ELTROMBOPAG USE IN SEVERE ITP AND BEYOND, A SINGLE CENTRE COHORT
(Abstract release date: 05/19/16)
EHA Library. Taylor A. 06/09/16; 134973; PB2073
Dr. Alice Taylor
Contributions
Contributions
Abstract
Abstract: PB2073
Type: Publication Only
Background
Thrombopoietin receptor (TPO) agonists are licensed for immune thrombocytopenia purpura ITP). Potential for use in other thrombocytopenic conditions is being explored.
Aims
Our aim was to demonstrate the efficacy of eltrombopag in ITP -whether for severe disease or bridging therapy for surgery or chemotherapy. We also examined the use of eltrombopag in malignancy-associated thrombocytopenia, whether from disease or chemotherapy.
Methods
We retrospectively identified all patients who had received eltrombopag from a single institution. Patients were identified who had received eltrombopag as part of standard treatment pathways, as well as those for whom special consideration was sought (ie refractory thrombocytopenia in advanced malignancy). Eltrombopag was commenced from between March 2012 (as the first documented prescription time point) to January 2016.For ITP, we included those deemed to have a ‘severe’ phenotype according to the international consensus guidelines. All patients fulfilled these criteria, warranting second-line treatment beyond steroids and IVIg. Many had failed multiple previous lines or had comorbidities limiting options.For non-ITP thrombocytopenic patients, indication for eltrombopag was examined on a case-by-case basis, according to benefit versus risk, since prescription was off-label. The primary indication was to enable chemotherapy, based on oncology requirements for platelet counts >100 x 109/L.
Results
Our total cohort of 62 patients (36 males and 26 females) was treated over 8 years. This included 50 ITP patients requiring treatment for severe/refractory disease (n= 36), or bridging therapy (n=14). In the refractory group, 25 patients had primary ITP and 11 secondary ITP; including HIV (n=5), viral (n=2) malignancy (n=1) and other autoimmune aetiologies (n=3). Follow up was 1 to 85 months (median 12.5 months), with median 4 previous lines of therapy. Median time between ITP diagnosis and eltrombopag commencement was 24 months in the refractory ITP group. The delay was partially due to eltrombopag availability.In the overall ITP cohort (n=50), complete response (CR) was achieved in 28 patients (56%), partial response (PR) in 19 (38%) and no response (NR) in 3 patients (6%). Median time to response was 3 weeks (1 to 44 weeks). At follow up, 17 patients were in CR (34%) and 29 patients in PR (58%). Eltrombopag dose ranged from 25 to 100mg daily, with a median of 25mg (n=12). The difference in response at follow up was due to dose reduction, whilst maintaining haemostatic platelet counts.The malignancy-associated group included 12 patients. Eltrombopag use achieved CR in 6 patients (50%), PR in 3 (25%) and NR in 3 (25%).
Conclusion
In conclusion, eltrombopag use in the severe ITP setting achieved a response in 94% cases. For severe or refractory disease, dose reduction was possible once response achieved. Although not CR by strict definition, a safe platelet count could be maintained- typically >50 x 109/L- without bleeding sequelae. In the bridging cohort, eltrombopag proved a reliable means of improving platelet counts for intervention, without the problems associated with steroids or IVIg. Beyond ITP, eltrombopag may be of practical benefit during chemotherapy and other myelosuppressive treatment, to ensure optimal dosing of these therapies via platelet count support. Although our non-ITP cohort was small, CR was achieved in half of these patients. Further trials are needed to demonstrate which patients may benefit from eltrombopag during treatment for malignancy-associated thrombocytopenia.
Session topic: E-poster
Keyword(s): ITP, Thrombocytopenia, TPO
Type: Publication Only
Background
Thrombopoietin receptor (TPO) agonists are licensed for immune thrombocytopenia purpura ITP). Potential for use in other thrombocytopenic conditions is being explored.
Aims
Our aim was to demonstrate the efficacy of eltrombopag in ITP -whether for severe disease or bridging therapy for surgery or chemotherapy. We also examined the use of eltrombopag in malignancy-associated thrombocytopenia, whether from disease or chemotherapy.
Methods
We retrospectively identified all patients who had received eltrombopag from a single institution. Patients were identified who had received eltrombopag as part of standard treatment pathways, as well as those for whom special consideration was sought (ie refractory thrombocytopenia in advanced malignancy). Eltrombopag was commenced from between March 2012 (as the first documented prescription time point) to January 2016.For ITP, we included those deemed to have a ‘severe’ phenotype according to the international consensus guidelines. All patients fulfilled these criteria, warranting second-line treatment beyond steroids and IVIg. Many had failed multiple previous lines or had comorbidities limiting options.For non-ITP thrombocytopenic patients, indication for eltrombopag was examined on a case-by-case basis, according to benefit versus risk, since prescription was off-label. The primary indication was to enable chemotherapy, based on oncology requirements for platelet counts >100 x 109/L.
