Skip to main content
Erschienen in: Indian Journal of Pediatrics 12/2012

01.12.2012 | Original Article

Immunosuppressive Therapy in Aplastic Anemia

verfasst von: Vineeta Gupta, Akash Kumar, Vijai Tilak, Isha Saini, Baldev Bhatia

Erschienen in: Indian Journal of Pediatrics | Ausgabe 12/2012

Einloggen, um Zugang zu erhalten

Abstract

Objective

To assess the response to antithymocyte globulin based immunosuppressive therapy (IST) in pediatric patients with idiopathic aplastic anemia.

Methods

Thirty patients (19 boys and 11 girls) with aplastic anemia received antithymocyte globulin and cyclosporine. Twenty-two patients had severe and 8 had very severe aplastic anemia.

Results

Mean age of the patients was 9.19 ± 2.56 y. Three patients died within 1 mo of therapy, two due to sepsis and one due to intracranial hemorrhage. Twenty-seven patients were analyzed for response to therapy. Eight patients (29.7%) responded at 3 mo: 3 complete response (CR) and 5 partial response (PR). Six mo after the therapy, overall response (OR) was seen in 9/27 (33.3%), with one more patient in no response group achieving partial response. At 1 year, patients in CR maintained their status and 1 patient in PR group relapsed. He again achieved partial response with repeat course of ATG. Responders had significantly shorter duration of illness and higher absolute neutrophil count as compared to non responders to IST. None of the patients developed acute leukemia in the follow up.

