RESPONSE TO IMMUNOSUPPRESSIVE THERAPY IN PATIENTS OF ACQUIRED APLASTIC ANAEMIA: A SINGLE CENTER EXPERIENCE FROM A DEVELOPING COUNTRY
Keywords:Key Words: Aplastic anemia, immunosuppressive therapy, Cyclosporine.
AbstractBackground: Aplastic Anaemia (AA) is characterized by pancytopenia and hypocellular marrow. Immunosuppressive therapy (IST) SHOWS impressive haematological response; however, risk of relapse and clonal evolution persists. The objective of the study is to assess response to IST in patients with aplastic anaemia. Methods: A retrospective single centre study at AFBMTC / NIBMT for patients of acquired AA was conducted from January 2005 to December 2019.Inclusion criteria included diagnosed cases of acquired AA receiving IST for at least 12 weeks and age >2 years. IST included cyclosporine (CsA) alone, CsA + androgens, CsA + rabbit anti thymocyte globulin (rATG), CsA + horse anti thymocyte globulin (hATG). Primary outcome measure was response to IST; secondary outcome measure was overall survival (OS). Results: A total of 513 patients received IST. Median age was 23 years (range 2-97 years). In study cohort, 155 (30.2%) patients responded to the IST, 63 (12.3%) achieved complete response (CR) while 92 (17.9%) achieved partial response (PR). The ORR of CsA in NSAA, SAA and VSAA was 52.6%, 28.10% and 10% respectively; whereas ORR of CsA + rATG in NSAA, SAA and VSAA was 50%, 35.1% and 22.5% respectively. OS was 38% at a median follow up of 36 months. There was a significant difference in the survival distributions of different treatment modalities (p=0.016). Median survival time 60 months (CsA), 9 months (CsA+ androgens) and 39 months (CsA+ rATG/hATG.) Conclusion: In resource constrained settings, single agent CsA remains a reasonable alternative with modest activity and acceptable side effect profile.
Young NS. Aplastic Anaemia. N Engl J Med 2018;379(17):1643–56. Ref no 1&13 are same
Ahmed P, Chaudhry QUN, Satti TM, Mahmood SK, Ghafoor T, Shahbaz N, et al. Epidemiology of aplastic anaemia: a study of 1324 cases. Hematology 2020;25(1):48–54.
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(2):187–207.
Vaht K, Göransson M, Carlson K, Isaksson C, Lenhoff S, Sandstedt A, et al. Incidence and outcome of acquired aplastic anaemia: real-world data from patients diagnosed in Sweden from 2000-2011. Haematologica 2017;102(10):1683–90.
Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, et al. Immunosuppressive therapy for pediatric aplastic anaemia: a North American Pediatric Aplastic Anaemia Consortium study. Haematologica 2019;104(10):1974–83.
Georges GE, Storb R. Hematopoietic stem cell transplantation for acquired aplastic anaemia. Curr Opin Hematol 2016;23(6):495–500.
Alashkar F, Oelmüller M, Herich-Terhürne D, Turki AT, Schmitz C, Vance C, et al. Immunosuppressive therapy (IST) in adult patients with acquired aplastic anaemia (AA): A single-center experience over the past 15 years. Eur J Haematol 2019;103(1):18–25.
Scheinberg P, Townsley D, Dumitriu B, Scheinberg P, Weinstein B, Daphtary M, et al. Moderate-dose cyclophosphamide for severe aplastic anaemia has significant toxicity and does not prevent relapse and clonal evolution. Blood 2014;124(18):2820–3.
Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, et al. Eltrombopag Added to Immunosuppression in Severe Aplastic Anaemia. N Engl J Med 2022;386(1):11–23.
Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, et al. Eltrombopag added to standard immunosuppression for aplastic anaemia. N Engl J Med 2017;376(16):1540–50.
Chuncharunee S, Wong R, Rojnuckarin P, Chang CS, Chang KM, Lu MY, et al. Efficacy of rabbit antithymocyte globulin as first-line treatment of severe aplastic anaemia: an Asian multicenter retrospective study. Int J Hematol 2016;104(4):454–61.
Mandal PK, Baul S, Dolai TK, De R, Chakrabarti P. Outcome of Cyclosporine Monotherapy in Patients of Aplastic Anaemia: Experience of a Tertiary Care Hospital in Eastern India. Indian J Hematol Blood Transfus 2017;33(1):144–7.
Iftikhar R, Ahmad P, de Latour R, Dufour C, Risitano A, Chaudhri N, et al. Special issues related to the diagnosis and management of acquired aplastic anaemia in countries with restricted resources, a report on behalf of the Eastern Mediterranean blood and marrow transplantation (EMBMT) group and severe aplastic anaemia working party of the European Society for blood and marrow transplantation (SAAWP of EBMT). Bone Marrow Transplant 2021;56(10):2518–32.
Boddu P, Garcia-Manero G, Ravandi F, Borthakur G, Jabbour E, DiNardo C, et al. Clinical outcomes in adult patients with aplastic anaemia: A single institution experience. Am J Hematol 2017;92(12):1295–302.
Agarwal MB, Jijina F, Shah S, Malhotra P, Damodar S, Ross C. Safety and efficacy of indigenous equine antithymocyte globulin along with cyclosporine in subjects with acquired aplastic anaemia. Indian J Hematol Blood Transfus 2015;31(2):174–9.
Shah S, Jain P, Shah K, Patel K, Parikh S, Patel A, et al. Immunosuppressive therapy for aplastic anaemia: a single-center experience from western India. Ann Hematol 2019;98(1):41–6.
Bacigalupo A, Oneto R, Schrezenmeier H, Hochsmann B, Dufour C, Kojima S, et al. First line treatment of aplastic anaemia with thymoglobuline in Europe and Asia: Outcome of 955 patients treated 2001-2012. Am J Hematol 2018;93(5):643–8.
Journal of Ayub Medical College, Abbottabad is an OPEN ACCESS JOURNAL which means that all content is FREELY available without charge to all users whether registered with the journal or not. The work published by J Ayub Med Coll Abbottabad is licensed and distributed under the creative commons License CC BY ND Attribution-NoDerivs. Material printed in this journal is OPEN to access, and are FREE for use in academic and research work with proper citation. J Ayub Med Coll Abbottabad accepts only original material for publication with the understanding that except for abstracts, no part of the data has been published or will be submitted for publication elsewhere before appearing in J Ayub Med Coll Abbottabad. The Editorial Board of J Ayub Med Coll Abbottabad makes every effort to ensure the accuracy and authenticity of material printed in J Ayub Med Coll Abbottabad. However, conclusions and statements expressed are views of the authors and do not reflect the opinion/policy of J Ayub Med Coll Abbottabad or the Editorial Board.
USERS are allowed to read, download, copy, distribute, print, search, or link to the full texts of the articles, or use them for any other lawful purpose, without asking prior permission from the publisher or the author. This is in accordance with the BOAI definition of open access.
AUTHORS retain the rights of free downloading/unlimited e-print of full text and sharing/disseminating the article without any restriction, by any means including twitter, scholarly collaboration networks such as ResearchGate, Academia.eu, and social media sites such as Twitter, LinkedIn, Google Scholar and any other professional or academic networking site.