Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

Staci D. Arnold, Ruta Brazauskas, Naya He, Yimei Li, Richard Aplenc, Zhezhen Jin, Matt Hall, Yoshiko Atsuta, Jignesh Dalal, Theresa Hahn, Nandita Khera, Carmem Bonfim, Navneet S. Majhail, Miguel Angel Diaz, Cesar O. Freytes, William A. Wood, Bipin N. Savani, Rammurti T. Kamble, Susan Parsons, Ibrahim AhmedKeith Sullivan, Sara Beattie, Christopher Dandoy, Reinhold Munker, Susana Marino, Menachem Bitan, Hisham Abdel-Azim, Mahmoud Aljurf, Richard F. Olsson, Sarita Joshi, Dave Buchbinder, Michael J. Eckrich, Shahrukh Hashmi, Hillard Lazarus, David I. Marks, Amir Steinberg, Ayman Saad, Usama Gergis, Lakshmanan Krishnamurti, Allistair Abraham, Hemalatha G. Rangarajan, Mark Walters, Joseph Lipscomb, Wael Saber, Prakash Satwani

Research output: Contribution to journalArticle

18 Scopus citations

Abstract

Advances in allogeneic hematopoietic cell transplantation for sickle cell disease have improved outcomes, but there is limited analysis of healthcare utilization in this setting. We hypothesized that, compared to late transplantation, early transplantation (at age <10 years) improves outcomes and decreases healthcare utilization. We performed a retrospective study of children transplanted for sickle cell disease in the USA during 2000-2013 using two large databases. Univariate and Cox models were used to estimate associations of demographics, sickle cell disease severity, and transplant-related variables with mortality and chronic graft-versus-host disease, while Wilcoxon, Kruskal-Wallis, or linear trend tests were applied for the estimates of healthcare utilization. Among 161 patients with a 2-year overall survival rate of 90% (95% confidence interval [CI] 85-95%) mortality was significantly higher in those who underwent late transplantation versus early (hazard ratio (HR) 21, 95% CI 2.8-160.8, P=0.003) and unrelated compared to matched sibling donor transplantation (HR 5.9, 95% CI 1.7-20.2, P=0.005). Chronic graftversus host disease was significantly more frequent among those translanted late (HR 1.9, 95% CI 1.0-3.5, P=0.034) and those who received an unrelated graft (HR 2.5, 95% CI 1.2-5.4; P=0.017). Merged data for 176 patients showed that the median total adjusted transplant cost per patient was $467,747 (range: $344,029-$799,219). Healthcare utilization was lower among recipients of matched sibling donor grafts and those with low severity disease compared to those with other types of donor and disease severity types (P<0.001 and P=0.022, respectively); no association was demonstrated with late transplantation (P=0.775). Among patients with 2-year pre- and post-transplant data (n=41), early transplantation was associated with significant reductions in admissions (P<0.001), length of stay (P<0.001), and cost (P=0.008). Early transplant outcomes need to be studied prospectively in young children without severe disease and an available matched sibling to provide conclusive evidence for the superiority of this approach. Reduced post-transplant healthcare utilization inpatient care indicates that transplantation may provide a sustained decrease in healthcare costs over time.

Original languageEnglish (US)
Pages (from-to)1823-1832
Number of pages10
JournalHaematologica
Volume102
Issue number11
DOIs
StatePublished - Oct 27 2017

ASJC Scopus subject areas

  • Hematology

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    Arnold, S. D., Brazauskas, R., He, N., Li, Y., Aplenc, R., Jin, Z., Hall, M., Atsuta, Y., Dalal, J., Hahn, T., Khera, N., Bonfim, C., Majhail, N. S., Diaz, M. A., Freytes, C. O., Wood, W. A., Savani, B. N., Kamble, R. T., Parsons, S., ... Satwani, P. (2017). Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases. Haematologica, 102(11), 1823-1832. https://doi.org/10.3324/haematol.2017.169581