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REVOLADE

Primary immune thrombocytopenia
Opinions on drugs - Posted on Mar 04 2020

Reason for request

MA amendments

  • Key points

Favourable opinion for maintenance of reimbursement in primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and refractory to other treatments (e.g. corticosteroids, immunoglobulins).

  • What therapeutic improvement?

In the same way as NPLATE (romiplostim), REVOLADE (eltrombopag) represents a slight therapeutic improvement in the treatment of primary ITP lasting 12 months or longer from diagnosis of patients aged 1 year and above who are refractory to first-line treatments (e.g. corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab or splenectomy).

REVOLADE (eltrombopag) represents a slight therapeutic improvement in the treatment of primary ITP lasting 6 to 12 months from diagnosis of patients aged 1 year and above who are refractory to first-line treatments (corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab or splenectomy).

However, the clinical relevance of making a distinction between ITP lasting 12 months or longer from diagnosis and ITP lasting 6 to 12 months from diagnosis is low.

  • Role in the care pathway?

Immune thrombocytopenia (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, 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 may be considered in the treatment of ITP lasting more than 6 months from diagnosis in patients who are refractory to first-line treatments (corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab or splenectomy).

Splenectomy is a last resort treatment in children.


Clinical Benefit

Substantial

The clinical benefit of REVOLADE remains substantial in the MA indication

 


Clinical Added Value

minor

Considering:

-    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 used in second-line therapy,

-    the absence of any demonstration of efficacy in patients refractory to first-line medicinal treatments and not responding to second-line treatments,

the Transparency Committee considers that REVOLADE (eltrombopag), in the same way as NPLATE (romiplostim), provides a minor clinical added value (CAV IV) in the treatment of primary ITP lasting 12 months or longer from diagnosis in patients one year of age and older who are refractory to first-line treatments (corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab or splenectomy).

REVOLADE (eltrombopag) provides a minor clinical added value (CAV IV) in the treatment of primary ITP lasting 6 to 12 months from diagnosis in patients one year of age and older who are refractory to first-line treatments (corticosteroids, immunoglobulins) and who have not responded to one of the second-line treatments (immunosuppressant, rituximab or splenectomy).

However, the clinical relevance of making a distinction between ITP lasting 12 months or longer from diagnosis and ITP lasting 6 to 12 months from diagnosis is low.


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