Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin

Detalhes bibliográficos
Autor(a) principal: de-Medeiros,C.R.
Data de Publicação: 2000
Outros Autores: Ribeiro,R.C., Bittencourt,M.A., Zanis-Neto,J., Pasquini,R.
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Brazilian Journal of Medical and Biological Research
Texto Completo: http://old.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2000000500010
Resumo: Severe aplastic anemia (SAA) is probably an immune-mediated disorder, and immunosuppressive therapy is recommended for patients with no available donor for bone marrow transplant. Between October 1984 and November 1987, 25 consecutive children and adolescents with SAA with no HLA-compatible marrow donor received equine antithymocyte globulin (ATG) (15 mg kg-1 day-1) for 10 days. The patients were evaluated 6 weeks, 6 months, and 12 months after starting ATG treatment. Thereafter, patients were evaluated yearly until July 1998. Median age was 10 years (range, 1.5-20 years), granulocyte counts on referral ranged from 0.032 to 1.4 x 10(9)/l (median 0.256 x 10(9)/l), and 12 patients had granulocyte counts <0.2 x 10(9)/l. At a median follow-up of 9.6 years (range, 8.6-11.8 years), 10 patients (40%) remained alive with good marrow function. No morphologic evidence of hematological clonal disorders has been observed, although two patients probably have acquired clonal chromosomal abnormalities (trisomy 8 and del(6)q21, respectively). Responses to ATG were observed between 6 weeks and 6 months from the start of treatment in 60% of evaluable patients. The response rate was not different in patients whose granulocyte count at diagnosis was <0.2 x 10(9)/l, or in those who were <10 years of age. This study supports the view that, when compared with supportive measures, ATG is an effective treatment for children or adolescents with SAA. Although these results are inferior to those reported for marrow transplantation or more intensive immunosuppressive regimens, these patients who responded to ATG are long-term survivors with stable peripheral blood counts and a low rate of relapse.
id ABDC-1_8b5faf526e180435adaa06d0a0c732c0
oai_identifier_str oai:scielo:S0100-879X2000000500010
network_acronym_str ABDC-1
network_name_str Brazilian Journal of Medical and Biological Research
repository_id_str
spelling Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulinaplastic anemiachildrenadolescentsantithymocyte globulinSevere aplastic anemia (SAA) is probably an immune-mediated disorder, and immunosuppressive therapy is recommended for patients with no available donor for bone marrow transplant. Between October 1984 and November 1987, 25 consecutive children and adolescents with SAA with no HLA-compatible marrow donor received equine antithymocyte globulin (ATG) (15 mg kg-1 day-1) for 10 days. The patients were evaluated 6 weeks, 6 months, and 12 months after starting ATG treatment. Thereafter, patients were evaluated yearly until July 1998. Median age was 10 years (range, 1.5-20 years), granulocyte counts on referral ranged from 0.032 to 1.4 x 10(9)/l (median 0.256 x 10(9)/l), and 12 patients had granulocyte counts <0.2 x 10(9)/l. At a median follow-up of 9.6 years (range, 8.6-11.8 years), 10 patients (40%) remained alive with good marrow function. No morphologic evidence of hematological clonal disorders has been observed, although two patients probably have acquired clonal chromosomal abnormalities (trisomy 8 and del(6)q21, respectively). Responses to ATG were observed between 6 weeks and 6 months from the start of treatment in 60% of evaluable patients. The response rate was not different in patients whose granulocyte count at diagnosis was <0.2 x 10(9)/l, or in those who were <10 years of age. This study supports the view that, when compared with supportive measures, ATG is an effective treatment for children or adolescents with SAA. Although these results are inferior to those reported for marrow transplantation or more intensive immunosuppressive regimens, these patients who responded to ATG are long-term survivors with stable peripheral blood counts and a low rate of relapse.Associação Brasileira de Divulgação Científica2000-05-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersiontext/htmlhttp://old.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2000000500010Brazilian Journal of Medical and Biological Research v.33 n.5 2000reponame:Brazilian Journal of Medical and Biological Researchinstname:Associação Brasileira de Divulgação Científica (ABDC)instacron:ABDC10.1590/S0100-879X2000000500010info:eu-repo/semantics/openAccessde-Medeiros,C.R.Ribeiro,R.C.Bittencourt,M.A.Zanis-Neto,J.Pasquini,R.eng2000-04-20T00:00:00Zoai:scielo:S0100-879X2000000500010Revistahttps://www.bjournal.org/https://old.scielo.br/oai/scielo-oai.phpbjournal@terra.com.br||bjournal@terra.com.br1414-431X0100-879Xopendoar:2000-04-20T00:00Brazilian Journal of Medical and Biological Research - Associação Brasileira de Divulgação Científica (ABDC)false
dc.title.none.fl_str_mv Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
title Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
spellingShingle Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
de-Medeiros,C.R.