Conclusions

The treatment of aplastic anemia in pediatric patients is a challenging task. One third of the patients achieved overall response which included both complete and partial response.
Literatur
1.
Zurück zum Zitat Young NS. Acquired aplastic anemia. Annals Int Med. 2002;136:534–46. Young NS. Acquired aplastic anemia. Annals Int Med. 2002;136:534–46.
2.
Zurück zum Zitat Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006;108:2509–19.PubMedCrossRef Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006;108:2509–19.PubMedCrossRef
3.
Zurück zum Zitat Rosenfeld SE, Kimball J, Vining D, Young NS. Intensive immunosuppression with Antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood. 1995;85:3058–65.PubMed Rosenfeld SE, Kimball J, Vining D, Young NS. Intensive immunosuppression with Antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood. 1995;85:3058–65.PubMed
4.
Zurück zum Zitat International Agranulocytosis and Aplastic Anaemia study. Incidence of aplastic anemia: relevance of diagnostic criteria. Blood. 1987;70:1718–21. International Agranulocytosis and Aplastic Anaemia study. Incidence of aplastic anemia: relevance of diagnostic criteria. Blood. 1987;70:1718–21.
5.
Zurück zum Zitat Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975;45:355–63.PubMed Camitta BM, Rappeport JM, Parkman R, Nathan DG. Selection of patients for bone marrow transplantation in severe aplastic anemia. Blood. 1975;45:355–63.PubMed
6.
Zurück zum Zitat Bacigalupo A, Hows JM, Gluckman E, et al. Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA Working Party. Br J Haematol. 1988;70:177–82.PubMedCrossRef Bacigalupo A, Hows JM, Gluckman E, et al. Bone marrow transplantation (BMT) versus immunosuppression for the treatment of severe aplastic anaemia (SAA): a report of the EBMT SAA Working Party. Br J Haematol. 1988;70:177–82.PubMedCrossRef
7.
Zurück zum Zitat Marsh JCW, Ball SE, Darbyshire P, et al. Guidelines for the diagnosis and management of acquired aplastic anemia. Br J Haematol. 2003;123:782–801.PubMedCrossRef Marsh JCW, Ball SE, Darbyshire P, et al. Guidelines for the diagnosis and management of acquired aplastic anemia. Br J Haematol. 2003;123:782–801.PubMedCrossRef
8.
Zurück zum Zitat Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H. German aplastic anemia study group. Blood. 2003;101:1236–42.PubMedCrossRef Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H. German aplastic anemia study group. Blood. 2003;101:1236–42.PubMedCrossRef
9.
Zurück zum Zitat Frickhofen N, Rosenfeld SJ. Immunosuppressive therapy of aplastic anemia with antithymocyte globulin and cyclosporine. Semin Hematol. 2000;37:56–68.PubMedCrossRef Frickhofen N, Rosenfeld SJ. Immunosuppressive therapy of aplastic anemia with antithymocyte globulin and cyclosporine. Semin Hematol. 2000;37:56–68.PubMedCrossRef
10.
Zurück zum Zitat Guinan EC. Acquired aplastic anemia in childhood. Haematol Oncol Clin North Am. 2009;23:171–91.CrossRef Guinan EC. Acquired aplastic anemia in childhood. Haematol Oncol Clin North Am. 2009;23:171–91.CrossRef
11.
Zurück zum Zitat Marwaha RK, Bansal D, Trehan A, Varma N. Androgens in childhood acquired aplastic anemia in Chandigarh, India. Trop Doc. 2004;34:149–52. Marwaha RK, Bansal D, Trehan A, Varma N. Androgens in childhood acquired aplastic anemia in Chandigarh, India. Trop Doc. 2004;34:149–52.
12.
Zurück zum Zitat Rai M, Singh VP, Shukla J, et al. Low dose cyclosporine- a therapy in severe aplastic anemia. J Assoc Physicians India. 2001;49:966–99.PubMed Rai M, Singh VP, Shukla J, et al. Low dose cyclosporine- a therapy in severe aplastic anemia. J Assoc Physicians India. 2001;49:966–99.PubMed
13.
Zurück zum Zitat Chandra J, Naithani R, Ravi R, et al. Antithymocyte globulin and cyclosporine in children with acquired aplastic anemia. Indian J Pediatr. 2008;75:229–33.PubMedCrossRef Chandra J, Naithani R, Ravi R, et al. Antithymocyte globulin and cyclosporine in children with acquired aplastic anemia. Indian J Pediatr. 2008;75:229–33.PubMedCrossRef
14.
Zurück zum Zitat George B, Mathews V, Viswabandya A, Lakshmi KM, Srivastava A, Chandy M. Allogeneic hematopoetic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemia-a single center analysis of 100 patients. Pediatr Hematol Oncol. 2010;27:122–31.PubMedCrossRef George B, Mathews V, Viswabandya A, Lakshmi KM, Srivastava A, Chandy M. Allogeneic hematopoetic stem cell transplantation is superior to immunosuppressive therapy in Indian children with aplastic anemia-a single center analysis of 100 patients. Pediatr Hematol Oncol. 2010;27:122–31.PubMedCrossRef
15.
Zurück zum Zitat Bacigalupo A, Bruno B, Saracco P. Anti thymocyte globulin, cyclosporine, prednisolone and granulocyte colony-stimulating factor for severe aplastic anemia: an update of the GITMO/EBMT study on 100 patients: European Group for Blood and Marrow Transplantation (EBMT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti di Midolio Osseo (GITMO). Blood. 2000;95:1931–4.PubMed Bacigalupo A, Bruno B, Saracco P. Anti thymocyte globulin, cyclosporine, prednisolone and granulocyte colony-stimulating factor for severe aplastic anemia: an update of the GITMO/EBMT study on 100 patients: European Group for Blood and Marrow Transplantation (EBMT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti di Midolio Osseo (GITMO). Blood. 2000;95:1931–4.PubMed
16.
Zurück zum Zitat Di Bona E, Rodeghiero F, Bruno BA, et al. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anemia unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol. 1999;107:330–4.PubMedCrossRef Di Bona E, Rodeghiero F, Bruno BA, et al. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anemia unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol. 1999;107:330–4.PubMedCrossRef
17.
Zurück zum Zitat Kamio T, Ito E, Ohara A, Kosaka Y, et al. Relapse of aplastic anemia in children after immunosuppressive therapy: A report from the Japan Childhood aplastic anemia study group. Haematologica. 2011;96:814–9.PubMedCrossRef Kamio T, Ito E, Ohara A, Kosaka Y, et al. Relapse of aplastic anemia in children after immunosuppressive therapy: A report from the Japan Childhood aplastic anemia study group. Haematologica. 2011;96:814–9.PubMedCrossRef
18.
Zurück zum Zitat Gupta V, Gordon-Smith EC, Cook G, et al. A third course of anti-thymocyte globulin in aplastic anemia is only beneficial in previous responders. Br J Haematol. 2005;129:110–7.PubMedCrossRef Gupta V, Gordon-Smith EC, Cook G, et al. A third course of anti-thymocyte globulin in aplastic anemia is only beneficial in previous responders. Br J Haematol. 2005;129:110–7.PubMedCrossRef
19.
Zurück zum Zitat Rosenfeld S, Follman D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003;289:1130–5.PubMedCrossRef Rosenfeld S, Follman D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003;289:1130–5.PubMedCrossRef
20.
Zurück zum Zitat Socie G, Rosenfeld S, Frickhofen N, Gluckman E, Tichelli A. Late clonal diseases of treated aplastic anemia. Semin Hematol. 2000;37:91–101.PubMedCrossRef Socie G, Rosenfeld S, Frickhofen N, Gluckman E, Tichelli A. Late clonal diseases of treated aplastic anemia. Semin Hematol. 2000;37:91–101.PubMedCrossRef
21.
Zurück zum Zitat Ohara A, Kojima S, Hamajima N, et al. Myelodysplastic syndrome and acute myeloid leukemia as a late complication in children with acquired aplastic anemia. Blood. 1997;90:1009–13.PubMed Ohara A, Kojima S, Hamajima N, et al. Myelodysplastic syndrome and acute myeloid leukemia as a late complication in children with acquired aplastic anemia. Blood. 1997;90:1009–13.PubMed
22.
Zurück zum Zitat Kojima S, Hibi S, Kosaka Y, et al. Immunosuppressive therapy using Antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood. 2000;96:2049–54.PubMed Kojima S, Hibi S, Kosaka Y, et al. Immunosuppressive therapy using Antithymocyte globulin, cyclosporine, and danazol with or without human granulocyte colony-stimulating factor in children with acquired aplastic anemia. Blood. 2000;96:2049–54.PubMed
Metadaten
Titel
Immunosuppressive Therapy in Aplastic Anemia
verfasst von
Vineeta Gupta
Akash Kumar
Vijai Tilak
Isha Saini
Baldev Bhatia
Publikationsdatum
01.12.2012
Verlag
Springer-Verlag
Erschienen in
Indian Journal of Pediatrics / Ausgabe 12/2012
Print ISSN: 0019-5456
Elektronische ISSN: 0973-7693
DOI
https://doi.org/10.1007/s12098-012-0691-2

