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scientific articles

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

key information

source: Haematologica

year: 2017

authors: Arnold 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 MA, Freytes CO, Wood WA, Savani BN, 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


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.

organization: Emory University Hospital, USA; Medical College of Wisconsin, USA; University of Pennsylvania, USA; Columbia University, USA; Columbia University Medical Center, USA; Japanese Data Center for Hematopoietic Cell Transplantation, Japan; Nagoya University Graduate School of Medicine, Japan; Rainbow Babies & Children's Hospital, USA; Roswell Park Cancer Institute, USA; Mayo Clinic, USA; Hospital de Clinicas-Federal University of Parana, Brazil; Cleveland Clinic Taussig Cancer Institute, USA; Hospital Infantil Universitario Nino Jesus, Spain; Texas Transplant Institute, USA; University of North Carolina, USA; Vanderbilt University Medical Center, USA; Baylor College of Medicine, USA; Tufts Medical Center, USA; Duke University Medical Center, USA; University of Ottawa, Canada; Cincinnati Children's Hospital Medical Center, USA; Louisiana State University Health Shreveport, USA; University of Chicago Hospitals, USA; Tel-Aviv Sourasky Medical Center, Israel; University of Southern California Keck School of Medicine, USA; King Faisal Specialist Hospital Center & Research, Saudi Arabia; Karolinksa Institutet, Sweden; Uppsala University, Sweden; Nationwide Children's Hospital and Ohio State University Wexner, USA; Children's Hospital of Orange County, USA; Levine Children's Hospital, USA; Mayo Clinic, USA; University Hospitals Case Medical Center, USA; University Hospitals Bristol NHS Trust, United Kingdom; Mount Saini Hospital, USA; University of Alabama at Birmingham, USA; New York Presbyterian Hospital/Weill Cornell Medical College, USA; Children's National Medical Center, USA; Children's Hospital & Research Center Oakland, USA

DOI: 10.3324/haematol.2017.169581

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