Results
Our total cohort of 62 patients (36 males and 26 females) was treated over 8 years. This included 50 ITP patients requiring treatment for severe/refractory disease (n= 36), or bridging therapy (n=14). In the refractory group, 25 patients had primary ITP and 11 secondary ITP; including HIV (n=5), viral (n=2) malignancy (n=1) and other autoimmune aetiologies (n=3). Follow up was 1 to 85 months (median 12.5 months), with median 4 previous lines of therapy. Median time between ITP diagnosis and eltrombopag commencement was 24 months in the refractory ITP group. The delay was partially due to eltrombopag availability.In the overall ITP cohort (n=50), complete response (CR) was achieved in 28 patients (56%), partial response (PR) in 19 (38%) and no response (NR) in 3 patients (6%). Median time to response was 3 weeks (1 to 44 weeks). At follow up, 17 patients were in CR (34%) and 29 patients in PR (58%). Eltrombopag dose ranged from 25 to 100mg daily, with a median of 25mg (n=12). The difference in response at follow up was due to dose reduction, whilst maintaining haemostatic platelet counts.The malignancy-associated group included 12 patients. Eltrombopag use achieved CR in 6 patients (50%), PR in 3 (25%) and NR in 3 (25%).
Conclusion
In conclusion, eltrombopag use in the severe ITP setting achieved a response in 94% cases. For severe or refractory disease, dose reduction was possible once response achieved. Although not CR by strict definition, a safe platelet count could be maintained- typically >50 x 109/L- without bleeding sequelae. In the bridging cohort, eltrombopag proved a reliable means of improving platelet counts for intervention, without the problems associated with steroids or IVIg. Beyond ITP, eltrombopag may be of practical benefit during chemotherapy and other myelosuppressive treatment, to ensure optimal dosing of these therapies via platelet count support. Although our non-ITP cohort was small, CR was achieved in half of these patients. Further trials are needed to demonstrate which patients may benefit from eltrombopag during treatment for malignancy-associated thrombocytopenia.
Session topic: E-poster
Keyword(s): ITP, Thrombocytopenia, TPO
Abstract: PB2073
Type: Publication Only
Background
Thrombopoietin receptor (TPO) agonists are licensed for immune thrombocytopenia purpura ITP). Potential for use in other thrombocytopenic conditions is being explored.
Aims
Our aim was to demonstrate the efficacy of eltrombopag in ITP -whether for severe disease or bridging therapy for surgery or chemotherapy. We also examined the use of eltrombopag in malignancy-associated thrombocytopenia, whether from disease or chemotherapy.
Methods
We retrospectively identified all patients who had received eltrombopag from a single institution. Patients were identified who had received eltrombopag as part of standard treatment pathways, as well as those for whom special consideration was sought (ie refractory thrombocytopenia in advanced malignancy). Eltrombopag was commenced from between March 2012 (as the first documented prescription time point) to January 2016.For ITP, we included those deemed to have a ‘severe’ phenotype according to the international consensus guidelines. All patients fulfilled these criteria, warranting second-line treatment beyond steroids and IVIg. Many had failed multiple previous lines or had comorbidities limiting options.For non-ITP thrombocytopenic patients, indication for eltrombopag was examined on a case-by-case basis, according to benefit versus risk, since prescription was off-label. The primary indication was to enable chemotherapy, based on oncology requirements for platelet counts >100 x 109/L.
Results
Our total cohort of 62 patients (36 males and 26 females) was treated over 8 years. This included 50 ITP patients requiring treatment for severe/refractory disease (n= 36), or bridging therapy (n=14). In the refractory group, 25 patients had primary ITP and 11 secondary ITP; including HIV (n=5), viral (n=2) malignancy (n=1) and other autoimmune aetiologies (n=3). Follow up was 1 to 85 months (median 12.5 months), with median 4 previous lines of therapy. Median time between ITP diagnosis and eltrombopag commencement was 24 months in the refractory ITP group. The delay was partially due to eltrombopag availability.In the overall ITP cohort (n=50), complete response (CR) was achieved in 28 patients (56%), partial response (PR) in 19 (38%) and no response (NR) in 3 patients (6%). Median time to response was 3 weeks (1 to 44 weeks). At follow up, 17 patients were in CR (34%) and 29 patients in PR (58%). Eltrombopag dose ranged from 25 to 100mg daily, with a median of 25mg (n=12). The difference in response at follow up was due to dose reduction, whilst maintaining haemostatic platelet counts.The malignancy-associated group included 12 patients. Eltrombopag use achieved CR in 6 patients (50%), PR in 3 (25%) and NR in 3 (25%).
