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Allogeneic stem cell transplantation for sickle cell disease. A study of patients' decisions
K van Besien1, M Koshy, L Anderson-Shaw
1Section of Hematology and Oncology, University of Illinois at Chicago, IL 60637, USA.
Insights
Many adults with sickle cell disease desire curative stem cell transplants despite risks. Patient values, not provider assessments, drive risk acceptance for this potentially life-saving treatment.
Area of Science:
- Hematology
- Transplantation Medicine
- Patient-Reported Outcomes
Background:
- Allogeneic stem cell transplantation (SCT) offers a potential cure for sickle cell disease (SCD) but involves significant risks.
- Understanding patient and healthcare provider (HCP) perspectives on SCT risks and benefits is crucial for informed decision-making.
Purpose of the Study:
- To investigate adult SCD patients' and their HCPs' attitudes towards SCT risks and benefits.
- To compare patient risk acceptance with HCP recommendations and identify factors influencing risk tolerance.
Main Methods:
- A survey involving 100 adult SCD patients and their HCPs was conducted.
- Risk assessment utilized a reference gamble paradigm.
- Patient characteristics and agreement between patient-accepted risk and HCP-recommended risk were analyzed.
Main Results:
- 63% of patients were willing to accept short-term mortality risk for a cure; 15% accepted >35% mortality risk.
- No significant differences in patient or disease variables were found between risk-accepting and non-risk-accepting groups.
- No agreement was observed between HCP recommendations and patient-accepted risk levels.
Conclusions:
- A considerable number of adult SCD patients are interested in curative SCT, accepting substantial risks.
- Individual patient values, which are difficult to quantify, heavily influence risk acceptance and are not aligned with HCP assessments.
- Enhanced education and consultation regarding allogeneic SCT for SCD patients are recommended due to high patient interest.
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.