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First-Line Immunosuppressive Treatment in Children with Aplastic Anemia: Rabbit Antithymocyte Globulin

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Respiratory Virology and Immunogenicity

Part of the book series: Advances in Experimental Medicine and Biology ((NR,volume 836))

Abstract

Immunosuppressive therapy is the treatment of choice in children with acquired severe aplastic anemia (AA) and no HLA-matched family donor. The paper presents results of a multicenter study of 63 children with AA treated with rabbit antithymocyte globulin (r-ATG) and cyclosporine A as the first line treatment in the years 1996–2012. Therapeutic effects were evaluated at Days 112, 180, and 360. At Day 112, remission was achieved in 28 out of the 63 patients (44.4 %), complete remission in 10 patients (15.9 %), and partial remission in 18 (28.5 %). At Day 180, 31 patients (49.2 %) were in remission including 15 cases in complete (23.8 %), and 16 cases in partial remission (25.4 %). One year after therapy onset, 34 patients (64.9 %) were in remission including 24 patients (38.0 %) in complete and 10 (15.9 %) in partial remission. Relapse occurred in 4 patients, from 8 months up to 2 years and 2 months after remission. One child, 5 years after remission, was diagnosed with paroxysmal nocturnal hemoglobinuria. The estimated 10-year overall survival rate and 10-year event-free survival rate were 67 % and 57 %, respectively.

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References

  • Afable MG, Shaik M, Sugimoto Y, Elson P, Clemente M, Makishima H, Sekeres MA, Lichtin A, Advani A, Kalaycio M, Tiu RV, O’Keefe CL, Maciejewski JP (2011) Efficacy of rabbit anti-thymocyte globulin in severe aplastic anemia. Haematologica 96(9):1269–1275

    Article  CAS  PubMed Central  PubMed  Google Scholar 

  • Atta EH, Dias DS, Marra VL, de Azevedo AM (2010) Comparison between horse and rabbit antithymocyte globulin as first-line treatment for patients with severe aplastic anemia: single center retrospective study. Ann Hematol 89(9):851–859

    Article  CAS  PubMed  Google Scholar 

  • Bagby GC, Lipton JM, Sloand EM, Schiffer CA (2004) Marrow failure. In: Hematology: American Society of Hematology education program book, pp 318–336

    Google Scholar 

  • Camitta BM (2000) What is the definition of cure for aplastic anemia? Acta Haematol 103:16–18

    Article  CAS  PubMed  Google Scholar 

  • Chang MH, Kim KH, Kim HS, Jun HJ, Kim DH, Jang JH, Kim K, Jung CW (2010) Predictors of response to immunosuppressive therapy with antithymocyte globulin and cyclosporine and prognostic factors for survival in patients with severe aplastic anemia. Eur J Haematol 84(2):154–159

    Article  CAS  PubMed  Google Scholar 

  • Chen C, Xue HM, Xu HG, Li Y, Huang K, Zhou DH, Guo HX, Fang JP, Huang SL (2012) Rabbit-antithymocyte globulin combined with cyclosporine A as a first-line therapy: improved, effective, and safe for children with acquired severe aplastic anemia. J Cancer Res Clin Oncol 138(7):1105–1111

    Article  CAS  PubMed  Google Scholar 

  • Deyell RJ, Shereck EB, Milner RA, Schultz KR (2011) Immunosuppressive therapy without hematopoietic growth factor exposure in pediatric acquired aplastic anemia. Pediatr Hematol Oncol 28(6):469–478

    Article  CAS  PubMed  Google Scholar 

  • Di Bona E, Rodeghiero F, Bruno B, Gabbas A, Foa P, Locasciulli A, Rosanelli C, Camba L, Saracco P, Lippi A, Lori AP, Porta F, De Rossi G, Comotti B, Lacopino P, Dufour C, Bacigalupo A (1999) Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol 107(2):330–334

    Article  PubMed  Google Scholar 

  • Führer M, Rampf U, Baumann I, Faldum A, Niemeyer C, Janka-Schaub G, Friedrich W, Ebell W, Borkhardt A, Bender-Goetze C (2005) Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. Blood 106(6):2102–2104

    Article  PubMed  Google Scholar 

  • Gluckman E, Rokicka-Milewska R, Hann I, Nikiforakis E, Tavakoli F, Cohen-Scali S, Bacigalupo A, European Group for Blood and Marrow Transplantation Working Party for Severe Aplastic Anemia (2002) Results and follow-up of a phase III randomized study of recombinant human-granulocyte stimulating factor as support for immunosuppressive therapy in patients with severe aplastic anemia. Br J Haematol 119(4):1075–1082

