Quality of Life Strategies After Stem Cell Transplant for Blood Diseases

No longer recruiting at 14 trial locations

Overseen By Mohamed Sorror, MD, MSc

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Fred Hutchinson Cancer Center

Must not be taking: Anti-cytotoxic therapies

Disqualifiers: Orthopedic, Neurologic, HRQOL trial, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores ways to improve life quality and survival for older or frail patients after a stem cell transplant for blood diseases. It compares supportive care, which focuses on symptom relief and overall well-being, with clinical management, which addresses other health issues like heart or lung diseases, to determine the most effective approach. Participants will receive supportive care, clinical management, a combination of both, or standard care. Individuals who have undergone allogeneic hematopoietic stem cell transplantation (donor stem cell transplant) and live with blood diseases, especially those who struggle with physical activities or managing multiple health issues, might be suitable candidates. As a Phase 2 and Phase 3 trial, this study evaluates the treatment's effectiveness in a smaller group and represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking advancements in care.

Do I have to stop taking my current medications for this trial?

The trial protocol does not specify if you need to stop taking your current medications. However, it mentions that certain anti-cytotoxic therapies are not allowed within 90 days post-transplant unless approved by the principal investigator. It's best to discuss your specific medications with the trial team.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, it mentions that certain anti-cytotoxic therapies are not allowed within 90 days post-transplant unless approved by the principal investigator.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that supportive and palliative care (SPC) and managing other health conditions (CMC) can aid patients after stem cell transplants. Addressing other health issues, such as heart or lung diseases, is crucial for those who have undergone these transplants. This care may include exercise, diet, and medication.

Studies indicate that pre-existing health problems can lead to complications after a transplant. However, managing these issues can improve health outcomes. SPC aims to reduce stress and manage symptoms, enhancing quality of life.

These treatments are generally well-tolerated, though patients may still face risks from the transplant itself. Combining SPC and CMC offers a balanced approach to care, potentially reducing organ damage and improving overall well-being. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about the Quality of Life Strategies After Stem Cell Transplant trial because it explores how different care approaches can enhance life after a stem cell transplant for blood diseases. Unlike the standard of care, which focuses mainly on the medical aspects of recovery, this trial examines the impact of Clinical Management Care (CMC), Supportive Palliative Care (SPC), and a combination of both. These approaches aim to address not just physical recovery, but also the emotional, social, and financial challenges patients face. The trial hopes to uncover which strategies best improve overall well-being and quality of life, offering a more holistic approach to treatment.

What evidence suggests that this trial's treatments could be effective for improving quality of life after a stem cell transplant?

Research has shown that addressing multiple health problems simultaneously can greatly enhance the quality of life for individuals who have undergone a stem cell transplant. Most patients report feeling good to excellent within a few years post-transplant. In this trial, some participants will receive Supportive Palliative Care (SPC), which eases symptoms and stress, improving life quality and reducing physical and mental distress during and after the transplant. Others will undergo Clinical Management of Comorbidities (CMC), focusing on managing multiple health issues. A treatment arm combines both SPC and CMC, potentially leading to even better outcomes in terms of quality of life and survival. While each method alone has demonstrated benefits, using them together could provide the most comprehensive support for patients. ^{6 7 8 9 10}

Who Is on the Research Team?

Mohamed Sorror, MD, MSc

Principal Investigator

Fred Hutch/University of Washington Cancer Consortium

Are You a Good Fit for This Trial?

This trial is for older, weak, or frail patients with blood diseases who are about to receive a donor stem cell transplant. Participants must be 65+, able to speak and read English, have good heart and lung function without needing extra oxygen, and be willing to consent. They should also have certain health scores indicating comorbidities or frailty based on walking speed tests.

Inclusion Criteria

I am being considered for a stem cell transplant to treat my blood disease.

Able to speak and read English - interaction with the interventionist trainer and endpoint measurement must occur in English

You can engage in light to moderate physical activity. In very rare cases, if you have a birth deformity or a previous injury that affects your ability to walk, you may not be able to participate.

