DREAMS Program for Pediatric Stem Cell Transplant Patients

(DREAMS Trial)

Recruiting at 2 trial locations

Overseen By Lucille Lokko, BA

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: Dana-Farber Cancer Institute

Disqualifiers: Primary team declines permission

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

Compensation: Varies

Number of Visits: 3

Duration: 2 weeks

10 Participants Needed

Trial Summary

What is the purpose of this trial?

Pediatric patients undergoing stem cell transplant (SCT) are hospitalized for extended periods and are at high risk for sleep disturbances. In order to begin to address the environmental issues that SCT recipients face during inpatient hospitalizations, investigators will conduct a single arm pilot study of a program entitled 'Developing Restful Environments and Management Strategies' (DREAMS). The program will provide children receiving SCT and families with information and a kit that includes tools which may support sleep and circadian health during an inpatient hospitalization.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications.

What data supports the effectiveness of the DREAMS Program treatment for pediatric stem cell transplant patients?

Research shows that stem cell transplantation is effective for serious pediatric diseases, with improvements in survival rates over time. Additionally, bone marrow transplantation, a similar treatment, has been shown to offer potential cures for otherwise fatal diseases, with low mortality due to better supportive care. ^{1 2 3 4 5}

Is the DREAMS Program for Pediatric Stem Cell Transplant Patients generally safe?

Stem cell transplants in children have shown improvements in survival rates over the years, but they can still lead to serious long-term health issues like chronic conditions and frail health. While transplant-related mortality has decreased, survivors often face higher risks of severe health problems compared to those who receive conventional treatments. ^{1 5 6 7 8}

How is the DREAMS Program treatment different from other treatments for pediatric stem cell transplant patients?

The DREAMS Program is unique because it focuses on providing home care for pediatric stem cell transplant patients, which is an alternative to traditional inpatient or outpatient recovery programs. This approach may reduce the stress and demands on patients and their families by allowing recovery in a familiar home environment, although it requires significant support for caregivers. ^{1 3 4 9 10}

Research Team

Eric Zhou, PhD

Principal Investigator

Dana-Farber Cancer Institute

Eligibility Criteria

This trial is for children aged 9-17 who are scheduled to receive a stem cell transplant at Boston Children's Hospital and speak English, along with their primary caregiver. It's not open to those whose medical team doesn't allow them to participate.

Inclusion Criteria

I am between 9 and 17 years old.

I am scheduled for a stem cell transplant at Boston Children's Hospital.

Exclusion Criteria

Primary team declines permission to approach

Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Baseline

Baseline visit with survey and standard of care stem cell infusion

1 day

1 visit (in-person)

Treatment

Introduction to DREAMS program, Sleep and Circadian Care Kit, Actigraph, and Fitbit

2 weeks

1 visit (in-person)

Follow-up

Participants complete follow-up surveys and assessments

1 week

1 visit (in-person)

Treatment Details

Interventions

• DREAMS Program

Trial Overview The DREAMS program, which includes an educational video and care kit designed to help pediatric SCT patients sleep better during hospital stays, is being tested in this single-arm pilot study.

Participant Groups

Group I: DREAMS Program Experimental Treatment 1 Intervention

Enrolled participants and parent(s) will complete: * Baseline visit with survey * Standard of care stem cell infusion * In-clinic visit with introduction to DREAMS program, Sleep and Circadian Care Kit, Actigraph, and Fitbit. * Follow up survey

Who Is Running the Clinical Trial?

Dana-Farber Cancer Institute

Lead Sponsor

Trials

1,128

Recruited

382,000+

American Cancer Society, Inc.

Collaborator

Trials

237

Recruited

110,000+

Findings from Research

In a study of 156 children undergoing stem cell transplantation (SCT) from 1998 to 2002, the transplant-related mortality (TRM) for autologous SCT significantly improved from 12.2% to 0%, indicating enhanced safety in recent years.

Overall survival (OS) rates also improved dramatically from 34% in the earlier period (1989-1998) to 80.4% in the later period (1999-2002), showing that SCT has become a more effective treatment for pediatric patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hemopoietic stem cell transplantation in childhood: reduction in mortality and improvement of survival over the years. Espigado, I., Marín-Niebla, A., Pérez-Hurtado, JM., et al.[2005]

A Pediatric Early Warning Score (PEWS) of 8 or higher is significantly associated with the need for PICU admission in pediatric hematopoietic cell transplant patients, showing a sensitivity of 76% and specificity of 90%.

Incorporating weight gain into the PEWS model enhances its predictive ability for critical care needs, increasing the area under the receiver operating characteristic curve from 0.83 to 0.88, suggesting that monitoring weight gain could improve patient assessments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Modification and Assessment of the Bedside Pediatric Early Warning Score in the Pediatric Allogeneic Hematopoietic Cell Transplant Population. Cater, DT., Tori, AJ., Moser, EAS., et al.[2019]

In a bone marrow transplant program at Children's Hospital in New Orleans, 20 children aged 17 months to 20 years were treated, achieving an overall 2-year survival rate of 40%.

