Contributions
Abstract: EP1125
Type: E-Poster Presentation
Session title: Platelet disorders
Background
Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), has been available in Europe for the treatment of chronic primary immune thrombocytopenia (ITP) since 2010. Since 2019, it has been indicated in patients with primary ITP, lasting 6 months or longer from diagnosis and who are refractory to other treatments, aged 1 year and above. However, French ITP management guidelines published in 2017, as well as international guidelines updated in 2019 consider the use of second-line treatments earlier in the disease course, including TPO-RAs.
Aims
The ELEXTRA study has been designed to assess the use, the efficacy and the safety of eltrombopag in adult patients with ITP in the real world in France. The present study focused on patients treated with eltrombopag off label before 6 months of ITP duration.
Methods
The source of study population was the multicenter CARMEN-France registry, aimed at the prospective follow-up of adults with incident ITP in France. Patients included in the registry between 2013 and 2019 who were exposed to eltrombopag during the first 6 months of ITP course were selected. Efficacy was assessed in patients who had a platelet count <30 x 109/L at eltrombopag initiation. Overall and complete responses were defined by platelet counts ≥30 x 109/L and ≥100 x 109/L, respectively. Adverse drug reactions (ADRs) with a World Health Organization causality score at least as “possible”were described.
Results
Out of 799 patients included in the registry, 156 (19.5%) had been exposed to eltrombopag. Among them, 95 (60.9%) were treated off-label with eltrombopag during the first 6 months of ITP duration. Mean age was 60.5 years; 50 (52.6%) were men; 33 (34.7%) had at least one comorbidity of the Charlson Comorbidity Index; 56 (58.9%) had at least one cardiovascular risk factor (not including age and sex). At ITP diagnosis, the median platelet count was 7 x 109/L and 76 (80.0%) patients had bleeding. Median time from ITP onset to eltrombopag exposure was 1.6 months: 74 (77.9%) patients were exposed before 3 months and 21 (22.1%) between 3 and 6 months. Treatments before eltrombopag were: corticosteroids in 91 (95.8%) patients, intravenous immunoglobulin (IVIg) in 66 (69.5%), dapsone in 11 (11.6%), hydroxychloroquine in 6 (6.3%), rituximab in 5 (5.3%), romiplostim in 5 (5.3%), vinblastine in 5 (5.3%), danazol in 2 (2.1%); no patient was splenectomized. Eltrombopag was used as second-line and third-line agent in 41 (43.2%) and 32 (33.7%) patients, respectively. Among the 95 patients, 48 had a platelet count <30 x 109/L at eltrombopag initiation; among them, 39 (81.3%) achieved overall response and 35 (79.2%) complete response. Among the 39 patients who achieved overall response, 25 (64.1%) had a concomitant exposure to another ITP treatment at eltrombopag initiation (including steroids, IVIg in the past month or rituximab in the past 6 months). At last follow-up, 57 had stopped eltrombopag, including 17 for absence or loss of response, 15 for ADR and 9 for sustained complete response. Overall, 17 ADRs were reported in 16 (16.8%) patients: thrombocytosis (n=5), venous thrombosis (n=6: 4 pulmonary embolism, 1 deep vein thrombosis, 1 soleal vein thrombosis), rash (n=3), hepatitis, gastrointestinal discomfort, and diarrhea (n=1 each).
Conclusion
Eltrombopag was mostly used off-label before 6 months of ITP duration in the French real-world practice. Efficacy was similar to that observed in ITP lasting >6 months. The pattern of ADRs was in accordance with previous reports.
Keyword(s): Immune thrombocytopenia (ITP), Treatment
Abstract: EP1125
Type: E-Poster Presentation
Session title: Platelet disorders
Background
Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), has been available in Europe for the treatment of chronic primary immune thrombocytopenia (ITP) since 2010. Since 2019, it has been indicated in patients with primary ITP, lasting 6 months or longer from diagnosis and who are refractory to other treatments, aged 1 year and above. However, French ITP management guidelines published in 2017, as well as international guidelines updated in 2019 consider the use of second-line treatments earlier in the disease course, including TPO-RAs.
Aims
The ELEXTRA study has been designed to assess the use, the efficacy and the safety of eltrombopag in adult patients with ITP in the real world in France. The present study focused on patients treated with eltrombopag off label before 6 months of ITP duration.
Methods
The source of study population was the multicenter CARMEN-France registry, aimed at the prospective follow-up of adults with incident ITP in France. Patients included in the registry between 2013 and 2019 who were exposed to eltrombopag during the first 6 months of ITP course were selected. Efficacy was assessed in patients who had a platelet count <30 x 109/L at eltrombopag initiation. Overall and complete responses were defined by platelet counts ≥30 x 109/L and ≥100 x 109/L, respectively. Adverse drug reactions (ADRs) with a World Health Organization causality score at least as “possible”were described.
Results
Out of 799 patients included in the registry, 156 (19.5%) had been exposed to eltrombopag. Among them, 95 (60.9%) were treated off-label with eltrombopag during the first 6 months of ITP duration. Mean age was 60.5 years; 50 (52.6%) were men; 33 (34.7%) had at least one comorbidity of the Charlson Comorbidity Index; 56 (58.9%) had at least one cardiovascular risk factor (not including age and sex). At ITP diagnosis, the median platelet count was 7 x 109/L and 76 (80.0%) patients had bleeding. Median time from ITP onset to eltrombopag exposure was 1.6 months: 74 (77.9%) patients were exposed before 3 months and 21 (22.1%) between 3 and 6 months. Treatments before eltrombopag were: corticosteroids in 91 (95.8%) patients, intravenous immunoglobulin (IVIg) in 66 (69.5%), dapsone in 11 (11.6%), hydroxychloroquine in 6 (6.3%), rituximab in 5 (5.3%), romiplostim in 5 (5.3%), vinblastine in 5 (5.3%), danazol in 2 (2.1%); no patient was splenectomized. Eltrombopag was used as second-line and third-line agent in 41 (43.2%) and 32 (33.7%) patients, respectively. Among the 95 patients, 48 had a platelet count <30 x 109/L at eltrombopag initiation; among them, 39 (81.3%) achieved overall response and 35 (79.2%) complete response. Among the 39 patients who achieved overall response, 25 (64.1%) had a concomitant exposure to another ITP treatment at eltrombopag initiation (including steroids, IVIg in the past month or rituximab in the past 6 months). At last follow-up, 57 had stopped eltrombopag, including 17 for absence or loss of response, 15 for ADR and 9 for sustained complete response. Overall, 17 ADRs were reported in 16 (16.8%) patients: thrombocytosis (n=5), venous thrombosis (n=6: 4 pulmonary embolism, 1 deep vein thrombosis, 1 soleal vein thrombosis), rash (n=3), hepatitis, gastrointestinal discomfort, and diarrhea (n=1 each).
Conclusion
Eltrombopag was mostly used off-label before 6 months of ITP duration in the French real-world practice. Efficacy was similar to that observed in ITP lasting >6 months. The pattern of ADRs was in accordance with previous reports.
Keyword(s): Immune thrombocytopenia (ITP), Treatment