Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes
Objective Blocking interleukin‐1 with anakinra in patients with the autoinflammatory syndrome neonatal‐onset multisystem inflammatory disease (NOMID) reduces systemic and organ‐specific inflammation. However, the impact of long‐term treatment has not been established. This study was undertaken to ev...
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| Veröffentlicht in: | Arthritis & rheumatology (Hoboken, N.J.) N.J.), 2012-07, Vol.64 (7), p.2375-2386 |
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| creator | Sibley, Cailin H. Plass, Nikki Snow, Joseph Wiggs, Edythe A. Brewer, Carmen C. King, Kelly A. Zalewski, Christopher Kim, H. Jeffrey Bishop, Rachel Hill, Suvimol Paul, Scott M. Kicker, Patrick Phillips, Zachary Dolan, Joseph G. Widemann, Brigitte Jayaprakash, Nalini Pucino, Frank Stone, Deborah L. Chapelle, Dawn Snyder, Christopher Butman, John A. Wesley, Robert Goldbach-Mansky, Raphaela |
| description | Objective
Blocking interleukin‐1 with anakinra in patients with the autoinflammatory syndrome neonatal‐onset multisystem inflammatory disease (NOMID) reduces systemic and organ‐specific inflammation. However, the impact of long‐term treatment has not been established. This study was undertaken to evaluate the long‐term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID.
Methods
We conducted a cohort study of 26 NOMID patients ages 0.80–42.17 years who were followed up at the NIH and treated with anakinra 1–5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C‐reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated.
Results
Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P < 0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P < 0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverse events other than viral infections were rare, and all patients continued to receive the medication.
Conclusion
These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall. |
| doi_str_mv | 10.1002/art.34409 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_pubme</sourceid><recordid>TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3474541</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>1544016461</sourcerecordid><originalsourceid>FETCH-LOGICAL-c5449-74b7ac11eeea0a411f2ce4d2f1ed9a3bca3be13cd7163c82ab89404dfce0c2d63</originalsourceid><addsrcrecordid>eNqNkl1rFDEUhgdRbK1e-AckIIJeTJuvmdnxQlgWW6VFoVa9DGczZ3bTziRrktm6P9F_Zfaj6wcIXoSQ5Dnve3J4s-wpo8eMUn4CPh4LKWl9LztkBa9zygS7nx1SSmUuipodZI9CuE5HLgrxMDvgnNcyVRxmPz4NIYKx2BCPYeFsQAK2IQuPS7TROEtcSxroYYbp0s0SFda3xpIFRJOYQG5NnBOLzkKELl9rRNIPXTRhFSL2iW076HuIzq9IYwJCcokeISbbTTFYuDHWw2syJtrNnY8kxKFZkehIgxF9n1okce4R801_rVlivkLwxA1Rux7D4-xBC13AJ7v9KPt8-vZq8i6_-Hj2fjK-yHUhZZ1XclqBZgwRgYJkrOUaZcNbhk0NYqrTQiZ0U7FS6BGH6aiWVDatRqp5U4qj7M1WdzFMe2x0moCHTi286cGvlAOj_nyxZq5mbqmErGQhWRJ4uRPw7tuAIareBI1dB2mEQ1As9UlZKcv_QCnno4KXvEro87_Qazd4myaRBFnFWFVJkahXW0p7F4LHdt83o2qdJZWypDZZSuyz3z-6J-_Ck4AXOwCChq71YLUJv7gyedJi_YuTLXdrOlz921GNL6_urPNthUn5-b6vAH-jykpUhfr64UxV5-eTU345UV_ET7aN9nQ</addsrcrecordid><sourcetype>Open Access Repository</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>1517117743</pqid></control><display><type>article</type><title>Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes</title><source>MEDLINE</source><source>Wiley Online Library Journals Frontfile Complete</source><creator>Sibley, Cailin H. ; Plass, Nikki ; Snow, Joseph ; Wiggs, Edythe A. ; Brewer, Carmen C. ; King, Kelly A. ; Zalewski, Christopher ; Kim, H. Jeffrey ; Bishop, Rachel ; Hill, Suvimol ; Paul, Scott M. ; Kicker, Patrick ; Phillips, Zachary ; Dolan, Joseph G. ; Widemann, Brigitte ; Jayaprakash, Nalini ; Pucino, Frank ; Stone, Deborah L. ; Chapelle, Dawn ; Snyder, Christopher ; Butman, John A. ; Wesley, Robert ; Goldbach-Mansky, Raphaela</creator><creatorcontrib>Sibley, Cailin H. ; Plass, Nikki ; Snow, Joseph ; Wiggs, Edythe A. ; Brewer, Carmen C. ; King, Kelly A. ; Zalewski, Christopher ; Kim, H. Jeffrey ; Bishop, Rachel ; Hill, Suvimol ; Paul, Scott M. ; Kicker, Patrick ; Phillips, Zachary ; Dolan, Joseph G. ; Widemann, Brigitte ; Jayaprakash, Nalini ; Pucino, Frank ; Stone, Deborah L. ; Chapelle, Dawn ; Snyder, Christopher ; Butman, John A. ; Wesley, Robert ; Goldbach-Mansky, Raphaela</creatorcontrib><description>Objective
Blocking interleukin‐1 with anakinra in patients with the autoinflammatory syndrome neonatal‐onset multisystem inflammatory disease (NOMID) reduces systemic and organ‐specific inflammation. However, the impact of long‐term treatment has not been established. This study was undertaken to evaluate the long‐term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID.
Methods
We conducted a cohort study of 26 NOMID patients ages 0.80–42.17 years who were followed up at the NIH and treated with anakinra 1–5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C‐reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated.
Results
Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P < 0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P < 0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverse events other than viral infections were rare, and all patients continued to receive the medication.
Conclusion
These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.</description><identifier>ISSN: 0004-3591</identifier><identifier>ISSN: 2326-5191</identifier><identifier>EISSN: 1529-0131</identifier><identifier>EISSN: 2326-5205</identifier><identifier>DOI: 10.1002/art.34409</identifier><identifier>PMID: 22294344</identifier><identifier>CODEN: ARHEAW</identifier><language>eng</language><publisher>Hoboken: Wiley Subscription Services, Inc., A Wiley Company</publisher><subject>Adolescent ; Adult ; Antirheumatic Agents - administration & dosage ; Antirheumatic Agents - therapeutic use ; Biological and medical sciences ; C-Reactive Protein ; Child ; Child, Preschool ; Cryopyrin-Associated Periodic Syndromes - drug therapy ; Cryopyrin-Associated Periodic Syndromes - pathology ; Disease Progression ; Diseases of the osteoarticular system ; Drug therapy ; Female ; Humans ; Immunomodulators ; Infant ; Infant, Newborn ; Inflammation - drug therapy ; Inflammation - pathology ; Interleukin 1 Receptor Antagonist Protein - administration & dosage ; Interleukin 1 Receptor Antagonist Protein - therapeutic use ; Male ; Malformations and congenital and or hereditary diseases involving bones. Joint deformations ; Medical research ; Medical sciences ; Patients ; Pharmacology. Drug treatments ; Surveys and Questionnaires ; Treatment Outcome</subject><ispartof>Arthritis & rheumatology (Hoboken, N.J.), 2012-07, Vol.64 (7), p.2375-2386</ispartof><rights>Copyright © 2012 by the American College of Rheumatology</rights><rights>2015 INIST-CNRS</rights><rights>Copyright © 2012 by the American College of Rheumatology.</rights><rights>2012, American College of Rheumatology 2012</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c5449-74b7ac11eeea0a411f2ce4d2f1ed9a3bca3be13cd7163c82ab89404dfce0c2d63</citedby><cites>FETCH-LOGICAL-c5449-74b7ac11eeea0a411f2ce4d2f1ed9a3bca3be13cd7163c82ab89404dfce0c2d63</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fart.34409$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fart.34409$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>230,314,776,780,881,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=26177051$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/22294344$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Sibley, Cailin H.