Research Article
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Use of Eltrombopag in Patients with Chronic Idiopathic Thrombocytopenic Purpura: A Single Center Experience

Year 2025, Volume: 51 Issue: 1, 59 - 65, 27.05.2025
https://doi.org/10.32708/uutfd.1635651

Abstract

This study aimed to evaluate the efficacy and safety of eltrombopag in the outpatient clinic of the Department of Hematology, Uludag University Faculty of Medicine. Treatment responses and demographic characteristics of 25 patients diagnosed with refractory chronic immune thrombocytopenia (ITP) and receiving eltrombopag between January 2011 and March 2014 were analyzed retrospectively. Both platelet ≥30,000/mm³ and ≥50,000/mm³ values were considered as responses. Prolonged response was defined as the maintenance of platelet 50,000/mm³ values for 12 weeks or longer without the need for any additional treatment, and sustained response was defined as the maintenance of platelet 50,000/mm³ and at least 2 times the baseline values without the need for additional treatment. Twenty-five patients were evaluated. Ten patients (40%) had an initial platelet value of <15,000/mm³, and 15 (60%) had ≥15,000/mm³. The baseline median platelet value increased from 15,700/mm³ to 30,000/mm³ at week 2 and to 51,000/mm³ at week 6, and this level was maintained throughout the 85-week observation period. Thirteen (52%) patients had a sustained response, with a median follow-up of 18 months. In the study, participants were grouped according to splenectomy status, platelet <15,000/mm³, and gender, and there was no statistically significant difference in response. Eltrombopag is an effective and well-tolerated therapeutic agent in patients with chronic ITP refractory to other therapies and at increased bleeding risk. Responsiveness is independent of splenectomy, concomitant therapy, or baseline platelet values.

