Educational Letters for Neurofibromatosis

Overseen By Evan Koch, BA

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Massachusetts General Hospital

Disqualifiers: Multiple household members, Consent issues

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

Compensation: Varies

Number of Visits: Unknown

360 Participants Needed

Trial Summary

What is the purpose of this trial?

The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1). The primary objective of the study is to determine which intervention leads to higher rates of evidenced-based health screenings for NF1 patients in primary care settings. Adults with NF1 and parents/guardians of children with NF1 from across the U.S. who do not go to a specialized NF clinic and who have an upcoming annual wellness visits (e.g. an annual physical, a well-child visit, etc.) scheduled with a primary care provider (PCP) are eligible to enroll in the study. To see if you might be eligible, fill out a prescreening survey here: https://redcap.link/mynfguide

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It seems focused on educational interventions, so it's unlikely that medication changes are required.

What data supports the effectiveness of the treatment Letters about NF1 Care for Neurofibromatosis?

The research highlights a significant quality-of-life burden for patients with Neurofibromatosis type 1, indicating a high unmet need for effective treatments. While the specific effectiveness of educational letters is not directly addressed, the need for improved care and management strategies is evident, suggesting potential benefits from enhanced patient education. ^{1 2 3 4 5}

How does the treatment 'Educational Letters for Neurofibromatosis' differ from other treatments for neurofibromatosis type 1?

The 'Educational Letters for Neurofibromatosis' treatment is unique because it focuses on providing psychoeducation and advocacy training to help families and schools support children with neurofibromatosis type 1 in their educational journey, rather than directly treating the medical symptoms of the condition. ^{6 7 8 9 10}

Research Team

Scott Plotkin, MD

Principal Investigator

Massachusetts General Hospital

Vanessa Merker, PhD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

This trial is for adults with Neurofibromatosis 1 (NF1) and parents or guardians of children with NF1 in the U.S. who don't attend a specialized NF clinic but have an upcoming annual check-up with their primary care provider.

Inclusion Criteria

Adult: Has an in-person, well-person visit with a primary care provider scheduled within 3 months of their consent

Parent/Guardian of a Child with NF1: Does not attend an NF clinic within the Children's Tumor Foundation NF Clinic Network

Adult: Currently lives in the United States (including Puerto Rico and other United States territories)

Exclusion Criteria

Only one person per household may participate in the study

Unwilling or unable to give informed consent

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Online prescreening survey

Baseline Assessment

Participants complete baseline survey assessments online before their PCP visit

1 week

Online survey

Intervention

Participants are randomized to one of two groups and receive letters describing NF1 care recommendations

1 week

Annual wellness visit with PCP

Follow-up

Participants complete an online follow-up survey and a subsample participates in a virtual qualitative interview

2 weeks

Online survey and virtual interview

Treatment Details

Interventions

• Letters about NF1 Care

Trial Overview The study aims to see which of two educational letters about NF1 care leads to more evidence-based health screenings at primary care visits. It's a randomized controlled trial, meaning people are put into groups by chance.

Participant Groups

Group I: Intervention Arm Experimental Treatment 1 Intervention

Group II: Enhanced Usual Care Arm Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Patient-Centered Outcomes Research Institute

Collaborator

Trials

592

Recruited

27,110,000+

Findings from Research

Neurofibromatosis type 1 (NF1) significantly impacts the quality of life for patients due to complications from plexiform neurofibromas (PNs), leading to pain, social and physical function issues, and emotional distress.

There is a critical need for effective treatments to reduce tumor burden and improve quality of life for NF1 patients, as current research highlights gaps in understanding the health economic burden and the measurement of quality of life in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Natural History and Disease Burden of Neurofibromatosis Type 1 with Plexiform Neurofibromas: A Systematic Literature Review. Copley-Merriman, C., Yang, X., Juniper, M., et al.[2021]

The guideline developed for Neurofibromatosis type 1 (NF1) emphasizes the importance of personalized and targeted tumor management to ensure appropriate care while minimizing unnecessary interventions, based on a comprehensive literature review and expert consensus.

This guideline also addresses the psychosocial and quality of life impacts of NF1, reflecting current care practices in Europe and allowing for adjustments based on local resources.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

ERN GENTURIS tumour surveillance guidelines for individuals with neurofibromatosis type 1. Carton, C., Evans, DG., Blanco, I., et al.[2023]

In a study of 3,530 patients with neurofibromatosis 1 in Japan, 82% required medical intervention for major complications, highlighting the severity of this condition.

The most common complications included dermatological issues (71.8%), neurological problems (38.1%), and bone complications (33.3%), with specific treatments needed for conditions like brain tumors and scoliosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidemiological Analysis of Major Complications Requiring Medical Intervention in Patients with Neurofibromatosis 1. Yoshida, Y., Ehara, Y., Koga, M., et al.[2018]

A survey of 170 patients with cutaneous neurofibromas (cNF) revealed that the visibility of these growths significantly impacts their quality of life, affecting mood and social interactions more than the number of lesions.

