Session Type: ACR Poster Session C
Session Time: 9:00AM-11:00AM
Background/Purpose: CANDLE and SAVI are two rare autoinflammatory interferonopathies without validated outcome measures to assess disease activity; however, daily diaries of prominent disease symptoms have become standard in assessing clinical disease load and reporting treatment related outcomes in patients with the autoinflammatory disease spectrum CAPS (Cryopyrin associated periodic syndrome) where diary scores were accepted by the FDA in the context of drug approval. For novel AIDs with distinct organ manifestations new disease specific diaries need to be generated and validated. In pediatrics inflammatory diseases the Childhood Health Assessment Questionnaire (CHAQ), which measures physical function and the patient and physician-derived outcome measures, patient global VAS (PATG) and physician global VAS (PHYG) respectively are used to assess disease severity and response to treatment and have been used to validate patient related outcomes in the past.
Methods: The daily diary developed for NOMID was modified to reflect disease symptoms of CANDLE and SAVI. For CANDLE these include: fever, rash, joint pain, headache and fatigue (possible range, 0 to 20) For SAVI these also include respiratory symptoms and ulcerative lesions. Each symptom is rated on a scale from 0 to 4 for increasing severity. We correlated the diary score in CANDLE and SAVI patients against 6 validated outcome measures: Childhood Health Assessment Questionnaire (CHAQ), which measures physical function; physical global VAS ( PHYG); global pain assessment ( PAINGL); parent global( PARG); the patent quality of life ( PATQL) and parent QL ( PARQL) by correlating each symptom and the summary score with PHYG, PAINGL, PARG, PATQL and PARQL respectively. We used Pearson correlation and 1-sample t- test to assess whether these values plausibly centered at 0. P – values were not corrected for multiple comparisons but p-values of <0.01 were considered significant.
Results: 11 CANDLE and 4 SAVI patients and 3 undifferentiated interfernopathy patients were included. Mean diary scores computed for all patients correlated well with CHAQ ( r-0.36, p<0.001).Mean diary score computed during the evaluation for CANDLE patients correlated well with CHAQ( r-0.36, p<0.001), pain global ( r- 0.48, p<0.001). Individual symptoms like fever, rash etc correlated well with these outcome measures in both CANDLE and SAVI. However, the SAVI specific parameters, respiratory symptoms and ulcerative lesions did not show any significant correlation.
Conclusion: Our study shows that diary scores exhibit validity across other outcome measures validated for unrelated pediatric inflammatory diseases, but outcome measures in SAVI need to be further refined.
To cite this abstract in AMA style:Sawhney M, Montealegre Sanchez GA, Wesley R, Bahar K, Goldbach-Mansky R. Validation of Diary Score for the Assessment of Disease Activity in Candle (Chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature) and SAVI (STING associated vasculopathy with onset in Infancy) Patients [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/validation-of-diary-score-for-the-assessment-of-disease-activity-in-candle-chronic-atypical-neutrophilic-dermatosis-with-lipodystrophy-and-elevated-temperature-and-savi-sting-associated-vascul/. Accessed July 13, 2020.
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ACR Meeting Abstracts - https://acrabstracts.org/abstract/validation-of-diary-score-for-the-assessment-of-disease-activity-in-candle-chronic-atypical-neutrophilic-dermatosis-with-lipodystrophy-and-elevated-temperature-and-savi-sting-associated-vascul/