Article
Treat-to-target in Interleukin-1 mediated autoinflammatory diseases
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Authors
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Tatjana Welzel
- Pediatric Pharmcology and Pharmacometrics, University Children’s Hospital Basel, Basel, Switzerland; Pediatric Rheumatology and autoinflammation reference center Tuebingen (arcT), University Children’s Hospital Tuebingen, Tuebingen, Germany
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Jens Klotsche
- Epidemiology Unit, German Rheumatism Reserach Center, Berlin, Germany
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Beate Magdalena Zapf
- Pediatric Rheumatology and autoinflammation reference center Tuebingen (arcT), Department of Pediatrics, University Children`s Hospital Tuebingen, Tuebingen, Germany
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Susanne M. Benseler
- Rheumatology, Department of Pediatrics, Alberta Children’s Hospital, Cumming School of Medicine, Alberta Children’s Hospital Research Institute, University of Calgary, Alberta, Canada
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Jasmin B Kuemmerle-Deschner
- Pediatric Rheumatology and autoinflammation reference center Tuebingen (arcT), Department of Pediatrics, University Children’s Hospital Tuebingen, Tuebingen, Germany
| Published: | August 31, 2022 |
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Outline
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Introduction: Uncontrolled disease activity (DA) in Interleukin-1 mediated autoinflammatory diseases (IL-1 AID) is potential life-threatening. Therefore, treatment should achieve no/minimal DA to prevent morbidity and mortality. This study analyzes DA and treatment in IL-1 AID to evaluate the need of DA targeted treatment adjustments over disease course.
Methods: A single-center longitudinal study of consecutive children aged ≤18 years with IL-1 AID and no evidence of organ damage was performed. DA changes and treatment adjustments were descriptively analyzed between 01/2016 and 12/2019. DA was defined as composite outcome of the physician global assessment (PGA, 10 cm VAS) and Serum Amyloid A (SAA)/C-reactive protein (CRP) categorized as minimal (PGA≤2+CRP<1.5mg/dl and/or SAA<30mg/l), moderate (PGA>2-5+CRP>1.5<2.5mg/dl and/or SAA≥30<50mg/l) and severe (PGA>5+CRP>2.5mg/dl and/or SAA≥50mg/l).
Results: A total of 56 children were included, 34% were female. Median age at onset was 2.5 years (0.5; 4.1), and 4.9 years (3.0; 7.7) at diagnosis. At first visit, 28.6% received treatment and DA was minimal in 18/56 (32%), moderate in 28/56 (50%), and severe in 10/56 (18%). Median follow-up included 361 visits and was 2.1 years (1.4; 2.7). During follow-up, 36/56 patients (64%) experienced one episode of DA increase (47.2% minimal to moderate, 33.3% minimal to severe, 19.5% moderate to severe). In 28/36 patients (77.8%), DA increase resulted in treatment escalation: medication change in 20 patients, dose increase in 8 patients. In addition, 17 patients experienced a second episode of DA increase during follow-up (76.5% minimal to moderate, 23.5% minimal to severe). Treatment was escalated in 7/17 patients (41.2%): medication change in 3 patients, dose increase in 3 patients, interval reduction in 1 patient. At last visit, 46/56 patients (82.1%) received treatment. Treatment start and adjustment resulted in minimal DA in 44/56 patients (78.6%), moderate DA in 10/56 patients (17.9%) and severe DA in 2/56 patients (3.6%).
Conclusion: DA can increase over disease course in IL-1 AID, irrespective of treatment. Therefore, regular DA assessment and treatment adjustments are important. This study highlights the importance of previously published treat-to-target strategies with regular DA assessments and treatment adjustments in daily practice for all IL-1 AID to further optimize and personalize patient’s care.
Disclosures: Tatjana Welzel and Jens Klotsche contributed equally and should be considered as shared first author.
Susanne M. Benseler and Jasmin Kuemmerle-Deschner contributed equally and should be considered as shared senior author.
