NOVEL PERSPECTIVES IN PATIENTS WITH REFRACTORY CHRONIC IMMUNE THROMBOCYTOPENIA FOLLOWING ELTROMBOPAG TREATMENT
(Abstract release date: 05/19/16)
EHA Library. Kaliou M. 06/09/16; 134989; PB2089
Mrs. Maria Kaliou
Contributions
Contributions
Abstract
Abstract: PB2089
Type: Publication Only
Background
Primary immune thrombocytopenia (ITP) is characterized by an antibody-mediated accelerated destruction and inadequate production of platelets. Therapeutic strategies in ITP aim not only to increase and maintain platelet counts in safe levels without bleeding episodes but also to improve quality of life in this chronically ill patient population. Eltrombopag is the first oral thrombopoietin agonist approved for patients with ITP refractory to at least one line of treatment.
Aims
The present study aims to evaluate the safety and efficacy of etrombopag in clinical practice.
Methods
Eltrombopag was administered in 13 patients, 5 male: 8 female with a median age of 46 years (range 19-48) for a median 6.4 months (0.5-32.4). Patients had received 1 to 7 lines of treatment (median=1), including corticosteroids (13 patients), immunoglobulins (4 patients), rituximab (1 patient), vincristine (1 patient), cyclosporine (1 patient), romiplostim (1 patient), danazol (1 patient) and splenectomy (1 patient). In accordance with previous clinical trials, complete response to treatment was defined as a platelet count of ≥100x109/L.
Results
At initiation of eltrombopag treatment, the majority of patients (7/13) showed thrombocytopenia (WHO grade 4, <25x109/L). Initial dose was 50 mg, increased to 75 mg daily in 3 patients for optimal treatment. In 10/13 patients eltrombopag was administered in combination with corticosteroids that were gradually tapered by the fifth week of eltrombopag administration. Median platelet value by the second week of administration was 125x109/L (5-450x109/L); whereas, by the fourth week platelets increased to 185x109/L (16-500x109/L). At the end of follow-up, median platelet count was 132x109/L (60-400x109/L). All patients achieved complete response to treatment (platelet count≥100x109/L), except for one patient with a platelet count of 60x109/L at the end of follow-up. Regarding adverse events, one patient developed grade 2 hepatobiliary abnormalities and one patient grade 1 hemolytic anemia. Both adverse events resolved when the drug was temporarily discontinued. Only one patient switched to another treatment due to pulmonary embolism during the first month of treatment.
Conclusion
In the daily clinical practice eltrombopag is safe, well tolerated and highly effective in maintaining a safe platelet count when administered in ITP patients refractory to at least one line of treatment. Eltrombopag role in other subgroups of ITP patients remains to be investigated. In addition, larger studies with longer follow-up are needed to determine the incidence, predisposing factors and prophylactic measures to prevent severe adverse events.
Session topic: E-poster
Keyword(s): Immune thrombocytopenia (ITP), Thrombopoietin (TPO)
Type: Publication Only
Background
Primary immune thrombocytopenia (ITP) is characterized by an antibody-mediated accelerated destruction and inadequate production of platelets. Therapeutic strategies in ITP aim not only to increase and maintain platelet counts in safe levels without bleeding episodes but also to improve quality of life in this chronically ill patient population. Eltrombopag is the first oral thrombopoietin agonist approved for patients with ITP refractory to at least one line of treatment.
Aims
The present study aims to evaluate the safety and efficacy of etrombopag in clinical practice.
Methods
Eltrombopag was administered in 13 patients, 5 male: 8 female with a median age of 46 years (range 19-48) for a median 6.4 months (0.5-32.4). Patients had received 1 to 7 lines of treatment (median=1), including corticosteroids (13 patients), immunoglobulins (4 patients), rituximab (1 patient), vincristine (1 patient), cyclosporine (1 patient), romiplostim (1 patient), danazol (1 patient) and splenectomy (1 patient). In accordance with previous clinical trials, complete response to treatment was defined as a platelet count of ≥100x109/L.
Results
At initiation of eltrombopag treatment, the majority of patients (7/13) showed thrombocytopenia (WHO grade 4, <25x109/L). Initial dose was 50 mg, increased to 75 mg daily in 3 patients for optimal treatment. In 10/13 patients eltrombopag was administered in combination with corticosteroids that were gradually tapered by the fifth week of eltrombopag administration. Median platelet value by the second week of administration was 125x109/L (5-450x109/L); whereas, by the fourth week platelets increased to 185x109/L (16-500x109/L). At the end of follow-up, median platelet count was 132x109/L (60-400x109/L). All patients achieved complete response to treatment (platelet count≥100x109/L), except for one patient with a platelet count of 60x109/L at the end of follow-up. Regarding adverse events, one patient developed grade 2 hepatobiliary abnormalities and one patient grade 1 hemolytic anemia. Both adverse events resolved when the drug was temporarily discontinued. Only one patient switched to another treatment due to pulmonary embolism during the first month of treatment.
Conclusion
In the daily clinical practice eltrombopag is safe, well tolerated and highly effective in maintaining a safe platelet count when administered in ITP patients refractory to at least one line of treatment. Eltrombopag role in other subgroups of ITP patients remains to be investigated. In addition, larger studies with longer follow-up are needed to determine the incidence, predisposing factors and prophylactic measures to prevent severe adverse events.
