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Table 1 CAPS registry - recommended data collection schedule

From: Methodological challenges in monitoring new treatments for rare diseases: lessons from the cryopyrin-associated periodic syndrome registry

Period

Baseline

Follow-up (until study end or premature discontinuation)*

Timing

Enrollment

Every 6 months

Informed consent

x

 

CAPS phenotype

x

 

CAPS genotype (NLRP-3 mutation), if available

x

 

Non-CAPS medical history

x

 

Vital signs (height, weight, blood pressure)

x

x

Ilaris dosing/status

x

x

Historical and concomitant medications for auto-inflammatory disease*

x

x

Selected AEs*

 

x

Other serious AEs and non-serious adverse reactions*

 

x

CAPS clinical assessment *

x

x

CAPS-related clinical testing results, if performed

x

x

Selected local laboratory testing

x

x

Sexual development status (paediatric only)

x

x

Neurocognitive status (paediatric only)

x

x

Cerebrospinal fluid analysis

x

x

Vaccination record and outcome

x

x

Pregnancy status (females of child-bearing potential only)

 

x

Registry disposition

 

x

  1. * These assessments may also be conducted during follow-up for patients who discontinue canakinumab but remain in the Registry.