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 Ahmed; Keith 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

doi:10.3324/haematol.2017.169581

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 journal › Article › peer-review

48 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 language	English
Pages (from-to)	1823-1832
Number of pages	10
Journal	Haematologica
Volume	102
Issue number	11
DOIs	https://doi.org/10.3324/haematol.2017.169581
State	Published - Oct 27 2017

Bibliographical note

Publisher Copyright:
© 2017 Ferrata Storti Foundation.

Funding

Special acknowledgments to our patients and families. The numerous mentors and advisors who helped develop and support this project. Dr. Arnold was supported in part by a Robert Wood Johnson Foundation Harold Amos Medical Faculty Development Program award. Dr. Aplenc was supported by 1R01CA166581. CIBMTR Support List: the CIBMTR is supported primarily by Public Health Service Grant/Cooperative Agreement 5U24-CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID); a Grant/Cooperative Agreement 5U10HL069294 from NHLBI and NCI; a contract HHSH250201200016C with Health Resources and Services Administration (HRSA/DHHS); two Grants N00014-15-1-0848 and N00014-16-1-2020 from the Office of Naval Research; and grants from *Actinium Pharmaceuticals, Inc.; Alexion; *Amgen, Inc.; Anonymous donation to the Medical College of Wisconsin; Astellas Pharma US; AstraZeneca; Atara Biotherapeutics, Inc.; Be the Match Foundation; *Bluebird Bio, Inc.; *Bristol Myers Squibb Oncology; *Celgene Corporation; Cellular Dynamics International, Inc.; Cerus Corporation; *Chimerix, Inc.; Fred Hutchinson Cancer Research Center; Gamida Cell Ltd.; Genentech, Inc.; Genzyme Corporation; Gilead Sciences, Inc.; Health Research, Inc. Roswell Park Cancer Institute; HistoGenetics, Inc.; Incyte Corporation; Janssen Scientific Affairs, LLC; *Jazz Pharmaceuticals, Inc.; Jeff Gordon Children’s Foundation; The Leukemia & Lymphoma Society; Medac, GmbH; MedImmune; The Medical College of Wisconsin; *Merck & Co, Inc.; *Mesoblast; MesoScale Diagnostics, Inc.; *Miltenyi Biotec, Inc.; National Marrow Donor Program; Neovii Biotech NA, Inc.; Novartis Pharmaceuticals Corporation; Onyx Pharmaceuticals; Optum Healthcare Solutions, Inc.; Otsuka America Pharmaceutical, Inc.; Otsuka Pharmaceutical Co, Ltd. – Japan; PCORI; Perkin Elmer, Inc.; Pfizer, Inc; *Sanofi US; *Seattle Genetics; *Spectrum Pharmaceuticals, Inc.; St. Baldrick’s Foundation; *Sunesis Pharmaceuticals, Inc.; Swedish Orphan Biovitrum, Inc.; Takeda Oncology; Telomere Diagnostics, Inc.; University of Minnesota; and *Wellpoint, Inc. The views expressed in this article do not reflect the official policy or position of the National Institute of Health, the Department of the Navy, the Department of Defense, Health Resources and Services Administration (HRSA) or any other agency of the U.S. Government. *Corporate Members.

Funders	Funder number
Actinium Pharmaceuticals, Inc.
AMGEN, Inc.
HRSA/DHHS	N00014-15-1-0848, N00014-16-1-2020
Office of Naval Research Naval Academy
National Heart, Lung, and Blood Institute (NHLBI)
National Childhood Cancer Registry – National Cancer Institute	U24CA076518
National Institute of Allergy and Infectious F32-AI286447 Cydney N. Johnson Diseases National Institute of Allergy and Infectious R01AI168214 Jason W. Rosch Diseases National Institute of Allergy and Infectious P30 Cydney N. Johnson Diseases National Institute of Allergy and Infectious R00-AI166116 Christopher D. Radka Diseases National Institute of Allergy and Infectious T32-AI106700 Cydney N. Johnson Diseases National Institute of Allergy and Infectious R01AI192221 Jason W. Rosch Diseases National Inst...	HHSH250201200016C, 5U10HL069294
Health Resources and Services Administration
Robert Wood Johnson Foundation	1R01CA166581
Gilead Sciences
National Institute for Health Research Health Protection Research Unit

ASJC Scopus subject areas

Hematology

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10.3324/haematol.2017.169581

Cite this

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

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

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.",

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

note = "Publisher Copyright: {\textcopyright} 2017 Ferrata Storti Foundation.",

year = "2017",

month = oct,

day = "27",

doi = "10.3324/haematol.2017.169581",

language = "English",

volume = "102",

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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 2017, 'Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases', Haematologica, vol. 102, no. 11, pp. 1823-1832. https://doi.org/10.3324/haematol.2017.169581

TY - JOUR

T1 - Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

AU - Arnold, Staci D.

AU - Brazauskas, Ruta

AU - He, Naya

AU - Li, Yimei

AU - Aplenc, Richard

AU - Jin, Zhezhen

AU - Hall, Matt

AU - Atsuta, Yoshiko

AU - Dalal, Jignesh

AU - Hahn, Theresa

AU - Khera, Nandita

AU - Bonfim, Carmem

AU - Majhail, Navneet S.

AU - Diaz, Miguel Angel

AU - Freytes, Cesar O.

AU - Wood, William A.

AU - Savani, Bipin N.

AU - Kamble, Rammurti T.

AU - Parsons, Susan

AU - Ahmed, Ibrahim

AU - Sullivan, Keith

AU - Beattie, Sara

AU - Dandoy, Christopher

AU - Munker, Reinhold

AU - Marino, Susana

AU - Bitan, Menachem

AU - Abdel-Azim, Hisham

AU - Aljurf, Mahmoud

AU - Olsson, Richard F.

AU - Joshi, Sarita

AU - Buchbinder, Dave

AU - Eckrich, Michael J.

AU - Hashmi, Shahrukh

AU - Lazarus, Hillard

AU - Marks, David I.

AU - Steinberg, Amir

AU - Saad, Ayman

AU - Gergis, Usama

AU - Krishnamurti, Lakshmanan

AU - Abraham, Allistair

AU - Rangarajan, Hemalatha G.

AU - Walters, Mark

AU - Lipscomb, Joseph

AU - Saber, Wael

AU - Satwani, Prakash

PY - 2017/10/27

Y1 - 2017/10/27

N2 - 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.

AB - 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.

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

Abstract

Bibliographical note

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