Format

Send to

Choose Destination
Haematologica. 2017 Nov;102(11):1823-1832. doi: 10.3324/haematol.2017.169581. Epub 2017 Aug 17.

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

Author information

1
Emory University Hospital, Atlanta, GA, USA.
2
CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
3
Division of Biostatistics, Institute for Health and Society, Medical College of Wisconsin, Milwaukee, WI, USA.
4
University of Pennsylvania, Philadelphia, PA, USA.
5
Department of Biostatistics, Mailman School of Public Health, Columbia University, New York, NY, USA.
6
Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, Department of Pediatrics, Columbia University Medical Center, New York, NY, USA.
7
Japanese Data Center for Hematopoietic Cell Transplantation, Nagoya, Japan.
8
Nagoya University Graduate School of Medicine, Japan.
9
Rainbow Babies & Children's Hospital, Cleveland, OH, USA.
10
Department of Medicine, Roswell Park Cancer Institute, Buffalo, NY, USA.
11
Department of Hematology/Oncology, Mayo Clinic, Phoenix, AZ, USA.
12
Hospital de Clinicas-Federal University of Parana, Curitiba, Brazil.
13
Blood & Marrow Transplant Program, Cleveland Clinic Taussig Cancer Institute, Cleveland, OH, USA.
14
Department of Hematology/Oncology, Hospital Infantil Universitario Nino Jesus, Madrid, Spain.
15
Texas Transplant Institute, San Antonio, TX, USA.
16
Division of Hematology/Oncology, Department of Medicine, University of North Carolina, Chapel Hill, NC, USA.
17
Division of Hematology/Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN, USA.
18
Divsion of Hematology and Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX, USA.
19
Tufts Medical Center, Boston, MA, USA.
20
Duke University Medical Center, Durham, NC, USA.
21
University of Ottawa, Canada.
22
Cincinnati Children's Hospital Medical Center, OH, USA.
23
Section of Hematology/Oncology, Department of Internal Medicine, Louisiana State University Health Shreveport, LA, USA.
24
University of Chicago Hospitals, Chicago, IL, USA.
25
Department of Pediatric Hematology/Oncology, Tel-Aviv Sourasky Medical Center, Israel.
26
Division of Hematology, Oncology and Blood & Marrow Transplantation, Children's Hospital of Los Angeles, University of Southern California Keck School of Medicine, CA, USA.
27
Department of Oncology, King Faisal Specialist Hospital Center & Research, Riydah, Saudi Arabia.
28
Division of Therapeutic Immunology, Department of Laboratory Medicine, Karolinksa Institutet, Stockholm, Sweden.
29
Centre for Clinical Research Sormland, Uppsala University, Sweden.
30
Pediatric Hematology, Oncology and BMT, Nationwide Children's Hospital and Ohio State University Wexner, Columbus, OH, USA.
31
Division of Pediatrics Hematology, Children's Hospital of Orange County, Orange, CA, USA.
32
Levine Children's Hospital, Charlotte, NC, USA.
33
Department of Internal Medicine, Mayo Clinic, Minneapolis, MN, USA.
34
Seidman Cancer Center, University Hospitals Case Medical Center, Cleveland, OH, USA.
35
Adult Bone Marrow Transplant, University Hospitals Bristol NHS Trust, Bristol, United Kingdom.
36
Department of Hematology-Oncology, Mount Saini Hospital, New York, NY, USA.
37
Division of Hematology/Oncology, Department of Medicine, University of Alabama at Birmingham, Birmingham, AL, USA.
38
Hematologic Malignancies & Bone Marrow Transplant, Department of Medical Oncology, New York Presbyterian Hospital/Weill Cornell Medical College, New York, NY, USA.
39
Division of Blood and Marrow Transplantation, Center for Cancer and Blood Disorders, Children's National Medical Center, Washington, DC, USA.
40
Children's Hospital & Research Center Oakland, Oakland, NY, USA.
41
Health Policy and Management, Rollins School of Public Health, Winship Cancer Institute, Emory University, Atlanta, GA, USA.
42
CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA wsaber@mcw.edu.

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.

PMID:
28818869
PMCID:
PMC5664386
DOI:
10.3324/haematol.2017.169581
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for HighWire Icon for PubMed Central
Loading ...
Support Center