THU0571 Preliminary Response To Janus Kinase Inhibition with Baricitinib in Chronic Atypical Neutrophilic Dermatosis with Lipodystrophy and Elevated Temperatures (CANDLE)
| Autor: | Robert A. Colbert, Susanne Schalm, Meryl Waldman, Adam L Reinhardt, Kristina I. Rother, Paul A. Brogan, T.L. Klausmeier, S. Judd, D. Brown, Jason Dare, Yackov Berkun, R. Goldbach-Mansky, Abraham Zlotogorski, Ling Gao, G Montealegre, Chyi-Chia Richard Lee, AA de Jesus, H. Kim, Michelle O'Brien, Alessandra Brofferio, B. Kost, S. Murias, Theo Heller, D. Chapelle, Colleen Hadigan, Scott M. Paul |
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| Rok vydání: | 2016 |
| Předmět: |
030203 arthritis & rheumatology
0301 basic medicine medicine.medical_specialty business.industry Baricitinib Immunology Treatment options Baseline data medicine.disease General Biochemistry Genetics and Molecular Biology Surgery Clinic visit 03 medical and health sciences 030104 developmental biology 0302 clinical medicine Rheumatology Neutrophilic dermatosis Prednisone Internal medicine medicine Immunology and Allergy Lipodystrophy business medicine.drug Therapeutic strategy |
| Zdroj: | Annals of the Rheumatic Diseases. 75:398.2-399 |
| ISSN: | 1468-2060 0003-4967 |
| DOI: | 10.1136/annrheumdis-2016-eular.2810 |
| Popis: | Background Elevated serum IP-10 (CXCL10) levels and gene expression studies showing a prominent “interferon (IFN) signature” suggested modulation of IFN signaling might be a therapeutic option in CANDLE patients. Objectives The objective of this compassionate use program is to provide baricitinib (JAK1/JAK2 inhibitor) to CANDLE patients who have no other comparable or satisfactory treatment options. Potential efficacy of treatment was assessed by a reduction in mean Autoinflammatory Diary Scores (ADS) to Methods Paired t-test was used to compare mean ADS and prednisone doses at the last NIH clinic visit to baseline data. Results Between October 2011 and January 1st, 2016, 11 CANDLE patients have been treated (mean duration 2.5 years (SD±1)). 9 of 11 patients achieved an ADS of than 50% from baseline (mean total prednisone dose decreased from 0.8 mg/kg/day (0.2–1.8) to 0.2 mg/kg/day (0–1.1)) (p Conclusions Preliminary efficacy data in 11 CANDLE patients treated with baricitinib are encouraging and suggest that targeting IFN signaling with a JAK1/JAK2 inhibitor may be a successful therapeutic strategy. Monitoring BK viral titers in blood and urine, in addition to other measures of safety and efficacy, may be important in dose selection and the benefit-risk assessment of baricitinib for CANDLE patients. Disclosure of Interest G. Montealegre: None declared, A. Reinhardt: None declared, P. Brogan: None declared, Y. Berkun: None declared, A. Zlotogorski: None declared, D. Brown: None declared, L. Gao: None declared, J. Dare: None declared, S. Schalm: None declared, T. Klausmeier: None declared, S. Murias: None declared, D. Chapelle: None declared, H. Kim: None declared, S. Judd: None declared, M. O9Brien: None declared, A. de Jesus: None declared, B. Kost: None declared, S. Paul: None declared, R. Colbert Grant/research support from: CRADA (NIH -Eli Lilly), A. Brofferio: None declared, C. Lee: None declared, C. Hadigan: None declared, T. Heller: None declared, M. Waldman: None declared, K. Rother: None declared, R. Goldbach-Mansky: None declared |
| Databáze: | OpenAIRE |
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