Review
Efficacy and safety of tocilizumab in polyarteritis nodosa and adenosine deaminase 2 deficiency: a systematic literature review
E.S. Torun1, S. Çelen2
- Division of Rheumatology, Department of Internal Medicine, Dr. Demiroğlu Science University, Istanbul Florence Nightingale Hospital, İstanbul, Turkey. egesinantorun@hotmail.com
- Department of Internal Medicine, Dr. Dörtyol Government Hospital, Istanbul, Turkey.
CER19096
Review
Received: 13/07/2025
Accepted : 16/09/2025
In Press: 08/10/2025
Abstract
OBJECTIVES:
There is a significant need for medications that offer improved effectiveness and safety in the treatment of polyarteritis nodosa (PAN). Research has shown that serum interleukin-6 levels were elevated in both PAN and adenosine deaminase 2 deficiency (DADA 2) patients, which supported the exploration of tocilizumab for patients with refractory cases of these conditions.
METHODS:
A comprehensive systematic literature review was conducted to investigate the effectiveness and safety of tocilizumab in patients with PAN and DADA 2.
RESULTS:
We reviewed 28 studies. Twenty-nine PAN patients received tocilizumab, resulting in favourable response for twenty-two patients. Five patients did not respond to the treatment. Tocilizumab was discontinued in two patients due to adverse effects before its effectiveness could be evaluated. Fifteen DADA 2 patients were treated with tocilizumab, with two achieving complete response, two showing a partial response, and nine not responding at all. In two cases, the assessment of effectiveness was not possible. Three patients experienced ischaemic vascular events while on tocilizumab, and ten patients transitioned to anti-tumour necrosis factor therapies. Side effects included infections, cytopenias, and hyperlipidemia, which were consistent with those observed in other rheumatic disease treatments involving tocilizumab.
CONCLUSIONS:
Tocilizumab appeared to be a promising and safe option for paediatric and adult PAN patients as a salvage therapy for those who did not respond to conventional and biologic immunosuppressive therapies. However, in patients with DADA 2, tocilizumab showed limited effectiveness and it generally performed worse than anti-tumour necrosis factor alpha agents in most cases.
