TAVLESSE (fostamatinib)

Key points

Favourable opinion for reimbursement in the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments.

What therapeutic improvement?

No clinical added value in the therapeutic strategy for chronic ITP on the basis of currently available data.

Role in the care pathway?

Chronic ITP requires the initiation of specialised care, in liaison with the general practitioner.

In the absence of clinical or haematological signs of ITP severity and consequences on quality of life (asthenia, impact on schooling, etc.), no treatment or corticosteroid/Ig IV therapy (on demand or programmed) may be proposed.

In the event of chronic ITP, i.e., present for more than 12 months, several second-line treatments may be proposed in adults; few of these treatments have been evaluated in randomised controlled studies. Only thrombopoietin receptor agonists (romiplostim, eltrombopag) and azathioprine have an indication in ITP validated by an MA. Rituximab has a temporary recommendation for use (RTU) in this indication. According to the 2017 French national diagnostic and care protocol (PNDS), splenectomy is also one of the second-line therapeutic options in the event of chronic ITP. Despite the increased risks of serious infections and venous and/or arterial thrombosis in adults, splenectomy is the only treatment that has been established as being curative. There is no consensus with respect to its role in the hierarchical sequence of second-line treatments and it may be performed before or after rituximab and/or  Thrombopoietin receptor  agonists (TPO-RA).

In the absence of a consensus-based care pathway, the choice of treatment among the available options must remain personalised and be discussed on a case-by-case basis.

In accordance with the most recent Committee opinions relative to TPO-RA (eltrombopag and romiplostim), the occasional use of TPO-RA may be considered in the treatment of marked thrombocytopenia refractory to first-line treatments, in the event of severe bleeding syndrome, in the context of preparation for a surgical procedure or pending the effect of already initiated second-line therapy (depending on expert opinion). Outside these occasional uses, given the primarily suspensive effect and uncertainties with respect to the long-term safety data of these medicinal products, they may be considered in the treatment of chronic ITP refractory to first-line treatments (e.g. corticosteroids, immunoglobulins) and following the failure of one of the second-line treatments (immunosuppressant, rituximab or splenectomy) or as an alternative to these treatments when they are not considered to be appropriate.

Role of the medicinal product in the care pathway:

On the basis of currently available data and taking into account the identified medical need, the Committee considers that the use of TAVLESSE (fostamatinib) may be considered only as a last-resort treatment for multirefractory chronic ITP resistant to second-line treatments (which, according to the 2017 PNDS include splenectomy TPO-RA [switches included] and rituximab). In patients not having undergone splenectomy, multirefractory to medicinal treatments, TAVLESSE may be a treatment option when splenectomy is deemed to be inappropriate by reference or expert centres.

The Committee points out that this medicine is subject to hospital prescription only by certain specialist physicians (PRS). In addition, renewal of prescription is limited to certain healthcare professionals.

Given the specificities of treatment of this rare disease, the Committee recommends that decisions to initiate or discontinue fostamatinib treatment be taken after a documented proposal resulting from a multidisciplinary team meeting within a reference or expert centre for the treatment of ITP. Regular follow-up of patients within one of these centres is essential to ensure the efficacy of treatment and monitor its safety.

Special recommendations

Given the specificities of treatment of this rare disease, the Committee recommends that decisions to initiate or discontinue fostamatinib treatment be taken after a documented proposal resulting from a multidisciplinary team meeting within a reference or expert center for the treatment of ITP. Regular follow-up of patients within one of these centers is essential to ensure the efficacy of treatment and monitor its safety.