</creatorcontrib><creatorcontrib>Plass, Nikki</creatorcontrib><creatorcontrib>Snow, Joseph</creatorcontrib><creatorcontrib>Wiggs, Edythe A.</creatorcontrib><creatorcontrib>Brewer, Carmen C.</creatorcontrib><creatorcontrib>King, Kelly A.</creatorcontrib><creatorcontrib>Zalewski, Christopher</creatorcontrib><creatorcontrib>Kim, H. Jeffrey</creatorcontrib><creatorcontrib>Bishop, Rachel</creatorcontrib><creatorcontrib>Hill, Suvimol</creatorcontrib><creatorcontrib>Paul, Scott M.</creatorcontrib><creatorcontrib>Kicker, Patrick</creatorcontrib><creatorcontrib>Phillips, Zachary</creatorcontrib><creatorcontrib>Dolan, Joseph G.</creatorcontrib><creatorcontrib>Widemann, Brigitte</creatorcontrib><creatorcontrib>Jayaprakash, Nalini</creatorcontrib><creatorcontrib>Pucino, Frank</creatorcontrib><creatorcontrib>Stone, Deborah L.</creatorcontrib><creatorcontrib>Chapelle, Dawn</creatorcontrib><creatorcontrib>Snyder, Christopher</creatorcontrib><creatorcontrib>Butman, John A.</creatorcontrib><creatorcontrib>Wesley, Robert</creatorcontrib><creatorcontrib>Goldbach-Mansky, Raphaela</creatorcontrib><title>Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes</title><title>Arthritis & rheumatology (Hoboken, N.J.)</title><addtitle>Arthritis & Rheumatism</addtitle><description>Objective
Blocking interleukin‐1 with anakinra in patients with the autoinflammatory syndrome neonatal‐onset multisystem inflammatory disease (NOMID) reduces systemic and organ‐specific inflammation. However, the impact of long‐term treatment has not been established. This study was undertaken to evaluate the long‐term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID.
Methods
We conducted a cohort study of 26 NOMID patients ages 0.80–42.17 years who were followed up at the NIH and treated with anakinra 1–5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C‐reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated.
Results
Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P < 0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P < 0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverse events other than viral infections were rare, and all patients continued to receive the medication.
Conclusion
These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Antirheumatic Agents - administration & dosage</subject><subject>Antirheumatic Agents - therapeutic use</subject><subject>Biological and medical sciences</subject><subject>C-Reactive Protein</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Cryopyrin-Associated Periodic Syndromes - drug therapy</subject><subject>Cryopyrin-Associated Periodic Syndromes - pathology</subject><subject>Disease Progression</subject><subject>Diseases of the osteoarticular system</subject><subject>Drug therapy</subject><subject>Female</subject><subject>Humans</subject><subject>Immunomodulators</subject><subject>Infant</subject><subject>Infant, Newborn</subject><subject>Inflammation - drug therapy</subject><subject>Inflammation - pathology</subject><subject>Interleukin 1 Receptor Antagonist Protein - administration & dosage</subject><subject>Interleukin 1 Receptor Antagonist Protein - therapeutic use</subject><subject>Male</subject><subject>Malformations and congenital and or hereditary diseases involving bones. Joint deformations</subject><subject>Medical research</subject><subject>Medical sciences</subject><subject>Patients</subject><subject>Pharmacology. Drug treatments</subject><subject>Surveys and