Drug survival of anakinra and canakinumab in monogenic autoinflammatory diseases : observational study from the International AIDA Registry
© The Author(s) 2021. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissionsoup.com..
OBJECTIVES: To investigate survival of IL-1 inhibitors in monogenic autoinflammatory disorders (mAID) through drug retention rate (DRR) and identify potential predictive factors of drug survival from a real-life perspective.
PATIENTS AND METHODS: Multicentre retrospective study analysing patients affected by the most common mAID treated with anakinra or canakinumab. Survival curves were analysed with the Kaplan-Meier method. Statistical analysis included a Cox-proportional hazard model to detect factors responsible for drug discontinuation.
RESULTS: Seventy-eight patients for a total of 102 treatment regimens were enrolled. The mean treatment duration was 29.59 months. The estimated DRR of IL-1 inhibitors at 12, 24 and 48 months of follow-up was 75.8%, 69.7% and 51.1%, respectively. Patients experiencing an adverse event had a significantly lower DRR (P=0.019). In contrast, no significant differences were observed between biologic-naïve patients and those previously treated with biologic drugs (P=0.985). Patients carrying high-penetrance mutations exhibited a significantly higher DRR compared with those with low-penetrance variants (P=0.015). Adverse events were the only variable associated with a higher hazard of treatment withdrawal [hazard ratio (HR) 2.573 (CI: 1.223, 5.411), P=0.013] on regression analysis. A significant glucorticoid-sparing effect was observed (P<0.0001).
CONCLUSIONS: IL-1 inhibitors display an excellent long-term effectiveness in terms of DRR, and their survival is not influenced by the biologic line of treatment. They display a favourable safety profile, which deserves, however, a close monitoring given its impact on treatment continuation. Special attention should be paid to molecular diagnosis and mutation penetrance, as patients carrying low-penetrance variants are more likely to interrupt treatment.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:60 |
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| Enthalten in: |
Rheumatology (Oxford, England) - 60(2021), 12 vom: 01. Dez., Seite 5705-5712 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Sota, Jurgen [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 29.12.2021 Date Revised 29.12.2021 published: Print Citation Status MEDLINE |
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| doi: |
10.1093/rheumatology/keab419 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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NLM325128367 |
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| 245 | 1 | 0 | |a Drug survival of anakinra and canakinumab in monogenic autoinflammatory diseases |b observational study from the International AIDA Registry |
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| 500 | |a Date Revised 29.12.2021 | ||
| 500 | |a published: Print | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © The Author(s) 2021. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For permissions, please email: journals.permissionsoup.com. | ||
| 520 | |a OBJECTIVES: To investigate survival of IL-1 inhibitors in monogenic autoinflammatory disorders (mAID) through drug retention rate (DRR) and identify potential predictive factors of drug survival from a real-life perspective | ||
| 520 | |a PATIENTS AND METHODS: Multicentre retrospective study analysing patients affected by the most common mAID treated with anakinra or canakinumab. Survival curves were analysed with the Kaplan-Meier method. Statistical analysis included a Cox-proportional hazard model to detect factors responsible for drug discontinuation | ||
| 520 | |a RESULTS: Seventy-eight patients for a total of 102 treatment regimens were enrolled. The mean treatment duration was 29.59 months. The estimated DRR of IL-1 inhibitors at 12, 24 and 48 months of follow-up was 75.8%, 69.7% and 51.1%, respectively. Patients experiencing an adverse event had a significantly lower DRR (P=0.019). In contrast, no significant differences were observed between biologic-naïve patients and those previously treated with biologic drugs (P=0.985). Patients carrying high-penetrance mutations exhibited a significantly higher DRR compared with those with low-penetrance variants (P=0.015). Adverse events were the only variable associated with a higher hazard of treatment withdrawal [hazard ratio (HR) 2.573 (CI: 1.223, 5.411), P=0.013] on regression analysis. A significant glucorticoid-sparing effect was observed (P<0.0001) | ||
| 520 | |a CONCLUSIONS: IL-1 inhibitors display an excellent long-term effectiveness in terms of DRR, and their survival is not influenced by the biologic line of treatment. They display a favourable safety profile, which deserves, however, a close monitoring given its impact on treatment continuation. Special attention should be paid to molecular diagnosis and mutation penetrance, as patients carrying low-penetrance variants are more likely to interrupt treatment | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Multicenter Study | |
| 650 | 4 | |a Observational Study | |
| 650 | 4 | |a IL-1 | |
| 650 | 4 | |a anakinra | |
| 650 | 4 | |a canakinumab | |
| 650 | 4 | |a innovative biotechnologies | |
| 650 | 4 | |a monogenic autoinflammatory disorders | |
| 650 | 4 | |a personalized medicine | |
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| 650 | 7 | |a Interleukin 1 Receptor Antagonist Protein |2 NLM | |
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| 650 | 7 | |a canakinumab |2 NLM | |
| 650 | 7 | |a 37CQ2C7X93 |2 NLM | |
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| 700 | 1 | |a Sicignano, Ludovico Luca |e verfasserin |4 aut | |
| 700 | 1 | |a Verrecchia, Elena |e verfasserin |4 aut | |
| 700 | 1 | |a Aragona, Emma |e verfasserin |4 aut | |
| 700 | 1 | |a Maggio, Maria Cristina |e verfasserin |4 aut | |
| 700 | 1 | |a Lopalco, Giuseppe |e verfasserin |4 aut | |
| 700 | 1 | |a Emmi, Giacomo |e verfasserin |4 aut | |
| 700 | 1 | |a Parronchi, Paola |e verfasserin |4 aut | |
| 700 | 1 | |a Cauli, Alberto |e verfasserin |4 aut | |
| 700 | 1 | |a Wiesik-Szewczyk, Ewa |e verfasserin |4 aut | |
| 700 | 1 | |a Hernández-Rodríguez, José |e verfasserin |4 aut | |
| 700 | 1 | |a Gaggiano, Carla |e verfasserin |4 aut | |
| 700 | 1 | |a Tarsia, Maria |e verfasserin |4 aut | |
| 700 | 1 | |a Mourabi, Mariam |e verfasserin |4 aut | |
| 700 | 1 | |a Ragab, Gaafar |e verfasserin |4 aut | |
| 700 | 1 | |a Vitale, Antonio |e verfasserin |4 aut | |
| 700 | 1 | |a Fabiani, Claudia |e verfasserin |4 aut | |
| 700 | 1 | |a Frediani, Bruno |e verfasserin |4 aut | |
| 700 | 1 | |a Lamacchia, Vittoria |e verfasserin |4 aut | |
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| 700 | 1 | |a Cantarini, Luca |e verfasserin |4 aut | |
| 700 | 0 | |a Autoinflammatory Diseases Alliance (AIDA) and the Autoinflammatory Diseases Working Group of the Italian Society of Rheumatology (SIR) |e verfasserin |4 aut | |
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