Nplate (romiplostim) for ITP

A paper in NEJM reported on an open label comparative trial against standard treatment last November:

BACKGROUND 

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

CONCLUSIONS

Patients treated with romiplostim had a higher rate of a platelet response, lower incidence of treatment failure and splenectomy, less bleeding and fewer blood transfusions, and a higher quality of life than patients treated with the standard of care.

It seems like a great drug, but the FDA has approved it only for restricted use (after unsatisfactory attempts at standard treatments). Why? Well, according to the product labeling there seems to be a risk of marrow fibrosis and cytopenias, requiring careful baseline heme assessment and post treatment monitoring.


Via Clinical Cases and Images.