Identifying Best Approach in Improving Quality of Life and Survival After a Donor Stem Cell Transplant in Older, Medically Infirm, or Frail Patients With Blood Diseases
Study Details
Study Description
Brief Summary
This phase II/III trial studies the best approach in improving quality of life and survival after a donor stem cell transplant in older, weak, or frail patients with blood diseases. Patients who have undergone a transplant often experience increases in disease and death. One approach, supportive and palliative care (SPC), focuses on relieving symptoms of stress from serious illness and care through physical, cultural, psychological, social, spiritual, and ethical aspects. While a second approach, clinical management of comorbidities (CMC) focuses on managing multiple diseases, other than cancer, such as heart or lung diseases through physical exercise, strength training, stress reduction, medication management, dietary recommendations, and education. Giving SPC, CMC, or a combination of both may work better in improving quality of life and survival after a donor stem cell transplant compared to standard of care in patients with blood diseases.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2/Phase 3 |
Detailed Description
OUTLINE: Patients are randomized to 1 of 4 arms.
ARM I: Patients undergo SPC on days -15 before to +56 after transplant.
ARM II: Patients undergo a CMC program on days -15 before to +56 after transplant.
ARM III: Patients undergo interventions as outlined in Arm I and Arm II.
ARM IV: Patients receive standard of care.
In all arms, patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment and 30, 90, 180, and 365 days post HCT. In all arms patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180, and 365 days post HCT.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Arm I (SPC) Patients undergo SPC on days -15 before to +56 after transplant. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment, 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180, and 365 days post HCT. |
Other: Supportive Palliative Care
focuses on relieving symptoms of stress from serious illness and care through physical, cultural, psychological, social, spiritual, and ethical aspects
Other Names:
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo HCT
Other Names:
Other: Questionnaire Administration
Ancillary studies
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
Other: Survey Administration
Ancillary studies
|
| Experimental: Arm II (CMC) Patients undergo a CMC program on days -15 to 56. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment, 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180, and 365 days post HCT.. |
Other: Clinical Management
physical exercise, strength training, stress reduction, medication management, dietary recommendations, and education
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo HCT
Other Names:
Other: Questionnaire Administration
Ancillary studies
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
Other: Survey Administration
Ancillary studies
|
| Experimental: Arm III (SPC and CMC) Patients undergo interventions as outlined in Arm I and Arm II. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180, and 365 days post HCT. |
Other: Supportive Palliative Care
focuses on relieving symptoms of stress from serious illness and care through physical, cultural, psychological, social, spiritual, and ethical aspects
Other Names:
Other: Clinical Management
physical exercise, strength training, stress reduction, medication management, dietary recommendations, and education
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Undergo HCT
Other Names:
Other: Questionnaire Administration
Ancillary studies
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
Other: Survey Administration
Ancillary studies
|
| Active Comparator: Arm IV (standard of care) Patients receive standard of care. Patients undergo HCT on day 0 and complete questionnaires and surveys at enrollment, 30, 90, 180, and 365 days post HCT. Patients complete a 4-meter walk test, 6-minute walk test, up and go test, measured strength test and cognitive assessment at enrollment, 90, 180, and 365 days post HCT. |
Other: Best Practice
Given standard of care
Other Names:
Other: Questionnaire Administration
Ancillary studies
Other: Quality-of-Life Assessment
Ancillary studies
Other Names:
Other: Survey Administration
Ancillary studies
|
Outcome Measures
Primary Outcome Measures
- Improvement in health-related quality of life (HRQOL) (Phase II) [First 90 days after HCT]
The arm with the largest mean change in Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) from baseline to day 90. The Wilcoxon rank-sum test will be used to compare change in FACT-BMT between arms, and this will also be the test to be used in computation of the conditional power at the end of phase II.
- Survival after hematopoietic cell transplantation (HCT) (Phase III) [At 1 year after HCT]
- Change in HRQOL (Phase III) [Baseline to 90 days post-HCT]
Will be measured by the FACT-BMT.
Secondary Outcome Measures
- Rate of overall survival [Up to 1 year]
Overall survival will be compared between each of the experimental arms and the usual care only (UCO) arm using the log-rank test. Arms that do not survive the screening phase will also be included for comparison.
