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Reason for request
- Key points
Favourable opinion for reimbursement in chronic immune (idiopathic) thrombocytopenic purpura (ITP) patients one year of age and older who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).
- What therapeutic improvement?
A slight therapeutic improvement in the treatment of chronic ITP patients one year of age and older who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab).
- Role in the care pathway?
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, no treatment or corticosteroid/IgIV therapy (on demand or programmed) may be proposed.
According to the expert opinion, the occasional use of TPO-R agonists (romiplostim and eltrombopag) 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.
Outside these occasional uses, given the primarily suspensive effect and uncertainties with respect to the long-term safety data for these products, TPO-R agonists (romiplostim and eltrombopag) may be considered in the treatment of chronic ITP refractory to first-line treatments (e.g. corticosteroids, immunoglobulins), following the failure of one of the second-line treatments (immunosuppressant, rituximab).
Clinical Benefit
| Substantial |
The clinical benefit of NPLATE 125 micrograms powder for solution for injection is substantial in the MA paediatric extension. |
Clinical Added Value
| minor |
Considering: - the demonstration of the superiority of NPLATE (romiplostim) compared to placebo on the durable platelet response (> 6 weeks), the primary outcome measure, in children one year of age and older with ITP, diagnosed at least 6 months previously, refractory to first-line treatments, - the mechanism of action of TPO-R agonists, - the absence of demonstration of efficacy on the number of bleeding episodes and the use of rescue medications, - the lack of long-term efficacy and safety data for the product, which has a primarily suspensive effect, - the absence of comparative data versus the other treatments of ITP used in second-line therapy, - the absence of any demonstration of efficacy in children refractory to first-line medicinal treatments and not responding to second-line treatments, the Transparency Committee considers that NPLATE provides a minor clinical added value (CAV IV) in the treatment of chronic ITP patients one year of age and older who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab). |