    Article  CAS  PubMed  Google Scholar 

  • Kojima S, Hibi S, Kosaka Y, Yamamoto M, Tsuchida M, Mugishima H, Sugita K, Yabe H, Ohara A, Tsukimoto I (2000) Immunosuppressive therapy using antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony stimulating factor in children with acquired aplastic anemia. Blood 96:2049–2054

    CAS  PubMed  Google Scholar 

  • Marsh JC, Bacigalupo A, Schrezenmeier H, Tichelli A, Risitano AM, Passweg JR, Killick SB, Warren AJ, Foukaneli T, Aljurf M, Al-Zahrani HA, Höchsmann B, Schafhausen P, Roth A, Franzke A, Brummendorf T, Dufour C, Oneto R, Sedgwick P, Barrois A, Kordasti S, Elebute MO, Mufti GJ, Socie G, European Blood and Marrow Transplant Group Severe Aplastic Anaemia Working Party (2012) Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood 119(23):5391–5396

    Article  CAS  PubMed  Google Scholar 

  • Ochocka M, Karwacki M, Matysiak M (1995) Results of acquired aplastic anemia in children. Pediatr Pol 30:205–209

    Google Scholar 

  • Passweg JR, Marsh JC (2010) Aplastic anemia: first-line treatment by immunosuppression and sibling marrow transplantation. In: Hematology: American Society of Hematology education program book, pp 36–42

    Google Scholar 

  • Pawelec K, Matysiak M, Niewiadomska E, Rokicka-Milewska R, Kowalczyk J, Stefaniak J, Balwierz W, Załecka-Czerpko E, Chybicka A, Szmyd K, Sońta-Jakimczyk D, Bubała H, Krauze A, Wysocki M, Kurylak A, Wachowiak J, Grund G, Młynarski W, Bulas M, Krawczuk-Rybak M, Leszczyńska E, Urasiński T, Peregud-Pogorzelski J, Balcerska A, Wlazłowski M (2008) Results of immunosupressive therapy in children with severe aplastic anaemia. Report of the Polish Paediatric Haematology Group. Med Wieku Rozwoj 12:1092–1097

    PubMed  Google Scholar 

  • Pulsipher MA, Young NS, Tolar J, Risitano AM, Deeg HJ, Anderlini P, Calado R, Kojima S, Eapen M, Harris R, Scheinberg P, Savage S, Maciejewski JP, Tiu RV, Di Fronzo N, Horowitz MM, Antin JH (2011) Optimization of therapy for severe aplastic anemia based on clinical, biologic, and treatment response parameters: conclusions of an international working group on severe aplastic anemia convened by the Blood and Marrow Transplant Clinical Trials Network, March 2010. Biol Blood Marrow Transplant 17(3):291–299

    Article  PubMed Central  PubMed  Google Scholar 

  • Salamonowicz M, Pawelec K, Matysiak M, Kowalczyk J, Balwierz W, Zaleska-Czepko E, Chybicka A, Szmyd K, Szczepanski T, Bubala H, Wysocki M, Kurylak A, Wachowiak J, Szpecht D, Mlynarski W, Bulas M, Krawczuk-Rybak M, Panasiuk A, Leszczynska E, Urasinski T, Peregud-Pogorzelski J, Balcerska A, Kaczorowska-Hac B (2011) Results of treatment of severe aplastic anaemia in children using rabbit antithymocyte globulin (r-ATG). Blood 118(21):3435

    Google Scholar 

  • Samarasinghe S, Webb DK (2012) How I manage aplastic anaemia in children. Br J Haematol 157(1):26–40

    Article  CAS  PubMed  Google Scholar 

  • Saracco P, Quarello P, Lori AP, Zecca M, Longoni D, Svahn J, Varotto S, Del Vecchio GC, Dufour C, Ramenghi U, Bacigalupo A, Locasciulli A, Bone Marrow Failure Study Group of the AIEOP (Italian Association of Paediatric Haematology Oncology) (2008) Cyklosporin A response and dependence in children with acquired aplastic anaemia: multicentre retrospective study with long –term observation follow-up. Br J Haematol 140:197–205

    Article  CAS  PubMed  Google Scholar 

  • Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS (2011) Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. New Engl J Med 365(5):430–438

    Article  CAS  PubMed Central  PubMed  Google Scholar 

  • Schrezenmeier H, Marin P, Raghavachar A, Mc Cann S, Hows J, Gluckman E, Nissen C, van’t Veer-Korthof ET, Ljungman P, Hinterberger W (1993) Relapse of aplastic anemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working Party. Br J Haematol 85(2):371–377

    Article  CAS  PubMed  Google Scholar 

  • Socie G, Mary JY, Schrezenmeier H, Marsh J, Bacigalupo A, Locasciulli A, Führer M, Bekassy A, Tichelli A, Passweg J (2007) Granulocyte-stimulating factor and severe aplastic anemia: a survey by the European Group for Blood and Marrow Transplantation (EBMT). Blood 109(7):2794–2796

