Contributions
Abstract: PB2361
Type: Publication Only
Background
Primary immune thrombocytopenia (ITP) is an autoimmune disease in which, along with a greater destruction of platelets, an insufficient marrow production is observed. This second mechanism confirms the existence of thrombopoietin levels (TPO); which reinforces the hypothesis that thrombopoietin levels were elevated in this population group. To improve the number of platelets in ITP, a group of drugs, the thrombopoietin receptor agonists (TPO-RA), were developed. Two clinical trials led to the approval of eltrombopag (RAISE and EXTEND studies), without determining the time of treatment, as well as the possibility of temporary interruptions of those patients who reached platelet numbers in stable ranges.
Aims
To analyze if the successful suspension of the drug is possible, for those patients who reach adequate platelet counts.
Methods
Prospective study conducted between January/2017 and February/2018, in which 2 patients diagnosed with primary immune thrombocytopenia are included. We analyze the time of active treatment, as well as the period of suspension of therapy. The cut-off point of the platelet count was considered optimal was 100x109/mm3, and was defined by recommendations of clinical guidelines and patient safety and validated according to the laboratory standards of our center (Range of normality: 150x109/mm3-450x109/mm3).
Results
Two patients diagnosed with ITP on treatment with thrombopoietin analogues were included. The first case is a 75-year-old woman, with comorbidities that made difficult the treatment with corticosteroids at full doses (diabetes mellitus and recurrent infections). At diagnosis 15x109/mm3 platelets. First-line treatment with poor tolerance is started, suspending it and starting in May 2017 second-line treatment with eltrombopag. The treatment was maintained for 10 weeks at standard doses with very good tolerance and progressive rise in platelet counts, reaching stable numbers in the different analytical controls. Given the stability, it was decided to suspend treatment and close clinical and analytical follow-up, maintaining platelet numbers above 100x109/mm3 to date. The current period without treatment is 7 months. The second case is an 80-year-old woman with a diagnosis of ITP that debuted with severe thrombocytopenia and associated clinical bleeding. First-line treatment with corticosteroids was initiated at high doses and IVIGs cycle, being refractory without reaching adequate platelet counts. In August/2017, second-line treatment with thromboprotein receptor agonists (eltrombopag) was started until October 2017, with analytical and clinical stability. On that date it was decided to suspend treatment by stable platelet counts. The patient continues without active treatment and asymptomatic from the point of view of bleeding complications. The current period without treatment is 4 months.
Conclusion
Currently there is published data on the interruption of the TPO-RA, but objective data and standardized recommendations have not yet been provided. It has not been possible to identify the predictive factors related to the response and maintenance or withdrawal of the drug. These two cases demonstrate and support that not only a stable response can be achieved in patients diagnosed with ITP, but that the withdrawal of the drug, once adequate platelet ranges have been reached, is possible, but always with the need for a clinical and analytical follow-up close of these patients.
Session topic: 33. Platelets disorders
Keyword(s): Immune thrombocytopenia (ITP), Thrombopoietin (TPO)
Abstract: PB2361
Type: Publication Only
Background
Primary immune thrombocytopenia (ITP) is an autoimmune disease in which, along with a greater destruction of platelets, an insufficient marrow production is observed. This second mechanism confirms the existence of thrombopoietin levels (TPO); which reinforces the hypothesis that thrombopoietin levels were elevated in this population group. To improve the number of platelets in ITP, a group of drugs, the thrombopoietin receptor agonists (TPO-RA), were developed. Two clinical trials led to the approval of eltrombopag (RAISE and EXTEND studies), without determining the time of treatment, as well as the possibility of temporary interruptions of those patients who reached platelet numbers in stable ranges.
Aims
To analyze if the successful suspension of the drug is possible, for those patients who reach adequate platelet counts.
Methods
Prospective study conducted between January/2017 and February/2018, in which 2 patients diagnosed with primary immune thrombocytopenia are included. We analyze the time of active treatment, as well as the period of suspension of therapy. The cut-off point of the platelet count was considered optimal was 100x109/mm3, and was defined by recommendations of clinical guidelines and patient safety and validated according to the laboratory standards of our center (Range of normality: 150x109/mm3-450x109/mm3).
Results
Two patients diagnosed with ITP on treatment with thrombopoietin analogues were included. The first case is a 75-year-old woman, with comorbidities that made difficult the treatment with corticosteroids at full doses (diabetes mellitus and recurrent infections). At diagnosis 15x109/mm3 platelets. First-line treatment with poor tolerance is started, suspending it and starting in May 2017 second-line treatment with eltrombopag. The treatment was maintained for 10 weeks at standard doses with very good tolerance and progressive rise in platelet counts, reaching stable numbers in the different analytical controls. Given the stability, it was decided to suspend treatment and close clinical and analytical follow-up, maintaining platelet numbers above 100x109/mm3 to date. The current period without treatment is 7 months. The second case is an 80-year-old woman with a diagnosis of ITP that debuted with severe thrombocytopenia and associated clinical bleeding. First-line treatment with corticosteroids was initiated at high doses and IVIGs cycle, being refractory without reaching adequate platelet counts. In August/2017, second-line treatment with thromboprotein receptor agonists (eltrombopag) was started until October 2017, with analytical and clinical stability. On that date it was decided to suspend treatment by stable platelet counts. The patient continues without active treatment and asymptomatic from the point of view of bleeding complications. The current period without treatment is 4 months.
Conclusion
Currently there is published data on the interruption of the TPO-RA, but objective data and standardized recommendations have not yet been provided. It has not been possible to identify the predictive factors related to the response and maintenance or withdrawal of the drug. These two cases demonstrate and support that not only a stable response can be achieved in patients diagnosed with ITP, but that the withdrawal of the drug, once adequate platelet ranges have been reached, is possible, but always with the need for a clinical and analytical follow-up close of these patients.
Session topic: 33. Platelets disorders
Keyword(s): Immune thrombocytopenia (ITP), Thrombopoietin (TPO)