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Updated: Aug 19, 2026
Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies
Published on: September 6, 2017
Diagnosis and treatment of children with aplastic anemia
Peter Kurre1, F Leonard Johnson, H Joachim Deeg
1Division of Pediatric Hematology/Oncology, Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239-3098, USA. kurrepe@ohsu.edu
Insights
Pediatric severe aplastic anemia (SAA) survival is high with hematopoietic stem cell transplantation (HSCT) and immunosuppressive treatment (IST). New treatments are needed for SAA patients unresponsive to current therapies.
Area of Science:
- Pediatric Hematology
- Oncology
- Immunology
Background:
- Survival rates for children with severe aplastic anemia (SAA) have significantly improved.
- Success is attributed to hematopoietic stem cell transplantation (HSCT), immunosuppressive treatment (IST), and supportive care.
- Treatment decisions involve balancing IST-related morbidity with HSCT-related toxicity.
Purpose of the Study:
- To provide an updated risk-based treatment algorithm for pediatric acquired SAA.
- To review recent advancements in alternative donor HSCT and strategies to broaden HSCT eligibility.
- To discuss IST response, relapse risks, and complications like clonal evolution.
Main Methods:
- Review of current literature and clinical experience.
- Analysis of outcomes for alternative donor HSCT.
- Discussion of advancements in donor matching, conditioning regimens, and graft engineering.
Main Results:
- Established treatments offer good outcomes for most pediatric SAA patients.
- Alternative donor HSCT and improved donor matching expand HSCT options.
- IST response rates, relapse, and clonal evolution remain important considerations.
Conclusions:
- Novel non-transplantation therapies are crucial for SAA patients with unresponsive or relapsed disease lacking suitable donors.
- Further understanding of aplastic anemia pathophysiology is essential for improved outcomes.
- Continued research is needed to refine treatment strategies for pediatric SAA.
Background:
Long-term survival rates among children diagnosed with severe aplastic anemia (SAA) are excellent due to the success of human leukocyte antigen (HLA)-identical related hematopoietic stem cell transplantation (HSCT), concurrent advances in immunosuppressive treatment (IST), and improved supportive care. The challenge in making treatment recommendations for children with SAA, therefore, is to balance the apparent chronicity and morbidity following IST, with the potential up-front toxicity and complications of HSCT.
Methods:
This review provides an update on the diagnosis and a risk-based treatment algorithm for children with acquired SAA. Recent experience using alternative donor HSCT and efforts to extend HSCT eligibility through advances in donor matching, de-escalation of conditioning regimens, and potential marrow graft engineering are highlighted. We discuss IST response rates, risks of relapse, and complications including clonal evolution.
Conclusions:
While good treatment options exist for a majority of children diagnosed with SAA, novel non-transplantation treatments for unresponsive and relapsed patients without suitable transplant donors are needed. Further improvements in outcome will ultimately require a more complete understanding of the pathophysiology of aplastic anemia (AA).
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