Eltrombopag – A Cost-Effective Single Oral Therapeutic Agent Avoiding Splenectomy in Steroid-Resistant Immune Thrombocytopenic Purpura
Introduction
Immune Thrombocytopenic Purpura (ITP) is an autoimmune disorder with isolated thrombocytopenia marked by peripheral blood platelet count <100 × 109 /L without any other etiology or disorders that may be associated with thrombocytopenia. The treatment of newly diagnosed ITP patients is steroids. However, about one-third of patients do not respond to steroids. In such cases, intravenous immunoglobulin or splenectomy is indicated. Apart from the evidence of platelet destruction in ITP, certain studies have shown decreased platelet production along with destruction. Eltrombopag is an oral Thrombopoietin Receptor Agonist (TPO-RA). Eltrombopag has been shown to provide a good response in chronic ITP patients not responding to steroid therapy.
Case Report
A 63-year-old hypothyroid, hypertensive, and diabetic female presented with a history of bruising of skin in multiple sites. She denied any history of hematuria, melena, epistaxis, and bleeding gums. There was no history of fever, weight loss, and trauma. General examination showed multiple echymotic patches over the trunk and extremities. System examinations were within the normal limits and did not show any hepatosplenomegaly and generalized lymph node enlargement. Investigations revealed a low platelet count of 8 × 109 /L [Normal range 150 × 109 /L - 450 × 109 /L] with normal white cell count and haemoglobin. Liver functions, renal functions, and coagulation profile were normal. Thus we excluded the possibilities of leukemia, aplastic anaemia, systemic lupus erythematosus, and sepsis.
Isolated thrombocytopenia suggested the possibility of immune thrombocytopenic purpura. This was confirmed by bone marrow biopsy, which demonstrated normal megakaryocytes, whereas peripheral blood smear showed decreased platelet count which was typical of ITP.
The patient was treated with platelet transfusion, intravenous, and oral steroids to prevent dreaded complications like intracranial haemorrhage. A transient increase in platelet count was noticed but failed to maintain the optimum platelet count. Immunotherapy was initiated with azathioprine along with steroids. But the platelet count remained sub-optimal and the patient developed cutaneous symptoms. Our patient did not respond to corticosteroids and immunosuppression. So we considered TPO-RA (Eltrombopag). It resulted in a drastic increase in platelet count and the patient was free of symptoms. Hence, Eltrombopag was continued and platelet count elevated up to 300 × 109 /L. She is on our follow-up for 1 year without worsening of symptoms.
Discussion
Immune Thrombocytopenic Purpura (ITP) is a diagnosis of exclusion. There are no specific clinical markers or laboratory investigations to establish the diagnosis of ITP with accuracy. Clinically, ITP has an increased risk of bleeding. If ITP is diagnosed within 3 months of its presentation, then it is said to be ‘‘newly diagnosed’’ [1]. Chronic ITP is diagnosed if symptoms last for more than 12 months [2]. Eltrombopag is an oral Thrombopoietin Receptor Agonist (TPORA) and has been shown to provide a good response in chronic ITP patients not responding to steroid therapy as it increases the platelet counts significantly in patients with chronic ITP and reduces the morbidity and mortality associated with the ITP [3,4].