Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation
Yıl: 2022 Cilt: 39 Sayı: 2 Sayfa Aralığı: 103 - 108 Metin Dili: İngilizce DOI: 10.4274/tjh.galenos.2022.2021.0675 İndeks Tarihi: 07-06-2022
Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation
Öz: Objective: This study aimed to evaluate the efficacy and safety of
eltrombopag (ELT) in the treatment of thrombocytopenia following
hematopoietic stem cell transplantation (HSCT).
Materials and Methods: Forty-eight patients treated with ELT for
thrombocytopenia after allogeneic or autologous transplantation at
the Erciyes University Bone Marrow Transplantation Center between
July 2017 and July 2021 were evaluated retrospectively.
Results: Forty-eight HSCT recipients were included in this study.
Thirty (62.5%) patients were evaluated as having experienced delayed
platelet recovery (DPR) and 18 (37.5%) patients as having experienced
secondary failure of platelet recovery (SFPR). The median platelet
count before ELT treatment was 13x109
/L (range: 3-20x109
/L). Twentythree patients responded to treatment and the cumulative incidence
of successful platelet recovery was 48%. Patients with both DPR
and SFPR responded, but patients with DPR had a higher response
rate (50% vs. 44%). The median platelet count of the 23 responding
patients was 12x109
/L (5-19x109
/L) before treatment and 68x109
/L
(52-266x109
/L) after treatment (p<0.0001). While the number of bone
marrow megakaryocytes before treatment was adequate in 22 (46%)
cases, it was decreased in 26 (54%) cases. Patients with adequate bone
marrow megakaryocytes had a better response rate than those without
(77% vs. 23%, p<0.0001). The group with adequate megakaryocytes
responded to treatment at a median of 33 days (range: 9-174 days).
Patients with decreased megakaryocytes responded at a median of
55 days (30-164 days) (p=0.002). No drug-related side effects were
observed in any patients.
Conclusion: This real-life experience demonstrates that ELT is an
effective and safe treatment option for thrombocytopenia after HSCT.
The adequacy of bone marrow megakaryocytes before ELT treatment
was an important factor affecting response to treatment.
Anahtar Kelime: Hematopoetik Kök Hücre Nakli Sonrası Trombositopeni Tedavisinde Eltrombopag Kullanımı
Öz: Amaç: Transplantasyon sonrası trombositopeni tedavisinde
eltrombopagın (ELT) etkinlik ve güvenliğini değerlendirmeyi amaçladık.
Gereç ve Yöntemler: Erciyes Üniversitesi Kemik İliği Nakil Merkezi’nde
Temmuz 2017-Temmuz 2021 tarihleri arasında allojenik veya otolog
transplantasyon sonrası trombositopeni nedeniyle ELT ile tedavi edilen
48 hasta retrospektif olarak değerlendirildi.
Bulgular: Bu çalışmaya 48 hematopoietik kök hücre nakli (HKHN)
alıcısı dahil edildi. Otuz (%62,5) hasta gecikmiş trombosit iyileşmesi
(DPR) ve 18 (%37,5) hasta ikincil trombosit iyileşme başarısızlığı (SFPR)
olarak değerlendirildi. ELT tedavisinden önce medyan trombosit sayısı
13x109
/L (aralık, 3-20x109
/L) idi. Yirmi üç hasta tedaviye yanıt verdi ve
başarılı trombosit iyileşmesinin kümülatif insidansı %48 idi. Hem DPR
hem de SFPR’li hastalar yanıt verdi, ancak DPR’li hastalarda yanıt oranı
daha yüksekti (%50’ye karşı %44). Yanıt veren 23 hastanın medyan
trombosit sayısı tedaviden önce ve sonra 12 (5-19)x109
/L ve 68 (52-
266)x109
/L idi (p<0,0001). Tedavi öncesi kemik iliği megakaryosit sayısı
22 (%46) hastada yeterli iken 26 (%54) hastada azalmıştı. Yeterli
kemik iliği megakaryositleri olan hastalar, azalmış olanlara göre daha
iyi bir yanıt oranına sahipti (%77’ye karşı %23, p<0,0001). Yeterli
megakaryositleri olan grup, tedaviye medyan 33 (aralık, 9-174) günde
yanıt verdi. Megakaryositleri azalmış hastalar, ortalama 55 (aralık, 30-
164) gün yanıt verdi (p=0,002). Hiçbir hastada ilaca bağlı yan etki
gözlenmedi.
