Contributions
Abstract: PB1831
Type: Publication Only
Background
Eltrombopag, a peroral thrombopoietin receptor agonist, has been introduced recently as a possible novel therapeutic option for adult patients with acquired severe aplastic anemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and were unsuitable for hematopoietic stem cell transplantation (HSCT). However, it is still not known how eltrombopag is used for that indication in the real-world setting.
Aims
In this work we analyzed results of eltrombopag usage in Croatia for the treatment of patients with SAA or very severe aplastic anemia (vSAA) outside of clinical trials.
Methods
We conducted a retrospective analysis on the use of eltrombopag for SAA or vSAA patients among Croatian hematology centers.
Results
From March 2015 until February 2018 seven adult patients (4 (57%) female, median age 63 (38-79) years) with acquired aplastic anemia were treated with eltrombopag in Croatian hematology centers: 3 among them with SAA, 3 with vSAA, and 1 with PNH with SAA fenotype. All patients received eltrombopag after at least one previous line of immunosuppressive treatment, either as a monotherapy (2/7) or in combination with CyA +/- low dose methylprednisolone. Complete remission (CR) (defined as Plt > 100/mcL and Hb >100 g/L and Neutrophils > 1.5/mcL) achieved 2 patients, partial remission (PR) (defined as transfusion independence) 1 patient, minimal response (defined as some improvement in 1 or more lineage but not fulfilling the criteria of PR) 1 patient, and 3 patients were refractory to eltrombopag. Median time of eltrombopag administration was 5 (3-25) months. Median eltrombopag dose was 150 mg per day and adverse events were consistent with the known safety data of the drug. Two patients in CR are receiving eltrombopag for 25 and 14 months, respectively. One patient who achieved minimal response relapsed after 8 months of treatment and developed new cytogenetic change (trisomy 8). 85.7 % of patients are alive at a median follow up of 12 (5-32) months. Only one patient died, 8 months after discontinuation of eltrombopag therapy, with refractory vSAA, comorbidities and infections.
Conclusion
Presented data from Croatian hematology centers in the real-world setting confirm that eltrombopag is interesting new modality to treat patients with acquired SAA, especially for those who are refractory to prior immunosuppressive therapy and/or unsuitable for HSCT.
Session topic: 12. Bone marrow failure syndromes incl. PNH - Clinical
Keyword(s): Aplastic anemia, Therapy, Thrombopoietin (TPO)
Abstract: PB1831
Type: Publication Only
Background
Eltrombopag, a peroral thrombopoietin receptor agonist, has been introduced recently as a possible novel therapeutic option for adult patients with acquired severe aplastic anemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and were unsuitable for hematopoietic stem cell transplantation (HSCT). However, it is still not known how eltrombopag is used for that indication in the real-world setting.
Aims
In this work we analyzed results of eltrombopag usage in Croatia for the treatment of patients with SAA or very severe aplastic anemia (vSAA) outside of clinical trials.
Methods
We conducted a retrospective analysis on the use of eltrombopag for SAA or vSAA patients among Croatian hematology centers.
Results
From March 2015 until February 2018 seven adult patients (4 (57%) female, median age 63 (38-79) years) with acquired aplastic anemia were treated with eltrombopag in Croatian hematology centers: 3 among them with SAA, 3 with vSAA, and 1 with PNH with SAA fenotype. All patients received eltrombopag after at least one previous line of immunosuppressive treatment, either as a monotherapy (2/7) or in combination with CyA +/- low dose methylprednisolone. Complete remission (CR) (defined as Plt > 100/mcL and Hb >100 g/L and Neutrophils > 1.5/mcL) achieved 2 patients, partial remission (PR) (defined as transfusion independence) 1 patient, minimal response (defined as some improvement in 1 or more lineage but not fulfilling the criteria of PR) 1 patient, and 3 patients were refractory to eltrombopag. Median time of eltrombopag administration was 5 (3-25) months. Median eltrombopag dose was 150 mg per day and adverse events were consistent with the known safety data of the drug. Two patients in CR are receiving eltrombopag for 25 and 14 months, respectively. One patient who achieved minimal response relapsed after 8 months of treatment and developed new cytogenetic change (trisomy 8). 85.7 % of patients are alive at a median follow up of 12 (5-32) months. Only one patient died, 8 months after discontinuation of eltrombopag therapy, with refractory vSAA, comorbidities and infections.
Conclusion
Presented data from Croatian hematology centers in the real-world setting confirm that eltrombopag is interesting new modality to treat patients with acquired SAA, especially for those who are refractory to prior immunosuppressive therapy and/or unsuitable for HSCT.
Session topic: 12. Bone marrow failure syndromes incl. PNH - Clinical
Keyword(s): Aplastic anemia, Therapy, Thrombopoietin (TPO)