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Last update 23 Jan 2025

Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

Last update 23 Jan 2025

Basic Info

Synonyms

POMP-related autoinflammation, POMP-related autoinflammation and immune dysregulation disease, PRAID

+ [2]

Introduction-

Drugs associated with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

Baricitinib

Target

JAK1 x JAK2

Mechanism

JAK1 inhibitors [+1]

Active Org.

Eli Lilly & Co. [+6]

Originator Org.

Incyte Corp.

Active Indication

Enthesitis-Related Arthritis [+23]

Inactive Indication

Conjunctivitis [+8]

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

EU [+3]

First Approval Date13 Feb 2017

Clinical Trials associated with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

NCT01724580

/ Approved for marketingNot Applicable

Compassionate Use Treatment Protocol I4V-MC-JAGA: Treatment of Conditions Expected to Benefit From JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI and Severe Juvenile Dermatomyositis

The Requesting Physician/Investigator contacts Lilly when, based on their medical opinion, a patient meets the criteria for inclusion in the expanded access program.

Start Date-

Sponsor / Collaborator

Eli Lilly & Co.

NCT04517253

/ Active, not recruitingPhase 2/3

A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients With NNS/CANDLE, SAVI, and AGS

The main purpose of this study is to evaluate the efficacy and safety of baricitinib in adult and pediatric Japanese participants with Nakajo-Nishimura Syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (NNS/CANDLE), STING-associated vasculopathy with onset during infancy (SAVI), and Aicardi-Goutières Syndrome (AGS).

Start Date27 Oct 2020

Sponsor / Collaborator

Eli Lilly & Co.

100 Clinical Results associated with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

100 Translational Medicine associated with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

0 Patents (Medical) associated with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

Literatures (Medical) associated with Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature

16 Feb 2025·Journal of Toxicology and Environmental Health, Part A

cGAS/STING pathway modulation in polyhexamethyleneguanidine phosphate-induced immune dysregulation and pulmonary fibrosis using human monocytic cells (THP-1) and male C57BL/6 mice

Article

Author: Jee, Jung in ; Seok, Jin Kyung ; Kim, Ha Ryong ; Chung, Kyu Hyuck ; Lim, Jungyun ; Baek, Yong-Wook ; Kang, HanGoo ; Bae, Ok-Nam ; Kim, Donghyun ; Lee, Joo Young ; Jeon, Minwoo

Polyhexamethyleneguanidine phosphate (PHMG), a widely used antimicrobial agent, has been implicated in humidifier disinfectant-associated lung injuries (HDLI). PHMG exposure suppressed interferon regulatory factor 3 (IRF3) activation and interferon-β (IFN-β) expression induced by a cGAS agonist or a STING agonist in human monocytic cells (THP-1), which are known to transition to alveolar macrophages during pulmonary fibrosis development. However, the mechanisms underlying PHMG-induced pulmonary toxicity in lung remain unclear. Thus, it was of interest to investigate the effects of PHMG on the innate immune system in male C57BL/6 mouse, focusing on the cyclic GMP-AMP synthase (cGAS)/stimulator of interferon genes (STING) pathway and potential role in pulmonary fibrosis. Intratracheal administration of PHMG (1 or 2 mg/kg) in mice resulted in lung fibrosis, as evidenced by H&E staining with Szapiel scoring, Masson's trichrome staining with Ashcroft scoring, and increased mRNA levels of TGF-β and collagen type I. Interestingly, lower dose of PHMG enhanced IFN-β production in the lungs, whereas higher dose decreased IFN-β levels, indicating a biphasic effect that initially promotes inflammation but ultimately impairs host defense mechanisms, leading to pulmonary fibrosis. Our findings demonstrate the critical role of the cGAS/STING pathway in PHMG-induced mouse lung injury and suggest that targeting this pathway might serve as a potential therapeutic strategy for treating pulmonary fibrosis.

01 Nov 2024·Pediatric Dermatology

A case of mother and child with CANDLE syndrome: Diagnosis and subsequent treatment with baricitinib

Article

Author: Kristal, Leonard ; Alehashemi, Sara ; Guo, William ; Lozeau, Daniel ; Tonnesen, Marcia ; de Jesus, Adriana ; Miller, Devin ; Schuval, Susan

AbstractChronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE) or proteasome‐associated autoinflammatory syndrome is a rare autoinflammatory disorder that typically presents in infancy with characteristic symptoms, including recurrent fever, panniculitis, and progressive lipodystrophy, among other findings. We present a case of mother and child with CANDLE syndrome. The child was eventually started on baricitinib with normalization of rash and systemic findings.

08 Nov 2023·Nephrology Dialysis Transplantation

The five types of glomerulonephritis classified by pathogenesis, activity and chronicity (GN-AC)

Article

Author: Romagnani, Paola ; Kitching, A Richard ; Leung, Nelson ; Anders, Hans-Joachim

ABSTRACTGlomerulonephritis (GN) is a diverse group of immune-mediated disorders. Currently, GN is classified largely by histological patterns that are difficult to understand and teach, and most importantly, do not indicate treatment choices. Indeed, altered systemic immunity is the primary pathogenic process and the key therapeutic target in GN. Here, we apply a conceptual framework of immune-mediated disorders to GN guided by immunopathogenesis and hence immunophenotyping: (i) infection-related GN require pathogen identification and control; (ii) autoimmunity-related GN, defined by presence of autoantibodies and (iii) alloimmunity-related GN in transplant recipients both require the suppression of adaptive immunity in lymphoid organs and bone marrow; (iv) autoinflammation-related GN, e.g. inborn errors of immunity diagnosed by genetic testing, requires suppression of single cytokine or complement pathways; and (v) Monoclonal gammopathy-related GN requires B or plasma cell clone-directed therapy. A new GN classification should include disease category, immunological activity to tailor the use of the increasing number of immunomodulatory drugs, and chronicity to trigger standard chronic kidney disease care including the evolving spectrum of cardio-renoprotective drugs. Certain biomarkers allow diagnosis and the assessment of immunological activity and disease chronicity without kidney biopsy. The use of these five GN categories and a therapy-focused GN classification is likely to overcome some of the existing hurdles in GN research, management and teaching by reflecting disease pathogenesis and guiding the therapeutic approach.

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