Article Sidebar
Views pdf: 34
EARLY TREATMENT OUTCOMES OF PEDIATRIC PATIENTS WITH IDIOPATHIC APLASTIC ANEMIA USING THE hATG COMBINED WITH CYCLOSPORIN A REGIMENT AT THE NATIONAL INSTITUTE OF HEMATOLOGY AND BLOOD TRANSFUTION DURING THE 2020–2025 PERIOD
Main Article Content
Abstract
Treatment of Idiopathic Aplastic Anemia in Pediatric Patients Using Horse ATG Combined with Cyclosporin A: A Primary Therapeutic Option for Patients Ineligible for Allogeneic Stem Cell Transplantation. Background: Immunosuppressive therapy (IST) using horse anti-thymocyte globulin (hATG) in combination with cyclosporin A (CSA) has proven to be highly effective and is considered the first-line treatment for pediatric patients with idiopathic aplastic anemia (AA) who are not eligible for allogeneic stem cell transplantation. Objective: To evaluate early treatment outcomes of pediatric patients with idiopathic aplastic anemia receiving hATG plus CSA at the National Institute of Hematology and Blood Transfusion (NIHBT) during the period from 2020 to 2025. Subjects and Methods: This is a descriptive case series study involving 27 pediatric patients diagnosed with idiopathic aplastic anemia who received first-line treatment with immunosuppressive therapy using hATG in combination with CSA. The study was conducted at the Pediatric Hematology Department, NIHBT, from January 2020 to March 2025. Results: The overall response rate was 37% at 3 months and 66.7% at 6 months. The probability of overall survival beyond 2 years was 95.8%, and the cumulative response rate at 9 and 12 months was 75%. Conclusion: Horse ATG combined with cyclosporin A is an effective and first-line therapeutic option for pediatric patients with idiopathic aplastic anemia who are not candidates for allogeneic stem cell transplantation.
Article Details
Keywords
Pediatric patient, aplastic anemia, treatment of aplastic anemia, horse ATG, cyclosporine A.
References
2. Camitta, B. M.; Rappeport, J. M.; Parkman, R.; Nathan, D. G. Selection of Patients for Bone Marrow Transplantation in Severe Aplastic Anemia. Blood 1975, 45 (3), 355–363.
3. Bacigalupo, A.; Hows, J.; Gluckman, E.; Nissen, C.; Marsh, J.; Van Lint, M. T.; Congiu, M.; De Planque, M. M.; Ernst, P.; McCann, S.; Ragavashar, A.; Frickhofen, N.; Wursch, A.; Marmont, A. M.; Gordon-Smith, E. C. Bone Marrow Transplantation (BMT) versus Immunosuppression for the Treatment of Severe Aplastic Anaemia (SAA): A Report of the EBMT* SAA Working Party. British Journal of Haematology 1988, 70 (2), 177–182. https://doi. org/10.1111/j.1365-2141.1988.tb02460.x.
4. Scheinberg, P.; Wu, C. O.; Nunez, O.; Scheinberg, P.; Boss, C.; Sloand, E. M.; Young, N. S. Treatment of Severe Aplastic Anemia with a Combination of Horse Antithymocyte Globulin and Cyclosporine, with or without Sirolimus: A Prospective Randomized Study. Haematologica 2009, 94 (3), 348–354. https://doi.org/10.3324/haematol.13829.
5. Kulasekararaj, A.; Cavenagh, J.; Dokal, I.; Foukaneli, T.; Gandhi, S.; Garg, M.; Griffin, M.; Hillmen, P.; Ireland, R.; Killick, S.; Mansour, S.; Mufti, G.; Potter, V.; Snowden, J.; Stanworth, S.; Zuha, R.; Marsh, J.; the BSH Committee. Guidelines for the Diagnosis and Management of Adult Aplastic Anaemia: A British Society for Haematology Guideline. Br J Haematol 2024, 204 (3), 784–804. https://doi.org/ 10.1111/bjh.19236.
6. Bacigalupo, A. How I Treat Acquired Aplastic Anemia. Blood 2017, 129 (11), 1428–1436. https://doi.org/10.1182/blood-2016-08-693481.
7. Rogers, Z. R.; Nakano, T. A.; Olson, T. S.; Bertuch, A. A.; Wang, W.; Gillio, A.; Coates, T. D.; Chawla, A.; Castillo, P.; Kurre, P.; Gamper, C.; Bennett, C. M.; Joshi, S.; Geddis, A. E.; Boklan, J.; Nalepa, G.; Rothman, J. A.; Huang, J. N.; Kupfer, G. M.; Cada, M.; Glader, B.; Walkovich, K. J.; Thompson, A. A.; Hanna, R.; Vlachos, A.; Malsch, M.; Weller, E. A.; Williams, D. A.; Shimamura, A. Immunosuppressive Therapy for Pediatric Aplastic Anemia: A North American Pediatric Aplastic Anemia Consortium Study. Haematologica 2019, 104 (10), 1974–1983. https://doi.org/10.3324/haematol.2018.206540.