Abstract
Allogeneic stem cell transplantation is increasingly considered as a curative though risky treatment option for adults with sickle cell disease. Little is known about attitudes of adult patients and their health care providers regarding the risks and benefits of transplantation. A survey of 100 patients and their health care providers was undertaken. Assessment of risk was by a reference gamble paradigm. Comparison was made of the characteristics of those accepting substantial risk vs those not accepting risk, as well as assessment of agreement on risks recommended by health care providers and accepted by patients. Sixty-three of 100 patients were willing to accept some short-term risk of mortality in exchange for the certainty of cure. Fifteen patients were willing to accept more than 35% mortality risk. No differences in patient or disease-related variables were identified between those accepting risk and those not accepting risk. There was no agreement between the recommendations of health care providers and the risk accepted by patients. A substantial proportion of adults with sickle cell disease are interested in curative treatment, at the expense of considerable risk. The decision to accept risk is influenced by individual patient values that cannot be easily quantified and that do not correlate with the assessment of the health care provider. Given the substantial interest in curative therapy, education about and consultation for allogeneic stem cell transplantation in sickle cell patients should be encouraged. Bone Marrow Transplantation (2001) 28, 545–549.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Similar content being viewed by others
References
Platt OS, Brambilla DJ, Rosse WF et al. Mortality in sickle cell disease. Life expectancy and risk factors for early death New Engl J Med 1994 330: 1639–1644
Vermylen C, Cornu G, Ferster A et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium Bone Marrow Transplant 1998 22: 1–6
Walters MC, Patience M, Leisenring W et al. Bone marrow transplantation for sickle cell disease New Engl J Med 1996 335: 369–376
van Besien K, Bartholomew A, Stock W et al. Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease Bone Marrow Transplant 2000 26: 445–449
Giralt S, Estey E, Albitar M et al. Engraftment of allogeneic hematopoietic progenitor cells with purine analog-containing chemotherapy: harnessing graft-versus-leukemia without myeloablative therapy Blood 1997 89: 4531–4536
Sass H . Moral aspects of risk and innovation Artific Org 1997 21: 1217–1221
Prayle D, Brazier M . Supply of medicines: paternalism, autonomy and reality J Med Ethics 1998 24: 93–98
Kodish E, Lantos J, Siegler M et al. Bone marrow transplantation in sickle cell disease: the trade-off between early mortality and quality of life Clin Res 1990 38: 694–700
Kodish E, Lantos J, Stocking C et al. Bone marrow transplantation for sickle cell disease – a study of parents' decisions New Engl J Med 1991 325: 1349–1353
Froberg DG, Kane RL . Methodology for measuring health-state preferences – II: Scaling methods J Clin Epidemiol 1989 42: 459–471
Altman DG . Practical Statistics for Medical Research Chapman and Hall: London 1995
Charache S, Terrin ML, Moore RH et al. and the investigators of the multicenter study of hydroxyurea in sickle cell anemia. Effect of hydroxyurea on the frequency of painful crisis in sickle cell disease New Engl J Med 1995 332: 1317–1322
Altman DG . Some common problems in medical research In: Practical Statistics for Medical Research Chapman and Hall: London 1995 pp 396–439
Walters MC, Patience M, Leisenring W et al. Barriers to bone marrow transplantation for sickle cell anemia Biol Blood Marrow Transplant 1996 2: 100–104
Chandy M . Management of hematological diseases: socio-economic aspects. In: Schechter GP, Hoffman R, Schrier SL, Bajus JL (eds). American Society of Hematology Education Program Book Washington 1999 pp 73–82
Platt OS, Guinan EC . Bone marrow transplantation in sickle cell anemia – the dilemma of choice New Engl J Med 1996 335: 426–427
Lee A, Thomas P, Cupidore L et al. Improved survival in homozygous sickle cell disease: lessons from a cohort study Br Med J 1995 311: 1600–1602
Blume KG, Amylon MD . The evaluation and counseling of candidates for hematopoietic cell transplantation In: Thomas ED, Blume KG, Forman SJ (eds) Hematopoietic Cell Transplantation Blackwell Science: Oxford 1999 371–380
Quill T, Brody H . Physician recommendations and patient autonomy: finding a balance between physician power and patient choice Ann Int Med 1996 125: 763–769
Ross I . Practice guidelines, patient interests and risky procedures Bioethics 1996 10: 310–322
Annas GJ . A national bill of patients' rights New Engl J Med 1998 338: 695–699
Brunet-Jailly J . The ethics of clinical research in developing countries IRB: a Review of Human Subjects Research 1999 21: 8–11
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
van Besien, K., Koshy, M., Anderson-Shaw, L. et al. Allogeneic stem cell transplantation for sickle cell disease. A study of patients' decisions. Bone Marrow Transplant 28, 545–549 (2001). https://doi.org/10.1038/sj.bmt.1703208
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/sj.bmt.1703208
Keywords
This article is cited by
-
Ethical development of stem-cell-based interventions
Nature Medicine (2019)
-
Traffic Light: prognosis-based eligibility for clinical trials of hematopoietic SCT in adults with sickle cell anemia
Bone Marrow Transplantation (2015)
-
Reduced intensity conditioning and allogeneic stem cell transplantation in childhood malignant and nonmalignant diseases
Bone Marrow Transplantation (2008)
-
A survey on patient perception of reduced-intensity transplantation in adults with sickle cell disease
Bone Marrow Transplantation (2007)
-
Has stem cell transplantation come of age in the treatment of sickle cell disease?
Bone Marrow Transplantation (2007)