence of infection in the general population (objective 1a),
• Characterise the “immune” portion of the population and the durability of immunity (objective 1b).

Primary objective : Describe in current medical practice the evolution of hemoglobin levels during the 9-month follow-up in each subgroup defined by the Charlson comorbidity index.

Secondary objectives :
- Describe the characteristics of patients treated with Mircera® in the total population and in the subgroups defined by the Charlson comorbidity index;
- Describe the evolution of the hemoglobin level during the observation period according to the Liu comorbidity index and to ESA resistance factors;
- Describe the changes in the Mircera® dosage during the observation period in the total population and in the subgroups defined by the Charlson comorbidity index;
- Describe the safety profile of Mircera® during the observation period in the total population and in the subgroups defined by the Charlson comorbidity index.

The main objective of this study is to describe the natural course of vascular Ehlers-Danlos syndrome, in particular the order of appearance of different types of complications (arterial, digestive, pulmonary and uterine).

The primary objective of the RaDiCo-ECYSCO cohort is to understand the natural history and major long-term manifestations and outcomes of cystinosis in paediatric and adult cases.

Secondary Objectives are to:
• Evaluate the impact of disease and treatments on patients’ quality of life
• Evaluate the effect of treatment on the complications
• appraise the long-term safety of treatment and compliance

Information Technology Objectives are to:
• Develop and diffuse an electronic tool of data collection from various sources linked to a database integrating a system of management and follow-up of data-management allowing collection of data for cystinosis paediatric and adult patients.
• Include data generated by patients and, where relevant, their parents and or carers.
• Expand the cohort to cover a broader European population.
• Promote the use of the RaDiCo-ECYSCO eCRF for mutualisation and harmonisation of data for cystinosis paediatric and adult patients within the expert sites.

Improvement of standard care objectives are to:
• Develop comprehensive evidence based guidelines for treatments as well as for follow-up of patients who will switch from paediatric to adult status,
• Propose a system of audit against the guidelines ensuring overall care is of the highest standard as well as identifying areas of concern for actions.