Storybook for Explaining Leukemia to Children

Overseen By Rosalia Soto

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Children's Hospital Los Angeles

Disqualifiers: Developmental delays, Cognitive delays, Chronic illnesses

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

140 Participants Needed

Trial Summary

What is the purpose of this trial?

The goal of this clinical trial is to measure the effects of using a storybook versus standard child life intervention with parents of children newly diagnosed with leukemia on parental stress. The main questions it aims to answer are: * What effect will the storybook have on parent/legal guardian stress at three timepoints: baseline, discharge, and follow up? * Will this storybook impact parent/legal guardian comfort levels and improve their child's understanding? Participants will be asked to complete surveys at three timepoints, prior to and following child life intervention and about 3.5 months later. During child life interventions, participants will receive resources and support to explain leukemia to their school aged, 3-16-year-old, child (patient or sibling). Researchers will compare Intervention and Control Groups to see if parental stress is lower in those who received the storybook in addition to the standard child life intervention versus the standard child life intervention alone.

Will I have to stop taking my current medications?

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

What data supports the effectiveness of the drug Who is Luke Eemia?, Gleevec, Imatinib mesylate for treating leukemia in children?

Imatinib mesylate (Gleevec) is effective in treating chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia in children, showing improved survival rates and being a standard first-line therapy due to its high response rates and safety profile. ^{1 2 3 4 5}

Is the treatment generally safe for children?

Imatinib mesylate, also known as Gleevec, is generally safe for children, but it can cause some side effects. Common mild side effects include skin rashes, while more serious ones like muscle and joint pain can occur, sometimes requiring stopping the treatment. ^{1 3 6 7 8}

How is the drug Who is Luke Eemia? (Gleevec, Imatinib mesylate) different from other treatments for leukemia?

Who is Luke Eemia? (Gleevec, Imatinib mesylate) is unique because it is a targeted therapy specifically designed to treat certain types of leukemia, like Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), by blocking a protein that helps cancer cells grow. This makes it different from traditional chemotherapy, which attacks all rapidly dividing cells. ^{1 2 4 9 10}

Research Team

Mandy Flores

Principal Investigator

Children's Hospital Los Angeles

Kaitlin Bennett

Principal Investigator

Children's Hospital Los Angeles

Erin Shields, MA

Principal Investigator

Children's Hospital Los Angeles

Eligibility Criteria

This trial is for parents or legal guardians of children aged 6-11 who have been newly diagnosed with leukemia. Participants will be involved in child life interventions and must be willing to complete surveys at three different times: when starting, at discharge, and about 3.5 months later.

Inclusion Criteria

I am the parent or guardian of a child aged 6-11 with a new leukemia diagnosis.

Parent/legal guardian is able to speak, read, and write English or Spanish, and give informed consent

I am the parent/guardian of a child under 6 or over 11, who has a sibling with new leukemia.

Exclusion Criteria

Their school-aged child does not give assent to participate

Their school-aged children have developmental or cognitive delays, and/or other chronic illnesses

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1 week

Baseline Assessment

Parents complete the Parenting Stress Index Short Form and Comfort Survey prior to child life intervention

1 week

1 visit (in-person)

Child Life Intervention

Parents receive standard child life care or the storybook intervention, and complete the Discharge Survey and Storybook Assessment (Intervention Group only)

1 week

1 visit (in-person)

Follow-up

Parents complete the Parenting Stress Index Short Form about 3.5 months after baseline to assess long-term effects

3.5 months

1 visit (virtual)

Treatment Details

Interventions

• Who is Luke Eemia?

Trial Overview The study is testing the impact of a storybook called 'Who is Luke Eemia?' on reducing parental stress compared to standard child life support alone. It also looks at whether the storybook helps improve understanding of leukemia for both parents and their children.

