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Updated: May 15, 2026
Induction of Alloantigen-specific Anergy in Human Peripheral Blood Mononuclear Cells by Alloantigen Stimulation with Co-stimulatory Signal Blockade
Published on: March 14, 2011
Management of acquired aplastic anemia in children
E T Korthof1, A N Békássy, A A Hussein
1Department of Pediatrics/Willem-Alexander Children's Hospital, Division of Stem Cell Transplantation, Leiden University Medical Center, Leiden, The Netherlands. l.korthof@sanquin.nl
Insights
Diagnosing pediatric aplastic anemia requires ruling out other bone marrow (BM) failure syndromes. Prompt diagnosis and treatment, including hematopoietic stem cell transplant (SCT) or immunosuppressive therapy, improve outcomes.
Area of Science:
- Pediatric Hematology
- Bone Marrow Failure Syndromes
- Aplastic Anemia
Background:
- Diagnosing aplastic anemia in children necessitates excluding inherited and acquired bone marrow (BM) failure syndromes with similar presentations.
- Timely diagnosis and initiation of treatment are critical, directly impacting patient outcomes irrespective of the chosen therapeutic strategy.
Purpose of the Study:
- To outline an efficient diagnostic plan for pediatric aplastic anemia.
- To review current therapeutic options and long-term management strategies for children with aplastic anemia.
Main Methods:
- Review of diagnostic criteria for aplastic anemia and related BM failure syndromes.
- Analysis of treatment outcomes for hematopoietic stem cell transplantation (SCT) and immunosuppressive therapy (IST).
- Evaluation of strategies for managing refractory or relapsed cases and long-term complications.
Main Results:
- Hematopoietic SCT is the gold standard for patients with matched sibling donors.
- Combined immunosuppressive therapy, particularly horse-derived anti-thymocyte globulin plus cyclosporine A, shows high response and survival rates in patients without sibling donors.
- Incomplete response, relapse, and progression to myelodysplasia/leukemia are significant long-term concerns.
Conclusions:
- Effective management of pediatric aplastic anemia relies on accurate diagnosis and timely, appropriate treatment.
- Alternative donor SCT and novel immunosuppressive agents offer options for refractory or relapsed disease.
- Centralized care at centers of excellence with ongoing monitoring is essential for managing immediate and long-term effects.
Abstract:
The diagnosis of aplastic anemia in children requires exclusion of a variety of inherited or acquired BM failure syndromes with similar phenotypes. An efficient diagnostic plan is important because time from diagnosis to 'final' treatment is directly related to outcome regardless of the therapeutic option chosen. The gold standard of therapy remains hematopoietic SCT with a graft of BM cells for those children with matched sibling donors. Conversely for children without a sibling donor the high response and markedly improved overall survival rates of combined immunosuppressive therapy have proven robust, especially when horse derived anti-thymocyte globuline plus ciclosporine A are used. Incomplete response, relapse and progression to myelodysplasia/leukemia however have emerged as significant long-term issues. Improvements in outcome of alternative donor transplantation and the use of established and novel immunosuppressive agents provide multiple alternatives for treating refractory or relapsed patients. Regardless of the type of therapeutic approach, patients require centralized treatment in a center of excellence, ongoing monitoring for recurrence of disease and/or therapy-related immediate side effects and long-term effects.
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