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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Outcomes and healthcare utilization in children and young adults with aplastic anemia: A multiinstitutional analysis
Ashish Gupta1, Pingfu Fu2, Hasan Hashem3
1Division of Pediatric Hematology and Oncology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Insights
Hematopoietic stem cell transplant (HCT) offers improved outcomes for severe aplastic anemia in young patients. Early consideration of HCT with alternative donors is recommended due to comparable survival rates and reduced healthcare utilization.
Area of Science:
- Pediatric Hematology
- Bone Marrow Failure Syndromes
- Transplantation Immunology
Background:
- Aplastic anemia is a life-threatening bone marrow failure syndrome predominantly affecting children and young adults.
- Current guidelines prioritize hematopoietic stem cell transplant (HCT) for patients with matched sibling donors.
- Outcomes for HCT using alternative donor sources are progressively improving.
Purpose of the Study:
- To evaluate the efficacy and outcomes of upfront versus salvage hematopoietic stem cell transplant (HCT) in pediatric and young adult patients with severe aplastic anemia.
- To analyze healthcare utilization associated with different HCT strategies.
- To assess trends in treatment mortality and graft failure rates over time.
Main Methods:
- Analysis of a multi-institutional pediatric cohort using the Pediatric Health Information System (PHIS) database (2006-2015).
- Inclusion of 2,169 patients diagnosed with aplastic anemia (AA).
- Comparison of outcomes between upfront and salvage HCT, including mortality, healthcare utilization, and transfusion requirements.
Main Results:
- Nearly 20% of patients underwent HCT; the remainder received immunosuppressive therapy.
- No significant difference in mortality was observed between upfront and salvage HCT (OR 1.24, P = 0.567).
- Each platelet transfusion correlated with increased mortality (OR 1.37, P = 0.002). Salvage transplants showed higher healthcare utilization but reduced mortality and graft failure in recent years (2011-2015).
Conclusions:
- Hematopoietic stem cell transplant (HCT) outcomes for severe aplastic anemia (AA) are improving.
- Upfront HCT using suitable alternative donors should be considered for pediatric and young adult patients.
- Reducing reliance on transfusions may be crucial for improving survival outcomes.
Background:
Aplastic anemia is a bone marrow failure syndrome with high mortality affecting children and young adults. Although current treatment guidelines recommend hematopoietic stem cell transplant (HCT) for patients with matched sibling donors, outcomes with alternate donor options have been improving.
Procedure:
We analyzed a validated multiinstitutional pediatric cohort using one of the largest pediatric and young adult database, the Pediatric Health Information System, for patients diagnosed with aplastic anemia (AA) from 2006 to 2015. Outcomes with upfront and salvage transplants were analyzed along with healthcare utilization.
Results:
Among 2,169 patients in the study period, almost 20% underwent HCT, while others received immunosuppressive therapy. In a multivariate model, there was no significant difference in mortality with upfront or salvage transplants (odds ratio [OR] 1.24, 95% confidence interval [CI] 0.6-2.58, P = 0.567), while every platelet transfusion was associated with higher mortality (OR 1.37, 95% CI 1.12-1.67, P = 0.002). Healthcare utilization was significantly higher in salvage transplants requiring frequent hospitalization and transfusion requirements. Treatment mortality and graft failure rates were significantly reduced in the salvage transplant group in recent years (2011-2015 as compared to 2006-2010).
Conclusion:
As outcomes with HCT continue to improve in severe AA, transplant with good alternate donors should be considered upfront in children and young adults.
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