References

  • 1. Pruemer J. Epidemiology, pathophysiology, and initial management of chronic immune thrombocytopenic purpura. Am J Health Syst Pharm. 2009;66(2 Suppl 2):S4-S10.
  • 2. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115(2):168-186.
  • 3. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866.
  • 4. Demir AM, Ümit EG, Ar MC, et al. Management of Adult Primary Immune Thrombocytopenia: Delphi-Based Consensus Recommendations. Turk J Haematol. 2024 May 30;41(2):97-104..
  • 5. Neunert CE, Arnold DM, Grace RF, Kuhne T, McCrae KR, Terrell DR. The 2022 review of the 2019 American Society of Hematology guidelines on immune thrombocytopenia. Blood Adv. 2024 Jul 9;8(13):3578-3582.
  • 6. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817.
  • 7. González-López TJ, Provan D. Proposal for a New Protocol for the Management of Immune Thrombocytopenia (ITP). Adv Ther. 2022 Jun;39(6):2287-2291.
  • 8. Garnock-Jones KP, Keam SJ. Eltrombopag. Drugs. 2009;69(5):567-576.
  • 9. Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomized, double-blind, placebo-controlled trial. Lancet. 2009;373(9664): 641-648.
  • 10. Cheng G, Saleh MN, Marcher C, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomized, phase 3 study. Lancet. 2011;377(9763):393-402.
  • 11. Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536.
  • 12. Bussel JB, Saleh MN, Vasey SY, et al. Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP). Br J Haematol. 2013 Feb;160(4):538-546.
  • 13. Tomiyama Y, Miyakawa Y, Okamoto S, et al. A lower starting dose of eltrombopag is efficacious in Japanese patients with previously treated chronic immune thrombocytopenia. J Thromb Haemost. 2012 May;10(5):799-806.
  • 14. Yoshida M, Kanashima H, Nakao T, et al. Retrospective analysis of eltrombopag for the treatment of refractory primary immune thrombocytopenia in Japan. Rinsho Ketsueki. 2013 May;54(5):444-450.
  • 15. Katsutani S, Tomiyama Y, Kimura A, et al. Oral eltrombopag for up to three years is safe and well-tolerated in Japanese patients with previously treated chronic immune thrombocytopenia: an open-label, extension study. Int J Hematol. 2013 Sep;98(3):323-330.
  • 16. Tripathi AK, Shukla A, Mishra S, et al. Eltrombopag therapy in newly diagnosed steroid non-responsive ITP patients. Int J Hematol. 2014 Apr;99(4):413-417.
  • 17. Gardellini A, Guidotti F, Feltri M, et al. Eltrombopag as second line treatment in patients with primary immune thrombocytopenia: A single center real life experience. Blood Cells Mol Dis. 2021 Dec;92:102620.
  • 18. Rosenberg A, Cashion C, Ali F, et al. Treatment of immune thrombocytopenia in Australian adults: A multicenter retrospective observational study. Res Pract Thromb Haemost. 2022 Sep 18;6(6):e12792.
  • 19. Eser A, Toptaş T, Tanrıkulu F, et al. Marmara University eltrombopag experience in chronic ITP patients. XXXIXth National Hematology Congress Book of Abstracts 2013;271-272.
  • 20. Özdemirkıran F, Payzın B, Kiper H, et al. Eltrombopag in the treatment of refractory chronic immune thrombocytopenic purpura: Aegean region experience. XXXIXth National Hematology Congress Book of Abstracts 2013;67-68.
  • 21. Çekdemir D, Hindilerden F, Güvenç S, et al. Use of eltrombopag in immune thrombocytopenia in Turkey: Experience of eleven centers. XXXIXth National Hematology Congress Book of Abstracts 2013;9.
  • 22. Dogan EE, Turan Erkek E, Elverdi T, et al. Eltrombopag in the Treatment of Immune Thrombocytopenia: Two-Center Experience from Istanbul. Indian J Hematol Blood Transfus. 2022 Apr;38(2):327-332.
  • 23. Pektaş G, Uncu İA, Dere Y, et al. Retrospective Evaluation of Survival and Prognostic Factors in Immune Thrombocytopenia: A Single-Center and Cross-Sectional Study. Medicina (Kaunas). 2024 Jul 17;60(7):1153.
  • 24. González-López TJ, Sánchez-González B, Pascual C, et al. Sustained response after discontinuation of short-and medium-term treatment with eltrombopag in patients with immune thrombocytopenia. Platelets. 2014 Feb 5. [Epub ahead of print]
  • 25. Mahévas M, Fain O, Ebbo M, et al. The temporary use of thrombopoietin-receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia. Results of a French observational study. Br J Haematol. 2014 Jun;165(6):865-869.
  • 26. Mishra K, Pramanik S, Jandial A, et al. Real-world experience of eltrombopag in immune thrombocytopenia. Am J Blood Res. 2020 Oct 15;10(5):240-251.
  • 27. Cooper N, Ghanima W, Vianelli N, et al. Sustained response off-treatment in eltrombopag-treated adult patients with ITP who are refractory or relapsed after first-line steroids: Primary, final, and ad-hoc analyses of the Phase II TAPER trial. Am J Hematol. 2024 Jan;99(1):57-67.
  • 28.Cooper N, Scully M, Percy C, et al. Real-world use ofthrombopoietin receptor agonists for the management of immune thrombocytopenia in adult patients in the United Kingdom: Results from the TRAIT study. Br J Haematol. 2024 Jun;204(6):2442-2452.

Kronik İdiyopatik Trombositopenik Purpura Tanılı Hastalarda Eltrombopag Kullanımı: Tek Merkez Deneyimi

Year 2025, Volume: 51 Issue: 1, 59 - 65, 27.05.2025
https://doi.org/10.32708/uutfd.1635651

