New Medications Are Needed for Children With Juvenile Idiopathic Arthritis.
New Medications Are Needed for Children With Juvenile Idiopathic Arthritis.
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DOI：
10.1002/art.41390
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发表时间：
2020-11
期刊：
影响因子：
--
通讯作者：
PRCSG Advisory Council and the CARRA Registry Investigators
中科院分区：
文献类型：
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作者：
Brunner HI;Schanberg LE;Kimura Y;Dennos A;Co DO;Colbert RA;Fuhlbrigge RC;Goldmuntz E;Kingsbury DJ;Patty-Resk C;Mintz S;Onel K;Rider LG;Schneider R;Watts A;von Scheven E;Lovell DJ;Beukelman T;PRCSG Advisory Council and the CARRA Registry Investigators
记录治疗幼年特发性关节炎（JIA）的其他FDA批准药物的需求。本研究纳入了在辛辛那提儿童医院医学中心（CCHMC）接受治疗的JIA患者的电子病历和儿童关节炎和风湿病研究联盟（CARRA）登记的JIA患者的数据。未满足的药物需求通过两种方式测量：（A）存在慢性不受控制的JIA，定义为医生对JIA活动的全球评估（0-10; 0=不活动）≥3个或≥3个活动关节或患者整体健康评估（0-10; 0=非常好）≥3，尽管序贯使用了≥2种生物疾病缓解抗风湿药物（bDMARD）;和（B）使用≥1个未批准用于任何JIA类别的bDMARD。在CCHMC，1，599例JIA患者中有829例（52%）接受了≥1种bDMARD治疗，19%（304/1，599）暴露于≥1种未经批准的bDMARD。在CARRA登记研究中，7，379名儿童中有4，766名（65%）接受了≥1种bDMARD治疗，1，122名（15%）接受了≥1种未经批准的bDMARD处方;在接受≥1次DMARD治疗的儿童中，CCHMC中52%（225/487）的患者和CARRA登记研究中45%（527/1159）的患者（数据完整）尽管使用≥2种bDMARD，但仍患有慢性不受控制的JIA。尽管bDMARD目前已被批准用于JIA，但仍持续需要其他治疗方法来控制JIA体征和症状。由于FDA的批准对于确保bDMARD的获得至关重要，因此新药的研究和许可对于解决未满足的药物需求和进一步改善JIA结果至关重要。
To document the need for additional FDA approved medications for the treatment of juvenile idiopathic arthritis (JIA). The electronic medical record of JIA patients treated at Cincinnati Children’s Hospital Medical Center (CCHMC) and data from JIA patients enrolled in the Childhood Arthritis & Rheumatology Research Alliance (CARRA) Registry were included in this study. Unmet medication need was measured in two ways: (A) presence of chronically uncontrolled JIA defined as a physician global-assessment of JIA activity (0–10; 0=inactive) ≥3 OR ≥3 active joints OR a patient global-assessment of well-being (0–10; 0=very well) ≥3, despite sequential use of ≥2 biologic disease-modifying anti-rheumatic drugs (bDMARDs); and (B) use of ≥1 bDMARD not approved for any JIA category. At CCHMC, 829 of 1,599 JIA patients (52%) were treated with ≥1 bDMARD and 19% (304/1,599) had been exposed to ≥1 unapproved bDMARD. In the CARRA Registry, 4,766 of 7,379 (65%) children had received ≥1 bDMARD and 1,122 (15%) had been prescribed ≥1 unapproved bDMARD; Of those children treated with ≥1 DMARD, 52% (225/487) at CCHMC and 45% (527/1159) of patients in the CARRA Registry with complete data had chronically uncontrolled JIA despite use of ≥2 bDMARDs. Despite the bDMARDs that are currently approved for JIA, there is persistent need for additional therapies to control JIA signs and symptoms. Since FDA approval is critical to insure bDMARD access, the study and licensing of new medications is critical to address the unmet medication need and to further improve JIA outcomes.
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DOI：
10.1186/s12969-017-0160-6
发表时间：
2017-04-17
期刊：
Pediatric rheumatology online journal
影响因子：
--
作者：
Beukelman T;Kimura Y;Ilowite NT;Mieszkalski K;Natter MD;Burrell G;Best B;Jones J;Schanberg LE;CARRA Registry Investigators
通讯作者：
CARRA Registry Investigators
影响因子：
3.9
作者：
Wallace, Carol A.;Giannini, Edward H.;Lovell, Daniel J.
通讯作者：
Lovell, Daniel J.
影响因子：
--
作者：
Ruperto, Nicolino;Lovell, Daniel J.;Giannini, Edward H.
通讯作者：
Giannini, Edward H.
影响因子：
--
作者：
Ringold S;Weiss PF;Beukelman T;DeWitt EM;Ilowite NT;Kimura Y;Laxer RM;Lovell DJ;Nigrovic PA;Robinson AB;Vehe RK;American Collge of Rheumatology
通讯作者：
American Collge of Rheumatology
影响因子：
13.3
作者：
Angeles-Han, Sheila T.;Ringold, Sarah;Reston, James
通讯作者：
Reston, James