Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases
Creators: name: Buchbinder, Dave
name: Hahn, Theresa
name: Bitan, Menachem
name: Atsuta, Yoshiko
name: Ahmed, Ibrahim
name: Hall, Matt
name: Majhail, Navneet S.
name: Sullivan, Keith
name: Savani, Bipin N.
name: Marks, David I.
name: Jin, Zhezhen
name: Freytes, Cesar O.
name: He, Naya
name: Hashmi, Shahrukh
name: Steinberg, Amir
name: Saad, Ayman
name: Saber, Wael
name: Eckrich, Michael J.
name: Brazauskas, Ruta
name: Li, Yimei
name: Khera, Nandita
name: Dandoy, Christopher
name: Dalal, Jignesh
name: Gergis, Usama
name: Munker, Reinhold
name: Krishnamurti, Lakshmanan
name: Satwani, Prakash
name: Diaz, Miguel Angel
name: Walters, Mark
name: Lazarus, Hillard
name: Parsons, Susan
name: Lipscomb, Joseph
name: Rangarajan, Hemalatha G.
name: Abraham, Allistair
name: Olsson, Richard F.
name: Kamble, Rammurti T.
name: Bonfim, Carmem
name: Aljurf, Mahmoud
name: Beattie, Sara
name: Wood, William A.
name: Aplenc, Richard
name: Abdel-Azim, Hisham
name: Marino, Susana
name: Arnold, Staci D.
name: Joshi, Sarita
Publisher: name: The University of North Carolina at Chapel Hill University Libraries
Year: 2017

description: 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.