Questionnaires</subject><subject>Treatment Outcome</subject><issn>0004-3591</issn><issn>2326-5191</issn><issn>1529-0131</issn><issn>2326-5205</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2012</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqNkl1rFDEUhgdRbK1e-AckIIJeTJuvmdnxQlgWW6VFoVa9DGczZ3bTziRrktm6P9F_Zfaj6wcIXoSQ5Dnve3J4s-wpo8eMUn4CPh4LKWl9LztkBa9zygS7nx1SSmUuipodZI9CuE5HLgrxMDvgnNcyVRxmPz4NIYKx2BCPYeFsQAK2IQuPS7TROEtcSxroYYbp0s0SFda3xpIFRJOYQG5NnBOLzkKELl9rRNIPXTRhFSL2iW076HuIzq9IYwJCcokeISbbTTFYuDHWw2syJtrNnY8kxKFZkehIgxF9n1okce4R801_rVlivkLwxA1Rux7D4-xBC13AJ7v9KPt8-vZq8i6_-Hj2fjK-yHUhZZ1XclqBZgwRgYJkrOUaZcNbhk0NYqrTQiZ0U7FS6BGH6aiWVDatRqp5U4qj7M1WdzFMe2x0moCHTi286cGvlAOj_nyxZq5mbqmErGQhWRJ4uRPw7tuAIareBI1dB2mEQ1As9UlZKcv_QCnno4KXvEro87_Qazd4myaRBFnFWFVJkahXW0p7F4LHdt83o2qdJZWypDZZSuyz3z-6J-_Ck4AXOwCChq71YLUJv7gyedJi_YuTLXdrOlz921GNL6_urPNthUn5-b6vAH-jykpUhfr64UxV5-eTU345UV_ET7aN9nQ</recordid><startdate>201207</startdate><enddate>201207</enddate><creator>Sibley, Cailin H.</creator><creator>Plass, Nikki</creator><creator>Snow, Joseph</creator><creator>Wiggs, Edythe A.</creator><creator>Brewer, Carmen C.</creator><creator>King, Kelly A.</creator><creator>Zalewski, Christopher</creator><creator>Kim, H. Jeffrey</creator><creator>Bishop, Rachel</creator><creator>Hill, Suvimol</creator><creator>Paul, Scott M.</creator><creator>Kicker, Patrick</creator><creator>Phillips, Zachary</creator><creator>Dolan, Joseph G.</creator><creator>Widemann, Brigitte</creator><creator>Jayaprakash, Nalini</creator><creator>Pucino, Frank</creator><creator>Stone, Deborah L.</creator><creator>Chapelle, Dawn</creator><creator>Snyder, Christopher</creator><creator>Butman, John A.</creator><creator>Wesley, Robert</creator><creator>Goldbach-Mansky, Raphaela</creator><general>Wiley Subscription Services, Inc., A Wiley Company</general><general>Wiley</general><general>Wiley Subscription Services, Inc</general><scope>BSCLL</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7QL</scope><scope>7QP</scope><scope>7T5</scope><scope>7TM</scope><scope>7U7</scope><scope>C1K</scope><scope>H94</scope><scope>K9.</scope><scope>7X8</scope><scope>7U9</scope><scope>5PM</scope></search><sort><creationdate>201207</creationdate><title>Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes</title><author>Sibley, Cailin H. ; Plass, Nikki ; Snow, Joseph ; Wiggs, Edythe A. ; Brewer, Carmen C. ; King, Kelly A. ; Zalewski, Christopher ; Kim, H. Jeffrey ; Bishop, Rachel ; Hill, Suvimol ; Paul, Scott M. ; Kicker, Patrick ; Phillips, Zachary ; Dolan, Joseph G. ; Widemann, Brigitte ; Jayaprakash, Nalini ; Pucino, Frank ; Stone, Deborah L. ; Chapelle, Dawn ; Snyder, Christopher ; Butman, John A. ; Wesley, Robert ; Goldbach-Mansky, Raphaela</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c5449-74b7ac11eeea0a411f2ce4d2f1ed9a3bca3be13cd7163c82ab89404dfce0c2d63</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2012</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Antirheumatic Agents - administration & dosage</topic><topic>Antirheumatic Agents - therapeutic use</topic><topic>Biological and medical sciences</topic><topic>C-Reactive Protein</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Cryopyrin-Associated Periodic Syndromes - drug therapy</topic><topic>Cryopyrin-Associated Periodic Syndromes - pathology</topic><topic>Disease Progression</topic><topic>Diseases of the osteoarticular system</topic><topic>Drug therapy</topic><topic>Female</topic><topic>Humans</topic><topic>Immunomodulators</topic><topic>Infant</topic><topic>Infant, Newborn</topic><topic>Inflammation - drug therapy</topic><topic>Inflammation - pathology</topic><topic>Interleukin 1 Receptor Antagonist Protein - administration & dosage</topic><topic>Interleukin 1 Receptor Antagonist Protein - therapeutic use</topic><topic>Male</topic><topic>Malformations and congenital and or hereditary diseases involving bones. Joint deformations</topic><topic>Medical research</topic><topic>Medical sciences</topic><topic>Patients</topic><topic>Pharmacology. Drug treatments</topic><topic>Surveys and Questionnaires</topic><topic>Treatment Outcome</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Sibley, Cailin H.