Background Takayasu arteritis (TA) is a chronic inflammatory vasculitis of unknown origin affecting large vessels, predominantly the aorta and its main branches. Early symptoms include systemic inflammation and ischaemia of involved organs.

It was thought to be a disorder that affected mostly young Asian females, but TA has now been identified in both sexes and many ethnic and racial groups worldwide.

Tocilizumab is a humanised monoclonal antibody inhibitor of IL-6 receptor without a licence for its use in TA.

Purpose To assess the safety and efficacy of tocilizumab in a pluripathological patient with TA.

Material and methods Observational retrospective study of the use of tocilizumab in a child diagnosed with TA and several pathologies for 1 year.

The information was obtained from the electronic clinical history (DIRAYA^®) and the pharmacy service managing software (ATHOS-PRISMA^®).

Results A 12-year-old female was admitted in our tertiary care centre in July 2016 for heart failure secondary to dilated cardiomyopathy, diagnosed with Takayasu Grade V disease (supraortic, thoracic and abdominal-renal OA). In addition she presented, as basic diseases, arterial hypertension and renal failure. The first treatment line was corticosteroid pulses at 30 mg/kg and subsequently cyclophosphamide. On the fourth day, concurring with cyclophosphamide administration, clinical deterioration and increased cardiac dysfunction were presented. It was related to cyclophosphamide administration and was solved after diuretics’ optimisation.

After receiving four cycles of cyclophosphamide the patient maintained high acute-phase reactants, anaemia, and elevated IL-6 levels, so switching to tocilizumab was decided to stop systemic inflammatory activity and avoid new obstructions.

Treatment with tocilizumab 8 mg/kg/biweekly was initiated after being processed by the pharmacy and authorised by the medical director. Concomitant treatment: corticosteroids, antihypertensives and diuretics.

Ten months’ later, Takayasu disease is inactive and most clinical manifestations have disappeared (she only referred to discrete and eventual pain in the flexion of the left elbow without limitation or associated swelling). The patient continues with the same dosage.

Conclusion Tocilizumab has proved to be effective and well tolerated in this patient with TA.

Although this is an isolated case, we consider it essential that health professionals share their experiences in rare diseases to improve the therapeutic approach, especially in paediatric patients.

No conflict of interest