Outcomes and healthcare utilization in children and young adults with aplastic anemia: A multiinstitutional analysis

Ashish Gupta, Pingfu Fu, Hasan Hashem, Anant Vatsayan, Steven Shein, Jignesh Dalal

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


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.

Original languageEnglish (US)
Article numbere26704
JournalPediatric Blood and Cancer
Issue number12
StatePublished - Dec 2017

Bibliographical note

Funding Information:
This publication was made possible by the Clinical and Translational Science Collaborative of Cleveland (4UL1TR000439) from the National Center for Advancing Translational Sciences (NCATS) component of the National Institutes of Health and NIH roadmap for Medical Research. Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the NIH.

Publisher Copyright:
© 2017 Wiley Periodicals, Inc.


  • aplastic anemia
  • healthcare utilization
  • transplant outcomes
  • treatment algorithm


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