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Design of a cost-effectiveness analysis alongside a randomized trial of transplantation using umbilical cord blood versus HLA-haploidentical related bone marrow in advanced hematologic cancer.

TitleDesign of a cost-effectiveness analysis alongside a randomized trial of transplantation using umbilical cord blood versus HLA-haploidentical related bone marrow in advanced hematologic cancer.
Publication TypeJournal Article
Year of Publication2014
AuthorsRoth, JA, Bensink, ME, O'Donnell, PV, Fuchs, EJ, Eapen, M, Ramsey, SD
JournalJ Comp Eff Res
Volume3
Issue2
Pagination135-44
Date Published2014 Mar
ISSN2042-6313
KeywordsAdolescent, Adult, Aged, Bone Marrow Transplantation, Comparative Effectiveness Research, Cord Blood Stem Cell Transplantation, Cost-Benefit Analysis, Female, Health Care Costs, Health Expenditures, Hematologic Neoplasms, Humans, Male, Middle Aged, Quality of Life, Young Adult
Abstract

BACKGROUND: BMT CTN 1101 is a Phase III randomized controlled trial evaluating the comparative effectiveness of double unrelated umbilical cord blood (dUCB) versus HLA-haploidentical related donor bone marrow (haplo-BM) donor cell sources for blood or bone marrow transplantation (BMT) in patients with hematologic malignancies. Herein, we present the rationale, design and methods of the first cost-effectiveness analysis to be conducted alongside a BMT trial.

METHODS: Consenting patients will provide health insurance information to allow calculation of direct medical costs from reimbursement records, and will provide out-of-pocket costs, time costs and health-related quality of life measures through an online survey. These outcomes will inform a cost-effectiveness analysis comparing dUCB and haplo-BM donor cell sources from patient, payer and societal perspectives.

CONCLUSION: Novel approaches may significantly change the cost, outcomes or availability of BMT. The results of this analysis will be the first to provide a comprehensive evaluation of the comparative effectiveness of these approaches from multiple perspectives.

DOI10.2217/cer.13.95
Alternate JournalJ Comp Eff Res
PubMed ID24645687
PubMed Central IDPMC4036637
Grant ListU10 HL069294 / HL / NHLBI NIH HHS / United States
U10 HL069290 / HL / NHLBI NIH HHS / United States
U24 CA076518 / CA / NCI NIH HHS / United States
U10 HL069301 / HL / NHLBI NIH HHS / United States
U10 HL069310 / HL / NHLBI NIH HHS / United States
R01 HL116291 / HL / NHLBI NIH HHS / United States
T32 AG027677 / AG / NIA NIH HHS / United States
U10 CA180828 / CA / NCI NIH HHS / United States
U10HL069294 / HL / NHLBI NIH HHS / United States
R01HL116291-01 / HL / NHLBI NIH HHS / United States
U10 HL069330 / HL / NHLBI NIH HHS / United States
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