Clinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases.

TitleClinical risks and healthcare utilization of haematopoietic cell transplantation for sickle cell disease in the U.S. using merged databases.
Publication TypeJournal Article
Year of Publication2017
AuthorsArnold SD, Brazauskas R, He N, Li Y, Aplenc R, Jin Z, Hall M, Atsuta Y, Dalal J, Hahn T, Khera N, Bonfim C, Majhail NS, Diaz MAngel, Freytes CO, Wood WA, Savani BM, Kamble RT, Parsons S, Ahmed I, Sullivan K, Beattie S, Dandoy C, Munker R, Marino S, Bitan M, Abdel-Azim H, Aljurf M, Olsson RF, Joshi S, Buchbinder D, Eckrich MJ, Hashmi S, Lazarus H, Marks DI, Steinberg A, Saad A, Gergis U, Krishnamurti L, Abraham A, Rangarajan HG, Walters M, Lipscomb J, Saber W, Satwani P
Start Page1823
Date Published2017 Nov

Allogeneic hematopoietic cell transplant advances for sickle cell disease improve outcomes, but limited healthcare utilization analysis exists. We hypothesized that early transplant (age < 10 years) improves outcomes and decreases healthcare utilization compared to late. We performed a retrospective study of US children transplanted for sickle cell during 2000-2013 using two large databases. Univariate and Cox models estimated associations between demographics, sickle cell severity, and transplant-related variables on mortality and chronic graft versus-host-disease; while, Wilcoxon, Kruskal-Wallis, or linear trend tests estimated against healthcare utilization. 161 patients with 90% 2-year overall survival (95% confidence interval [CI] 85-95%) displayed significantly higher mortality late versus early (hazard ratio (HR) 21, 95% CI 2.8-160.8, p=0.003) and unrelated compared to matched sibling donor (HR 5.9, 95% CI 1.7-20.2, p=0.005) and chronic graft versus host disease late (HR 1.9, 95% CI 1.0-3.5, p=0.034) and unrelated (HR 2.5, 95%CI 1.2-5.4; p=0.017). 176 merged patients had $467,747 median total adjusted transplant cost per patient (range: $344,029-$799,219). Matched sibling donor and low severity had the lowest healthcare utilization compared to other donor and severity types (p<0.001 and p=0.022, respectively); late transplant demonstrated no association (p=0.775). Patients with 2-yr pre- and post-transplant data (N=41) showed significant admission (p<0.001), length of stay (p<0.001), and cost (p=0.008) reductions. Superior early transplant outcomes require prospective studies in young children without severe disease and an available matched sibling to provide conclusive evidence for this approach. Reduced post-transplant healthcare utilization inpatient care indicates that transplant may provide a sustained decrease in healthcare costs over time.

Alternate JournalHaematologica
PubMed ID28818869
PubMed Central IDPMC5664386