Transplantation of stem cells from umbilical cord blood

Findings by SBU Alert

Version: 1

Technology and target group

Transplantation of stem cells is an established, potentially curative method of treatment for leukemia and other blood diseases. Stem cells are usually harvested from bone marrow or peripheral blood. Stem cells from umbilical cord blood have, in recent decade, been used when donators of bone marrow or peripheral blood cannot be found. The volume of umbilical cord blood that can be obtained is limited, and thereby so is the number of cells that can be harvested. Consequently, the main target group has been children, but adult patients have also been treated. Transplantation of stem cells from umbilical cord blood can be used in metabolic and immune deficiency diseases, malignant blood diseases (including leukemia), and bone marrow failure syndrome. The size of the target group in Sweden is estimated at 6 to 12 patients annually, but this figure may increase if a higher level of mismatch between donor and recipient can be accepted.

Patient benefits

There are no prospective randomized controlled trials that compare the outcome from transplanting stem cells of different types. There are followup studies on over 1 000 patients on the medical effects and complications following transplantation of stem cells from umbilical cord blood. These studies show that survival following transplantation is comparable regardless of whether the stem cells are harvested from umbilical cord blood or bone marrow. The clinical importance of potential advantages and disadvantages in using stem cells from umbilical cord blood is not yet clear. The prevalence of acute and chronic GvHD (an immunologically triggered reaction) is lower when stem cells from umbilical cord blood are used, but engraftment is delayed compared to transplantation of other types of stem cells.

Economic aspects

The price per unit of stem cells from umbilical cord blood when purchased from a blood bank is currently 150 000 SEK, which approximately corresponds to the cost of harvesting bone marrow or peripheral blood from unrelated donors. Primary economic studies are lacking, and currently there is no evidence to show the cost effectiveness of the method. Although the cost for each transplantation is significant, the total economic impact on the healthcare system is minor since the target group is small.

Scientific evidence

There is moderate* scientific documentation concerning the medical effects and complications from transplantation of stem cells from umbilical cord blood. There is no* scientific documentation concerning the cost effectiveness.

Until further notice, the method should be considered experimental and be performed only in accordance with established treatment protocols. It is important to report the outcomes of all transplantations in Sweden to a shared European registry and/or the international bone marrow transplantation registry.

*This assessment by SBU Alert uses a 4-point scale to grade the quality and evidence of the scientific documentation. The grades indicate: (1) good, (2) moderate, (3) poor, or (4) no scientific evidence on the subject.

This summary is based on a report prepared at SBU in collaboration with Albert N Békássy, MD PhD, and Bengt Sallerfors MD PhD, both from Lund University Hospital and has been reviewed by Prof Olle Ringdén, MD PhD, Huddinge University Hospital.

The full report is available only in Swedish.

Alert is a joint effort by the Swedish Council on Technology Assessment in Health Care (SBU), the Medical Products Agency, the National Board of Health and Welfare, and the Federation of Swedish County Councils.

References

1. American Academy of Pediatrics, Work Group on Cord Banking. Cord Blood Banking for Potential Future Transplantation: Subject Review. Pediatrics 1999;104:116-118.
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3. Broxmeyer HE, Douglas GW, Hangoc G, Cooper S, Bard J, English D, et al. Human umbilical cord blood as potential source of transplantable hematopoietic stem/progenitor cells. Proc Natl Acad Sci USA 1989;86:3828-32.
4. Broxmeyer HE, ed. Cellular Characteristics of Cord Blood and Cord Blood Transplantation. Bethesda: AABB Press. 1998:227.
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6. Eurocord WEB site http://www.cbtg.com/
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12. Netcord WEB site http://www.msz.uni-duesseldorf.de/NETCORD/Welcome.html
13. Rocha V, Cornish J, Sievers EL, Filipovich A, Locatelli F, et al. Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood 2001;(10): 2962-2971.
14. van Rood JJ. Bone Marrow Donors Worldwide, WEB site http://www.bmdw.org/. Annual Report 1999. RC Leiden: Drukkerij Groen BV, 2000.
15. Rubinstein P, Stevens CE. Placental blood for bone marrow replacement: The New York Blood Center´s program and clinical results. Baillieres Best Pract Res Clin Haematol 2000;13:565-84.
16. SBU-rapport: Benmärgstransplantation. Stockholm 1991.
17. Wahlin A (2000): Personligt meddelande
18. Wagner JE, Kernan NA Steinbuch M, Broxmeyer HE, Gluckman E. Allogenic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet 1995;343:214-219.
    European Blood- and MarrowTransplantation Registry WEB site http://ebmt.clinicalresearch.nl/EBMTNEW/4Registry/Promise.html