Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
NOMID	Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.

Participant Flow:   Overall Study

	NOMID
STARTED	43
COMPLETED	41
NOT COMPLETED	2

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups

	Description
NOMID	Patients who met the criteria for NOMID and treated with escalating doses of anakinra 1-5 mg/kg/day to achieve laboratory and organ inflammation remission.

Baseline Measures

	NOMID
Overall Participants Analyzed  [Units: Participants]	43
Age  [Units: Participants]
<=18 years	36
Between 18 and 65 years	7
>=65 years	0
Gender  [Units: Participants]
Female	25
Male	18
Ethnicity (NIH/OMB)  [Units: Participants]
Hispanic or Latino	8
Not Hispanic or Latino	33
Unknown or Not Reported	2
Race (NIH/OMB)  [Units: Participants]
American Indian or Alaska Native	0
Asian	4
Native Hawaiian or Other Pacific Islander	0
Black or African American	1
White	29
More than one race	1
Unknown or Not Reported	8

1.  Primary:

Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)   [ Time Frame: Baseline ]

2.  Primary:

Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)   [ Time Frame: 36 months ]

3.  Primary:

Diary Symptom Sum Score (DSSS) (Fever, Rash, Joint Pain, Vomiting, and Headaches)   [ Time Frame: 60 months ]

4.  Primary:

Patient / Parent Global Score of Overall Disease Activity   [ Time Frame: Baseline ]

5.  Primary:

Patient / Parent Global Score of Overall Disease Activity   [ Time Frame: 36 months ]

6.  Primary:

Patient / Parent Global Score of Overall Disease Activity   [ Time Frame: 60 months ]

7.  Primary:

Parent /Patient Pain Rating   [ Time Frame: Baseline ]

8.  Primary:

Parent /Patient Pain Rating   [ Time Frame: 36 months ]

9.  Primary:

Parent /Patient Pain Rating   [ Time Frame: 60 months ]

10.  Primary:

Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: Baseline ]

11.  Primary:

Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: 36 months ]

12.  Primary:

Childhood Health Assessment Questionnaire (CHAQ)   [ Time Frame: 60 months ]

13.  Primary:

Serum Amyloid A (SAA) Measurement   [ Time Frame: Baseline ]

14.  Primary:

Serum Amyloid A (SAA) Measurement   [ Time Frame: 36 months ]

15.  Primary:

Serum Amyloid A (SAA) Measurement   [ Time Frame: 60 months ]

16.  Primary:

C-reactive Protein (CRP) Measurement   [ Time Frame: Baseline ]

17.  Primary:

C-reactive Protein (CRP) Measurement   [ Time Frame: 36 months ]

18.  Primary:

C-reactive Protein (CRP) Measurement   [ Time Frame: 60 months ]

19.  Primary:

Erythrocyte Sedimentation Rate (ESR) Measurement   [ Time Frame: Baseline ]

20.  Primary:

Erythrocyte Sedimentation Rate (ESR) Measurement   [ Time Frame: 36 months ]

21.  Primary:

Erythrocyte Sedimentation Rate (ESR) Measurement   [ Time Frame: 60 months ]

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Data reported is on 26 patients who completed their 3 year assessment and 20 of these patients also completed their 5 year assessment.