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ELTROMBOPAG FOR CHRONIC IMMUNE THROMBOCYTOPENIA: A SINGLE CENTER EXPERIENCE
Author(s): ,
Evgeniya Arshanskaya
Affiliations:
Hematology,City Clinical Hospital n.a. S.P. Botkin,Moscow,Russian Federation;Adolescent and Young Hematology,Federal Research Clinical Center for Pediatric n.a. D.Rogachev,Moscow,Russian Federation
,
Sergey Semochkin
Affiliations:
Oncology, Hematology and Radiotherapy,Pirogov Russian National Research Medical University,Moscow,Russian Federation
,
Valentina Ivanova
Affiliations:
Hematology,City Clinical Hospital n.a. S.P. Botkin,Moscow,Russian Federation
Vadim Ptushkin
Affiliations:
Hematology,City Clinical Hospital n.a. S.P. Botkin,Moscow,Russian Federation;Adolescent and Young Hematology,Federal Research Clinical Center for Pediatric n.a. D.Rogachev,Moscow,Russian Federation;Oncology, Hematology and Radiotherapy,Pirogov Russian Nat
(Abstract release date: 05/19/16) EHA Library. Semochkin S. 06/09/16; 134992; PB2092
Prof. Dr. Sergey Semochkin
Prof. Dr. Sergey Semochkin
Contributions
Abstract
Abstract: PB2092

Type: Publication Only

Background
Immune thrombocytopenia (ITP) is an autoimmune disorder that leads to peripheral destruction, as well as a decreased production of platelets. The incidence of ITP in adults is around 3-4 per 100000 people per year. The probability of spontaneous remission in adults is low. Up to 90% of cases become chronic, and 10% do not respond to standard therapy. Eltrombopag is a non-peptide thrombopoietin receptor agonist that has been approved for second-line treatment of ITP.

Aims
The primary aims of the study were to determine the efficacy and safety of long-term eltrombopag treatment in their own clinical practice.

Methods
Fifty adult chronic ITP patients who were refractory to previous ITP treatments (platelet count <30,000 cells/µL) were enrolled in the study in the period from October 2012 to December 2015. Treatment was started in all patients with 50 mg/d oral eltrombopag. Response was assessed at 1, 2, 3 and 12 months. The target platelet response was defined as an increase in the platelet count ≥50,000 cells/µL. The analysis is performed at the time of Februare 2016.

Results
The median age was 57.4 years (range, 18.2-87.1 years), and 35 patients were women. The median number of prior ITP treatments was 2 (range, 0-4), including 9 (18%) patients who had undergone a splenectomy and 14 (28%) - rituximab. The median ITP duration before eltrombopag treatment was 3.1 (range, 0.6-37.9) years. The median platelet count at baseline was 12,000 (range, 1,000-27,000) cells/µL. After 1 month 25 (50%) achieved the target platelet count (>50,000 cells/µL). The median platelet count reached 50,000 (range, 9,000-334,000) cells/µL. After 3 month 35 (70%) achieved the target platelet count with median platelet count is 84,000 (range, 9,000-334,000) cells/µL. After 12 month 37 (74%) achieved the target platelet count with median platelet count is 107,000 (range, 22,000-330,000) cells/µL. The current median duration of eltrombopag treatment was 14.8 (range, 1.1-40.3) months. 43 (86%) patients continued therapy, and 7 (14%) patients who have not achieved a response were excluded from the study for 2-3 months. On the likelihood of achieving a platelet response at 3 months do not affect gender, age, previous therapy (rituximab, splenectomy) and an initial platelet counts of less than or more 15,000 cells/µL (p>0.05). Twelve patients had bleeding episodes of mild to moderate severity. Thromboembolic events were not reported. Eltrombopag is a well-tolerated treatment. Most reported adverse effects have been mild–moderate and have not led to cessation of treatment.

Conclusion
Eltrombopag is well tolerated and effectively achieves target platelet counts in refractory adult chronic ITP patients.

Session topic: E-poster

Keyword(s): Idiopathic thombocytopenic purpura (ITP), Thrombopoietin (TPO), Treatment
Abstract: PB2092

Type: Publication Only

Background
Immune thrombocytopenia (ITP) is an autoimmune disorder that leads to peripheral destruction, as well as a decreased production of platelets. The incidence of ITP in adults is around 3-4 per 100000 people per year. The probability of spontaneous remission in adults is low. Up to 90% of cases become chronic, and 10% do not respond to standard therapy. Eltrombopag is a non-peptide thrombopoietin receptor agonist that has been approved for second-line treatment of ITP.

Aims
The primary aims of the study were to determine the efficacy and safety of long-term eltrombopag treatment in their own clinical practice.

Methods
Fifty adult chronic ITP patients who were refractory to previous ITP treatments (platelet count <30,000 cells/µL) were enrolled in the study in the period from October 2012 to December 2015. Treatment was started in all patients with 50 mg/d oral eltrombopag. Response was assessed at 1, 2, 3 and 12 months. The target platelet response was defined as an increase in the platelet count ≥50,000 cells/µL. The analysis is performed at the time of Februare 2016.

Results
The median age was 57.4 years (range, 18.2-87.1 years), and 35 patients were women. The median number of prior ITP treatments was 2 (range, 0-4), including 9 (18%) patients who had undergone a splenectomy and 14 (28%) - rituximab. The median ITP duration before eltrombopag treatment was 3.1 (range, 0.6-37.9) years. The median platelet count at baseline was 12,000 (range, 1,000-27,000) cells/µL. After 1 month 25 (50%) achieved the target platelet count (>50,000 cells/µL). The median platelet count reached 50,000 (range, 9,000-334,000) cells/µL. After 3 month 35 (70%) achieved the target platelet count with median platelet count is 84,000 (range, 9,000-334,000) cells/µL. After 12 month 37 (74%) achieved the target platelet count with median platelet count is 107,000 (range, 22,000-330,000) cells/µL. The current median duration of eltrombopag treatment was 14.8 (range, 1.1-40.3) months. 43 (86%) patients continued therapy, and 7 (14%) patients who have not achieved a response were excluded from the study for 2-3 months. On the likelihood of achieving a platelet response at 3 months do not affect gender, age, previous therapy (rituximab, splenectomy) and an initial platelet counts of less than or more 15,000 cells/µL (p>0.05). Twelve patients had bleeding episodes of mild to moderate severity. Thromboembolic events were not reported. Eltrombopag is a well-tolerated treatment. Most reported adverse effects have been mild–moderate and have not led to cessation of treatment.

Conclusion
Eltrombopag is well tolerated and effectively achieves target platelet counts in refractory adult chronic ITP patients.

Session topic: E-poster

Keyword(s): Idiopathic thombocytopenic purpura (ITP), Thrombopoietin (TPO), Treatment

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