For patients classified as good-risk, the projected 2-year disease-free survival rate was significantly higher at 85%, highlighting the effectiveness of bone marrow transplantation as a potential cure for pediatric malignancies and congenital disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Bone marrow transplantation: a children's hospital of New Orleans experience. Yu, LC., Ode, D., Warrier, RP.[2004]

The homebound hematopoietic stem cell transplant (HSCT) program is perceived as safe and effective by caregivers, providing high-quality medical care in a familiar environment that enhances the patient's sense of normalcy and privacy.

Caregivers reported unmet needs, particularly in feeling unprepared for nursing responsibilities and managing emotional distress, highlighting the need for additional psychotherapeutic support and training to better equip them for home care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Exploring the caregiver's experience in an innovative homebound hematopoietic stem cell transplantation program. Jenkelowitz, J., Garzon, MG., Lynch, K., et al.[2022]

In a study of 41 pediatric patients undergoing bone marrow transplantation, the overall survival rate was 70% for allogeneic transplants and 59.4% for autologous transplants, indicating comparable outcomes to existing literature.

Acute graft-versus-host disease (GVHD) was a significant complication, with 4 patients experiencing severe forms, and infections were common, affecting 58.5% of patients, highlighting the need for careful monitoring and management during recovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical and epidemiological analysis of bone marrow transplantation in a pediatric oncology unit]. de Castro, CG., Gregianin, LJ., Brunetto, AL.[2020]

Survivors of hematopoietic stem cell transplantation (HSCT) have a significantly higher burden of severe chronic conditions compared to those treated with conventional therapy, with 81.3% of HSCT survivors experiencing grade 3-4 conditions versus 69.2% in the conventional therapy group.

By age 25, HSCT survivors had a much higher incidence of serious health issues, including second cancers (17.0% vs 7.9%), cardiovascular (19.6% vs 10.2%), and pulmonary conditions (16.1% vs 4.6%), highlighting the need for ongoing clinical follow-up and targeted prevention strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chronic disease burden and frailty in survivors of childhood HSCT: a report from the St. Jude Lifetime Cohort Study. Eissa, HM., Lu, L., Baassiri, M., et al.[2019]

In a study of 31 children undergoing hematopoietic stem cell transplantation (HSCT), 94% experienced early infectious complications, with the first infections typically occurring around 6 days post-transplant and lasting about 9 days.

The use of antibiotic prophylaxis, specifically Tienam, was effective in delaying the onset of these early infections, highlighting its potential role in managing infection risks during the critical post-transplant period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical analysis of 29 children with early infectious complications following hematopoietic stem cells transplantation]. Li, Y., Huang, SL., Fang, JP., et al.[2019]

From 2000 to 2012, the number of pediatric hematopoietic stem cell transplants (HSCT) for nonmalignant conditions doubled, indicating a significant increase in the use of this treatment.

In-hospital mortality rates for these procedures decreased from 13.4% to 7.1%, while infections like bacteremia and vascular catheter infections also declined, suggesting improved safety and outcomes over the years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

In-hospital mortality of hematopoietic stem cell transplantation among children with nonmalignancies: A nationwide study in the United States from 2000 to 2012. Takahashi, T., Pereda, MA., Bala, N., et al.[2019]

Bone marrow transplantation (BMT) is a critical treatment for children with severe hematological or oncological diseases, but it raises unique ethical and legal challenges, especially when a related donor is not available.

The process requires informed consent from both the donor and the recipient's parents, and it is essential that BMT units meet specific requirements to ensure patient safety and successful outcomes, including having experienced medical staff and support services in place.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ethical problems in bone marrow transplantation in children. Massimo, L.[2006]

Hematopoietic stem cell transplantation (HSCT) is a critical treatment option for children with serious blood cancers, often being their only chance for survival.

The process of HSCT involves complex financial, ethical, and emotional challenges for both the donor siblings and the recipients, highlighting the need for support during this difficult time.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ethical issues facing hematopoietic stem cell transplantation in India. Raturi, M., Das, K.[2021]

References

1. United States pubmed.ncbi.nlm.nih.gov

Hemopoietic stem cell transplantation in childhood: reduction in mortality and improvement of survival over the years. [2005]

2. United States pubmed.ncbi.nlm.nih.gov

Modification and Assessment of the Bedside Pediatric Early Warning Score in the Pediatric Allogeneic Hematopoietic Cell Transplant Population. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Bone marrow transplantation: a children's hospital of New Orleans experience. [2004]

4. England pubmed.ncbi.nlm.nih.gov

Exploring the caregiver's experience in an innovative homebound hematopoietic stem cell transplantation program. [2022]

5. Brazil pubmed.ncbi.nlm.nih.gov

[Clinical and epidemiological analysis of bone marrow transplantation in a pediatric oncology unit]. [2020]

6. United States pubmed.ncbi.nlm.nih.gov

Chronic disease burden and frailty in survivors of childhood HSCT: a report from the St. Jude Lifetime Cohort Study. [2019]

7. China pubmed.ncbi.nlm.nih.gov

[Clinical analysis of 29 children with early infectious complications following hematopoietic stem cells transplantation]. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

In-hospital mortality of hematopoietic stem cell transplantation among children with nonmalignancies: A nationwide study in the United States from 2000 to 2012. [2019]

9. England pubmed.ncbi.nlm.nih.gov

Ethical problems in bone marrow transplantation in children. [2006]

10. France pubmed.ncbi.nlm.nih.gov

Ethical issues facing hematopoietic stem cell transplantation in India. [2021]