Exclusion Criteria

I am scheduled for a donor lymphocyte infusion within 3 months after my transplant.

I can walk safely and follow study rules despite my health issues.

You are currently participating in another research study where the main focus is on measuring your quality of life.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Transplant Preparation

Participants undergo supportive and palliative care (SPC) or clinical management of comorbidities (CMC) from days -15 to 0 before transplant

2 weeks

Transplant and Immediate Post-Transplant

Participants undergo hematopoietic cell transplantation (HCT) on day 0 and receive SPC, CMC, or both until day +56

8 weeks

Multiple visits for assessments and interventions

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments at 30, 90, 180, and 365 days post HCT

1 year

4 visits (in-person) at specified intervals

What Are the Treatments Tested in This Trial?

Interventions

• Allogeneic Hematopoietic Stem Cell Transplantation
• Best Practice
• Clinical Management
• Quality-of-Life Assessment
• Questionnaire Administration
• Supportive Palliative Care
• Survey Administration

Trial Overview

The study compares supportive palliative care (SPC), clinical management of comorbidities (CMC), or both against standard care in improving life quality and survival after stem cell transplants. SPC addresses stress relief from illness while CMC manages other diseases through exercise, medication, diet, etc.

How Is the Trial Designed?

Group I: Arm III (SPC and CMC) Experimental Treatment 6 Interventions

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

Group II: Arm II (CMC) Experimental Treatment 5 Interventions

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

Group III: Arm I (SPC) Experimental Treatment 5 Interventions

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

Group IV: Arm IV (standard of care) Active Control 4 Interventions

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

Allogeneic Hematopoietic Stem Cell Transplantation is already approved in European Union, United States, Canada, Japan for the following indications:

Approved in European Union as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in United States as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Canada as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Approved in Japan as Allogeneic Hematopoietic Stem Cell Transplantation for:

• Primary immunodeficiency disorders
• Immune dysregulatory disorders
• Hemophagocytic lymphohistiocytosis
• Bone marrow failure syndromes
• Hemoglobinopathies

Who Is Running the Clinical Trial?

Fred Hutchinson Cancer Center

Lead Sponsor

Trials

583

Recruited

1,341,000+

Fred Hutchinson Cancer Research Center

Lead Sponsor

Trials

444

Recruited

148,000+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

Leukemia patients who underwent allogeneic hematopoietic cell transplantation (allo-HCT) with a Mexican reduced-intensity conditioning regimen reported a better mental quality of life compared to matched reference subjects, indicating potential psychological benefits of this treatment approach.

The physical quality of life scores were similar between allografted patients and reference subjects, suggesting that while mental health may improve, physical health outcomes may not differ significantly after this type of transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Health-Related Quality of Life in leukemia Survivors of Allogeneic Hematopoietic Stem Cell Transplantation Employing the Mexican Reduced-Intensity Conditioning. González-Ramírez, MP., Miravete-Lagunes, K., Gómez-de-León, A., et al.[2018]

Allogeneic stem cell transplantation (allo-SCT) can offer a chance for cure but also poses significant risks, including high mortality and long-term complications like fatigue and psychological distress, which can persist for years after the procedure.

Integrating specialized palliative care early in the allo-SCT process has shown promising effectiveness in improving patient outcomes, although there are still barriers to its implementation that need to be addressed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

What is known about palliative care in adult patients with allogeneic stem cell transplantation (allo-SCT)? Simon, ST., Pralong, A., Hallek, M., et al.[2021]

Hematopoietic stem-cell transplantation (HSCT) can offer a chance for cure but also comes with high risks of morbidity and mortality, leading to significant symptom burden and decreased quality of life for patients.

Integrating palliative care early in the HSCT process can improve the well-being of patients and their families, as it focuses on comfort and quality of life, even when curative intent is present.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Strange Bedfellows No More: How Integrated Stem-Cell Transplantation and Palliative Care Programs Can Together Improve End-of-Life Care. Levine, DR., Baker, JN., Wolfe, J., et al.[2020]

Integrating palliative care with treatment during allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to effectively reduce symptom burden and mood disturbances, improving health-related quality of life for patients.

