OFEV - PID-ScS chez l’adulte (nintedanib)

Key points

Favourable opinion for reimbursement in the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD).

What therapeutic improvement?

Therapeutic improvement in the management of the disease.

Role in the care pathway?

The therapeutic management of systemic sclerosis (SSc) is difficult due to its clinical heterogeneity, the involvement of several organs and the absence of global treatment acting simultaneously on each of the different pathogenic mechanisms. In a multidisciplinary approach, the treatment of organ involvement is the primary objective and depends on the type and severity of this involvement.

Questioning seeks to identify any occupational exposure to silica or solvents, leading to specification of risk factors and performance of an occupational investigation. It is also necessary to identify any factors liable to exacerbate vascular disease (hammering, vibrations), as well as exposure to tobacco smoke.

The diagnosis should be considered in the event of combination of several criteria defined in the ACR/EULAR classification: Raynaud’s phenomenon (present in more than 95% of cases), cutaneous sclerosis, trophic disorders, such as fingertip ulcers or pitting scars, calcinosis and joint or muscle and tendon involvement.

Assessment should be routinely conducted to identify the presence of any pulmonary involvement. If pulmonary involvement is detected, regular monitoring should be put in place to measure the evolution of interstitial lung disease (ILD).

The severity of ILD is measured by assessing dyspnoea, transcutaneous oxygen saturation, forced vital capacity (FVC) and total lung capacity, carbon monoxide diffusing capacity (DLCO), chest high resolution computed tomography (HRCT) scan data (extent and characteristics of lung lesions).

The French national diagnostic and care protocol (PNDS) recommends treating patients with progressive ILD (10% loss of FVC or ≥ 200 mL and/or 15% of DLCO) or ILD that is severe from the outset.

Mycophenolate Mofetil (MMF) is recommended as first-line treatment, and cyclophosphamide IV as second-line treatment or as first-line treatment in fast-progressing forms or those with a poor prognosis. Rituximab is reserved for third-line treatment.

Each treatment line should be reassessed after 6 months of treatment on the basis of clinical data (NYHA functional class, 6 min walking test) and pulmonary function tests. A HRCT scan should be performed at the end of the treatment sequence and in the event of clinical worsening.

In the event of stabilisation or improvement of clinical condition, respiratory function tests or scan results, immunosuppressant therapy is continued for at least 2 years (there is a lack of published data beyond this period). Cyclophosphamide IV can be continued for 12 months and is then followed by azathioprine or MMF.

In ILD forms with severe respiratory failure despite the previously cited treatments, and in the absence of other severe organ involvement, lung transplantation may be considered.

Role of the medicinal product in the care pathway

OFEV can be used alone or in combination with immunomodulators and/or oral corticosteroids in patients with a clinical and radiological diagnosis of SSc-ILD.

It should be highlighted that the efficacy and safety data were only obtained in patients with a pulmonary fibrosis extension of more than 10%, confirmed with the following respiratory function criteria: FVC ≥ 40% and DLCO ≥ 30%.

Special recommendations

Due to the complexity of diagnosing and managing these diseases, the Committee recommends that the decision to initiate OFEV (nintedanib) treatment be discussed during a multidisciplinary team (MDT) meeting and that the decision be tracked, then submitted and explained to the patient.