Sonuç: Bu gerçek yaşam deneyimi, ELT’nin HKHN sonrası
trombositopeni için etkili ve güvenli bir tedavi seçeneği olduğunu
göstermektedir. ELT tedavisi öncesi kemik iliği megakaryositlerinin
yeterliliği tedaviye yanıtı etkileyen önemli bir faktördü.
Anahtar Kelime: Belge Türü: Makale Makale Türü: Araştırma Makalesi Erişim Türü: Erişime Açık
- 1. Kuzmina Z, Eder S, Böhm A, Pernicka E, Vormittag L, Kalhs P, Petkov V, Stary G, Nepp J, Knobler R. Significantly worse survival of patients with NIH-defined chronic graft-versus-host disease and thrombocytopenia or progressive onset type: results of a prospective study. Leukemia 2012;26:746-756.
- 2. Bruno B, Gooley T, Sullivan KM, Davis C, Bensinger WI, Storb R, Nash RA. Secondary failure of platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2001;7:154-162.
- 3. Yamazaki R, Kuwana M, Mori T, Okazaki Y, Kawakami Y, Ikeda Y, Okamoto S. Prolonged thrombocytopenia after allogeneic hematopoietic stem cell transplantation: associations with impaired platelet production and increased platelet turnover. Bone Marrow Transplant 2006;38:377-384.
- 4. Dominietto A, Raiola AM, Van Lint MT, Lamparelli T, Gualandi F, Berisso G, Bregante S, Frassoni F, Casarino L, Verdiani S. Factors influencing haematological recovery after allogeneic haemopoietic stem cell transplants: graft‐versus‐host disease, donor type, cytomegalovirus infections and cell dose. Br J Haematol 2001;112:219-227.
- 5. Culligan D. Clinical bone marrow and blood stem cell transplantation. Br J Haematol 2001;112:254-255.
- 6. Bolwell B, Pohlman B, Sobecks R, Andresen S, Brown S, Rybicki L, Wentling V, Kalaycio M. Prognostic importance of the platelet count 100 days post allogeneic bone marrow transplant. Bone Marrow Transplant 2004;33:419- 423.
- 7. Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007;357:2237-2247.
- 8. Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood 2009;113:2161-2171.
- 9. Bussel JB, de Miguel PG, Despotovic JM, Grainger JD, Sevilla J, Blanchette VS, Krishnamurti L, Connor P, David M, Boayue KB. Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo-controlled study. Lancet Haematol 2015;2:e315-e325.
- 10. Mishra K, Pramanik S, Jandial A, Sahu KK, Sandal R, Ahuja A, Yanamandra U, Kumar R, Kapoor R, Verma T. Real-world experience of eltrombopag in immune thrombocytopenia. Am J Blood Res 2020;10:240.
- 11. Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med 2017;376:1540- 1550.
- 12. Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med 2012;367:11-19.
- 13. Tanaka T, Inamoto Y, Yamashita T, Fuji S, Okinaka K, Kurosawa S, Kim SW, Tanosaki R, Fukuda T. Eltrombopag for treatment of thrombocytopenia after allogeneic hematopoietic cell transplantation. Biol Blood Marrow Transplant 2016;22:919-924.
- 14. Dyba J, Tinmouth A, Bredeson C, Matthews J, Allan D. Eltrombopag after allogeneic haematopoietic cell transplantation in a case of poor graft function and systematic review of the literature. Transfus Med 2016;26:202- 207.
- 15. Bosch‐Vilaseca A, García‐Cadenas I, Roldán E, Novelli S, Barba P, Esquirol A, Valcárcel D, Martino R, Sierra J. Usefulness of thrombopoietin receptor agonists for persistent clinically relevant thrombocytopenia after allogeneic stem cell transplantation. Eur J Haematol 2018;101:407-414.
- 16. Marano L, Marotta S, Cacace F, Frieri C, Simeone L, Trastulli F, Vitiello S, Cardano F, Pane F, Risitano AM. Eltrombopag for prolonged post-transplant hyporigenerative cytopenias. Blood 2018;132:5711.
- 17. Nash RA, Kurzrock R, DiPersio J, Vose J, Linker C, Maharaj D, Nademanee AP, Negrin R, Nimer S, Shulman H. A phase I trial of recombinant human thrombopoietin in patients with delayed platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2000;6:25-34.
