Let's Get REAL Tool for Pediatric Blood Cancers

Overseen By Ginny L Schulz, Ph.D., RN, CPNP-PC

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Washington University School of Medicine

Disqualifiers: Severe medical problems, Cognitive incapacity, others

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

Compensation: Varies

Number of Visits: 1

60 Participants Needed

Trial Summary

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

The trial information does not specify whether participants need to stop taking their current medications.

What safety data exists for the Let's Get REAL Tool for Pediatric Blood Cancers?

The safety of treatments for pediatric blood cancers, like those in the Let's Get REAL Tool, often involves monitoring adverse events (unwanted side effects). Studies show that children with cancer frequently experience adverse events, with many being severe, but some can be prevented or reduced in severity. This highlights the importance of careful monitoring and intervention to improve safety. ^{1 2 3 4 5}

What makes the Let's Get REAL treatment unique for pediatric blood cancers?

The Let's Get REAL treatment is unique because it likely involves precision medicine, which uses genetic profiling to tailor therapies specifically to the genetic makeup of a child's cancer, potentially improving outcomes and reducing side effects compared to traditional treatments. ^{6 7 8 9 10}

What is the purpose of this trial?

The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).

Research Team

Ginny Schulz, Ph.D., RN, CPNP-PC

Principal Investigator

Washington University School of Medicine

Eligibility Criteria

This trial is for young patients with conditions like metabolic disorders, diabetes, blood cancers, and immune deficiencies who are undergoing stem cell transplants or cellular therapy. It's open to kids aged 8-17 along with their parents. Specific eligibility details aren't provided.

Inclusion Criteria

* Children or adolescents 8-17 years of age referred for SCTCT.

* Diagnosis of malignant or nonmalignant disorder.

* Referred for any type of SCTCT. Autologous and allogeneic stem cell and cellular therapies are eligible.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Consultation Preparation

Participants and their parents use the Let's Get REAL family health communication tool up to one month prior to their SCTCT consultation visit.

4 weeks

1 visit (in-person) for SCTCT consultation

Consultation and Immediate Follow-up

SCTCT consultation visits are conducted and audio-recorded. Participants complete surveys up to one month after the consultation.

4 weeks

Post-Discharge Follow-up

Participants complete additional surveys up to one month post-discharge from SCTCT. Optional semi-structured interviews may be conducted up to 8 weeks after SCTCT consultation.

8 weeks

Treatment Details

Interventions

• Let's Get REAL

Trial Overview The 'Let's Get REAL' tool is being tested to see if it helps kids involved in the study make real-time decisions about their stem cell transplant and cellular therapy treatments. The study will involve two age groups: one for children aged 8-12 and another for teens aged 13-17.

Participant Groups

Group I: Let's Get REAL family health communication tool: Patients (8-12 years of age) Experimental Treatment 1 Intervention

Patients and parent(s) will be given the Let's Get REAL family health communication tool to use up to one month prior to their SCTCT consultation visit. It is a guide for pediatric patients and their families to learn about and discuss SCTCT. Participants will complete demographic and baseline surveys prior to using the tool, and then additional surveys up to one month after SCTCT consultation visit and up to one month post-discharge from SCTCT. SCTCT consultation visits will be audio-recorded. Participants may also participate in an optional semi-structured interview up to 8 weeks after SCTCT consultation.

Group II: Let's Get REAL family health communication tool: Patients (13-17 years of age) Experimental Treatment 1 Intervention

Patients and parent(s) will be given the Let's Get REAL family health communication tool to use up to one month prior to their SCTCT consultation visit. It is a guide for pediatric patients and their families to learn about and discuss SCTCT. Participants will complete demographic and baseline surveys prior to using the tool, and then additional surveys up to one month after SCTCT consultation visit and up to one month post-discharge from SCTCT. SCTCT consultation visits will be audio-recorded. Participants may also participate in an optional semi-structured interview up to 8 weeks after SCTCT consultation.