</creatorcontrib><creatorcontrib>Plass, Nikki</creatorcontrib><creatorcontrib>Snow, Joseph</creatorcontrib><creatorcontrib>Wiggs, Edythe A.</creatorcontrib><creatorcontrib>Brewer, Carmen C.</creatorcontrib><creatorcontrib>King, Kelly A.</creatorcontrib><creatorcontrib>Zalewski, Christopher</creatorcontrib><creatorcontrib>Kim, H. Jeffrey</creatorcontrib><creatorcontrib>Bishop, Rachel</creatorcontrib><creatorcontrib>Hill, Suvimol</creatorcontrib><creatorcontrib>Paul, Scott M.</creatorcontrib><creatorcontrib>Kicker, Patrick</creatorcontrib><creatorcontrib>Phillips, Zachary</creatorcontrib><creatorcontrib>Dolan, Joseph G.</creatorcontrib><creatorcontrib>Widemann, Brigitte</creatorcontrib><creatorcontrib>Jayaprakash, Nalini</creatorcontrib><creatorcontrib>Pucino, Frank</creatorcontrib><creatorcontrib>Stone, Deborah L.</creatorcontrib><creatorcontrib>Chapelle, Dawn</creatorcontrib><creatorcontrib>Snyder, Christopher</creatorcontrib><creatorcontrib>Butman, John A.</creatorcontrib><creatorcontrib>Wesley, Robert</creatorcontrib><creatorcontrib>Goldbach-Mansky, Raphaela</creatorcontrib><collection>Istex</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Bacteriology Abstracts (Microbiology B)</collection><collection>Calcium & Calcified Tissue Abstracts</collection><collection>Immunology Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Toxicology Abstracts</collection><collection>Environmental Sciences and Pollution Management</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>MEDLINE - Academic</collection><collection>Virology and AIDS Abstracts</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Arthritis & rheumatology (Hoboken, N.J.)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Sibley, Cailin H.</au><au>Plass, Nikki</au><au>Snow, Joseph</au><au>Wiggs, Edythe A.</au><au>Brewer, Carmen C.</au><au>King, Kelly A.</au><au>Zalewski, Christopher</au><au>Kim, H. Jeffrey</au><au>Bishop, Rachel</au><au>Hill, Suvimol</au><au>Paul, Scott M.</au><au>Kicker, Patrick</au><au>Phillips, Zachary</au><au>Dolan, Joseph G.</au><au>Widemann, Brigitte</au><au>Jayaprakash, Nalini</au><au>Pucino, Frank</au><au>Stone, Deborah L.</au><au>Chapelle, Dawn</au><au>Snyder, Christopher</au><au>Butman, John A.</au><au>Wesley, Robert</au><au>Goldbach-Mansky, Raphaela</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes</atitle><jtitle>Arthritis & rheumatology (Hoboken, N.J.)</jtitle><addtitle>Arthritis & Rheumatism</addtitle><date>2012-07</date><risdate>2012</risdate><volume>64</volume><issue>7</issue><spage>2375</spage><epage>2386</epage><pages>2375-2386</pages><issn>0004-3591</issn><issn>2326-5191</issn><eissn>1529-0131</eissn><eissn>2326-5205</eissn><coden>ARHEAW</coden><abstract>Objective
Blocking interleukin‐1 with anakinra in patients with the autoinflammatory syndrome neonatal‐onset multisystem inflammatory disease (NOMID) reduces systemic and organ‐specific inflammation. However, the impact of long‐term treatment has not been established. This study was undertaken to evaluate the long‐term effect of anakinra on clinical and laboratory outcomes and safety in patients with NOMID.