- 18. Akahoshi Y, Kanda J, Gomyo A, Hayakawa J, Komiya Y, Harada N, Kameda K, Ugai T, Wada H, Ishihara Y. Risk factors and impact of secondary failure of platelet recovery after allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2016;22:1678-1683.
- 19. Reid R, Bennett JM, Becker M, Chen Y, Milner L, Phillips GL 2nd, Liesveld J. Use of eltrombopag, a thrombopoietin receptor agonist, in post‐transplantation thrombocytopenia. Am J Hematol 2012;87:743-745.
- 20. Raut SS, Shah SA, Sharanangat VV, Shah KM, Patel KA, Anand AS, Talati SS, Panchal HP, Patel AA, Parikh SK. Safety and efficacy of eltrombopag in post-hematopoietic stem cell transplantation (HSCT) thrombocytopenia. Indian J Hematol Blood Transfus 2015;31:413-415.
- 21. Yuan C, Boyd AM, Nelson J, Patel RD, Varela JC, Goldstein SC, Ahmad S, Zhu X, Mori S. Eltrombopag for treating thrombocytopenia after allogeneic stem cell transplantation. Biol Blood Marrow Transplant 2019;25:1320-1324.
- 22. Mori S, Patel RD, Boyd A, Simon S, Nelson J, Goldstein SC. Eltrombopag treatment for primary and secondary thrombocytopenia post allogeneic and autologous stem cell transplantation is effective and safe. Biol Blood Marrow Transplant 2018;24:S343-S344.
APA | Guven Z, ÇELİK S, Eser B, ÇETİN M, ÜNAL A, Kaynar L (2022). Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. , 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
Chicago | Guven Zeynep Tugba,ÇELİK Serhat,Eser Bülent,ÇETİN Mustafa,ÜNAL Ali,Kaynar Leylagul Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. (2022): 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
MLA | Guven Zeynep Tugba,ÇELİK Serhat,Eser Bülent,ÇETİN Mustafa,ÜNAL Ali,Kaynar Leylagul Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. , 2022, ss.103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
AMA | Guven Z,ÇELİK S,Eser B,ÇETİN M,ÜNAL A,Kaynar L Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. . 2022; 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
Vancouver | Guven Z,ÇELİK S,Eser B,ÇETİN M,ÜNAL A,Kaynar L Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. . 2022; 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
IEEE | Guven Z,ÇELİK S,Eser B,ÇETİN M,ÜNAL A,Kaynar L "Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation." , ss.103 - 108, 2022. 10.4274/tjh.galenos.2022.2021.0675 |
ISNAD | Guven, Zeynep Tugba vd. "Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation". (2022), 103-108. https://doi.org/10.4274/tjh.galenos.2022.2021.0675 |
APA | Guven Z, ÇELİK S, Eser B, ÇETİN M, ÜNAL A, Kaynar L (2022). Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. Turkish Journal of Hematology, 39(2), 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
Chicago | Guven Zeynep Tugba,ÇELİK Serhat,Eser Bülent,ÇETİN Mustafa,ÜNAL Ali,Kaynar Leylagul Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. Turkish Journal of Hematology 39, no.2 (2022): 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
MLA | Guven Zeynep Tugba,ÇELİK Serhat,Eser Bülent,ÇETİN Mustafa,ÜNAL Ali,Kaynar Leylagul Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. Turkish Journal of Hematology, vol.39, no.2, 2022, ss.103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
AMA | Guven Z,ÇELİK S,Eser B,ÇETİN M,ÜNAL A,Kaynar L Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. Turkish Journal of Hematology. 2022; 39(2): 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
Vancouver | Guven Z,ÇELİK S,Eser B,ÇETİN M,ÜNAL A,Kaynar L Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation. Turkish Journal of Hematology. 2022; 39(2): 103 - 108. 10.4274/tjh.galenos.2022.2021.0675 |
IEEE | Guven Z,ÇELİK S,Eser B,ÇETİN M,ÜNAL A,Kaynar L "Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation." Turkish Journal of Hematology, 39, ss.103 - 108, 2022. 10.4274/tjh.galenos.2022.2021.0675 |
ISNAD | Guven, Zeynep Tugba vd. "Eltrombopag for Treatment of Thrombocytopenia Following Hematopoietic Stem Cell Transplantation". Turkish Journal of Hematology 39/2 (2022), 103-108. https://doi.org/10.4274/tjh.galenos.2022.2021.0675 |