Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Duke Medical Center, Durham, North Carolina.
Arthritis Rheumatol. 2020 Nov;72(11):1945-1951. doi: 10.1002/art.41390. Epub 2020 Oct 8.
To document the need for additional Food and Drug Administration (FDA)-approved medications for the treatment of juvenile idiopathic arthritis (JIA).
The electronic medical records of JIA patients treated at Cincinnati Children's Hospital Medical Center (CCHMC) and data from JIA patients enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry were included in this study. Unmet medication need was defined in 2 ways: (a) the presence of chronically uncontrolled JIA, defined as a physician global assessment of JIA activity ≥3 (on a 0-10 scale, where 0 = inactive) OR ≥3 joints with active arthritis OR a patient global assessment of well-being ≥3 (on a 0-10 scale, where 0 = very well), despite sequential use of ≥2 biologic disease-modifying antirheumatic drugs (bDMARDs); and (b) the 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 304 (19%) had been exposed to ≥1 unapproved bDMARD. In the CARRA Registry, 4,766 of 7,379 children (65%) had received ≥1 bDMARD, and 1,122 (15%) had been prescribed ≥1 unapproved bDMARD. Of those children treated with ≥2 bDMARDs for whom complete data were available, 52% (255 of 487) at CCHMC and 45% (527 of 1,159) in the CARRA Registry had chronically uncontrolled JIA despite the use of ≥2 bDMARDs.
Despite the availability of bDMARDs currently approved for JIA, there is persistent need for additional therapies to control JIA signs and symptoms. Since FDA approval is critical to ensure access to bDMARDs, the study and licensing of new medications is critical to address the unmet medication need and to further improve JIA outcomes.
记录治疗青少年特发性关节炎（JIA）所需的更多美国食品和药物管理局（FDA）批准的药物。
本研究纳入了在辛辛那提儿童医院医疗中心（CCHMC）接受治疗的 JIA 患者的电子病历和在儿童关节炎和风湿病研究联盟（CARRA）注册中心登记的 JIA 患者的数据。未满足的药物需求通过以下两种方式定义：（a）存在慢性未控制的 JIA，定义为医师整体评估 JIA 活动度≥3（0-10 分，其中 0 表示不活动）或≥3 个关节存在活动性关节炎，或患者整体评估健康状况≥3（0-10 分，其中 0 表示非常好），尽管已连续使用≥2 种生物疾病修正抗风湿药物（bDMARD）；（b）使用≥1 种未批准用于任何 JIA 类别的 bDMARD。
在 CCHMC，1599 例 JIA 患者中有 829 例（52%）接受了≥1 种 bDMARD 治疗，304 例（19%）接受了≥1 种未批准的 bDMARD 治疗。在 CARRA 注册中心，7379 例儿童中有 4766 例（65%）接受了≥1 种 bDMARD，1122 例（15%）接受了≥1 种未批准的 bDMARD 治疗。在接受≥2 种 bDMARD 治疗且可获得完整数据的儿童中，CCHMC 中有 52%（255 例）和 CARRA 注册中心中有 45%（527 例）存在慢性未控制的 JIA，尽管已使用≥2 种 bDMARD。
尽管目前有用于 JIA 的 bDMARD，但仍需要额外的治疗方法来控制 JIA 的体征和症状。由于 FDA 批准对于获得 bDMARD 至关重要，因此研究和许可新药物对于满足未满足的药物需求以及进一步改善 JIA 结局至关重要。