Session topic: E-poster
Keyword(s): Immune thrombocytopenia (ITP), Thrombopoietin (TPO)
Abstract: PB2089
Type: Publication Only
Background
Primary immune thrombocytopenia (ITP) is characterized by an antibody-mediated accelerated destruction and inadequate production of platelets. Therapeutic strategies in ITP aim not only to increase and maintain platelet counts in safe levels without bleeding episodes but also to improve quality of life in this chronically ill patient population. Eltrombopag is the first oral thrombopoietin agonist approved for patients with ITP refractory to at least one line of treatment.
Aims
The present study aims to evaluate the safety and efficacy of etrombopag in clinical practice.
Methods
Eltrombopag was administered in 13 patients, 5 male: 8 female with a median age of 46 years (range 19-48) for a median 6.4 months (0.5-32.4). Patients had received 1 to 7 lines of treatment (median=1), including corticosteroids (13 patients), immunoglobulins (4 patients), rituximab (1 patient), vincristine (1 patient), cyclosporine (1 patient), romiplostim (1 patient), danazol (1 patient) and splenectomy (1 patient). In accordance with previous clinical trials, complete response to treatment was defined as a platelet count of ≥100x109/L.
Results
At initiation of eltrombopag treatment, the majority of patients (7/13) showed thrombocytopenia (WHO grade 4, <25x109/L). Initial dose was 50 mg, increased to 75 mg daily in 3 patients for optimal treatment. In 10/13 patients eltrombopag was administered in combination with corticosteroids that were gradually tapered by the fifth week of eltrombopag administration. Median platelet value by the second week of administration was 125x109/L (5-450x109/L); whereas, by the fourth week platelets increased to 185x109/L (16-500x109/L). At the end of follow-up, median platelet count was 132x109/L (60-400x109/L). All patients achieved complete response to treatment (platelet count≥100x109/L), except for one patient with a platelet count of 60x109/L at the end of follow-up. Regarding adverse events, one patient developed grade 2 hepatobiliary abnormalities and one patient grade 1 hemolytic anemia. Both adverse events resolved when the drug was temporarily discontinued. Only one patient switched to another treatment due to pulmonary embolism during the first month of treatment.
Conclusion
In the daily clinical practice eltrombopag is safe, well tolerated and highly effective in maintaining a safe platelet count when administered in ITP patients refractory to at least one line of treatment. Eltrombopag role in other subgroups of ITP patients remains to be investigated. In addition, larger studies with longer follow-up are needed to determine the incidence, predisposing factors and prophylactic measures to prevent severe adverse events.
Session topic: E-poster
Keyword(s): Immune thrombocytopenia (ITP), Thrombopoietin (TPO)
Type: Publication Only
Background
Primary immune thrombocytopenia (ITP) is characterized by an antibody-mediated accelerated destruction and inadequate production of platelets. Therapeutic strategies in ITP aim not only to increase and maintain platelet counts in safe levels without bleeding episodes but also to improve quality of life in this chronically ill patient population. Eltrombopag is the first oral thrombopoietin agonist approved for patients with ITP refractory to at least one line of treatment.
Aims
The present study aims to evaluate the safety and efficacy of etrombopag in clinical practice.
Methods
Eltrombopag was administered in 13 patients, 5 male: 8 female with a median age of 46 years (range 19-48) for a median 6.4 months (0.5-32.4). Patients had received 1 to 7 lines of treatment (median=1), including corticosteroids (13 patients), immunoglobulins (4 patients), rituximab (1 patient), vincristine (1 patient), cyclosporine (1 patient), romiplostim (1 patient), danazol (1 patient) and splenectomy (1 patient). In accordance with previous clinical trials, complete response to treatment was defined as a platelet count of ≥100x109/L.
Results
At initiation of eltrombopag treatment, the majority of patients (7/13) showed thrombocytopenia (WHO grade 4, <25x109/L). Initial dose was 50 mg, increased to 75 mg daily in 3 patients for optimal treatment. In 10/13 patients eltrombopag was administered in combination with corticosteroids that were gradually tapered by the fifth week of eltrombopag administration. Median platelet value by the second week of administration was 125x109/L (5-450x109/L); whereas, by the fourth week platelets increased to 185x109/L (16-500x109/L). At the end of follow-up, median platelet count was 132x109/L (60-400x109/L). All patients achieved complete response to treatment (platelet count≥100x109/L), except for one patient with a platelet count of 60x109/L at the end of follow-up. Regarding adverse events, one patient developed grade 2 hepatobiliary abnormalities and one patient grade 1 hemolytic anemia. Both adverse events resolved when the drug was temporarily discontinued. Only one patient switched to another treatment due to pulmonary embolism during the first month of treatment.
Conclusion
In the daily clinical practice eltrombopag is safe, well tolerated and highly effective in maintaining a safe platelet count when administered in ITP patients refractory to at least one line of treatment. Eltrombopag role in other subgroups of ITP patients remains to be investigated. In addition, larger studies with longer follow-up are needed to determine the incidence, predisposing factors and prophylactic measures to prevent severe adverse events.
Session topic: E-poster
Keyword(s): Immune thrombocytopenia (ITP), Thrombopoietin (TPO)
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