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

J Comp Eff Res. 2014 Mar;3(2):135-44. doi: 10.2217/cer.13.95.


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.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Bone Marrow Transplantation / economics*
  • Comparative Effectiveness Research / methods
  • Cord Blood Stem Cell Transplantation / economics*
  • Cost-Benefit Analysis
  • Female
  • Health Care Costs / statistics & numerical data
  • Health Expenditures / statistics & numerical data
  • Hematologic Neoplasms / economics*
  • Hematologic Neoplasms / surgery*
  • Humans
  • Male
  • Middle Aged
  • Quality of Life
  • Young Adult