Introduction

Neuroblastoma is the most common extracranial solid tumor diagnosed in children, presenting complex treatment challenges1.

Throughout diagnosis and treatment, many patients and caregivers report a lack of adequate tools and information to navigate therapy options. Addressing these gaps is essential to improving care experiences and outcomes.

This consensus program was initiated to establish best practices for enhancing patient and caregiver experiences by:

  • Empowering caregivers with the knowledge and tools to engage in transparent, shared decision-making with healthcare providers
  • Supporting patient advocacy groups and treatment centers in bridging gaps in education on clinical trials, treatment, options, and long-term side effect management
  • Bringing the patient/caregiver voice to the forefront
  • Reflecting the commitment of the healthcare team and ethicist to promote compassionate, ethical, patient-centered care through enhanced collaborative dialogue and shared decision-making

Objective

To develop a set of consensus statements that address the needs of patients with neuroblastoma and their caregivers, while identifying opportunities to improve their experiences through enhanced dialogue and shared decision-making with physicians and care team members.

Methods

  • A multidisciplinary advisory panel was convened, consisting of neuroblastoma healthcare professionals (n=3), allied health professionals (n=3), and advocates/caregivers (n=3)
  • A literature review identified key information gaps and needs across the treatment journey, informing the development of 31 draft consensus statements
  • Panelists independently rated their agreement with each statement using a 5-point Likert scale (5=strongly agree)
  • Consensus was defined as a median rating of ≥4 on a 5-point Likert scale, indicating agreement to accept the statement; statements with a median score below this threshold were considered to lack consensus and were subject to further review or refinement
  • A virtual consensus meeting held on 15 November 2024, allowed panelists to refine, adjust, and propose new statements
  • Panelists then re-rated and revised new statements to confirm consensus on content and tone

Results

  • Thirty final consensus statements were developed, spanning the six domains
  • All revised and newly proposed statements achieved strong consensus, each scoring an average of 4.1 or greater out of 5 points on the Likert scale
    • Statements and consensus scores can be viewed below
  • Key themes of the panel discussion emphasized the need for:
    • The importance of transparency and comprehensiveness in patient-provider interaction
    • Recognition of families’ evolving priorities over the diagnosis and treatment journey
    • A stronger commitment to shared decision-making
  • Advisors selected one key recommendation within each domain that they considered to be most impactful
  • Proposed solutions included:
    • Education for providers and other stakeholders on the consensus statements
    • Development of hospital and community-based resources to support families from diagnosis through treatment
    • Engagement of patient advocacy organizations to become familiar with and actively disseminate the consensus statements

Conclusions

  • The high level of consensus among advisors highlights critical opportunities to enhance patient and caregiver education in neuroblastoma care
  • These results represent an important step toward bridging educational gaps and establishing best practices in shared decision-making between patients, families, and providers
  • Future efforts will focus on raising awareness in clinical settings of the issues highlighted in the consensus recommendations and creating solutions to address them

Domains and Key Statements

Critical Encounters

At diagnosis, relapse, or progression, caregivers should receive referrals to support networks, contact information for second opinions and key opinion leaders, educational resources, and information on all treatment options—regardless of availability at their institution or the patient's insurance status.*

Caregivers often feel overwhelmed at the point of diagnosis/progression/relapse, feel significant time pressure, struggle to compare possible therapeutic options effectively, and lack the proper support to make informed decisions.*

*These statements were identified as the "most important" within their respective domains, as determined by a vote of the steering committee member.

Mean
Consensus
Score
4.1
Caregivers may not receive information on all available treatment options at the time of diagnosis or relapse, for various reasons.
4.2
Caregivers often feel overwhelmed at the point of diagnosis/progression/relapse, feel significant time pressure, struggle to compare possible therapeutic options effectively, and lack the proper support to make informed decisions.
4.3
At diagnosis, relapse, or progression, caregivers should receive referrals to support networks, contact information for second opinions and key opinion leaders, educational resources, and information on all treatment options—regardless of availability at their institution or the patient’s insurance status.
4.7
Families should be assessed for their needs and goals, psychosocial and socioeconomic needs, health literacy, and desired level of involvement in decision-making, with frequent reassessment throughout the treatment journey.
4.2
Healthcare teams should receive training on appropriate, compassionate delivery of difficult diagnoses, explanation of disease pathology, and the child’s prognosis.

Unmet Educational Needs

Educated and engaged caregivers are more likely to feel empowered and satisfied both during and after the decision-making process.*

When asking about alternative treatment options, clinical trials, or second opinions, caregivers should receive explanations of the options and their appropriateness and availability, without outright dismissal of these alternatives.*

*These statements were identified as the "most important" within their respective domains, as determined by a vote of the steering committee member.

Mean
Consensus
Score
4.1
Neuroblastoma advocacy organizations can provide supplemental education to patients and their siblings.
4.4
Caregivers often have unmet educational needs and want their healthcare team to provide them with high-quality, thorough, trustworthy information about neuroblastoma and its treatment options.
4.7
Educated and engaged caregivers are more likely to feel empowered and satisfied both during and after the decision-making process.
4.3
When asking about alternative treatment options, clinical trials, or second opinions, caregivers should receive explanations of the options and their appropriateness and availability, without outright dismissal of these alternatives.
4.8
Healthcare teams vary in experience related to neuroblastoma diagnosis and treatment, and face a complex, evolving landscape of treatment options and guidelines.