Methods
We conducted a cohort study of 26 NOMID patients ages 0.80–42.17 years who were followed up at the NIH and treated with anakinra 1–5 mg/kg/day for at least 36 months. Disease activity was assessed using daily diaries, questionnaires, and C‐reactive protein level. Central nervous system (CNS) inflammation, hearing, vision, and safety were evaluated.
Results
Sustained improvements in diary scores, parent's/patient's and physician's global scores of disease activity, parent's/patient's pain scores, and inflammatory markers were observed (all P < 0.001 at 36 and 60 months). At 36 and 60 months, CNS inflammation was suppressed, with decreased cerebrospinal fluid white blood cell counts (P = 0.0026 and P = 0.0076, respectively), albumin levels, and opening pressures (P = 0.0012 and P < 0.001, respectively). Most patients showed stable or improved hearing. Cochlear enhancement on magnetic resonance imaging correlated with continued hearing loss. Visual acuity and peripheral vision were stable. Low optic nerve size correlated with poor visual field. Bony lesions progressed. Adverse events other than viral infections were rare, and all patients continued to receive the medication.
Conclusion
These findings indicate that anakinra provides sustained efficacy in the treatment of NOMID for up to 5 years, with the requirement of dose escalation. Damage progression in the CNS, ear, and eye, but not bone, is preventable. Anakinra is well tolerated overall.</abstract><cop>Hoboken</cop><pub>Wiley Subscription Services, Inc., A Wiley Company</pub><pmid>22294344</pmid><doi>10.1002/art.34409</doi><tpages>12</tpages><oa>free_for_read</oa></addata></record> |
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| ispartof | Arthritis & rheumatology (Hoboken, N.J.), 2012-07, Vol.64 (7), p.2375-2386 |
| issn | 0004-3591 2326-5191 1529-0131 2326-5205 |
| language | eng |
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| source | MEDLINE; Wiley Online Library Journals Frontfile Complete |
| subjects | Adolescent Adult Antirheumatic Agents - administration & dosage Antirheumatic Agents - therapeutic use Biological and medical sciences C-Reactive Protein Child Child, Preschool Cryopyrin-Associated Periodic Syndromes - drug therapy Cryopyrin-Associated Periodic Syndromes - pathology Disease Progression Diseases of the osteoarticular system Drug therapy Female Humans Immunomodulators Infant Infant, Newborn Inflammation - drug therapy Inflammation - pathology Interleukin 1 Receptor Antagonist Protein - administration & dosage Interleukin 1 Receptor Antagonist Protein - therapeutic use Male Malformations and congenital and or hereditary diseases involving bones. Joint deformations Medical research Medical sciences Patients Pharmacology. Drug treatments Surveys and Questionnaires Treatment Outcome |
| title | Sustained response and prevention of damage progression in patients with neonatal-onset multisystem inflammatory disease treated with anakinra: A cohort study to determine three- and five-year outcomes |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2025-02-09T11%3A30%3A30IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_pubme&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Sustained%20response%20and%20prevention%20of%20damage%20progression%20in%20patients%20with%20neonatal-onset%20multisystem%20inflammatory%20disease%20treated%20with%20anakinra:%20A%20cohort%20study%20to%20determine%20three-%20and%20five-year%20outcomes&rft.jtitle=Arthritis%20&%20rheumatology%20(Hoboken,%20N.J.)&rft.au=Sibley,%20Cailin%20H.&rft.date=2012-07&rft.volume=64&rft.issue=7&rft.spage=2375&rft.epage=2386&rft.pages=2375-2386&rft.issn=0004-3591&rft.eissn=1529-0131&rft.coden=ARHEAW&rft_id=info:doi/10.1002/art.34409&rft_dat=%3Cproquest_pubme%3E1544016461%3C/proquest_pubme%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=1517117743&rft_id=info:pmid/22294344&rfr_iscdi=true |