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Outcomes and healthcare utilization in children and young adults with aplastic anemia: A multiinstitutional analysis
Author(s) -
Gupta Ashish,
Fu Pingfu,
Hashem Hasan,
Vatsayan Anant,
Shein Steven,
Dalal Jignesh
Publication year - 2017
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26704
Subject(s) - medicine , aplastic anemia , salvage therapy , odds ratio , hematopoietic stem cell transplantation , confidence interval , anemia , cohort , young adult , pediatrics , retrospective cohort study , surgery , transplantation , bone marrow , chemotherapy
Abstract 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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