Thrombopoietin-receptor agonists

Thrombopoietin-receptor agonists (TPO-RAs) have been approved for use in immune thrombocytopenia (ITP) after showing safety and efficacy. There is increasing interest to expand the role of TPO-RAs, both in ITP as well as in other thrombocytopenic disorders.

In ITP, more studies are providing evidence of TPO-RA efficacy and safety, as well as their applicability to various patient groups, including children. Use of TPO-RAs in hepatitis C has shown early success in allowing treatments in patients who would otherwise be excluded due to thrombocytopenia. Use in congenital thrombocytopenias has also shown early success. The use of TPO-RAs in myelodysplastic syndrome (MDS) is questionable after reports of increasing blasts and leukemic transformation, whereas in other chemotherapy-induced thrombocytopenias (C-ITs) reports are few. Bone marrow fibrosis remains an area of active study, although the data to date suggest this is seen in a small minority of patients, and is reversible and of questionable clinical relevance. Thrombotic complications are also an area of concern and need further close follow-up.

The use of TPO-RAs continues to grow as more evidence of safety and efficacy is found. More studies are needed to determine their utility in other diseases as well as to better characterize adverse events observed to date.