Rationale and Objective: Patients with neurofibromatosis (NF) as a group report difficulties coping with NF symptoms, high levels of stress (stemming primarily from the uncertainty of disease progression and NF symptoms), depression, anxiety, and low quality of life (QoL). The psychosocial profile of patients with NF is similar to or worse than that of patients with other chronic illnesses and cancers, who face similar stressors including much uncertainty about progression of the disease. The care of most chronic illnesses is comprehensive and addresses both the medical and psychosocial manifestations of the disease. However, the care for patients with NF has been historically biomedical, focused only on the medical manifestations of the disease. By ignoring psychosocial factors, patients with NF are deprived from learning coping skills to manage this condition, which could greatly improve their QoL as well as the success of medical treatment, while waiting for a medical cure.
The objective of this study is to test within a scientifically sound, randomized controlled trial (RCT) whether a mind-body intervention, The Relaxation Response Resiliency Program for adult patients with NF (3RP-NF) improves four aspects of QoL (physical health, psychological, social relationships, and environment), pain interference, pain intensity, and other psychosocial factors (e.g., depression, anxiety), much more than a control intervention, Health Enhancement Program for patients with NF (HEP-NF), that provides only educational information without teaching coping skills. Both interventions will be delivered via videoconferencing with Vidyo, in order to reduce burden of traveling to clinic for weekly meetings and to ensure that the intervention is accessible to patients from across the United States including those in remote areas. Vidyo is a HIPAA-approved, secure video web platform that is currently used to deliver clinical care at Massachusetts General Hospital (MGH). Vidyo is easy to use -- patients receive a link via email and click on it to download it on their computer, tablet, or phone. For each session, patients simply click on the Vidyo icon to join the group meeting. All participants undergo a test call prior to the start of the groups, and support for any unlikely technical issues is available. Clinical visits through Vidyo are now reimbursable by major insurance companies. Participants will not know which is the intervention and which is the control. We have already preliminarily tested the intervention in a subset of patients with NF1, NF2, and schwannomatosis (N=63) and found that patients are very interested in participating in the 3RP-NF through live video. Patients in both groups improved, but those in the intervention improved significantly more than those in the control on all QoL measures, as well as anxiety and pain intensity. Improvements were clinically meaningful and durable. This excellent preliminary data provides a strong rationale for the proposed fully powered RCT.
Applicability and Impact of Research: Because this research addresses coping with NF and not underlining disease mechanisms, it can be helpful to all patients with NF, regardless of NF type. In fact, research has shown that patients with NF1, NF2, and schwannomatosis report similar levels of emotional distress. Because the intervention requires only a 6th grade reading level, the intervention groups are small, and skills are learned and practiced within each group session, patients with learning and cognitive disabilities also benefit from it. The Vidyo web platform is user friendly and has received high patient satisfaction ratings at MGH for the past several years from patients with various mental and cognitive concerns. The intervention has the potential to change the manner in which care for NF patients is delivered by addressing the person (e.g., coping, QoL) and not only the body (e.g., tumor). The 3RP-NF is economical (only eight sessions), generalizable, risk free (e.g., no side effects), accepted, usable, and appreciated by patients with NF and has been found to improve QoL and other psychosocial variables significantly more than the control condition.
In order to be sure that the 3RP-NF intervention works better than the HEP-NF control and that its effects are durable, we need to test it in a larger RCT with enough patients. If found efficacious within a large RCT, the 3RP-NF intervention can completely change the model of care for patients with NF. Once efficacy is established, we aim to disseminate the intervention to other NF-specific centers with help from the Children's Tumor Foundation (CTF) and to train providers in the delivery of the intervention. We are also currently conducting preliminary RCTs of an adapted 3RP-NF for adolescents with NF via live video and for patients with NF2 who are deaf, using live video and CART. We are also hoping to incorporate the 3RP within clinical drug trials to buttress adherence and efficacy. This will surely advance not only the QoL of patients with NF, but also patients' satisfaction with care, and medical utilization and cost.