Biomaterial Database

Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. 2019

Zora R Rogers, and Taizo A Nakano, and Timothy S Olson, and Alison A Bertuch, and Winfred Wang, and Alfred Gillio, and Thomas D Coates, and Anjulika Chawla, and Paul Castillo, and Peter Kurre, and Christopher Gamper, and Carolyn M Bennett, and Sarita Joshi, and Amy E Geddis, and Jessica Boklan, and Grzegorz Nalepa, and Jennifer A Rothman, and James N Huang, and Gary M Kupfer, and Michaela Cada, and Bertil Glader, and Kelly J Walkovich, and Alexis A Thompson, and Rabi Hanna, and Adrianna Vlachos, and Maggie Malsch, and Edie A Weller, and David A Williams, and Akiko Shimamura

Pediatric Hematology/Oncology, University of Texas Southwestern Medical Center, Dallas, TX, USA.

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 ≥50×109L. 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.

UI	MeSH Term	Description	Entries
D007165	Immunosuppression Therapy	Deliberate prevention or diminution of the host's immune response. It may be nonspecific as in the administration of immunosuppressive agents (drugs or radiation) or by lymphocyte depletion or may be specific as in desensitization or the simultaneous administration of antigen and immunosuppressive drugs.	Antirejection Therapy,Immunosuppression,Immunosuppressive Therapy,Anti-Rejection Therapy,Therapy, Anti-Rejection,Therapy, Antirejection,Anti Rejection Therapy,Anti-Rejection Therapies,Antirejection Therapies,Immunosuppression Therapies,Immunosuppressions,Immunosuppressive Therapies,Therapies, Immunosuppression,Therapies, Immunosuppressive,Therapy, Immunosuppression,Therapy, Immunosuppressive
D007223	Infant	A child between 1 and 23 months of age.	Infants
D008297	Male		Males
D002675	Child, Preschool	A child between the ages of 2 and 5.	Children, Preschool,Preschool Child,Preschool Children
D005260	Female		Females
D005500	Follow-Up Studies	Studies in which individuals or populations are followed to assess the outcome of exposures, procedures, or effects of a characteristic, e.g., occurrence of disease.	Followup Studies,Follow Up Studies,Follow-Up Study,Followup Study,Studies, Follow-Up,Studies, Followup,Study, Follow-Up,Study, Followup
D006801	Humans	Members of the species Homo sapiens.	Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000741	Anemia, Aplastic	A form of anemia in which the bone marrow fails to produce adequate numbers of peripheral blood elements.	Anemia, Hypoplastic,Aplastic Anaemia,Aplastic Anemia,Anaemia, Aplastic,Aplastic Anaemias,Aplastic Anemias,Hypoplastic Anemia,Hypoplastic Anemias
D000961	Antilymphocyte Serum	Serum containing GAMMA-GLOBULINS which are antibodies for lymphocyte ANTIGENS. It is used both as a test for HISTOCOMPATIBILITY and therapeutically in TRANSPLANTATION.	ATGAM,Antilymphoblast Globulins,Antilymphocyte Antibodies,Antilymphocyte Globulin,Lymphocytotoxic Antibodies,Anti-Thymocyte Globulin,Antilymphocyte Immunoglobulin,Antithymocyte Globulin,Antithymoglobulin,Lymphocyte Immune Globulin, Anti-Thymocyte Globulin,Lymphocyte Immune Globulin, Anti-Thymocyte Globulin (Equine),Pressimmune,Anti Thymocyte Globulin,Anti-Thymocyte Globulins,Antibodies, Antilymphocyte,Antibodies, Lymphocytotoxic,Antibody, Antilymphocyte,Antibody, Lymphocytotoxic,Antilymphoblast Globulin,Antilymphocyte Antibody,Antilymphocyte Globulins,Antilymphocyte Immunoglobulins,Antilymphocyte Serums,Antithymocyte Globulins,Antithymoglobulins,Globulin, Anti-Thymocyte,Globulin, Antilymphoblast,Globulin, Antilymphocyte,Globulin, Antithymocyte,Globulins, Anti-Thymocyte,Globulins, Antilymphoblast,Globulins, Antilymphocyte,Globulins, Antithymocyte,Immunoglobulin, Antilymphocyte,Immunoglobulins, Antilymphocyte,Lymphocyte Immune Globulin, Anti Thymocyte Globulin,Lymphocytotoxic Antibody,Serum, Antilymphocyte,Serums, Antilymphocyte
D012189	Retrospective Studies	Studies used to test etiologic hypotheses in which inferences about an exposure to putative causal factors are derived from data relating to characteristics of persons under study or to events or experiences in their past. The essential feature is that some of the persons under study have the disease or outcome of interest and their characteristics are compared with those of unaffected persons.	Retrospective Study,Studies, Retrospective,Study, Retrospective