Romiplostim for Treatment on Immune Thrombocytopenia (ITP)

Romiplostim, a thrombopoietin mimetic, increases platelet counts in patients with immune thrombocytopenia, with few adverse effects.

In this open-label, 52-week study (funded by Amgen), 234 adult patients with immune thrombocytopenia, who had not undergone splenectomy, were randomized to receive the standard of care or weekly subcutaneous injections of romiplostim.

The rate of a platelet response in the romiplostim group was 2.3 times that in the standard-of-care group.

Patients receiving romiplostim had a significantly lower incidence of treatment failure [11%] than those receiving the standard of care [30%].

Splenectomy also was performed less frequently in patients receiving romiplostim [9%]) than in those receiving the standard of care [36%].

The romiplostim group had a lower rate of bleeding events, fewer blood transfusions, and greater improvements in the quality of life.

Romiplostim is a fusion protein analog of thrombopoietin. It is marketed under the trade name Nplate through a restricted usage program. Romiplostim was designated an orphan drug by the FDA in 2003, as the chronic ITP population in the USA is under 200,000.

In 2008, the FDA approved romiplostim as a long-term treatment for chronic ITP in adults who have not responded to other treatments, such as:

- corticosteroids

- intravenous immunoglobulin (IVIG)

- Rho(D) immune globulin

- splenectomy

References:

Romiplostim or Standard of Care in Patients with Immune Thrombocytopenia. NEJM, 2010.
Immune Thrombocytopenia - 2010 Review in Hematology http://goo.gl/tXT2U

Immune thrombocytopenia: No longer ‘idiopathic’ http://goo.gl/z2Ks0
Immune thrombocytopenia in adults: An update. CCJM, 2012. 

Image source: Nplate.com