儿童慢性非细菌性骨髓炎——生物时代之前一项前瞻性观察队列的五年标准化随访

Chronic non-bacterial osteomyelitis in children- five-year standardized follow-up of a prospective observational cohort in the pre-biological era.

作者信息

Hofmann Christine, Holl-Wieden Annette, Reiser Christiane, Beer Meinrad, Raab Peter, Morbach Henner, Girschick Hermann J

机构信息

Pediatric Rheumatology, Immunology, Osteology, Children's Hospital, University of Wuerzburg, Wuerzburg, Germany.

Clinic of Pediatric Rheumatology and Rare diseases, Children's Hospital, University of Tuebingen, Tuebingen, Germany.

出版信息

Pediatr Rheumatol Online J. 2025 May 13;23(1):50. doi: 10.1186/s12969-025-01106-2.

DOI:10.1186/s12969-025-01106-2

PMID:40361097

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12076821/

Abstract

BACKGROUND

This prospective, long-term observational study, initiated in 2002, aimed to characterize clinical and laboratory data, whole body MRI detected lesions, and treatment responses in 37 juvenile patients with chronic non-bacterial osteomyelitis at a time when biological DMARDs were not yet standard therapy.

METHODS

Patients were assessed at baseline and at 1 (without MRI), 3, 6, 12, 18, 24, 36, 48, 60 months. All patients received naproxen as first-line therapy. Clinical management allowed for escalation to sulfasalazine, pamidronate, and glucocorticoids as needed. Treatment response was evaluated using the pedCNO disease activity score (30/50/70/90% improvement). Further composite numeric disease activity (DA) scores- the CARRA CDAS and a new MRI DAS - were applied.

RESULTS

The mean age at disease onset was 10.8 years, with a diagnostic delay of 5.8 months. Naproxen was the initial treatment in all patients. Second-line therapy was initiated in 10 patients due to inadequate improvement in physician global assessment of disease activity, patient-reported overall wellbeing or MRI lesions. Escalated therapies included sulfasalazine (n = 10), bisphosphonates (n = 1), methotrexate (n = 1), and short- (< 4 wks) or long-term oral glucocorticoids (n = 5 and n = 3, respectively). The mean number of clinical lesions decreased from 2.1 to 0.4 at 12 months and reached 0.15 at 60 months. MRI-detected lesions declined from 5.0 to 2.25 at 12 months and to 1.1 at 60 months.

CONCLUSION

Most children experienced favourable long-term outcomes. Clinical improvement occurred more rapidly than radiologic resolution. Patients with insufficient response to NSAIDs should be considered for a treat-to-target approach, including the use of conventional and biologic DMARDs.

TRIAL REGISTRATION

A trial registration EUDRA CT was not available at the time the study was started. Informed consent was given by all parents.

摘要

背景

这项前瞻性长期观察性研究始于2002年，旨在描述37例慢性非细菌性骨髓炎青少年患者的临床和实验室数据、全身MRI检测到的病变以及治疗反应，当时生物性改善病情抗风湿药（bDMARDs）尚未成为标准治疗方法。

方法

在基线以及第1个月（无MRI检查）、3个月、6个月、12个月、18个月、24个月、36个月、48个月、60个月时对患者进行评估。所有患者均接受萘普生作为一线治疗。临床管理允许根据需要升级为柳氮磺胺吡啶、帕米膦酸盐和糖皮质激素。使用儿童慢性非细菌性骨髓炎疾病活动评分（改善30%/50%/70%/90%）评估治疗反应。还应用了进一步的综合数字疾病活动（DA）评分——儿童风湿病研究联盟（CARRA）综合疾病活动评分（CDAS）和一种新的MRI疾病活动评分。

结果

疾病发病的平均年龄为10.8岁，诊断延迟5.8个月。萘普生是所有患者的初始治疗药物。由于医生对疾病活动的整体评估、患者报告的总体健康状况或MRI病变改善不足，10例患者开始二线治疗。升级后的治疗包括柳氮磺胺吡啶（n = 10）、双膦酸盐（n = 1）、甲氨蝶呤（n = 1）以及短期（<4周）或长期口服糖皮质激素（分别为n = 5和n = 3）。临床病变的平均数量在12个月时从2.1降至0.4，在60个月时降至0.15。MRI检测到的病变在12个月时从5.0降至2.25，在60个月时降至1.1。