Conclusion
In conclusion, eltrombopag use in the severe ITP setting achieved a response in 94% cases. For severe or refractory disease, dose reduction was possible once response achieved. Although not CR by strict definition, a safe platelet count could be maintained- typically >50 x 109/L- without bleeding sequelae. In the bridging cohort, eltrombopag proved a reliable means of improving platelet counts for intervention, without the problems associated with steroids or IVIg. Beyond ITP, eltrombopag may be of practical benefit during chemotherapy and other myelosuppressive treatment, to ensure optimal dosing of these therapies via platelet count support. Although our non-ITP cohort was small, CR was achieved in half of these patients. Further trials are needed to demonstrate which patients may benefit from eltrombopag during treatment for malignancy-associated thrombocytopenia.
Session topic: E-poster
Keyword(s): ITP, Thrombocytopenia, TPO
Type: Publication Only
Background
Thrombopoietin receptor (TPO) agonists are licensed for immune thrombocytopenia purpura ITP). Potential for use in other thrombocytopenic conditions is being explored.
Aims
Our aim was to demonstrate the efficacy of eltrombopag in ITP -whether for severe disease or bridging therapy for surgery or chemotherapy. We also examined the use of eltrombopag in malignancy-associated thrombocytopenia, whether from disease or chemotherapy.
Methods
We retrospectively identified all patients who had received eltrombopag from a single institution. Patients were identified who had received eltrombopag as part of standard treatment pathways, as well as those for whom special consideration was sought (ie refractory thrombocytopenia in advanced malignancy). Eltrombopag was commenced from between March 2012 (as the first documented prescription time point) to January 2016.For ITP, we included those deemed to have a ‘severe’ phenotype according to the international consensus guidelines. All patients fulfilled these criteria, warranting second-line treatment beyond steroids and IVIg. Many had failed multiple previous lines or had comorbidities limiting options.For non-ITP thrombocytopenic patients, indication for eltrombopag was examined on a case-by-case basis, according to benefit versus risk, since prescription was off-label. The primary indication was to enable chemotherapy, based on oncology requirements for platelet counts >100 x 109/L.
Results
Our total cohort of 62 patients (36 males and 26 females) was treated over 8 years. This included 50 ITP patients requiring treatment for severe/refractory disease (n= 36), or bridging therapy (n=14). In the refractory group, 25 patients had primary ITP and 11 secondary ITP; including HIV (n=5), viral (n=2) malignancy (n=1) and other autoimmune aetiologies (n=3). Follow up was 1 to 85 months (median 12.5 months), with median 4 previous lines of therapy. Median time between ITP diagnosis and eltrombopag commencement was 24 months in the refractory ITP group. The delay was partially due to eltrombopag availability.In the overall ITP cohort (n=50), complete response (CR) was achieved in 28 patients (56%), partial response (PR) in 19 (38%) and no response (NR) in 3 patients (6%). Median time to response was 3 weeks (1 to 44 weeks). At follow up, 17 patients were in CR (34%) and 29 patients in PR (58%). Eltrombopag dose ranged from 25 to 100mg daily, with a median of 25mg (n=12). The difference in response at follow up was due to dose reduction, whilst maintaining haemostatic platelet counts.The malignancy-associated group included 12 patients. Eltrombopag use achieved CR in 6 patients (50%), PR in 3 (25%) and NR in 3 (25%).
Conclusion
In conclusion, eltrombopag use in the severe ITP setting achieved a response in 94% cases. For severe or refractory disease, dose reduction was possible once response achieved. Although not CR by strict definition, a safe platelet count could be maintained- typically >50 x 109/L- without bleeding sequelae. In the bridging cohort, eltrombopag proved a reliable means of improving platelet counts for intervention, without the problems associated with steroids or IVIg. Beyond ITP, eltrombopag may be of practical benefit during chemotherapy and other myelosuppressive treatment, to ensure optimal dosing of these therapies via platelet count support. Although our non-ITP cohort was small, CR was achieved in half of these patients. Further trials are needed to demonstrate which patients may benefit from eltrombopag during treatment for malignancy-associated thrombocytopenia.
Session topic: E-poster
Keyword(s): ITP, Thrombocytopenia, TPO
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