RESPONSE TO IMMUNOSUPPRESSIVE THERAPY IN PATIENTS OF ACQUIRED APLASTIC ANAEMIA: A SINGLE CENTER EXPERIENCE FROM A DEVELOPING COUNTRY

Authors

  • Maryam Khan Armed Forces Bone Marrow Transplant Cemtre,Rawalpindi.
  • Raheel Iftikhar Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Qamar un Nisa Chaudhry Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Syed Kamran Mehmood Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Tehniat Faraz Dow University of Health Sciences,Karachi,Pakistan
  • Tariq Ghafoor Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Nighat Shahbaz Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Mehreen Ali Khan Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Ghassan Umair Shamshad Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan
  • Tariq Azam Khattak Armed Forces Bone Marrow Transplant Centre,Rawalpindi,Pakistan

DOI:

https://doi.org/10.55519/JAMC-04-S4-9885

Keywords:

Key Words: Aplastic anemia, immunosuppressive therapy, Cyclosporine.

Abstract

Background: 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.

References

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.

Downloads

Published

2022-10-11