MedPath

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

Phase 2
Active, not recruiting
Conditions
Hematopoietic and Lymphoid Cell Neoplasm
Non-Neoplastic Hematologic and Lymphocytic Disorder
Interventions
Other: Clinical Management
Other: Supportive Palliative Care
Other: Best Practice
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Other: Questionnaire Administration
Other: Quality-of-Life Assessment
Other: Survey Administration
Registration Number
NCT03870750
Lead Sponsor
Fred Hutchinson Cancer Center
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.

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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
455
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 50 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)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm I (SPC)Survey AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm II (CMC)Clinical ManagementPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm II (CMC)Allogeneic Hematopoietic Stem Cell TransplantationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm III (SPC and CMC)Survey AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm I (SPC)Supportive Palliative CarePatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm I (SPC)Allogeneic Hematopoietic Stem Cell TransplantationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm I (SPC)Questionnaire AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm I (SPC)Quality-of-Life AssessmentPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm III (SPC and CMC)Clinical ManagementPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm III (SPC and CMC)Allogeneic Hematopoietic Stem Cell TransplantationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm III (SPC and CMC)Supportive Palliative CarePatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm II (CMC)Questionnaire AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm II (CMC)Survey AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm II (CMC)Quality-of-Life AssessmentPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm III (SPC and CMC)Questionnaire AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm IV (standard of care)Questionnaire AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm III (SPC and CMC)Quality-of-Life AssessmentPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm IV (standard of care)Quality-of-Life AssessmentPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm IV (standard of care)Survey AdministrationPatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Arm IV (standard of care)Best PracticePatients 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. Patients may also complete surveys on medical and non-medical (transportation, lodging) costs related to transplant after HCT.
Primary Outcome Measures
NameTimeMethod
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.

Change in HRQOL (Phase III)Baseline to 90 days post-HCT

Will be measured by the FACT-BMT.

Survival after hematopoietic cell transplantation (HCT) (Phase III)At 1 year after HCT
Secondary Outcome Measures
NameTimeMethod
Rate of overall survivalUp 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.

Relapse-free survivalUp 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.

Duration of each hospitalizationUp 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.

Non-relapse mortalityAt 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.

Duration of admissions to intensive care unitUp 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.

Cumulative incidence of relapseUp 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 frailtyUp 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.

Days out of hospital aliveUp 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.

Cumulative incidence of disabilityUp 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 hospitalizationUp 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 unitUp 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.

Trial Locations

Locations (11)

Northwell Health Cancer Institute

🇺🇸

New Hyde Park, New York, United States

Stanford Cancer Institute Palo Alto

🇺🇸

Palo Alto, California, United States

University of California San Francisco

🇺🇸

San Francisco, California, United States

Wayne State University/Karmanos Cancer Institute

🇺🇸

Detroit, Michigan, United States

University of Minnesota/Masonic Cancer Center

🇺🇸

Minneapolis, Minnesota, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

🇺🇸

Cleveland, Ohio, United States

Oregon Health and Science University

🇺🇸

Portland, Oregon, United States

Vanderbilt University

🇺🇸

Nashville, Tennessee, United States

Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

🇺🇸

Houston, Texas, United States

Fred Hutch/University of Washington Cancer Consortium

🇺🇸

Seattle, Washington, United States

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