Weitere Artikel der Ausgabe 12/2012

Indian Journal of Pediatrics 12/2012 Zur Ausgabe

Symposium on Pediatric HIV/AIDS

Editorial: Recent Advances in HIV Infection

Häufigste Gründe für Brustschmerzen bei Kindern

06.05.2024 Pädiatrische Diagnostik Nachrichten

Akute Brustschmerzen sind ein Alarmsymptom par exellence, schließlich sind manche Auslöser lebensbedrohlich. Auch Kinder klagen oft über Schmerzen in der Brust. Ein Studienteam ist den Ursachen nachgegangen.

Endlich: Zi zeigt, mit welchen PVS Praxen zufrieden sind

IT für Ärzte Nachrichten

Darauf haben viele Praxen gewartet: Das Zi hat eine Liste von Praxisverwaltungssystemen veröffentlicht, die von Nutzern positiv bewertet werden. Eine gute Grundlage für wechselwillige Ärzte und Psychotherapeuten.

Durch übermäßige Internetnutzung wird oft die Schule verpasst

Häufige Fehlzeiten in der Schule können durch physische und psychische Probleme verursacht werden. Wie in einer Studie aus Finnland nun belegt wird, führt auch die exzessive Nutzung des Internets gehäuft zu Abwesenheiten.

Kinder mit anhaltender Sinusitis profitieren häufig von Antibiotika

30.04.2024 Rhinitis und Sinusitis Nachrichten

Persistieren Sinusitisbeschwerden bei Kindern länger als zehn Tage, ist eine Antibiotikatherapie häufig gut wirksam: Ein Therapieversagen ist damit zu über 40% seltener zu beobachten als unter Placebo.

Update Pädiatrie

Bestellen Sie unseren Fach-Newsletter und bleiben Sie gut informiert.