Abstract

Bu çalışmada, Bursa Uludağ Üniversitesi Tıp Fakültesi Hematoloji Bilim Dalı polikliniğine başvuran hastalarda eltrombopag tedavisinin etkinliği ve güvenilirliğini değerlendirmeyi amaçladık. Ocak 2011 ile Mart 2014 tarihleri arasında refrakter kronik immün trombositopeni (ITP) tanısı konan ve eltrombopag alan toplam 25 hastanın tedavi yanıtları ve demografik özellikleri retrospektif olarak analiz edildi. Hem trombosit ≥30.000/mm3 hem de ≥50.000/mm3 değerleri yanıt olarak kabul edilmiştir. Uzamış yanıt, trombosit 50.000/mm3 değerlerinin herhangi bir ek tedaviye ihtiyaç duyulmadan 12 hafta veya daha uzun süre korunması ve sürekli yanıt, trombosit 50.000/mm3 ve başlangıç değerlerinin en az 2 katının ek tedaviye ihtiyaç duyulmadan korunması olarak tanımlandı. Yirmi beş hasta değerlendirildi. Başlangıç median trombosit değerinin 15,700 /mm3’den 2. haftada 30,000/mm3’e, 6. haftada 51,000/mm3’e yükseldiği ve 85 haftalık gözlem periyodu süresince bu düzeyin korunduğu görüldü. Hastaların 13’ünün (%52) devam eden yanıta sahip olduğu; median izlem sürelerinin 18 ay, eltrombopag kullanım sürelerinin ise 11 ay olduğu görüldü. Çalışmada katılımcılar splenektomi durumuna, trombosit <15,000/mm3 olup olmamasına ve cinsiyete göre gruplandırıldı ve yanıt açısından istatistiksel olarak anlamlı fark olmadığı görüldü. İlaç genel olarak iyi tolere edildi. Eltrombopag, diğer tedavilere dirençli ve artmış kanama riski olan kronik ITP hastalarında etkili ve iyi tolere edilen bir terapötik ajandır. Çalışmamızın sonuçları, yanıt durumunun splenektomi, eşzamanlı tedavi alma veya başlangıç trombosit değerlerinden bağımsız olduğunu ortaya koymuştur.

