Sixty-four (85%) of the 75 families returned the completed survey (n = 54 by mail and n = 10 online). Of those respondents, 86% were mothers and 14% were fathers. Slightly more than half of the children with NF1 were male (59%). At study enrollment, 25% of patients were less than 6 years of age, 33% were 6 to 10 years, and 42% were 11 to 24 years of age. Eighty-nine percent (n = 57) of children had entered a study at the NCI, while 11% had been evaluated for potential enrollment on a study but did not enroll (see ).
Enrollment status of patients whose families responded to the survey (N = 64).
Most families had learned about NF1 studies being conducted at the NIH through their local doctor (59%) or online (23%). More than half (58%) of the families had been involved with research at the NCI for more than two years. For 75% of respondents, no other family members had been diagnosed with NF1 other than the child evaluated.
Understanding of Study
Parents whose child entered a treatment study for his or her PNs (n = 51) were asked how well they understood various factors prior to enrollment. The majority reported that they thoroughly understood the main purpose of the study (86%, 95% Confidence Interval [CI] 77–95%), what medical procedures were involved (92%, 95% CI 84–99%), possible side effects (86%, 95% CI 77–95%), the time required for participation (86%, 95% CI 77–95%), and the right to stop the study at any time (96%, 95% CI 90–100%). Thirty-three respondents whose child had participated in a PCT were asked how well they understood the reason for the placebo, and 100% said they understood the reason “somewhat” or “a lot.” Moreover, 52% (95% CI 35–69%) of these parents said they “had no problem with [the experience of participating in a PCT]”, while 48% (95% CI 31–65%) reported finding it hard not knowing if their child was taking the study drug or the placebo.
Reasons for Participation
The most common reasons for initially enrolling on the study included to shrink the child’s tumors (77%, 95% CI 67–87%), to access medical care that otherwise would not have been available (72%, 95% CI 62–82%), and to help others (72%, 95% CI 62–82%). Less common reasons for enrollment included increasing one’s knowledge about NF1 (56%, 95% CI 44–68%) and meeting other families living with NF1 (28%, 95% CI 17–39%). In addition to the multiple-choice options on this item, parents were given the opportunity to write in other responses. One parent stated that a reason for their participation was “to make sure I was doing everything I could possibly do for my child.” Another parent commented that she had felt hopeless regarding her daughter’s condition, and they participated because “[the study] gave me hope.”
Experiences of Participation
Almost half (47%, 95% CI 35–59%) of respondents felt that study participation interfered with their family’s daily routine “some” or “a lot.” A few (6%, 95% CI 0 – 12%) reported that it interfered with their family relationships, although 75% (95% CI 65–85%) stated that participation impacted their family relationships in a positive way “some” or “a lot”. Most respondents reported that they did not have significant financial difficulties related to coming to the NCI (78%, 95% CI 68–88%), and that their child did not have notable difficulties from missing school due to coming to the NCI (77%, 95% CI 67–87). However, almost half (47%, 95% CI 35–59%) felt that their co-workers or supervisors were not always understanding about the time they had to take off from work.
With respect to interactions with research staff, 92% (95% CI 85–99%) of respondents agreed that the amount of contact was appropriate. Eighty-four percent (95% CI 75–93%) reported feeling “very comfortable” calling the research staff with questions, and 80% (95% CI 70–90%) reported that the staff members were “very easy” to reach.
When asked about the best parts of study participation, the most frequently endorsed options included the opportunity to help others (72%, 95% CI 62–82%), having access to medical care that would have been expensive outside the NCI (69%, 95% CI 58–80%), and the fact that they learned more about NF1 (58%, 95% CI 46–70%). Parents also cited the benefits of meeting other families living with NF1 (33%, 95% CI 22–44%), and their child’s tumors becoming more stable (25%, 95% CI 14–36%). Seventy-two percent (95% CI 62–82%) of respondents felt that study participation helped their child “some” or “a lot”. Several respondents provided additional comments about the best parts of participation in an open-ended section of this question. These comments referenced the fact that specific conditions (e.g., malignant tumors, other NF1-related complications) were identified that had not been found previously, and the availability and “caring” nature of the staff.