Treatment Goals and Management Plan

Caregiver education should be ongoing throughout the treatment journey and should include information about risks and benefits of treatment options, impact on quality of life, acute side effects, and late effects/long-term risks.*

Caregivers should be given adequate information on all potential approaches to treatment, with an unrushed opportunity for discussion and questions, to allow for their personal preferences, family goals, and treatment goals to be considered.*

*These statements were identified as the "most important" within their respective domains, as determined by a vote of the steering committee member.

Mean
Consensus
Score
4.3
Families should be made aware that different institutions have different approaches to the treatment and follow-up of neuroblastoma, across the disease course.
4.3
Caregivers should be made aware of clinical trial availability regardless of location, as well as the criteria for joining, practical considerations associated with travel, and the possible risks and benefits of research participation.
4.4
Caregivers should be given adequate information on all potential approaches to treatment, with an unrushed opportunity for discussion and questions, to allow for their personal preferences, family goals, and treatment goals to be considered.
4.7
Caregivers should feel empowered to ask questions of their healthcare team, pursue a second opinion, seek an alternative yet practical location for treatment, or request support or other resources.
4.8
Whenever possible, treatment planning and/or administration should be done by a multidisciplinary pediatric oncology team with expertise in neuroblastoma.
4.9
Caregiver education should be ongoing throughout the treatment journey and should include information about risks and benefits of treatment options, impact on quality of life, acute side effects, and late effects/long-term risks.

Ethics

When presenting and discussing care options, no assumptions should be made about family treatment preferences, willingness to travel, ability to pay, ability to comprehend information, or willingness to participate in research.*

Neuroblastoma healthcare providers have an obligation to patients to stay up to date on best practices, national guidelines, and clinical trials and to supply comprehensive information to families about available treatments. *

*These statements were identified as the "most important" within their respective domains, as determined by a vote of the steering committee member.

Mean
Consensus
Score
4.1
Ethical guidance should be established to ensure treatment centers present all treatment options and their potential benefits and limitations, in the context of a patient’s diagnosis and health status.
4.6
When presenting and discussing care options, no assumptions should be made about family treatment preferences, willingness to travel, ability to pay, ability to comprehend information, or willingness to participate in research.
4.8
Neuroblastoma healthcare providers have an obligation to patients to stay up to date on best practices, national guidelines, and clinical trials and to supply comprehensive information to families about available treatments.
4.8
Caregivers should be informed of their right to stop treatment or research participation, or request an alternative care option, and feel empowered to do so without fear of negative repercussions in their relationship with the care team.
4.6
Collaborative discussion between caregivers and healthcare providers can help determine the level of involvement the child or adolescent should have in care decision-making, and how health information should be conveyed to the child.
4.9
Healthcare teams should routinely evaluate their treatment practices and offerings to determine what adjustments should be made to reflect best practices and the evolving standards of care.

Health Equity

Caregivers should be aware that there may be socioeconomic, financial, institutional and/or geopolitical factors that can impact their child's care, and they should receive support in navigating these issues.*

Neuroblastoma clinical trials should include patients that represent the diversity of the patient population, and results from trials should be interpreted with care when research participants do not reflect the characteristics of a patient in the clinic.*

*These statements were identified as the "most important" within their respective domains, as determined by a vote of the steering committee member.

Mean
Consensus
Score
4.2
Neuroblastoma clinical trials should include patients that represent the diversity of the patient population, and results from trials should be interpreted with care when research participants do not reflect the characteristics of a patient in the clinic.
4.4
There is a prevalent need to better serve marginalized communities and families to reduce disparities in neuroblastoma care and outcomes.
4.7
Caregivers should be aware that there may be socioeconomic, financial, institutional and/or geopolitical factors that can impact their child’s care, and they should receive support in navigating these issues.

Patient/Provider Communication

Patients and/or families should be educated and empowered to ask questions and healthcare providers should guide them appropriately.*

Caregivers should have the opportunity to discuss treatment decisions and expectations and should be informed about options the treatment center can and cannot offer throughout the treatment journey.*

*These statements were identified as the "most important" within their respective domains, as determined by a vote of the steering committee member.

Mean
Consensus
Score
4.2
Patients and/or families should be educated and empowered to ask questions and healthcare providers should guide them appropriately.
4.1
Caregivers should be assessed for the extent of shared-decision making they desire, with reassessment of their desired involvement throughout the treatment journey.
4.6
Caregivers should have the opportunity to discuss treatment decisions and expectations and should be informed about options the treatment center can and cannot offer throughout the treatment journey.
4.6
Healthcare teams should assess and accommodate the family’s preferred learning and communication methods, to support them at their desired level of understanding and participation in informed shared decision-making.
4.7
Healthcare teams should offer information on or referrals to appropriate caregiver/family support resources

Reference: 1. Qiu B, Matthay KK. Nat Rev Clin Oncol. 2022;19(8):515-33.

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