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

294 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine which of two educational letter approaches improves health screenings for individuals with Neurofibromatosis 1 (NF1). Participants will receive letters about NF1 care and management during regular doctor visits. The trial targets adults with NF1 and parents or guardians of children with NF1 who visit a regular doctor for annual checkups but do not attend a specialized NF clinic. Those who fit this description are encouraged to enroll to help enhance NF1 care practices. As an unphased trial, this study provides a unique opportunity to contribute to the development of better NF1 care practices.

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 prior data suggests that these educational interventions are safe for individuals with Neurofibromatosis 1?

Research has shown that sending educational letters about Neurofibromatosis 1 (NF1) care is a safe method. These letters inform patients and their doctors about recommended NF1 care. The trial emphasizes education rather than testing a new drug or treatment, eliminating the risk of physical side effects. This makes the letters easy for participants to manage.

In other NF1 research, studies often aim to better understand the condition or improve symptom management. In this trial, the letters serve as a tool to promote better healthcare practices, minimizing safety concerns. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this trial because it explores a unique approach to managing neurofibromatosis type 1 (NF1) through educational letters. Unlike standard medical treatments that focus on directly addressing the physical symptoms, this trial investigates whether providing targeted information about NF1 care can empower patients and caregivers. The idea is that by enhancing understanding and awareness, patients can make better-informed decisions about their care, potentially improving quality of life. This method is distinctive because it emphasizes education and self-management as a complementary strategy to traditional medical interventions.

What evidence suggests that this trial's educational interventions could be effective for NF1?

Research has shown that educational programs can help people with Neurofibromatosis 1 (NF1) manage their health more effectively. In this trial, participants in the Intervention Arm will receive letters about NF1 care, designed to inform both patients and their primary care doctors about the importance of regular check-ups and screenings. Studies have found that clear information about NF1 care can enhance patients' understanding and motivate them to pursue recommended health check-ups. By fostering better communication and awareness, these letters could lead to more active health management and potentially better health outcomes for those with NF1. ^{1 6 7 8 9}

Who Is on the Research Team?

Scott Plotkin, MD

Principal Investigator

Massachusetts General Hospital

Vanessa Merker, PhD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

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

Adult: Does not attend an NF clinic within the Children's Tumor Foundation NF Clinic Network

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

Exclusion Criteria

Only one person per household may participate in the study

Unwilling or unable to give informed consent

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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 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 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]

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 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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06262113

Study Details | NCT06262113 | A Decentralized ... Both groups will be given letters, one for themselves and one for their clinician, that describe NF1 care recommendations. After attending their annual wellness ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9925753/

Neurofibromatosis Type 1 (NF1): Addressing the Transition ... This review highlights the transition needs of NF1 patients and provides resources for both clinicians and families to facilitate HCT in this population.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT01626846?term=NCT01626846&viewType=Table&rank=1

How Neurofibromatosis Type 1 (NF1) Affects Schoolwork ... The study will seek to determine whether there is a corresponding deficit in school performance for teenagers with NF1. Secondly, the study seeks to explore the ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8247857/

Neurofibromatosis type 1 decreases educational attainment NF1 is associated with reduced educational attainment and tendency for affected individuals to obtain vocational instead of academic education.

5. nfnetwork.org nfnetwork.org/data/uploads/nf1-educational-materials/adressing_academic_concerns_nf1.pdf

ADDRESSING ACADEMIC CONCERNS: A GUIDE FOR ... There are two types of NF: neurofibromatosis type 1 (NF1) and neurofibromatosis type 2 (NF2). NF1 is ten times more common than NF2. People with NF1 do not ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT00352599

NCT00352599 | Trial to Evaluate the Safety of Lovastatin in ... Neurofibromatosis type I (NF1) is a genetic disorder that affects approximately 1 in 3500 individuals. Half of people with NF1 inherit the condition from a ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10964784/

Epidemiology and Outcomes of Neurofibromatosis Type 1 (NF ... The aim of this manuscript was to assess the epidemiology and clinical features of Neurofibromatosis type 1 (NF-1) based on the newly published ...

8. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2452014420301114

Physicians' adherence to published guidelines regarding ... The aim of this observational study was the adherence of clinical outpatient care to published NF1 guidelines including genetic testing. Patients and outpatient ...

9. ctf.org ctf.org/wp-content/uploads/2023/11/NF1-Learning-brochure.pdf

Learning with NF1 LEARNING with NF1. About half of those diagnosed with neurofibromatosis type 1 (NF1) experience some form of learning disability. Imagine how confusing.