Worst parts of participation included having to take time off work (28%, 95% CI 18–38%), traveling to the NIH (25%, 95% CI 15–35%), the child having to miss school (25%, 95% CI 15–35%), and seeing no benefits to the child’s health (22%, 95% CI 12–32%). Factors endorsed less frequently were the child having to go through painful or uncomfortable medical procedures (14%, 95% CI 6–22%), the child having to take medicine (11%, 95% CI 3–18%), the child having to go through too many evaluations or procedures (9%, 95% CI 2–16%), and the child experiencing side effects from the medicine (6%, 95% CI 0–12%). In an open-ended part of this question, respondents were able to name other factors they considered to be the worst parts of participation. One parent noted that it was difficult to leave her other children while visiting the NIH. Additional factors specified by other parents included worrying that the child could be found ineligible to continue on study, the stress of waiting for medical test results, and having difficulty getting the child to take the study drug due to its unappealing taste.
Most respondents said they would “definitely” (84%, 95% CI 75–93%) or “probably” (9%, 95% CI 2–16%) participate in a future study at the NIH. A smaller but still substantial proportion of respondents said they would “definitely” (40%, 95% CI 28–52%) or “probably” (26%, 95% CI 14–36%) be willing to participate in a study with a placebo control group. Among respondents whose child had previously participated in a PCT, 84% (95% CI 72–96%) said they would “definitely” or “probably” participate in a PCT in the future. By contrast, among those whose child had participated in a study not involving a placebo group, only 54% (95% CI 31–77%) said they would “definitely” or “probably” participate in a PCT.
From a list of seven choices, respondents were asked to indicate areas they would like assessed in future studies. The majority expressed interest in studies assessing cognitive functioning (69%, 95% CI 58–80%), pain (63%, 95% CI 51–75%), scoliosis and other bone changes (61%, 95% CI 49–73%), and emotional/behavioral functioning (55%, 95% CI 43–67%). Respondents also expressed interest in studies assessing social skills (45%, 95% CI 33–57%), motor skills (44%, 95% CI 32–56%), and career/vocational skills (41%, 95% CI 29–53%). Among four future treatment studies, the most interest was expressed in a study targeting learning problems (55%, 95% CI 43–67%), followed by studies involving treatment for skin tumors (48%, 95% CI 35–59), NF1-related cancer tumors (41%, 95% CI 29–53%), and brain tumors (30%, 95% CI 19–41%).
Overall Satisfaction with Participation
On a Likert scale question assessing overall satisfaction with study participation (0 = very negative experience, 10 = very positive experience), the mean rating was 9.5 (SD = .78; 95% CI 8.0–10.0), and ratings ranged from 7 to 10. As shown in , higher overall satisfaction was significantly correlated with fewer transportation problems (e.g., airport shuttles), fewer financial difficulties related to study participation, and fewer school problems for the child resulting from study participation (ps < .05). Respondents’ overall satisfaction was not related to the impact of study participation on family relationships, having long wait times before or between study appointments, whether the respondents’ co-workers or supervisors were understanding about the time they took off work to come to the NIH, perceptions of whether study participation helped their child, disruptions to the family’s routine, or the age of the child during study participation (ps > .05).
Correlations between satisfaction ratings and other variables.
Respondents were divided into two groups based on whether their child had participated in the PCT or in a treatment study without a placebo control group. The mean satisfaction ratings were very similar across these groups (9.48 ± .80, 95% CI 7.91–10, and 9.69 ± .63, 95% CI 8.45–10, respectively) and not significantly different from each other (F = 0.71, p > .05).
Suggestions for Improvements
In an optional open-ended question, respondents were asked what things could be improved about participating in a study at the NCI. Forty-one percent (n = 26) of parents provided comments. Suggestions included improvements in transportation (n = 3), increased communication between the NCI medical team and home physicians (n = 2), more contact from staff regarding scheduling upcoming appointments (n = 2), providing accommodations and/or covering travel costs for additional family members (a second parent or patient’s siblings; n = 2), providing more information on new or upcoming studies (n = 2), providing more information about what to expect during the visits (n = 1), and decreasing long wait times for one particular clinic (n = 1). In addition, one parent commented that study visits were difficult because of the location of the NIH (being far from their home), and two parents whose children had participated in a placebo-controlled study expressed a desire to know whether their child was on placebo or the drug. (This was communicated to all parents when the study was completed and unblinded).
A number of parents offered positive comments about their experience when responding to this open-ended question. Specific factors cited included the “clinical experience” of the child, the lodging, the helpfulness of the information received throughout the study, and the friendliness and supportiveness of the staff.