Pamidronate in chronic non-bacterial osteomyelitis: a randomized, double-blinded, placebo-controlled pilot trial

Abstract

Objective

This is the first randomized double-blinded, placebo-controlled pilot trial to investigate the efficacy of pamidronate in reducing radiological and clinical disease activity in chronic non-bacterial osteomyelitis (CNO).

Method

Patients received pamidronate or placebo at baseline and weeks 12 and 24. Whole-body magnetic resonance imaging was performed at baseline and weeks 12 and 36, and computed tomography of the anterior chest wall (ACW) at baseline and week 36. Radiological disease activity was systematically scored in the ACW and spine. Patient-reported outcomes [visual analogue scale (VAS) pain, VAS global health, Health Assessment Questionnaire (HAQ), EuroQol-5 Dimensions (EQ-5D), and 36-item Short-Form Health Survey (SF-36)] and biomarkers of bone turnover and inflammation were assessed at baseline and weeks 1, 4, 12, 24, and 36. Data are expressed as median [interquartile range].

Results

Fourteen patients were randomized and 12 were analysed. From baseline to week 36, the radiological disease activity score in the ACW decreased from 5 [4–7] to 2.5 [1–3] in the pamidronate group, but did not change in the placebo group (p = 0.04). From baseline to week 36, VAS pain and VAS global health tended to decrease more in the pamidronate than in the placebo group (p = 0.11, p = 0.08). Physical functioning (HAQ) and health-related quality of life (EQ-5D, SF-36) did not change. Biomarkers of bone turnover decreased only in the pamidronate group (p ≤ 0.02).

Conclusion

Pamidronate may improve radiological and clinical disease activity in CNO. Methods to score radiological disease activity in adult CNO were suggested.

Clinical Trials: NCT02594878

Acknowledgements

This work was supported by Aarhus University; the Danish Rheumatism Association [grant number R121-A3015]; Aase and Ejnar Danielsen Foundation; the AP Møller Foundation for the Advancement of Medical Science; and the Hede Nielsen Foundation [no grant number].

The authors thank all participating patients, study nurses, and technicians. The rheumatologists T Lorenzen, K Mai, L Svendsen, S Aaboe, U Weber, M Varvounis, A Linauskas, RD Østgaard, and AG Loft are acknowledged for their contribution to the recruitment of patients to the study.

Disclosure statement

No potential conflict of interest was reported by the authors.

Supporting information

Additional Supporting Information may be found in the online version of this article.

Supplementary file S1: Study criteria.

Supplementary file S2: Imaging material and methods

Supplementary file S3: CT images of the anterior chest wall and MRI of the spine.

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