Participant Groups

Group I: Intervention (Storybook) Group Experimental Treatment 1 Intervention

Parents in this group will receive the "Who is Luke Eemia?" storybook, and will receive guidance from the child life specialist on how to utilize the storybook with their school-aged child. Parents in this study will complete surveys at three timepoints, these surveys include: (1) Comfort Survey (prior to child life intervention), (2) Discharge Survey and (3) Storybook Assessment survey to review the parent's impression of the storybook intervention tool (about 1 week following the child life intervention), and the (4) Parenting Stress Index -4-Short Form (at baseline, at the time of the discharge survey, and about 3.5 months later).

Group II: Control Active Control 1 Intervention

Parents in this group will receive the standard child life interventional support for explaining the leukemia diagnosis to the school-aged patient/sibling. Parents in this study will complete surveys at three timepoints, these surveys include: (1) Comfort Survey (prior to child life intervention), (2) Discharge Survey (about 1 week following the child life intervention), and the (3) Parenting Stress Index-4-Short Form (at baseline, at the time of the discharge survey, and about 3.5 months later).

Who is Luke Eemia? is already approved in United States, European Union for the following indications:

Approved in United States as Gleevec for:

• Chronic myeloid leukemia (CML)
• Gastrointestinal stromal tumors (GISTs)
• Acute lymphoblastic leukemia (ALL)

Approved in European Union as Glivec for:

• Chronic myeloid leukemia (CML)
• Gastrointestinal stromal tumors (GISTs)
• Acute lymphoblastic leukemia (ALL)

Who Is Running the Clinical Trial?

Children's Hospital Los Angeles

Lead Sponsor

Trials

257

Recruited

5,075,000+

Findings from Research

A population pharmacokinetic (PPK) model for imatinib and its active metabolite in children and young adults was developed using data from 41 subjects, revealing that total body weight significantly influences drug clearance and volume of distribution.

The findings suggest that while current pediatric dosing guidelines are based on adult data, this new model can help optimize dosing and manage toxicities in children, ensuring safer and more effective treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Population pharmacokinetics of imatinib mesylate and its metabolite in children and young adults. Menon-Andersen, D., Mondick, JT., Jayaraman, B., et al.[2015]

Imatinib mesylate is an effective treatment for pediatric patients with chronic myeloid leukemia, but none of the patients achieved complete molecular remission with this therapy alone, indicating that it may not be sufficient for a cure.

Stem cell transplantation appears to be necessary for a potential cure, as 3 out of 6 patients who underwent the procedure achieved undetectable BCR-ABL levels, and the relapse-free survival rate at four years was 65.6%.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical characteristics and treatment outcome of pediatric patients with chronic myeloid leukemia. Belgaumi, AF., Al-Shehri, A., Ayas, M., et al.[2021]

Imatinib mesylate (Gleevec) is recognized as the gold standard treatment for chronic myeloid leukemia, but it can cause cutaneous reactions that may restrict its use in some patients.

The case study presented demonstrates a successful progressive challenge for a patient who experienced a drug-induced rash from imatinib, suggesting that some patients may tolerate the medication after careful management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Successful progressive challenge after a cutaneous reaction to imatinib mesylate (Gleevec): a case report and review of the literature. Park, MA., Volcheck, GW., Guarderas, JC.[2016]

In a study of 92 children and adolescents with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), the addition of imatinib to intensive chemotherapy significantly improved 3-year event-free survival (EFS) to 80%, compared to historical controls at 35%.

The study found that adding imatinib did not increase toxicity compared to chemotherapy alone, and that the combination treatment was as effective as blood and marrow transplantation (BMT) in terms of EFS, suggesting a potential alternative to BMT for these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improved early event-free survival with imatinib in Philadelphia chromosome-positive acute lymphoblastic leukemia: a children's oncology group study. Schultz, KR., Bowman, WP., Aledo, A., et al.[2022]

Imatinib mesylate (Glivec) is the standard first-line treatment for chronic myeloid leukemia (CML) due to its high response rates and favorable safety profile, but switching to a copy version of the drug may lead to treatment failure, as seen in two case studies.

