How Eltrombopag Can Change Thrombocytopenia Treatment

On November 20, 2008, the U. S. Food and Drug Administration (FDA) approved eltrombopag tablets (Promacta ®, GlaxoSmithKline Inc.) for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Eltrombopag is an orally-administered thrombopoietin receptor agonist that stimulates bone marrow megakaryocytes to produce platelets. Data supporting this indication were derived from a controlled clinical study of patients treated over a six week period, and a single-arm extension study treating patients for multiple months.

Further studies for long-term efficacy and safety have since been performed.  In September 2010, NICE (The National Institute for Health and Clinical Excellence, the drug efficacy and certifying agency for England and Wales) decided to “not recommend” the drug for the above indications, citing high costs as the primary reason.  The drug now reaches Indian shores being marketed as RevoladeTM.

We take the issue to leading Hematology experts for their views.

Q.1. How important is decreased platelet production in the etiopathogenesis of Chronic ITP, a disease traditionally thought to be one of primarily increased platelet destruction?

Decreased production is an important mechanism of aetiopathogenesis and well-validated in the experimental model.

Q.2. How robust is the data for the efficacy of this drug in Chronic ITP?

The data is robust ONLY in terms of rise in platelet count to the drug.

Q.3. What is its safety and efficacy in children?

Data in children is not available yet, Presently, it isn’t recommended for use by the company. Doctors could, however, use it on compassionate grounds.

Q.4. Could this drug become an alternative to “permanent” actions like splenectomy, or more toxic or manipulative measures like immunomodulation or immunosuppression, considering its more biological basis of action?

Yes, Eltrombopag may be a safer alternative to splenectomy (which cannot be undone). However, the limitation here would be the cost. Issues that will be required to answer are:

  • When to start and stop the treatment?
  • What target platelet counts to achieve?
Q.5. What will be the impact of “cost-factor” in the use of this drug in ITP management in India?

Undeniably, it will be the most important factor for patients in India. Fortunately, we would require a lower dose, i.e. 25 mg OD, in adults.

Q.6. Do you see this drug as a “game- changer” in ITP management, or as an “also-ran”?

Yes, it will be a “game-changer” and will have an impact in second line therapy. It will definitely be used in third line therapy in chronic ITP in adults. It will also impact the decision for splenectomy as in a shared decision-making process, many patients would prefer a non-surgical option. In children, it will definitely impact the decision for splenectomy but, more data is needed.

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Surya group of hospitals are a pioneer in modern healthcare services for women and children since 1985. We offer cutting-edge facilities along with a staff that’s highly acclaimed in their field. To book an appointment in Mumbai with Dr. Mukesh Desai or call 022-6153-8989.

Dr. Mukesh Desai

Dr. Mukesh Desai is a Paediatric Haematology, Oncology & Immunology specialist at Surya Mother and Child Super Speciality Hospital. He holds an MBBS, MD, DCH (HAEMATOLOGY-ONCOLOGY). He is one of the top Paeadiatric haematology doctors in Mumbai.

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