An update on romiplostim for treatment of acute radiation syndrome

Detonation of an improvised nuclear weapon, or a radiological dispersal device by terrorists, or an unintended radiological/nuclear accident in populated areas would result in a mass casualty scenario with radiation exposures of different severities. Such incidences are perceived as national security threats of major consequence. Acute radiation syndrome (ARS) is triggered by an exposure to a high dose of penetrating ionizing radiation during a short time window. In humans, moderate exposure to 2 to 4 Gy of ionizing radiation results in clinically manageable hematopoietic ARS (H-ARS), characterized by severe depletion of vital blood cells and bone marrow progenitors. Since 2015, the United States Food and Drug Administration (U.S. FDA) has approved four radiation medical countermeasures for H-ARS following the Animal Rule; namely, Neupogen, Neulasta, Leukine and Nplate (romiplostim). Here, we briefly present the treatment modalities for H-ARS. We have discussed the latest FDA-approved agent, romiplostim, as a treatment modality for H-ARS. The nature of this agent and the preclinical and clinical work that preceded its FDA approval as a radiation medical countermeasure are discussed, as are the development and use of related thrombopoietic agents for the treatment of radiation-exposed victims.