Authors' objectives:

The aim of this report is to evaluate the use of umbilical cord blood as an alternative source for hematopoietic stem cell transplantation.

Authors' results and conclusions: Hematopoietic stem cell transplantation from both related and unrelated donors is an accepted treatment option for patients with life-threatening malignant and non-malignant hematologic disorders. It constitutes the only available treatment for some hematological malignancies. Approximately 75% of the patients who could benefit from stem cell transplantation do not have a histocompatible related donor. For these patients, stem cell transplantation from an unrelated donor can be appropriate. However, for 20% of Caucasian and greater than 40% of non-Caucasian patients a suitable unrelated donor cannot be found. Expert opinion and some evidence support the centralization of such procedures in centres with a significant turnover. In the future, both accreditation and the supply of donor cells will require institutional centralization at the MUHC. Advantages of cord blood stem cells include 1) increased probability of transplantation since HLA-matching requirements are less strict, 2) more prompt availability (average 1-2 months difference), an obvious advantage when the time suitable for transplantation is strictly limited, 3). Less potential for transmission of viral infections such as CMV and EBV with cord blood. Disadvantages of cord blood stem cells include 1) generally longer time to hematopoieitic recovery, which may theoretically increase the hospital length of stay, 2) possible failure of the graft due to insufficient stem cells, an outcome that can usually be avoided by giving more than one unit. On average, although there are differences in the clinical course, there is no consistent difference in the overall survival between cord blood and bone marrow transplants. Mortality in the early post-transplant years is high (50-65%), with 5-year mortality rates of approximately 75%. Although the number of patients with longer follow-up is small, survival after the fifth year is probably close to a near normal life expectancy. Based on our stated assumptions, we estimate that stem cell transplantation undertaken in a mix of cases similar to those currently treated at the MUHC, might result in an average 2.94 (95% CI: 2.58- 4.30) life-years gained per patient.

Authors' recommendations: In spite of its high initial mortality and high costs, stem cell transplantation from either bone marrow/ peripheral blood or cord blood is a clinically effective therapy for appropriately selected patients, with an acceptable cost-effectiveness ratio. - Recommendation 1. The MUHC should urgently seek designated funding to enable it to offer this technology to appropriate adult patients and to support a transplant centre of sufficient quality to maintain good clinical outcomes and to assure the accreditation on which the future supply of donor cells will depend. The opportunity costs associated with even a modest increase in activity of 20 patients per year (approximately $1.8 million) would be too great a budgetary commitment in the absence of additional funding. - Recommendation 2. It is therefore recommended that no significant increase in stem cell transplantation be authorized in the absence of additional funding. Hematopoietic stem cell transplantation is an accepted therapy at the MUHC. In spite of the higher cost of cord blood transplantation it would be ethically difficult to refuse it to appropriate recipients for whom matching bone marrow stem cells can not be found. - Recommendation 3. The present practice of applying for special funding for each such case is fully justified and should be continued. The projected volume of stem cell transplantation activity would fully justify the existence of a stem cell centre at the MUHC. In the future it will not be possible to maintain transplant centers in each of the component hospitals of the MUHC. - Recommendation 4. All adult stem cell transplants carried out at the MUHC should take place in one designated centre.

Authors' methods: Systematic review

Project Status: Completed

URL for project: http://www.mcgill.ca/tau/publications/2005/

Year Published: 2005

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Canada

Organisation Name: Technology Assessment Unit of the McGill University Health Centre (MUHC)

Contact Address: Technology Assessment Unit of the MUHC, 536-5100 Boul. Maisonneuve O, Montreal, H4A 3T2

Contact Name: eva.suarthana@mcgill.ca

Contact Email: nisha.almeida@muhc.mcgill.ca

Copyright: Technology Assessment Unit of the McGill University Health Centre (MUHC)