In one case, a patient relapsed after switching to the copy drug, while another patient experienced severe complications and died shortly after the switch, highlighting the need for caution and further research on the safety and efficacy of copy versions of imatinib.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Changing therapy from Glivecto a "copy" imatinib results in a worsening of chronic myeloid leukemia disease status: two case reports. Asfour, IA., Elshazly, SA.[2023]

Imatinib mesylate, a tyrosine kinase inhibitor used for treating chronic myeloid leukemia and gastrointestinal stromal tumors, is generally well-tolerated, with most patients experiencing only mild side effects (grade 1 or 2).

Approximately 30% of patients on imatinib develop skin toxicity, with 5% experiencing high-grade reactions, including cases of extensive skin hyperpigmentation, which is linked to the drug's effect on melanocytes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hyperpigmentation due to imatinib: A rare case of cutaneous involvement. Rehman, H., Hakim, N., Sugarman, R., et al.[2020]

In a review of 9 pediatric patients treated with imatinib mesylate for chronic myelogenous leukemia over 8 years, 88.9% experienced musculoskeletal pain, indicating a significant nonhematologic toxicity profile in children.

One-third of the patients had to discontinue therapy due to severe musculoskeletal pain, which is notably higher than the rates observed in adults, suggesting that pediatric patients may experience different and more severe side effects from imatinib.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nonhematologic toxicity of imatinib mesylate in pediatric patients with chronic myelogenous leukemia: a predominance of musculoskeletal pain. Heym, KM., Gressett Ussery, SM., Trinkman, H., et al.[2015]

A retrospective study involving 41 pediatric patients with Neurofibromatosis type 1 (NF-1) treated with the MEK inhibitor selumetinib highlights the issue of cutaneous toxicity, which may affect patient adherence to treatment.

The authors propose a treatment algorithm to manage the skin-related side effects of selumetinib, aiming to improve adherence and overall treatment outcomes for these young patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

MEK inhibitor-induced paronychia in a paediatric population: A tertiary centre experience. Palmeiro, AG., Silva, L., Pimentel, B., et al.[2023]

Adherence to oral chemotherapy regimens is crucial for achieving a cure in children with acute lymphoblastic leukemia (ALL), highlighting the importance of following treatment plans.

The study identifies race-specific factors that influence adherence to these regimens, suggesting that tailored support may be necessary to improve treatment outcomes across different racial groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

It takes a village. Winick, N.[2021]

Leukemia is the most common type of cancer in children in both Britain and the USA, highlighting its significant impact on pediatric health.

The report provides essential information for dentists on how to manage the dental care of children with leukemia, emphasizing the importance of understanding the medical aspects of the disease and its treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Childhood leukemia and dental considerations. Williams, MC., Lee, GT.[2005]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Population pharmacokinetics of imatinib mesylate and its metabolite in children and young adults. [2015]

2. Italy pubmed.ncbi.nlm.nih.gov

Clinical characteristics and treatment outcome of pediatric patients with chronic myeloid leukemia. [2021]

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

Successful progressive challenge after a cutaneous reaction to imatinib mesylate (Gleevec): a case report and review of the literature. [2016]

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

Improved early event-free survival with imatinib in Philadelphia chromosome-positive acute lymphoblastic leukemia: a children's oncology group study. [2022]

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

Changing therapy from Glivecto a "copy" imatinib results in a worsening of chronic myeloid leukemia disease status: two case reports. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Hyperpigmentation due to imatinib: A rare case of cutaneous involvement. [2020]

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

Nonhematologic toxicity of imatinib mesylate in pediatric patients with chronic myelogenous leukemia: a predominance of musculoskeletal pain. [2015]

8. Australia pubmed.ncbi.nlm.nih.gov

MEK inhibitor-induced paronychia in a paediatric population: A tertiary centre experience. [2023]

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

It takes a village. [2021]

10. Singapore pubmed.ncbi.nlm.nih.gov

Childhood leukemia and dental considerations. [2005]