Group III: Let's Get REAL family health communication tool: Parents Experimental Treatment 1 Intervention

Patients and parent(s) will be given the Let's Get REAL family health communication tool to use up to one month prior to their SCTCT consultation visit. It is a guide for pediatric patients and their families to learn about and discuss SCTCT. Participants will complete demographic and baseline surveys prior to using the tool, and then additional surveys up to one month after SCTCT consultation visit and up to one month post-discharge from SCTCT. SCTCT consultation visits will be audio-recorded. Participants may also participate in an optional semi-structured interview up to 8 weeks after SCTCT consultation.

Who Is Running the Clinical Trial?

Washington University School of Medicine

Lead Sponsor

Trials

2,027

Recruited

2,353,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Findings from Research

Out of 236 Individual Case Safety Reports (ICSRs) analyzed, only 18 (7.6%) were classified as off-label cases, indicating a low incidence of off-label use of antineoplastic drugs in pediatric patients, primarily related to therapeutic indications.

Among the off-label cases, 29 adverse drug reactions (ADRs) were reported, with common reactions including diarrhea and neutropenia, suggesting that while off-label use is limited, it can still lead to significant safety concerns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System. Mascolo, A., Scavone, C., Bertini, M., et al.[2022]

Adverse event (AE) reporting in pediatric acute myeloid leukemia trials often underestimates the actual rates, with sensitivity for many AEs being less than 50%, indicating a need for improved reporting methods.

Using external electronic data sources, like Pediatric Health Information System (PHIS) billing and microbiology data, significantly enhances the accuracy of AE detection, particularly for specific toxicities such as viridans group streptococcal bacteremia, which showed high sensitivity and positive predictive value.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Accuracy of Adverse Event Ascertainment in Clinical Trials for Pediatric Acute Myeloid Leukemia. Miller, TP., Li, Y., Kavcic, M., et al.[2020]

In a study of 181 pediatric patients with acute lymphoblastic leukemia, the incidence of healthcare-associated adverse events was found to be 51.8 per 1000 patient-days, affecting 81.2% of patients during the induction phase, with most events being severe or higher in severity.

Infectious processes were the most common severe adverse events, accounting for 30.5%, and 80.2% of these adverse events led to hospital readmissions, highlighting the need for improved safety measures to reduce preventable and ameliorable adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Incidence, severity, and preventability of adverse events during the induction of patients with acute lymphoblastic leukemia in a tertiary care pediatric hospital in Mexico. Vázquez-Cornejo, E., Morales-Ríos, O., Hernández-Pliego, G., et al.[2022]

In 2017, pharmacists conducted 2361 interventions for 381 pediatric cancer patients, achieving a high acceptance rate of 97.2%, with over half of the interventions deemed clinically significant.

The interventions not only helped prevent adverse drug events but also resulted in substantial cost savings, with a cost-benefit of $28,705 from the hospital's perspective and $35,611 from the patient's perspective, indicating a positive economic impact.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical and Economic Impact of Pharmacists' Intervention on Care of Pediatric Hematology and Oncology Patients. Kim, HJ., Lee, S., Lee, YJ., et al.[2023]

The ExtractEHR automated package effectively identified and graded laboratory adverse events in a cohort of 1,077 children with acute myeloid leukaemia and acute lymphoblastic leukaemia, demonstrating its scalability and accuracy in capturing adverse event rates.

The study found that ExtractEHR reported significantly higher rates of laboratory adverse events compared to those manually reported in clinical trials, suggesting that automated systems can enhance the understanding of chemotherapy risks in pediatric patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rates of laboratory adverse events by course in paediatric leukaemia ascertained with automated electronic health record extraction: a retrospective cohort study from the Children's Oncology Group. Miller, TP., Getz, KD., Li, Y., et al.[2023]

Survival rates for children with cancer have significantly improved due to advancements in treatment strategies, including intensified chemotherapy and better risk stratification using both traditional and novel biological markers.

