Apriori feasibility testing of randomized clinical trial design in patients with cleft deformities and Class III malocclusion

Abstract

Objectives: To assess the feasibility of randomizing treatment (surgical vs. non-surgical) for correction of a Class III malocclusion (underbite) resulting from an earlier repair of cleft lip and palate.

Materials and methods: Surveys about willingness to accept randomized treatment during adolescence were mailed to the parents of cleft lip and palate patients under the care of Children's Hospital Los Angeles between 2005 and 2010. The inclusion criteria were patients with cleft lip and palate, Class III malocclusion due to maxillary deficiency, and absence of medical and cognitive contraindications to treatment.

Results: Out of 287 surveys, 82 (28%) were completed and returned; 47% of the subjects held a strong treatment preference (95% CI, 35-58%), while 30% were willing to accept randomization (95% CI, 20-41%). Seventy-eight percent would drop out of a randomized trial if dissatisfied with the assigned treatment (95% CI, 67-86%). The three most commonly cited reasons for being unwilling to accept random treatment assignment were 1) the desire for doctors to choose the best treatment, 2) the desire for parents to have input on treatment, and 3) the desire to correct the underbite as early as possible.

Conclusion: Based on this study, parents and patients would be unwilling to accept a randomly assigned treatment and would not remain in an assigned group if treatment did not meet expectations. This highlight the limitations associated with randomization trials involving surgical modalities and provide justification for other research models (e.g., cohort studies) to compare two treatment options when randomization is not feasible.

Keywords: Feasibility; Observational cohort study; Randomized trial.

Copyright © 2014 Elsevier Ireland Ltd. All rights reserved.

Figures

Fig. 1

(a) Treatment preference and number of patients in each group (n = 82). (b) Distribution of treatment preferences according to age group (n = 82): Group 1, age <13 years pre-treatment; Group 2, age >13 years, but <18 years mid-treatment; Group 3, age >18 years after treatment.

Fig. 2

(a) Acceptance of randomization and number of patients in each group. (b) Distribution according to age group: Group 1, age 13 years, but 18 years after treatment.

Fig. 3

(a) Distribution of willingness to stay with the original treatment group for the duration of the trial. (b) Distribution of willingness to stay with the original treatment group for the duration of the trial according to age group: Group 1, age 13 years, but 18 years after treatment.