Clinical outcomes of pediatric patients with severe acquired aplastic anemia treated at Siriraj Hospital

Nachapol  Jatuten; Bunchoo Pongtanakul

doi:10.69898/jhtm.36.2026.285795

Authors

Dr.Nachapol Jatuten Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand 10700
Dr.Bunchoo Pongtanakul Division of Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand 10700

DOI:

https://doi.org/10.69898/jhtm.36.2026.285795

Keywords:

Severe aplastic anemia, Immunosuppressive, children, outcome, hematopoietic stem cell transplantation

Abstract

Background: Severe acquired aplastic anemia (SAA) is a life-threatening condition. Thai treatment guidelines recommend matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT) and immunosuppressive therapy (IST) combined with eltrombopag (EPAG).

Objective: This study aimed to investigate the clinical outcomes of pediatric patients with SAA treated at Siriraj Hospital.

Method: This retrospective study included pediatric patients with SAA treated at Siriraj Hospital from 1 January 2013 to 31 December 2023.

Result: Twenty-seven patients were enrolled. Treatment modalities included IST in 13 patients (48.2%), IST+EPAG in 7 patients (25.9%) and MSD-HSCT in 7 patients (25.9%). At 3 and 6 months, overall response (OR) rates were 28.6% and 42.9% in the IST+EPAG group and 0% and 7.7% in the IST-only group, respectively. However, these differences were not statistically significant (p = 0.111 and 0.197, respectively). Overall survival (OS) was similar between the IST+EPAG (62.5%, 95%CI: 0.14-0.89) and the IST group (63.3%, 95%CI: 0.29–0.84). The mean time to response and mean time to transfusion independence were shorter in the IST+EPAG group compared with the IST group, but the differences were not statically significance (p = 0.085 and 0.162, respectively). The OS for the MSD-HSCT group was 42.9%.

Conclusion: The combination of EPAG with IST was associated with a trend toward higher OR rates at 3 and 6 months. The IST+EPAG group also demonstrated shorter mean times to hematologic response and transfusion independence. OS was comparable between groups.

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References

Young NS. Aplastic Anemia. New England Journal of Medicine. 2018;379:1643-56.

Ahmed P, Chaudhry QuN, Satti TM, Mahmood SK, Ghafoor T, Shahbaz N, et al. Epidemiology of aplastic anemia: a study of 1324 cases. Hematology. 2020;25:48-54.

Issaragrisil S, Leaverton PE, Chansung K, Thamprasit T, Porapakham Y, Vannasaeng S, et al. Regional patterns in the incidence of aplastic anemia in Thailand. The Aplastic Anemia Study Group. Am J Hematol. 1999;61:164-8

Nao Y, Ryoji K, Hiromasa Y, Yoshiyuki K, Hiroshi Y, Ken-ichiro W, et al. First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy. Haematologica. 2014;99:1784-91.

Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017;129:1428-36.

Erickson-Miller CL, Delorme E, Tian S-S, Hopson CB, Landis AJ, Valoret EI, et al. Preclinical Activity of Eltrombopag (SB-497115), an Oral, Nonpeptide Thrombopoietin Receptor Agonist. Stem Cells. 2009;27:424-30.

Marrapodi MM, Mascolo A, Roberti D, Martino MD, Rafaniello C, Riccardi C, et al. The efficacy and the safety of eltrombopag in pediatric patients with severe aplastic anemia: a systematic review. Frontiers in Pediatrics. 2023;11.1149718. doi: 10.3389/fped.2023.1149718. eCollection 2023.

Goronkova O, Novichkova G, Salimova T, Kalinina I, Baidildina D, Petrova U, et al. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia. Blood Adv. 2023;7:953-62.

Uaprasert N CK, Pongtanakul B, Lauhasurayotin S, Sirachainan N, Visuthisakchai S, et al. . Guideline for Diagnosis and Management of Aplastic Anemia in Thailand 2020. J Hematol Transfus Med 2020;30:405-13.

Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172:187-207.

Jie M, Fu L, Li S, He Y, Yao J, Cheng X, et al. Efficacy and safety of eltrombopag in the first-line therapy of severe aplastic anemia in children. Pediatr Hematol Oncol. 2021;38:647-57.

Khattachan O, Poparn H, Lauhasurayothin S, Techavichit P, Sosothikul D, Seksarn P. Clinical outcomes of pediatric patients with severe idiopathic aplastic anemia in King Chulalongkorn Memorial Hospital. J Hematol Transfus Med. 2020;30:263–70.

Groarke EM, Patel BA, Gutierrez-Rodrigues F, Rios O, Lotter J, Baldoni D, et al. Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia. Br J Haematol. 2021;192:605-14.

Shimamura A, Maschan A, Bennett C, Samarasinghe S, Farrar JE, Li CK, et al. Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial. Blood Adv. 2025 Aug 12;9:3728-3738.

Latour RPd, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, et al. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. New England Journal of Medicine. 2022;386:11-23.

Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365:430-8.

Jeong DC, Nack Gyun C, Bin C, Yao Z, Min R, Yoshiyuki T, et al. Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children. Haematologica. 2014;99:664-71.

Zhao Y, Yang W, Zhao X, Hu X, Hu J, Liu X, et al. Efficacy of eltrombopag with immunosuppressive therapy for children with acquired aplastic anemia. Front Pediatr. 2022;10:1095143.

Shimano KA, Rothman JA, Allen SW, Castillo P, de Jong JLO, Dror Y, et al. Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations. Pediatric Blood & Cancer. 2024;71:e31070.

Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. New England Journal of Medicine. 2017;376:1540-50.

Lesmana H, Jacobs T, Boals M, Gray N, Lewis S, Ding J, et al. Eltrombopag in children with severe aplastic anemia. Pediatr Blood Cancer. 2021;68:e29066.

Chaudhry QUN, Iftikhar R, Satti TM, Mahmood SK, Ghafoor T, Shamshad GU, et al. Outcome of Fludarabine-Based Conditioning in High-Risk Aplastic Anemia Patients Undergoing Matched Related Donor Transplantation: A Single-Center Study from Pakistan. Biology of Blood and Marrow Transplantation. 2019;25:2375-82.