Patients expressed dissatisfaction with current treatments (surgery and laser), particularly due to high rates of regrowth, and showed a strong interest in new therapeutic options, preferring well-tolerated topical or oral medications that could effectively reduce the number and growth of cNF.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cutaneous neurofibromas: patients' medical burden, current management and therapeutic expectations: results from an online European patient community survey. Guiraud, M., Bouroubi, A., Beauchamp, R., et al.[2020]

In a study of 162 adults with Neurofibromatosis type 1, women reported significantly worse quality of life related to the disease compared to men, particularly in areas like perceived physical appearance, anxiety, and emotional health.

The study found that while women’s perceived physical appearance correlated with disease visibility, this was not the case for men, highlighting the need for healthcare providers to address body image and mental health concerns, especially in women.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A cross-sectional study of gender differences in quality of life domains in patients with neurofibromatosis type 1. Hamoy-Jimenez, G., Elahmar, HA., Mendoza, M., et al.[2022]

The School Liaison Program (SLP) significantly improved parents' perceptions of their child's academic potential and satisfaction with their educational progress compared to a control group of parents of children with neurofibromatosis type 1 who did not receive these services.

Parents who participated in the SLP reported a better understanding of their child's learning needs and increased ability to access school supports, indicating that this model of psychoeducation and advocacy could benefit other children with chronic medical conditions affecting neurocognitive functioning.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Effectiveness of a Hospital-Based School Liaison Program: A Comparative Study of Parental Perception of School Supports for Children With Pediatric Cancer and Neurofibromatosis Type 1. Northman, L., Morris, M., Loucas, C., et al.[2019]

Therapeutic education is a comprehensive approach that empowers cancer patients and their caregivers to manage daily life skills, but its implementation in cancer surgery is limited due to strict administrative regulations.

The article outlines the principles of therapeutic education as defined by the French Haute Autorité de Santé and presents a specific program for patients with tracheostomy and/or gastrostomy, aiming to facilitate the authorization process for such educational initiatives.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[How to reconcile therapeutic patient education and care pathology in oncology: Application in head and neck neoplams]. Buiret, G., Chidiac, F., Pujo, K., et al.[2019]

Patients who underwent lung transplants for cystic fibrosis reported significant improvements in health and quality of life, indicating the efficacy of the transplant procedure.

There is a clear need for ongoing education regarding transplant-related complications and immunosuppressive drug management, as well as support for psychological adjustment and information about new social rights post-transplant.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Identifying the educational needs of lung transplant recipients with cystic fibrosis. David, V., Feldman, D., Danner-Boucher, I., et al.[2015]

Therapeutic education in cancer aims to actively involve patients in their treatment decisions and disease management, which can enhance their adjustment to living with cancer as a chronic disease.

Pilot studies suggest that educational programs can improve the quality of life for cancer patients and reduce treatment side effects, highlighting the need for further research to develop and evaluate cancer-specific education programs in France.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Therapeutic education in oncology: involving patient in the management of cancer]. Pérol, D., Toutenu, P., Lefranc, A., et al.[2011]

Therapeutic education significantly improves patients' quality of life and treatment compliance, leading to remarkable outcomes such as an 80% reduction in amputations over 10 years for diabetic patients.

Motivational interviewing techniques are crucial in helping patients change their behaviors gradually, making them active partners in their treatment and enhancing their ability to manage their health effectively.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Therapeutic education of the diabetic patient]. Golay, A., Lagger, G., Chambouleyron, M., et al.[2015]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Natural History and Disease Burden of Neurofibromatosis Type 1 with Plexiform Neurofibromas: A Systematic Literature Review. [2021]

2. England pubmed.ncbi.nlm.nih.gov

ERN GENTURIS tumour surveillance guidelines for individuals with neurofibromatosis type 1. [2023]

3. Sweden pubmed.ncbi.nlm.nih.gov

Epidemiological Analysis of Major Complications Requiring Medical Intervention in Patients with Neurofibromatosis 1. [2018]

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

Cutaneous neurofibromas: patients' medical burden, current management and therapeutic expectations: results from an online European patient community survey. [2020]

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

A cross-sectional study of gender differences in quality of life domains in patients with neurofibromatosis type 1. [2022]

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

The Effectiveness of a Hospital-Based School Liaison Program: A Comparative Study of Parental Perception of School Supports for Children With Pediatric Cancer and Neurofibromatosis Type 1. [2019]

7. France pubmed.ncbi.nlm.nih.gov

[How to reconcile therapeutic patient education and care pathology in oncology: Application in head and neck neoplams]. [2019]

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

Identifying the educational needs of lung transplant recipients with cystic fibrosis. [2015]

9. France pubmed.ncbi.nlm.nih.gov

[Therapeutic education in oncology: involving patient in the management of cancer]. [2011]

10. Belgium pubmed.ncbi.nlm.nih.gov

[Therapeutic education of the diabetic patient]. [2015]