aplastic anemia
children
adolescents
antithymocyte globulin
title_short Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
title_full Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
title_fullStr Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
title_full_unstemmed Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
title_sort Long-term outcome of 25 children and adolescents with severe aplastic anemia treated with antithymocyte globulin
author de-Medeiros,C.R.
author_facet de-Medeiros,C.R.
Ribeiro,R.C.
Bittencourt,M.A.
Zanis-Neto,J.
Pasquini,R.
author_role author
author2 Ribeiro,R.C.
Bittencourt,M.A.
Zanis-Neto,J.
Pasquini,R.
author2_role author
author
author
author
dc.contributor.author.fl_str_mv de-Medeiros,C.R.
Ribeiro,R.C.
Bittencourt,M.A.
Zanis-Neto,J.
Pasquini,R.
dc.subject.por.fl_str_mv aplastic anemia
children
adolescents
antithymocyte globulin
topic aplastic anemia
children
adolescents
antithymocyte globulin
description Severe aplastic anemia (SAA) is probably an immune-mediated disorder, and immunosuppressive therapy is recommended for patients with no available donor for bone marrow transplant. Between October 1984 and November 1987, 25 consecutive children and adolescents with SAA with no HLA-compatible marrow donor received equine antithymocyte globulin (ATG) (15 mg kg-1 day-1) for 10 days. The patients were evaluated 6 weeks, 6 months, and 12 months after starting ATG treatment. Thereafter, patients were evaluated yearly until July 1998. Median age was 10 years (range, 1.5-20 years), granulocyte counts on referral ranged from 0.032 to 1.4 x 10(9)/l (median 0.256 x 10(9)/l), and 12 patients had granulocyte counts <0.2 x 10(9)/l. At a median follow-up of 9.6 years (range, 8.6-11.8 years), 10 patients (40%) remained alive with good marrow function. No morphologic evidence of hematological clonal disorders has been observed, although two patients probably have acquired clonal chromosomal abnormalities (trisomy 8 and del(6)q21, respectively). Responses to ATG were observed between 6 weeks and 6 months from the start of treatment in 60% of evaluable patients. The response rate was not different in patients whose granulocyte count at diagnosis was <0.2 x 10(9)/l, or in those who were <10 years of age. This study supports the view that, when compared with supportive measures, ATG is an effective treatment for children or adolescents with SAA. Although these results are inferior to those reported for marrow transplantation or more intensive immunosuppressive regimens, these patients who responded to ATG are long-term survivors with stable peripheral blood counts and a low rate of relapse.
publishDate 2000
dc.date.none.fl_str_mv 2000-05-01
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://old.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2000000500010
url http://old.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2000000500010
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 10.1590/S0100-879X2000000500010
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv text/html
dc.publisher.none.fl_str_mv Associação Brasileira de Divulgação Científica
publisher.none.fl_str_mv Associação Brasileira de Divulgação Científica
dc.source.none.fl_str_mv Brazilian Journal of Medical and Biological Research v.33 n.5 2000
reponame:Brazilian Journal of Medical and Biological Research
instname:Associação Brasileira de Divulgação Científica (ABDC)
instacron:ABDC
instname_str Associação Brasileira de Divulgação Científica (ABDC)
instacron_str ABDC
institution ABDC
reponame_str Brazilian Journal of Medical and Biological Research
collection Brazilian Journal of Medical and Biological Research
repository.name.fl_str_mv Brazilian Journal of Medical and Biological Research - Associação Brasileira de Divulgação Científica (ABDC)
repository.mail.fl_str_mv bjournal@terra.com.br||bjournal@terra.com.br
_version_ 1754302930459033600