Eltrombopag Improves Aplastic Anemia Treatment, Study Shares

Just 50 years ago, receiving a diagnosis of severe aplastic anemia could be devastating. While therapeutic options have greatly improved over the past five decades, some doctors have still found it difficult to effectively treat, or improve treatment for, patients with aplastic anemia. However, this may soon change. According to Medical XPress, results from the Phase 3 RACE clinical trial highlighted how adding eltrombopag to immunosuppressive therapies could improve response rates for those with aplastic anemia.

Interested in learning more? The full study findings are published in the New England Journal of Medicine.


To begin, let’s take a look at what eltrombopag is. This orally administered thrombopoietic medication helps to increase platelet counts. It is used, in conjunction with other therapies, for aplastic anemia; eltrombopag can also be used as a monotherapy for chronic immune thrombocytopenia, or to help ready patients with hepatitis C prepare for other treatments.

Currently, the standard-of-care for severe aplastic anemia is either hematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy (hATG and CsA). But one of the concerns from medical professionals is that not all patients respond well to treatment. For example, an estimated 35% of those with severe aplastic anemia do not have adequate treatment responses. Plus, for those who do respond to treatment, an estimated 33% will have a relapse and 33% will require chronic immunosuppressives. Therefore, additional therapeutic options are urgently needed.

Researchers evaluated one of these options, eltrombopag, in the Phase 3 RACE study. Altogether, 197 patients (aged 15+) enrolled. Participants were treatment-naive, meaning they had not previously received any immunosuppressive therapy. During the trial, patients either received immunosuppressive therapy on its own, or immunosuppressives alongside eltrombopag, for up to 6 months. Findings include:

  • Patients who also received eltrombopag saw significantly higher complete response rates compared to those just receiving immunosuppressives. For example, 68% of those taking eltrombopag saw a complete response, compared to 41% from the other cohort.
  • Eltrombopag was found to be relatively safe and well-tolerated.
  • Adding eltrombopag to treatment also hastened response rates to around 3 months.
  • While eltrombopag was found to be safe overall, some researchers believe treatment could cause secondary malignancies to occur. While the data so far has not supported this, further research will be carried out to determine whether this is true.

Aplastic Anemia

Aplastic anemia is a rare and serious blood disorder, which can also be considered a form of bone marrow failure. In this condition, the bone marrow stops producing enough healthy blood cells (including red and white blood cells, as well as platelets). Aplastic anemia may be acquired through gene mutations, or caused by viral infections, radiation or chemotherapy, or certain medication use. Either way, the bone marrow becomes damaged and stops producing enough blood and stem cells. This condition may develop over time or occur suddenly. While it may appear in those of all ages, aplastic anemia typically manifests in teens and early 20s, or in the elderly. Symptoms include:

  • Fatigue
  • Pallor (extremely pale skin)
  • Shortness of breath that worsens with exertion
  • Rapid or irregular heartbeat
  • Frequent nosebleeds or bleeding gums
  • Recurrent or lengthy infections
  • Prolonged bleeding from cuts
  • Dizziness
  • Chest pain
  • Rash
  • Fever

Learn more about aplastic anemia.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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