Contributions
Abstract: PB2435
Type: Publication Only
Background
Thrombocytopenia after allogeneic hematopoetic cell transplantation is a common complication. The underlying causes are often multifactorial in this patient population including impaired platelet production, increased platelet destruction and combination of these mechanisms. Eltrombopag is an oral thrombopoietin receptor agonist which interacts with the transmembrane domain of the receptor on bone marrow megakartocytes and upstream progenitor stem cells. Eltrombopag use has been suggested chronic idiopathic thrombocytopenic purpura and aplastic anemia. There are few reports eltrombopag treatment after allogeneic hematopoetic cell transplantation.
Aims
We aim to report our eltrombopag experiences with 7 pediatric patients treated with eltrombopag for severe thrombocytopenia after allogeneic hematopoetic cell transplantation at our center.
Methods
A total of 198 allogeneic hematopoetic cell transplantation were performed at Acıbadem Adana Hospital Pediatric Bone Marrow Transplantation Unit in Turkey from 2013 to 2017. Seven cases of 198 patients were treated eltrombopag for persistan thrombocytopenia after allogeneic hematopoetic cell transplantation. Bone marrow aspiration and biyopsy were performed in all patients before starting eltrombopag treatment. Medical records and treatment modalities were evaluated retrospectively.
Results
In this study, 7 patients were treated with eltrombopag, age ranging from 5 to 17 years with a average of 12 years. There were 3 patients with thalassemia major, 2 patients with acute lymphoblastic leukemia, one patients with aplastic anemia and one patient with myelodysplastic syndrome. Four patients were males, three were female. Five patients had HCT from an unrelated donor. Two patients had haploidentical transplantation from mother. Five patients had bone marrow transplantation,2 had peripheral blood stem cell transplantation. Eltrombopag was started in 6 patients with prolonged isolated thrombocytopenia and one patient with secondary failure of platelet recovery. The number of megacaryocytes was decreased in 6 patients and was within the normal range in one patient. Before starting eltrombopag treatment 7 patients were dependent on platelet transfusions weekly. Eltrombopag was started at a median 228 days after HSCT (range, 77 to 582 days) in patients with PIT. Eltrombopag were started at the dose of 25-50 mg once daily. Median duration for treatment was 177 days. No serious side effects were seen. The incidence rate of successful platelet recovery to > 50000/mikrolitre without transfusion support was 71% (5 of 7 patients).
Conclusion
Eltrombopag is currently available oral thrombopoietin receptor agonist. We showed that our experience using of eltrombopag is very effective with minimal side effects for persistent thrombocytopenia after allogeneic hematopoetic cell transplantation in pediatric patients who had depended on platelet transfusion.
Session topic: 23. Stem cell transplantation - Clinical
Keyword(s): Childhood, Stem cell transplant, Thrombocytopenia
Abstract: PB2435
Type: Publication Only
Background
Thrombocytopenia after allogeneic hematopoetic cell transplantation is a common complication. The underlying causes are often multifactorial in this patient population including impaired platelet production, increased platelet destruction and combination of these mechanisms. Eltrombopag is an oral thrombopoietin receptor agonist which interacts with the transmembrane domain of the receptor on bone marrow megakartocytes and upstream progenitor stem cells. Eltrombopag use has been suggested chronic idiopathic thrombocytopenic purpura and aplastic anemia. There are few reports eltrombopag treatment after allogeneic hematopoetic cell transplantation.
Aims
We aim to report our eltrombopag experiences with 7 pediatric patients treated with eltrombopag for severe thrombocytopenia after allogeneic hematopoetic cell transplantation at our center.
Methods
A total of 198 allogeneic hematopoetic cell transplantation were performed at Acıbadem Adana Hospital Pediatric Bone Marrow Transplantation Unit in Turkey from 2013 to 2017. Seven cases of 198 patients were treated eltrombopag for persistan thrombocytopenia after allogeneic hematopoetic cell transplantation. Bone marrow aspiration and biyopsy were performed in all patients before starting eltrombopag treatment. Medical records and treatment modalities were evaluated retrospectively.
Results
In this study, 7 patients were treated with eltrombopag, age ranging from 5 to 17 years with a average of 12 years. There were 3 patients with thalassemia major, 2 patients with acute lymphoblastic leukemia, one patients with aplastic anemia and one patient with myelodysplastic syndrome. Four patients were males, three were female. Five patients had HCT from an unrelated donor. Two patients had haploidentical transplantation from mother. Five patients had bone marrow transplantation,2 had peripheral blood stem cell transplantation. Eltrombopag was started in 6 patients with prolonged isolated thrombocytopenia and one patient with secondary failure of platelet recovery. The number of megacaryocytes was decreased in 6 patients and was within the normal range in one patient. Before starting eltrombopag treatment 7 patients were dependent on platelet transfusions weekly. Eltrombopag was started at a median 228 days after HSCT (range, 77 to 582 days) in patients with PIT. Eltrombopag were started at the dose of 25-50 mg once daily. Median duration for treatment was 177 days. No serious side effects were seen. The incidence rate of successful platelet recovery to > 50000/mikrolitre without transfusion support was 71% (5 of 7 patients).
Conclusion
Eltrombopag is currently available oral thrombopoietin receptor agonist. We showed that our experience using of eltrombopag is very effective with minimal side effects for persistent thrombocytopenia after allogeneic hematopoetic cell transplantation in pediatric patients who had depended on platelet transfusion.
Session topic: 23. Stem cell transplantation - Clinical
Keyword(s): Childhood, Stem cell transplant, Thrombocytopenia