Clinical characteristics and outcomes of paediatric CNO: a retrospective cohort study with focus on zoledronic acid.
In a single-center retrospective cohort of 57 children with chronic non-bacterial osteomyelitis, 22 received off-label zoledronic acid, with reported clinical improvement in 77%, radiological improvement in 8 of 10 patients with paired whole-body MRI, and no serious adverse events.
Open original publication →What the AI sees
In a single-center retrospective cohort of 57 children with chronic non-bacterial osteomyelitis, 22 received off-label zoledronic acid, with reported clinical improvement in 77%, radiological improvement in 8 of 10 patients with paired whole-body MRI, and no serious adverse events.
Research significance
The cohort provides preliminary evidence that intermittently dosed zoledronic acid may improve clinical and MRI manifestations of severe or refractory pediatric chronic non-bacterial osteomyelitis; whether it is superior to other treatments or improves long-term outcomes remains an inference requiring controlled prospective study.
Source abstract
OBJECTIVES: Chronic non-bacterial osteomyelitis is a rare autoinflammatory bone disease of childhood. Non-steroidal anti-inflammatory drugs are often used first-line, while conventional and biologic disease-modifying antirheumatic drugs and bisphosphonates are reserved for severe or refractory disease. Zoledronic acid is used off-label, but robust data on its role remain limited. This study aimed to describe the clinical characteristics, management, and outcomes of children with Chronic non-bacterial osteomyelitis, with primary focus on the effectiveness and tolerability of Zoledronic acid. METHOD: A retrospective review was conducted of children diagnosed with chronic non-bacterial osteomyelitis between 2014 and 2024 at the Royal Children's Hospital, Melbourne. Clinical and magnetic resonance imaging responses to ZA, as well as adverse effects, were evaluated. RESULTS: Fifty-seven patients were included (mean age 10.3 years; 70% female; median follow-up 3.7 years [range 1.4-8.9]). Multifocal disease was present in 86%, and 63% underwent biopsy. All received non-steroidal anti-inflammatory drugs initially. Twenty-two patients (37%) received zoledronic acid (mean 2.6 doses); 77% showed significant clinical improvement. Three later required tumour necrosis factor inhibitors, and two received tumour necrosis factor inhibitors without prior zoledronic acid. Among 10 patients with pre- and post-zoledronic acid whole body magnetic resonance imaging, 80% demonstrated significant radiological improvement. Mild, transient flu-like symptoms were reported in 54% of patients treated with zoledronic acid; no serious adverse events occurred. CONCLUSION: Zoledronic acid was associated with high clinical and radiological response rates and was well tolerated. Its infrequent dosing schedule, typically one infusion every six months, offers a practical alternative to other bisphosphonates.