Contributions
Abstract: PB1823
Type: Publication Only
Background
Aplastic anemia (AA) is characterized by immune-mediated bone marrow hypoplasia and pancytopenia, and can be effectively treated with immunosuppressive therapy or allogeneic transplantation. Nevertheless, one third of patients are refractory to immunosuppression, with persistent, severe cytopenia and transfusion requirement.
Eltrombopag (ELT) is an orally bioavailable thrombopoietin receptor (TPO-R) agonist approved for the treatment of refractory idiopathic thrombocytopenia and aplastic anemia. Additionally It was shown that ELT is also able to decrease labile iron within the cells. Since in many studies hematological response was reported during iron chelation therapy (ICT) in patients with AA, similarly, in principle, some beneficial effects of ELT on hematopoiesis could also derive from its property to remove iron”
Aims
The study was a retrospective collection of clinical data of patients with AA treated concomitantly with eltrombopag and deferasirox (DFX) with the aim of investigating the tolerability of the association and the response rate of AA patients treated in 11 Italian hematological centers
Methods
We collected 14 AA, 2 cases of MDS and 1 case of B-CLL. Four patients with AA were excluded because the follow-up was less than 2 months or because the ICT and ELT were not concomitant. The median time of ELT therapy in the 10 AA patients was 15 months. In 8 out of 10 patients DFX therapy was started before ELT. The median time from diagnosis to ELT therapy was 9 months. All the patients were heavily transfused before ELT therapy. The median number of transfusions before ELT therapy was 33.5 U of RBC and 31 U of platelets. The median serum ferritin level was 2600 ng/mL at ELT starting and 1480 ng/mL at the end of ELT and DFX treatment. All the patients gradually reached the dose of 150 mg/d of ELT and were treated with a median dose of 10 mg/kg of DFX.
Results
Seven out of 10 AA patients (70%) obtained complete hematological response with transfusion independence, one a partial erythroid response and one has a significant reduction of platelets transfusion. One patient did not obtain any benefit. The two MDS cases and the B-CLL case obtained transfusion independence. The combination was well tolerated and no unexpected side effects or significant alterations of blood chemistry occurred.
Conclusion
Although the retrospective nature of our observation and the limited number of patients included, our multicenter experience with the association of ELT and DFX results in a good rate of tolerability and a surprising rate of responses. If these data will be conformed in a larger cohort of patients, these data will pave the way to prospective trials and biological studies to investigate the eventual role of iron removal in favoring eltrombopag response
Session topic: 12. Bone marrow failure syndromes incl. PNH - Clinical
Keyword(s): Aplastic anemia, iron chelation, Thrombopoietin (TPO), transfusion
Abstract: PB1823
Type: Publication Only
Background
Aplastic anemia (AA) is characterized by immune-mediated bone marrow hypoplasia and pancytopenia, and can be effectively treated with immunosuppressive therapy or allogeneic transplantation. Nevertheless, one third of patients are refractory to immunosuppression, with persistent, severe cytopenia and transfusion requirement.
Eltrombopag (ELT) is an orally bioavailable thrombopoietin receptor (TPO-R) agonist approved for the treatment of refractory idiopathic thrombocytopenia and aplastic anemia. Additionally It was shown that ELT is also able to decrease labile iron within the cells. Since in many studies hematological response was reported during iron chelation therapy (ICT) in patients with AA, similarly, in principle, some beneficial effects of ELT on hematopoiesis could also derive from its property to remove iron”
Aims
The study was a retrospective collection of clinical data of patients with AA treated concomitantly with eltrombopag and deferasirox (DFX) with the aim of investigating the tolerability of the association and the response rate of AA patients treated in 11 Italian hematological centers
Methods
We collected 14 AA, 2 cases of MDS and 1 case of B-CLL. Four patients with AA were excluded because the follow-up was less than 2 months or because the ICT and ELT were not concomitant. The median time of ELT therapy in the 10 AA patients was 15 months. In 8 out of 10 patients DFX therapy was started before ELT. The median time from diagnosis to ELT therapy was 9 months. All the patients were heavily transfused before ELT therapy. The median number of transfusions before ELT therapy was 33.5 U of RBC and 31 U of platelets. The median serum ferritin level was 2600 ng/mL at ELT starting and 1480 ng/mL at the end of ELT and DFX treatment. All the patients gradually reached the dose of 150 mg/d of ELT and were treated with a median dose of 10 mg/kg of DFX.
Results
Seven out of 10 AA patients (70%) obtained complete hematological response with transfusion independence, one a partial erythroid response and one has a significant reduction of platelets transfusion. One patient did not obtain any benefit. The two MDS cases and the B-CLL case obtained transfusion independence. The combination was well tolerated and no unexpected side effects or significant alterations of blood chemistry occurred.
Conclusion
Although the retrospective nature of our observation and the limited number of patients included, our multicenter experience with the association of ELT and DFX results in a good rate of tolerability and a surprising rate of responses. If these data will be conformed in a larger cohort of patients, these data will pave the way to prospective trials and biological studies to investigate the eventual role of iron removal in favoring eltrombopag response
Session topic: 12. Bone marrow failure syndromes incl. PNH - Clinical
Keyword(s): Aplastic anemia, iron chelation, Thrombopoietin (TPO), transfusion