Contributions
Abstract: PB1484
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Acquired aplastic anemia results from immune-mediated destruction of haematopoietic stem cells of bonemarrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.
Aims
To evaluate the Effectivness and Safety of Eltrombopag, Added to Standard Immunosuppression in Indian patients with Acquired Aplastic Anemia.
Methods
Patients (>5 years) of either gender with Acquired Aplastic Anaemia, who had received Eltrombopag, in addition to standard Immunosuppression with ATG/Cyclosporin from Jul-2019 to Oct-2020, at Department of Clinical Hematology, Nizam's Institute of Medical Sciences, Hyderabad, India, were included. The primary outcome was complete/partial hematologic response at 6 months. Secondary end points included overall response, survival, adverse events -platelet response (platelet count: ≥50000/μL).
Results
Of 17 patients, majority (%) were females. The mean age was… years. Seven (…%) patients had severe Aplastic Anaemia, two (12.5%) had non-severe aplastic Anaemia, 6 (37.5%) had Megakaryocytic thrombocytopenia. For newly diagnosed patients, the frontline use of eltrombopag (concomitant medications: anti-thymocyte globulin, ATG, and cyclosporin, N = 4; cyclosporin, N = 5; nil, N = 1) at a median maximum dose of 150 (50–300) mg/day led to an overall response rate (ORR) of 90% (trilineage: 60%; neutrophil: 20%; platelet: 10%). Adverse effects included dyspepsia, and liver function derangement. Eltrombopag was well tolerated without significant side-effects mandating drug withdrawal.
Conclusion
In a routine haematological practice, the use of eltrombopag in AA patients was feasible, safe, and associated with favourable responses.
Keyword(s): Aplastic anemia
Abstract: PB1484
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Acquired aplastic anemia results from immune-mediated destruction of haematopoietic stem cells of bonemarrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.
Aims
To evaluate the Effectivness and Safety of Eltrombopag, Added to Standard Immunosuppression in Indian patients with Acquired Aplastic Anemia.
Methods
Patients (>5 years) of either gender with Acquired Aplastic Anaemia, who had received Eltrombopag, in addition to standard Immunosuppression with ATG/Cyclosporin from Jul-2019 to Oct-2020, at Department of Clinical Hematology, Nizam's Institute of Medical Sciences, Hyderabad, India, were included. The primary outcome was complete/partial hematologic response at 6 months. Secondary end points included overall response, survival, adverse events -platelet response (platelet count: ≥50000/μL).
Results
Of 17 patients, majority (%) were females. The mean age was… years. Seven (…%) patients had severe Aplastic Anaemia, two (12.5%) had non-severe aplastic Anaemia, 6 (37.5%) had Megakaryocytic thrombocytopenia. For newly diagnosed patients, the frontline use of eltrombopag (concomitant medications: anti-thymocyte globulin, ATG, and cyclosporin, N = 4; cyclosporin, N = 5; nil, N = 1) at a median maximum dose of 150 (50–300) mg/day led to an overall response rate (ORR) of 90% (trilineage: 60%; neutrophil: 20%; platelet: 10%). Adverse effects included dyspepsia, and liver function derangement. Eltrombopag was well tolerated without significant side-effects mandating drug withdrawal.
Conclusion
In a routine haematological practice, the use of eltrombopag in AA patients was feasible, safe, and associated with favourable responses.
Keyword(s): Aplastic anemia