    CAS  PubMed  Google Scholar 

  • Takahashi Y, Muramatsu H, Sakata N, Hyakuna N, Hamamoto K, Kobayashi R, Ito E, Yagasaki H, Ohara A, Kikuchi A, Morimoto A, Yabe H, Kudo K, Watanabe K, Ohga S, Kojima S, Japan Childhood Aplastic Anemia Study Group (2013) Rabbit antithymocyte globulin and cyclosporine as first-line therapy for children with acquired aplastic anemia. Blood 121(5):862–863

    Article  CAS  PubMed  Google Scholar 

  • Yoshida N, Yagasaki H, Hama A, Takahashi Y, Kosaka Y, Kobayashi R, Yabe H, Kaneko T, Tsuchida M, Ohara A, Nakahata T, Kojima S (2011) Predicting response to immunosuppressive therapy in childhood aplastic anemia. Haematologica 96(5):771–774

    Article  PubMed Central  PubMed  Google Scholar 

  • Zheng Y, Liu Y, Chu Y (2006) Immunosuppressive therapy for acquired severe aplastic anemia (SAA) a prospective comparison of four different regiments. Exp Hematol 34:826–831

    Article  CAS  PubMed  Google Scholar 

Download references

Conflicts of Interest

The authors report no conflicts of interest in relation to this article.

Author information

Authors and Affiliations

  1. Department of Pediatric, Hematology and Oncology, Medical University of Warsaw, 24 Marszalkowska St., Warsaw, 00-576, Poland

    K. Pawelec, M. Salamonowicz & M. Matysiak

  2. Department of Pediatrics, Oncology and Hematology, Medical University of Bialystok, Bialystok, Poland

    A. Panasiuk, M. Krawczuk-Rybak & E. Leszczynska

  3. Department of Laboratory Diagnostics and Clinical Immunology of Developmental Age, Medical University of Warsaw, Warsaw, Poland

    U. Demkow

  4. Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, Lublin, Poland

    J. Kowalczyk

  5. Department of Pediatric Oncology and Hematology, Jagiellonian University Medical College, Krakow, Poland

    W. Balwierz & E. Zaleska-Czepko

  6. Department and Clinic of Pediatric Oncology, Hematology and Bone Marrow Transplantation, Medical University of Wroclaw, Wroclaw, Poland

    A. Chybicka & K. Szmyd

  7. Department of Pediatric Hematology and Oncology, Medical University of Silesia, Zabrze, Poland

    T. Szczepanski & H. Bubala

  8. Department of Pediatric Hematology, Oncology and Transplantology, Collegium Medicum in Bydgoszcz, Mikolaj Kopernik University, Bydgoszcz, Poland

    M. Wysocki & A. Kurylak

  9. Department of Pediatric Hematology, Oncology and Transplantology, University of Medical Sciences, Poznan, Poland

    J. Wachowiak & D. Szpecht

  10. Department of Pediatric Oncology, Hematology and Diabetology, Medical University of Lodz, Lodz, Poland

    W. Młynarski & M. Bulas

  11. Department of Pediatrics, Hematology and Oncology, Pomeranian Medical University, Szczecin, Poland

    T. Urasinski & J. Peregud-Pogorzelski

  12. Department of Pediatric, Hematology, Oncology and Endocrinology, Medical University of Gdansk, Gdansk, Poland

    A. Balcerska & B. Kaczorowska-Hac

Authors
  1. K. Pawelec
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  2. M. Salamonowicz
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  3. A. Panasiuk
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  6. W. Balwierz
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  7. E. Zaleska-Czepko
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  8. A. Chybicka
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  9. K. Szmyd
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  10. T. Szczepanski
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  11. H. Bubala
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  12. M. Wysocki
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  13. A. Kurylak
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  14. J. Wachowiak
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  15. D. Szpecht
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  16. W. Młynarski
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  17. M. Bulas
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  18. M. Krawczuk-Rybak
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  19. E. Leszczynska
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  20. T. Urasinski
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  21. J. Peregud-Pogorzelski
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  22. A. Balcerska
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  23. B. Kaczorowska-Hac
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  24. M. Matysiak
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Corresponding author

Correspondence to K. Pawelec .

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Editors and Affiliations

  1. Institute of Psychology, Opole University, Poland

    Mieczyslaw Pokorski

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Pawelec, K. et al. (2014). First-Line Immunosuppressive Treatment in Children with Aplastic Anemia: Rabbit Antithymocyte Globulin. In: Pokorski, M. (eds) Respiratory Virology and Immunogenicity. Advances in Experimental Medicine and Biology(), vol 836. Springer, Cham. https://doi.org/10.1007/5584_2014_38

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