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Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric patients is also limited. The clinical features and...

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Autores principales: Rogers, Zora R., Nakano, Taizo A., Olson, Timothy S., Bertuch, Alison A., Wang, Winfred, Gillio, Alfred, Coates, Thomas D., Chawla, Anjulika, Castillo, Paul, Kurre, Peter, Gamper, Christopher, Bennett, Carolyn M., Joshi, Sarita, Geddis, Amy E., Boklan, Jessica, Nalepa, Grzegorz, Rothman, Jennifer A., Huang, James N., Kupfer, Gary M., Cada, Michaela, Glader, Bertil, Walkovich, Kelly J., Thompson, Alexis A., Hanna, Rabi, Vlachos, Adrianna, Malsch, Maggie, Weller, Edie A., Williams, David A., Shimamura, Akiko
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Ferrata Storti Foundation 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6886407/
https://www.ncbi.nlm.nih.gov/pubmed/30948484
http://dx.doi.org/10.3324/haematol.2018.206540
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author Rogers, Zora R.
Nakano, Taizo A.
Olson, Timothy S.
Bertuch, Alison A.
Wang, Winfred
Gillio, Alfred
Coates, Thomas D.
Chawla, Anjulika
Castillo, Paul
Kurre, Peter
Gamper, Christopher
Bennett, Carolyn M.
Joshi, Sarita
Geddis, Amy E.
Boklan, Jessica
Nalepa, Grzegorz
Rothman, Jennifer A.
Huang, James N.
Kupfer, Gary M.
Cada, Michaela
Glader, Bertil
Walkovich, Kelly J.
Thompson, Alexis A.
Hanna, Rabi
Vlachos, Adrianna
Malsch, Maggie
Weller, Edie A.
Williams, David A.
Shimamura, Akiko
author_facet Rogers, Zora R.
Nakano, Taizo A.
Olson, Timothy S.
Bertuch, Alison A.
Wang, Winfred
Gillio, Alfred
Coates, Thomas D.
Chawla, Anjulika
Castillo, Paul
Kurre, Peter
Gamper, Christopher
Bennett, Carolyn M.
Joshi, Sarita
Geddis, Amy E.
Boklan, Jessica
Nalepa, Grzegorz
Rothman, Jennifer A.
Huang, James N.
Kupfer, Gary M.
Cada, Michaela
Glader, Bertil
Walkovich, Kelly J.
Thompson, Alexis A.
Hanna, Rabi
Vlachos, Adrianna
Malsch, Maggie
Weller, Edie A.
Williams, David A.
Shimamura, Akiko
author_sort Rogers, Zora R.
collection PubMed
description Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric patients is also limited. The clinical features and outcomes for 314 children treated from 2002 to 2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin (hATG) plus cyclosporine (CyA) with a median 61 months follow up. Following hATG/CyA, 71.2% (95%CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response achieved in pediatric patients was high, with 59.8% (95%CI: 53.7,65.8) complete response and 68.2% (95%CI: 62.2,73.8) achieving at least a very good partial response with a platelet count ≥50×10(9)L. At five years post-hATG/CyA, overall survival was 93% (95%CI: 89,96), but event-free survival without subsequent treatment was only 64% (95%CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis after a median 25.2 months (range: 4.3-71 months) post treatment. Myelodysplastic syndrome or leukemia developed in 6 of 314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95%CI: 0.08,0.47; P=0.0003). This study highlights the need for improved therapies to achieve sustained high-quality remission for children with severe aplastic anemia.
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spelling pubmed-68864072019-12-09 Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study Rogers, Zora R. Nakano, Taizo A. Olson, Timothy S. Bertuch, Alison A. Wang, Winfred Gillio, Alfred Coates, Thomas D. Chawla, Anjulika Castillo, Paul Kurre, Peter Gamper, Christopher Bennett, Carolyn M. Joshi, Sarita Geddis, Amy E. Boklan, Jessica Nalepa, Grzegorz Rothman, Jennifer A. Huang, James N. Kupfer, Gary M. Cada, Michaela Glader, Bertil Walkovich, Kelly J. Thompson, Alexis A. Hanna, Rabi Vlachos, Adrianna Malsch, Maggie Weller, Edie A. Williams, David A. Shimamura, Akiko Haematologica Article Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric patients is also limited. The clinical features and outcomes for 314 children treated from 2002 to 2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin (hATG) plus cyclosporine (CyA) with a median 61 months follow up. Following hATG/CyA, 71.2% (95%CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response achieved in pediatric patients was high, with 59.8% (95%CI: 53.7,65.8) complete response and 68.2% (95%CI: 62.2,73.8) achieving at least a very good partial response with a platelet count ≥50×10(9)L. At five years post-hATG/CyA, overall survival was 93% (95%CI: 89,96), but event-free survival without subsequent treatment was only 64% (95%CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis after a median 25.2 months (range: 4.3-71 months) post treatment. Myelodysplastic syndrome or leukemia developed in 6 of 314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95%CI: 0.08,0.47; P=0.0003). This study highlights the need for improved therapies to achieve sustained high-quality remission for children with severe aplastic anemia. Ferrata Storti Foundation 2019-10 /pmc/articles/PMC6886407/ /pubmed/30948484 http://dx.doi.org/10.3324/haematol.2018.206540 Text en Copyright© 2019 Ferrata Storti Foundation Material published in Haematologica is covered by copyright. All rights are reserved to the Ferrata Storti Foundation. Use of published material is allowed under the following terms and conditions: https://creativecommons.org/licenses/by-nc/4.0/legalcode. Copies of published material are allowed for personal or internal use. Sharing published material for non-commercial purposes is subject to the following conditions: https://creativecommons.org/licenses/by-nc/4.0/legalcode, sect. 3. Reproducing and sharing published material for commercial purposes is not allowed without permission in writing from the publisher.
spellingShingle Article
Rogers, Zora R.
Nakano, Taizo A.
Olson, Timothy S.
Bertuch, Alison A.
Wang, Winfred
Gillio, Alfred
Coates, Thomas D.
Chawla, Anjulika
Castillo, Paul
Kurre, Peter
Gamper, Christopher
Bennett, Carolyn M.
Joshi, Sarita
Geddis, Amy E.
Boklan, Jessica
Nalepa, Grzegorz
Rothman, Jennifer A.
Huang, James N.
Kupfer, Gary M.
Cada, Michaela
Glader, Bertil
Walkovich, Kelly J.
Thompson, Alexis A.
Hanna, Rabi
Vlachos, Adrianna
Malsch, Maggie
Weller, Edie A.
Williams, David A.
Shimamura, Akiko
Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
title Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
title_full Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
title_fullStr Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
title_full_unstemmed Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
title_short Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study
title_sort immunosuppressive therapy for pediatric aplastic anemia: a north american pediatric aplastic anemia consortium study
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6886407/
https://www.ncbi.nlm.nih.gov/pubmed/30948484
http://dx.doi.org/10.3324/haematol.2018.206540
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