Authors' objectives:

The purpose of this report has been to evaluate all relevant literature that can elucidate the basis for the therapeutic use of cord blood stem cells. Central issues were possible differences in the clinical effect between the use of stem cells from cord blood and bone marrow or peripheral blood and possible differences in the clinical effects between the use of allogeneic or autologous stem cells from cord blood. The expert group was also asked to elucidate the documented safe storage time for cryopreserved stem cells in regard to the clinical effect. In addition, the size of the relevant recipient group as well as organisational, economical, ethical and social aspects in relation to the method should be discussed.

Authors' results and conclusions: There are no published results of the therapeutic use of autologous stem cells from cord blood. For allogeneic transplants there are a few cohort studies and a number of larger and small patient series. There are no published randomised studies comparing cord blood transplants with transplants of bone marrow or peripheral blood stem cells. The cohort studies have a retrospective design with historical controls. The two largest patient series are registry studies that report results from a large patient material, altogether more than 1400 patients. None of the studies are population based. Of the 2000 patients that have been transplanted with cord blood about 1600 are children and about 400 adult individuals. Results from transplants with related (mainly sibling) and unrelated donors where the donors either are HLA-identical or have different degrees of HLA-mismatches have been reported. While published results for children exist for all categories, the cord blood transplantations in adults have almost exclusively been performed with unrelated donors. No publications have been designed to elucidate maximal safe storage time for cryopreserved stem cells in regard to the clinical effect. Cord blood stem cell transplantation has been in use for more than 10 years, thus, cord blood stem cells have so far shown effective after being cryopreserved for about 10 year.

Authors' recommendations: Autologous transplants of stem cells from cord blood have not been reported or documented. The clinical benefit of allogeneic transplants of stem cells from cord blood has not yet been directly compared to the results of allogeneic stem cells from bone marrow or peripheral blood in prospective studies. Published studies, all with retrospective design, suggest that the clinical effect of cord blood transplants at least in children may be comparable to transplants with stem cells from bone marrow or peripheral blood. The number of stem cells in the cord blood graft is often not sufficient to obtain adequate engraftment in adults and the risk for non-engraftment is larger in cord blood transplants than for transplants with stem cells from bone marrow or peripheral blood. The risk of graft-versus-host disease is probably less in transplants with cord blood than with the use of allogeneic stem cells from peripheral blood or bone marrow with comparable HLA-matching. This treatment option is expensive. With the present indications for the use of stem cells from cord blood it seems not to be cost-effective to establish Norwegian biobanks. The access to the Netcord network and established biobanks abroad provide for cord blood if needed for Norwegian patients. There are difficult ethical questions associated to the storage and use of as well as the proprietary rights to cryopreserved cord blood.

Authors' methods: Systematic review

Project Status: Completed

URL for project: http://www.nokc.no/

Year Published: 2003

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Norway

Organisation Name: Norwegian Institute of Public Health

Contact Address: Universitetsgata 2, Postbox 7004 St. Olavs plass, NO-0310 Oslo NORWAY. Tel: +47 23 25 50 00; Fax: +47 23 25 50 10;

Contact Name: Berit.Morland@nokc.no, dagny.fredheim@nokc.no

Contact Email: Berit.Morland@nokc.no, dagny.fredheim@nokc.no

Copyright: The Norwegian Knowledge Centre for the Health Services