What was the research about?
Hematopoietic cell transplant, also called HCT, bone marrow transplant, or stem cell transplant, is a type of cancer treatment. HCT replaces damaged cells after chemotherapy. Doctors can use HCT to treat acute myeloid leukemia, or AML, which is a type of blood cancer. Doctors also use other types of treatments for AML, such as more chemotherapy or supportive care.
In this study, the research team looked at factors that made patients less likely to have HCT. The team also compared how HCT or other treatments affected patients’
- Survival after one year
- Quality of life
- Ability to do daily activities, such as bathing or dressing
- Frailty, or the risk of a major drop in health and function for older adults
What were the results?
Patients were less likely to get treated with HCT if they
- Had low risk for relapse based on genetic analysis of their cancer
- Started treatment with less intensive chemotherapy
- Were older than 69
- Were less able to do everyday tasks
- Had cancer come back after remission
Patients who had HCT lived longer than those who didn’t have HCT. But when the research team took into account factors like age and other health problems, patients who did and didn’t have HCT were just as likely to survive after one year. Patients’ quality of life, depression, and ability to do daily activities didn’t differ between the two groups.
Patients in the two groups differed in how their frailty levels changed. At the end of a year, patients who didn’t have HCT were less frail than when they started the study. For patients who did have HCT, their frailty levels didn’t change.
Who was in the study?
The study included 692 adults with AML, or a similar illness treated like AML. Of the patients, 86 percent were white, 6 percent were African American, 3 percent were Asian, and 4 percent were Hispanic. The average age was 59, and 43 percent were women. Patients received care at one of 13 treatment centers across the United States.
What did the research team do?
The research team enrolled patients newly diagnosed with AML before they started treatment. Patients took surveys about quality of life and health. They performed a walking test at the start of the study and again seven times over the next two years. The team also looked at patients’ health records.
People with AML, doctors, researchers, and people from health insurance companies and advocacy groups gave input on the study.
What were the limits of the study?
The research team took into account many factors about patient’s health. But other factors that they weren’t able to include could have affected the results. Some patients who could have taken part in the study chose not to. Results may have differed if more patients had taken part in the study.
Future research could assign patients by chance to have HCT or other types of treatment that take the patient’s specific health needs into account.
How can people use the results?
Patients and their doctors can use the results when considering treatment options for AML.
To assess predictors for receiving allogeneic hematopoietic cell transplantation (HCT) and compare the effectiveness of HCT versus alternative treatments on overall mortality, quality of life (QOL), and functional status among adults with acute myeloid leukemia (AML) or similar diseases
|Design||Observational: cohort study|
|Population||692 newly diagnosed adults under age 80 with AML except those with the M3 subtype, high-risk myelodysplastic syndrome, myeloproliferative neoplasms, or myelofibrosis|
Primary: receipt of an allogeneic HCT within 9 months after diagnosis, overall mortality
Secondary: QOL, depression, ability to complete ADLs, and frailty
|Timeframe||Up to 2-year follow-up for primary outcomes|
This prospective cohort study assessed factors associated with a lower likelihood of receiving HCT and compared overall mortality, QOL, depression, ability to complete activities of daily living (ADLs), and frailty between patients who had HCT versus non-HCT treatments.
Patients received induction chemotherapy. Doctors monitored patients for treatment response. After a median of four months, of those that survived, doctors and patients decided whether patients would receive HCT or non-HCT treatments.
The study included 692 newly diagnosed adults with AML or similar diseases who received AML-like therapy at one of 13 treatment centers across the United States. Of these, 86% were white, 6% were African American, 3% were Asian, and 4% were Hispanic. The average age was 59, and 43% were female.
Patients completed surveys, a walking test to measure frailty, and other geriatric assessments at baseline and seven additional times within two years. Researchers reviewed medical records for predictors of receiving HCT and compared one-year mortality risk and changes over time in other outcomes between the two groups.
Patients with AML, clinicians, researchers, and representatives from health insurance companies and advocacy and professional organizations gave input throughout the study.
Factors associated with a lower likelihood of receiving HCT within nine months of diagnosis included
- Being older than 69 (hazard ratio [HR] = 0.40; 95% confidence interval [CI]: 0.27, 0.60)
- Having a low risk for relapse based on genetic analysis of patients’ cancer (HR = 0.28; 95% CI: 0.19, 0.42)
- Having less intensive induction chemotherapy (HR = 0.56; 95% CI: 0.34, 0.93)
- Having low functional status (HR = 0.49; 95% CI: 0.33, 0.73)
- Relapsing after initial complete remission (HR = 0.41; 95% CI: 0.24, 0.70)
In patients older than 59, predictors of a lower likelihood of receiving HCT included
- Impaired cognition (HR = 0.45; p=0.007)
- Impaired hearing (HR = 0.71; p=0.009)
Patients receiving HCT had a lower mortality risk than patients receiving non-HCT treatments (HR = 0.63; 95% CI: 0.50, 0.80). After accounting for other risk factors, HCT and non-HCT treatments did not differ significantly in mortality risk.
Patients receiving non-HCT treatments experienced a significantly greater improvement in walk tests over time than patients who received HCT (p=0.0167). The groups did not differ significantly in QOL, depression, or ADLs.
Researchers adjusted for a number of confounding variables, but others that were not accounted for could have affected the study results. Some eligible patients declined to participate; results may have differed if they had taken part in the study.
Conclusions and Relevance
Researchers identified several modifiable factors that could increase the likelihood of HCT treatment. For patients being treated with HCT, doctors may consider ways to decrease patient frailty.
Future Research Needs
Future research could randomize patients to receive HCT or alternative treatments, accounting for clinical factors.
Final Research Report
View this project's final research report.
Related Journal Citations
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
- The reviewers expressed concern about engagement activities involving patient stakeholders the researchers recruited from among the study participants. The reviewers noted that these individuals tended to have short tenure as stakeholder participants because of their disease progression, which could indicate insufficient involvement in study decision-making by patient stakeholders. The researchers clarified that they had a large group of patient stakeholders who contributed to all aspects of study design and follow-up before the study began. The researchers explained that they recruited patient partners from among study participants to enhance the pool of stakeholders, but they were not the only patients or patient advocates among the stakeholders.
- The reviewers asked the researchers to address whether trying to create better risk stratification models is the best way to improve clinical decision-making for patients with acute myeloid leukemia (AML). The researchers added an analysis of the predictive power of their model to the report, saying that the model was clearly better than relying on physician perception after an initial clinic visit in predicting patient survival. The researchers said their work provided very specific survival rates for different risk groups, which did not exist before this study and which physicians can use to counsel patients. The researchers also stated that risk models can help improve decision making about treatments. The researchers also noted that their model was the first AML prognosis model to incorporate comorbidities.
- The reviewers asked for more justification for choosing one-year survival as the primary outcome and questioned whether it was the best outcome to consider especially for younger patients. The researchers agreed that younger patients may be interested in longer survival timepoints but said that their study largely focused on older patients and patients with comorbidities. The researchers also noted that when comparing more and less intensive therapies, they used two-year survival as an endpoint, and in one analysis they used all survival data accumulated over the more than four years since the study began.
Conflict of Interest Disclosures
Study Registration Information
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