An interdisciplinary approach involving both transplant clinicians and palliative care specialists can address the physical and psychological challenges faced by patients and their families throughout the HSCT process.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Palliative care during and following allogeneic hematopoietic stem cell transplantation. Mitchell, SA.[2023]

This study aims to explore the supportive and palliative care needs of patients undergoing allogeneic stem cell transplantation (allo-SCT) by collecting qualitative and quantitative data from patients, informal carers, and healthcare providers, involving 20 patients and 100 informal carers in interviews and surveys.

The findings will help develop targeted interventions to reduce symptom burden and improve quality of life for allo-SCT recipients, addressing their existential concerns and changing goals of care, which are currently under-researched.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Developing a supportive and palliative care intervention for patients with allogeneic stem cell transplantation: protocol of a multicentre mixed-methods study (allo-PaS). Pralong, A., Herling, M., Holtick, U., et al.[2023]

Allogeneic hematopoietic stem cell transplantation (HSCT) can potentially replace a diseased immune system with healthy donor cells, showing promise in treating conditions like severe aplastic anemia and autoimmune diseases, but results can vary widely with both complete remissions and treatment failures reported.

Despite its potential benefits, allogeneic HSCT is associated with significant risks, including morbidity and mortality, leading to the conclusion that it cannot be generally recommended without further carefully conducted studies to identify the best patient populations and treatment technologies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Allogeneic hematopoietic stem cell transplantation for severe autoimmune diseases. Gratwohl, A.[2009]

In a study of 103 long-term survivors of allogeneic hematopoietic stem cell transplantation (allo-HSCT), nearly half exhibited pathological findings on high-resolution CT scans, indicating significant pulmonary complications, including chronic graft-versus-host disease in 33% of participants.

Survivors showed lower pulmonary function compared to healthy controls, with mean lung volumes over 80% of predicted values, highlighting the need for lifelong monitoring of pulmonary health in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Late-Onset, Noninfectious Pulmonary Complications following Allogeneic Hematopoietic Stem Cell Transplantation: A Nationwide Cohort Study of Long-Term Survivors. Myrdal, OH., Aaløkken, TM., Diep, PP., et al.[2022]

In a study of 10,632 patients who survived 2 years after allogeneic hematopoietic cell transplantation (HCT), the median follow-up was 9 years, revealing an 85% probability of being alive 10 years post-transplant.

Despite the promising long-term survival rates, patients still face a higher risk of late deaths compared to the general population, particularly due to factors like older age and chronic graft-versus-host disease (GVHD), indicating a need for improved management strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term survival and late deaths after allogeneic hematopoietic cell transplantation. Wingard, JR., Majhail, NS., Brazauskas, R., et al.[2022]

Integrating palliative care early for hematopoietic cell transplant (HCT) recipients can significantly enhance the quality of care by addressing complex symptoms and improving communication about care goals, especially for those at high risk of complications.

Palliative care services can assist HCT nurses in managing the significant side effects and challenges faced by transplant recipients, ultimately benefiting both patients and their families.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Palliative care of hematopoietic cell transplant recipients and families. Tierney, DK., Passaglia, J., Jenkins, P.[2014]

A 2-year initiative by the National Marrow Donor Program engaged patients, caregivers, and researchers to establish a patient-centered outcomes research (PCOR) agenda for hematopoietic cell transplantation (HCT), identifying six key areas of interest for future research.

The initiative highlighted the need for consistent measures and homogenous patient populations in HCT research to improve the comparability of outcomes, and it provided recommendations for priority research questions to address existing knowledge gaps.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Engaging Patients in Setting a Patient-Centered Outcomes Research Agenda in Hematopoietic Cell Transplantation. Burns, LJ., Abbetti, B., Arnold, SD., et al.[2021]

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sciencedirect.com

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