What is the role of triple therapy in treating Aplastic Anemia?

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From the Research

Role of Triple Therapy in Treating Aplastic Anemia

• The treatment of aplastic anemia involves supportive therapy and aplastic anemia-specific therapy to recover from hematopoiesis 1.
• Aplastic anemia-specific treatments include immunosuppressive therapy, thrombopoietin receptor agonist (TPO-RA) treatment, allogeneic hematopoietic stem cell transplantation, and anabolic hormone therapy 1.
• The most effective drug therapy is the combination of TPO-RA and immunotherapy combined with anti-thymocyte globulin and cyclosporine 1.
• Immunosuppressive therapy (IST) is the first choice for severe aplastic anemia (SAA) patients with hematopoietic stem cell transplantation (HSCT) limitation 2.
• Eltrombopag (EPAG), a small molecule thrombopoietin receptor agonist, can stimulate the proliferation of residual hematopoietic stem/progenitor cells (HSPC) and restore the bone marrow hematopoietic function of patients 2.
• The combination of IST and EPAG has been shown to have higher overall response rate (ORR) and complete response rate (CRR) compared to IST alone 2, 3.
• The addition of eltrombopag to immunosuppressive therapy was associated with markedly higher rates of hematologic response among patients with severe aplastic anemia than in a historical cohort 3.
• Triple therapy, including immunosuppressive therapy, TPO-RA, and other agents, may be effective in treating aplastic anemia, but the optimal combination and duration of therapy need to be determined 2, 3, 4.

Key Findings

• The overall response rates at 6 months were higher in patients treated with IST and EPAG compared to IST alone 2, 3.
• The complete response rates at 6 months were also higher in patients treated with IST and EPAG compared to IST alone 2, 3.
• The addition of EPAG to IST enabled patients to achieve earlier and faster hematologic responses with a higher rate of complete response 2.
• The treatment choice for aplastic anemia is made based on the disease severity and patient age 1, 5.

Treatment Outcomes

• The probability of an overall 10-year survival rate was 90% in patients treated with combined immunosuppressive therapy 4.
• The survival rate was 97% at a median follow-up of 2 years in patients treated with IST and EPAG 3.
• The rates of relapse and clonal evolution were similar to historical experience in patients treated with IST and EPAG 3.