References

  • 1. Pruemer J. Epidemiology, pathophysiology, and initial management of chronic immune thrombocytopenic purpura. Am J Health Syst Pharm. 2009;66(2 Suppl 2):S4-S10.
  • 2. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115(2):168-186.
  • 3. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866.
  • 4. Demir AM, Ümit EG, Ar MC, et al. Management of Adult Primary Immune Thrombocytopenia: Delphi-Based Consensus Recommendations. Turk J Haematol. 2024 May 30;41(2):97-104..
  • 5. Neunert CE, Arnold DM, Grace RF, Kuhne T, McCrae KR, Terrell DR. The 2022 review of the 2019 American Society of Hematology guidelines on immune thrombocytopenia. Blood Adv. 2024 Jul 9;8(13):3578-3582.
  • 6. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019 Nov 26;3(22):3780-3817.
  • 7. González-López TJ, Provan D. Proposal for a New Protocol for the Management of Immune Thrombocytopenia (ITP). Adv Ther. 2022 Jun;39(6):2287-2291.
  • 8. Garnock-Jones KP, Keam SJ. Eltrombopag. Drugs. 2009;69(5):567-576.
  • 9. Bussel JB, Provan D, Shamsi T, et al. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomized, double-blind, placebo-controlled trial. Lancet. 2009;373(9664): 641-648.
  • 10. Cheng G, Saleh MN, Marcher C, et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomized, phase 3 study. Lancet. 2011;377(9763):393-402.
  • 11. Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536.
  • 12. Bussel JB, Saleh MN, Vasey SY, et al. Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP). Br J Haematol. 2013 Feb;160(4):538-546.
  • 13. Tomiyama Y, Miyakawa Y, Okamoto S, et al. A lower starting dose of eltrombopag is efficacious in Japanese patients with previously treated chronic immune thrombocytopenia. J Thromb Haemost. 2012 May;10(5):799-806.
  • 14. Yoshida M, Kanashima H, Nakao T, et al. Retrospective analysis of eltrombopag for the treatment of refractory primary immune thrombocytopenia in Japan. Rinsho Ketsueki. 2013 May;54(5):444-450.
  • 15. Katsutani S, Tomiyama Y, Kimura A, et al. Oral eltrombopag for up to three years is safe and well-tolerated in Japanese patients with previously treated chronic immune thrombocytopenia: an open-label, extension study. Int J Hematol. 2013 Sep;98(3):323-330.
  • 16. Tripathi AK, Shukla A, Mishra S, et al. Eltrombopag therapy in newly diagnosed steroid non-responsive ITP patients. Int J Hematol. 2014 Apr;99(4):413-417.
  • 17. Gardellini A, Guidotti F, Feltri M, et al. Eltrombopag as second line treatment in patients with primary immune thrombocytopenia: A single center real life experience. Blood Cells Mol Dis. 2021 Dec;92:102620.
  • 18. Rosenberg A, Cashion C, Ali F, et al. Treatment of immune thrombocytopenia in Australian adults: A multicenter retrospective observational study. Res Pract Thromb Haemost. 2022 Sep 18;6(6):e12792.
  • 19. Eser A, Toptaş T, Tanrıkulu F, et al. Marmara University eltrombopag experience in chronic ITP patients. XXXIXth National Hematology Congress Book of Abstracts 2013;271-272.
  • 20. Özdemirkıran F, Payzın B, Kiper H, et al. Eltrombopag in the treatment of refractory chronic immune thrombocytopenic purpura: Aegean region experience. XXXIXth National Hematology Congress Book of Abstracts 2013;67-68.
  • 21. Çekdemir D, Hindilerden F, Güvenç S, et al. Use of eltrombopag in immune thrombocytopenia in Turkey: Experience of eleven centers. XXXIXth National Hematology Congress Book of Abstracts 2013;9.
  • 22. Dogan EE, Turan Erkek E, Elverdi T, et al. Eltrombopag in the Treatment of Immune Thrombocytopenia: Two-Center Experience from Istanbul. Indian J Hematol Blood Transfus. 2022 Apr;38(2):327-332.
  • 23. Pektaş G, Uncu İA, Dere Y, et al. Retrospective Evaluation of Survival and Prognostic Factors in Immune Thrombocytopenia: A Single-Center and Cross-Sectional Study. Medicina (Kaunas). 2024 Jul 17;60(7):1153.
  • 24. González-López TJ, Sánchez-González B, Pascual C, et al. Sustained response after discontinuation of short-and medium-term treatment with eltrombopag in patients with immune thrombocytopenia. Platelets. 2014 Feb 5. [Epub ahead of print]
  • 25. Mahévas M, Fain O, Ebbo M, et al. The temporary use of thrombopoietin-receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia. Results of a French observational study. Br J Haematol. 2014 Jun;165(6):865-869.
  • 26. Mishra K, Pramanik S, Jandial A, et al. Real-world experience of eltrombopag in immune thrombocytopenia. Am J Blood Res. 2020 Oct 15;10(5):240-251.
  • 27. Cooper N, Ghanima W, Vianelli N, et al. Sustained response off-treatment in eltrombopag-treated adult patients with ITP who are refractory or relapsed after first-line steroids: Primary, final, and ad-hoc analyses of the Phase II TAPER trial. Am J Hematol. 2024 Jan;99(1):57-67.
  • 28.Cooper N, Scully M, Percy C, et al. Real-world use ofthrombopoietin receptor agonists for the management of immune thrombocytopenia in adult patients in the United Kingdom: Results from the TRAIT study. Br J Haematol. 2024 Jun;204(6):2442-2452.
There are 28 citations in total.

Details

Primary Language English
Subjects Haematology
Journal Section Research Article
Authors

Emre Hafızoğlu 0000-0001-6291-851X

Vildan Ozkocaman 0000-0003-0014-7398

Fahir Özkalemkaş 0000-0001-9710-134X

Submission Date February 8, 2025
Acceptance Date March 21, 2025
Publication Date May 27, 2025
Published in Issue Year 2025 Volume: 51 Issue: 1

Cite

AMA Hafızoğlu E, Ozkocaman V, Özkalemkaş F. Use of Eltrombopag in Patients with Chronic Idiopathic Thrombocytopenic Purpura: A Single Center Experience. Journal of Uludağ University Medical Faculty. May 2025;51(1):59-65. doi:10.32708/uutfd.1635651

ISSN: 1300-414X, e-ISSN: 2645-9027

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