Recent discoveries in cancer genomics have identified specific genomic alterations and pathways in childhood cancers, paving the way for precision medicine that targets these alterations to enhance treatment effectiveness while reducing side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Precision Medicine in Pediatric Oncology: Translating Genomic Discoveries into Optimized Therapies. Tran, TH., Shah, AT., Loh, ML.[2021]

A hybrid-capture next-generation sequencing (NGS) panel was developed for detecting genetic alterations in pediatric solid tumors, achieving a high quality control pass rate of 82% from 255 samples submitted, indicating its reliability for clinical use.

The study found that 70% of sequenced samples had at least one genetic alteration, with 51% of samples showing clinically actionable alterations, demonstrating the potential of this approach for personalized treatment strategies in children with cancer.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A tailored molecular profiling programme for children with cancer to identify clinically actionable genetic alterations. George, SL., Izquierdo, E., Campbell, J., et al.[2020]

The Pediatric Molecular Tumor Board (PMTB) at MSKCC reviewed 41 cases from 39 patients, primarily focusing on gliomas, acute myeloid leukemia, and neuroblastoma, demonstrating the potential of genomic profiling to guide targeted therapies.

Out of the therapeutic recommendations made, 46% were implemented, with 70% involving targeted therapies, highlighting the practical application of genomic data in pediatric oncology, although most recommendations were based on varying levels of evidence, indicating a need for improved tools and protocols for future clinical use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Integrating Genomics Into Clinical Pediatric Oncology Using the Molecular Tumor Board at the Memorial Sloan Kettering Cancer Center. Ortiz, MV., Kobos, R., Walsh, M., et al.[2022]

Integrating genomic profiling into the clinical management of 56 children with high-risk hematologic malignancies revealed that 90% of patients had clinically impactful findings, with 80% having at least one targetable gene variant or fusion identified.

The study demonstrated that comprehensive genomic testing not only aids in identifying potential targeted therapies but also significantly refines diagnosis and prognosis for patients, with 34% benefiting from improved clinical insights.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience. Marks, LJ., Oberg, JA., Pendrick, D., et al.[2022]

St. Jude Cloud is a groundbreaking data-sharing platform that provides access to over 1.25 petabytes of genomic data from more than 10,000 pediatric cancer patients and survivors, which is crucial for improving research and treatment outcomes.

The platform includes advanced tools for data analysis and visualization, enabling researchers to classify pediatric cancer subtypes and understand mutational signatures, thereby enhancing the overall knowledge base for pediatric cancer treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

St. Jude Cloud: A Pediatric Cancer Genomic Data-Sharing Ecosystem. McLeod, C., Gout, AM., Zhou, X., et al.[2022]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System. [2022]

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

Accuracy of Adverse Event Ascertainment in Clinical Trials for Pediatric Acute Myeloid Leukemia. [2020]

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

Incidence, severity, and preventability of adverse events during the induction of patients with acute lymphoblastic leukemia in a tertiary care pediatric hospital in Mexico. [2022]

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

Clinical and Economic Impact of Pharmacists' Intervention on Care of Pediatric Hematology and Oncology Patients. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Rates of laboratory adverse events by course in paediatric leukaemia ascertained with automated electronic health record extraction: a retrospective cohort study from the Children's Oncology Group. [2023]

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

Precision Medicine in Pediatric Oncology: Translating Genomic Discoveries into Optimized Therapies. [2021]

7. England pubmed.ncbi.nlm.nih.gov

A tailored molecular profiling programme for children with cancer to identify clinically actionable genetic alterations. [2020]

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

Integrating Genomics Into Clinical Pediatric Oncology Using the Molecular Tumor Board at the Memorial Sloan Kettering Cancer Center. [2022]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Precision Medicine in Children and Young Adults with Hematologic Malignancies and Blood Disorders: The Columbia University Experience. [2022]

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

St. Jude Cloud: A Pediatric Cancer Genomic Data-Sharing Ecosystem. [2022]