- Non-relapse mortality [At 90 days and up to 1 year]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; analysis of variance (ANOVA) or Kruskal-Wallis test for comparisons involving more than two groups). Will use generalized estimating equations (GEEs) approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Cumulative incidence of relapse [Up to 1 year]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Relapse-free survival [Up to 1 year]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Cumulative incidence of frailty [Up to 1 year]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Cumulative incidence of disability [Up to 1 year]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Frequency of hospitalization [Up to 90 days after HCT]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Duration of each hospitalization [Up to 90 days after HCT]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Number of admissions to intensive care unit [Up to 90 days after HCT]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Duration of admissions to intensive care unit [Up to 90 days after HCT]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
- Days out of hospital alive [Up to 90 days after HCT]
Will be compared between arms using appropriate tests for continuous data (two-sample t-test or Wilcoxon rank-sum test, as appropriate for two-group comparisons; ANOVA or Kruskal-Wallis test for comparisons involving more than two groups). Will use GEEs approach for regression models, which can accommodate the within patient correlation structure and arbitrary patterns of missing data and also allow for the population average interpretation.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Vulnerable patients as defined by one or more of the following criteria
-
Age 65 years or older
-
Having Hematopoietic Cell Transplantation - Comorbidity Index (HCT-CI) scores of
= 3 (for patients that could be 20 years old and older)
-
Having frailty as determined by walk speed of < 0.8 m/s using 4-meter walk test (for patients that could be 40 years old and older)
-
Patients considered or referred for allogeneic HCT to treat a hematological malignant or non-malignant disease
-
Able to speak and read English - interaction with the interventionist trainer and endpoint measurement must occur in English
-
Willing and able to provide informed consent
-
Planned allogeneic HCT within 3 weeks - all types of donors and all sorts of conditioning regimens are allowed. Patients with suspected active disease (relatively old disease staging or relatively old intervention) or significant comorbidity (e.g. suspicious untreated pulmonary nodules) based on prior evaluations, that could delay the transplant would be considered for enrollment within a tighter window (10-14 days before allogeneic HCT) to allow for completed pre-HCT work-up evaluations that would confirm readiness to proceed with transplant
-
Able to exercise at low to moderate intensity, specifically taking into consideration the rare circumstances where subjects are not able to exercise due to either birth deformity or prior traumatic injury that affects their gait
-
Adequate cardiopulmonary reserve, as judged by data from the patient's electronic medical record as to whether a patient could walk up one flight of stairs, no need for supplemental oxygen, and/or physician judgment
Exclusion Criteria:
-
Orthopedic, neurologic or other problems which prevent safe ambulation and protocol adherence. Information on prior falls and other recent orthopedic or neurologic problems will be used to make judgment about protocol eligibility
-
Participation in another intervention clinical trial with HRQOL as a primary endpoint
-
Planned donor lymphocyte infusion (DLI) within 90 days post-transplant
-
Planned anti-cytotoxic therapies, other than tyrosine kinase inhibitors or single-agent monoclonal antibody, or FLT-3 inhibitors within 90 days of post-transplant unless pre-approved by the protocol principal investigator (PI)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Stanford Cancer Institute Palo Alto | Palo Alto | California | United States | 94304 |
| 2 | University of California, San Francisco | San Francisco | California | United States | 94143 |
| 3 | Wayne State University/Karmanos Cancer Institute | Detroit | Michigan | United States | 48201 |
| 4 | University of Minnesota/Masonic Cancer Center | Minneapolis | Minnesota | United States | 55455 |
| 5 | Mayo Clinic | Rochester | Minnesota | United States | 55905 |
| 6 | Oregon Health and Science University | Portland | Oregon | United States | 97239 |
| 7 | Baylor College of Medicine | Houston | Texas | United States | 77030 |
| 8 | Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | United States | 98109 |
Sponsors and Collaborators
- Fred Hutchinson Cancer Center
- National Cancer Institute (NCI)
- National Institutes of Health (NIH)
Investigators
- Principal Investigator: Mohamed Sorror, MD, MSc, Fred Hutch/University of Washington Cancer Consortium
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RG1004746
- NCI-2019-01097
- 9885
- R01CA227092