Moutschen Michel, Boulanger Cécile, Dehoorne Joke, Joos Rik, Roufosse Florence, Sabato Vito, van der Hilst Jeroen, Maury Eleonore, Rabijns Hilde, Witterzeel Marijn, Wouters Carine
Clinical Immunology and Internal Medicine, CHU de Liège, ULiège, Liège, Belgium.
Department of Pediatric Hematology and Oncology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
BMC Rheumatol. 2025 May 29;9(1):64. doi: 10.1186/s41927-025-00515-w.
Canakinumab, an IL-1β inhibitor, has demonstrated long-term efficacy and safety in patients with sJIA, FMF, TRAPS, and MKD/HIDS who experience inadequate disease control with conventional treatments. This non-interventional study aimed to gain insights into canakinumab use and treatment patterns for these diseases in Belgium.
Between July 1, 2018 and June 30, 2023, this national, non-interventional, retrospective/prospective study enrolled patients aged ≥ 2 years with sJIA, FMF, TRAPS, or MKD/HIDS reimbursed for, and treated with, canakinumab in Belgium. Part 1: retrospective data collection from first canakinumab administration in the initial 6-month reimbursement period until date of study inclusion. Part 2: prospective data collection following study inclusion. Canakinumab treatment and safety data were collected throughout.
At data cut-off, 96 patients (7 sJIA, 70 FMF, 13 TRAPS, 6 MKD/HIDS) were enrolled, of whom 54.2% were female and 87.5% were adults (aged ≥ 18 years). Median age at first canakinumab administration was 34.0 years (20.0, 35.0, 37.0, and 42.0 years in sJIA, FMF, TRAPS, and MKD/HIDS, respectively). Eighteen patients discontinued treatment (3 sJIA, 11 FMF, 4 TRAPS), which was due to lack of efficacy (per investigator's judgment) in 10 (10.4%) patients. Median dose per administration was 289.1 mg in patients with sJIA, and 150.0 mg in patients with FMF, TRAPS, and MKD/HIDS, while median interval between two consecutive administrations was 28.0 days. Thirty-five (36.5%) patients with FMF, TRAPS, or MKD/HIDS received ≥ 1 dose increase (≥ 150 mg). No safety events were reported.
These non-interventional study data highlight that canakinumab treatment patterns are generally aligned with the summary of product characteristics (SmPC) and reimbursement criteria in Belgium and further support the well-tolerated safety profile of canakinumab. However, Belgian reimbursement criteria require long-term glucocorticoids prior to canakinumab therapy; if it were possible to align treatment more closely with EULAR/PReS guidance, which recommends early initiation of anti-IL-1 or anti-IL-6 therapy, glucocorticoid treatment would be limited and improved outcomes for these patients would likely be possible.
Not applicable.
卡那单抗是一种白细胞介素-1β抑制剂，已在全身型幼年特发性关节炎（sJIA）、家族性地中海热（FMF）、肿瘤坏死因子受体相关周期性综合征（TRAPS）以及甲羟戊酸激酶缺乏症/高免疫球蛋白D综合征（MKD/HIDS）患者中显示出长期疗效和安全性，这些患者接受传统治疗后疾病控制不佳。这项非干预性研究旨在深入了解比利时这些疾病的卡那单抗使用情况和治疗模式。
在2018年7月1日至2023年6月30日期间，这项全国性、非干预性、回顾性/前瞻性研究纳入了年龄≥2岁、在比利时接受卡那单抗报销及治疗的sJIA、FMF、TRAPS或MKD/HIDS患者。第1部分：从首次使用卡那单抗的初始6个月报销期到纳入研究之日的回顾性数据收集。第2部分：纳入研究后的前瞻性数据收集。全程收集卡那单抗治疗和安全性数据。
在数据截止时，共纳入96例患者（7例sJIA、70例FMF、13例TRAPS、6例MKD/HIDS），其中54.2%为女性，87.5%为成年人（年龄≥18岁）。首次使用卡那单抗时的中位年龄为34.0岁（sJIA、FMF、TRAPS和MKD/HIDS患者分别为20.0岁、35.0岁、37.0岁和42.0岁）。18例患者停止治疗（3例sJIA、11例FMF、4例TRAPS），其中10例（10.4%）患者是由于（根据研究者判断）缺乏疗效。sJIA患者每次给药的中位剂量为289.1mg，FMF、TRAPS和MKD/HIDS患者为150.0mg，而连续两次给药的中位间隔时间为28.0天。35例（36.5%）FMF、TRAPS或MKD/HIDS患者接受了≥1次剂量增加（≥150mg）。未报告安全事件。
这些非干预性研究数据表明，卡那单抗的治疗模式总体上与比利时的产品特性摘要（SmPC）和报销标准一致，并进一步支持了卡那单抗良好的耐受性安全性。然而，比利时的报销标准要求在使用卡那单抗治疗前长期使用糖皮质激素；如果能够使治疗更紧密地符合欧洲抗风湿病联盟（EULAR）/儿科风湿病学会（PReS）的指南，该指南建议早期启动抗白细胞介素-1或抗白细胞介素-6治疗，那么糖皮质激素治疗将受到限制，这些患者可能会取得更好的治疗效果。
不适用。
