Traditionally considered a disease of adults, type 2 diabetes mellitus (T2DM) is increasingly diagnosed in children and adolescents in parallel to rising obesity rates. Currently, only insulin and metformin are approved as pharmacotherapy for T2DM in patients less than 18 years of age. It is well known that adolescent patients often have difficulty adhering to medication regimens, and may have trouble with the lifestyle interventions required to effectively treat T2DM. The consequences of early-onset diabetes and lack of adequate glycemia control have been demonstrated;i.e. nephropathy, cardiovascular disease, and ophthalmopathy may all occur early in these young patients. It has been shown that improved personalized support may be beneficial to patients with other chronic illnesses such as type 1 diabetes and in patients with HIV infection. The use of lay volunteers (without prior professional health care training) has been documented in several studies conducted in the USA as well as in the developing world, and has been systematically reviewed. In addition, British studies have suggested that using non-professional volunteers to support parents can be an effective strategy for improving the uptake of immunization in children younger than 2 years. A Seattle group has previously shown in a randomized controlled trial that enhanced tracking and outreach increased the proportion of persons with elevated blood pressure detected during community measurement who followed up with medical care. We have found no studies to date documenting the use of lay volunteers in the treatment of adolescent T2DM in the US or abroad. Thus, we would like to test whether a low-cost, simple, personal intervention (the buddy study, in which an individual volunteer partner is assigned to each participant) can be effective in improving hemoglobin A1c (HbA1c) levels, adherence to treatment regimens, and quality of life in adolescents with T2DM. Relevance of Existing Guidelines for Care The American Diabetes Association (ADA) recommends initial therapy of T2DM in youth with diet, exercise and metformin if patients present asymptomatically, and with insulin if they present with ketoacidosis. If monotherapy fails, then combination therapy should be initiated with metformin plus a sulfonylurea or insulin. However, other insulin secretagogues such as meglitinide, insulin sensitizers (e.g. thiazolidinediones), and glucosidase inhibitors (acarbose) are acceptable alternatives. Insulin therapy may be initiated at any point, or as a final step after combination therapy fails. It is important, however, to note that all drugs with the exception of metformin and insulin are being used off-label in pediatric patients. In this study, since only patients with HbA1c >7% will be included, treatment for all subjects will have been initiated or will be initiated with diet and metformin. If glycemia goals are not achieved on metformin, other medications will be added based on existing clinical guidelines for adults and children. Relevance of Study Results to Care Demonstration of a change in HbA1c levels, and secondarily, in weight, overall diabetes care (i.e., number of glucose monitor checks, medication compliance, and adherence to clinic visit schedule), and psychological well-being in this pilot study would be an important development in the approach to T2DM therapy in this often difficult-to-treat adolescent cohort. Given the limited number of FDA-approved therapies in the pediatric population, and the nature of T2DM as disease requiring significant lifestyle modifications, an approach using personalized follow-up from a lay volunteer would be particularly applicable in resource-poor and underserved settings. Study Objectives Primary outcome: To evaluate the effect of a minimally invasive intervention (the buddy system) on hemoglobin A1c levels in adolescents with T2DM. Secondary outcomes: To assess changes between groups in body weight, number of home glucose monitor checks, compliance with medications, adherence to visit schedule, and psychological well-being. Inclusion Criteria Any patient with type 2 diabetes mellitus as documented by his or her primary physician 1. Age 12-20 years at enrollment 2. Most recent HbA1c equal or greater than 7% Exclusion Criteria 1. Significant comorbidity that, in the opinion of the investigators, may limit adequate study participation 2. Significant psychiatric or cognitive disorder, or communication difficulty that will, in the opinion of the investigators, limit the subject's ability to interact with the patient assistant or may present a danger to the patient assistant;patients with current suicidal or homicidal ideation, history of arrest for violent behavior, or history of school suspension/expulsion for violent behavior will be excluded. 3. Pregnancy or intention to become pregnant within 6 months Study Design and Statistical Analysis Patients will be randomized to conventional treatment versus interventional group (buddy study). Patients will be followed for 6 months. During this period, all patients should undergo two follow-up visits including physical examination, detailed medical history, and laboratory testing (hemoglobin A1c) approximately 3 months apart. All patients will complete an initial screening questionnaire and quality of life questionnaires after the initial and 6 month clinic visits. Patients assigned to the interventional (buddy) group will also have a home visit conducted by the buddy once a month. This visit will last approximately 30 minutes to 1 hour. Its purpose is to assist with bonding of the buddy and the patient in his or her familiar environment and to further the understanding of the patients psychosocial situation. The home environment will not be systematically assessed but any observations about unsafe conditions will be disclosed by the buddy to the investigator(s). However, should a home visit be declined by the patient or his/her family, acceptable alternative buddy-patient meeting places include schools, cafes, or libraries chosen by both parties at a mutually acceptable time. If necessary, these additional face-to-face encounters may also take place at NIH or at CNMC if this is most convenient for the study participants. Current recruitment status: 10 adolescents with type 2 diabetes have been enrolled and randomized (5 to the conventional arm and 5 to the 'Buddy'arm. Due to the slow recruitment, the study is now inactive and the protocol remains open for analysis only.
|Nguyen, Tammy T; Jayadeva, Vikas; Cizza, Giovanni et al. (2014) Challenging recruitment of youth with type 2 diabetes into clinical trials. J Adolesc Health 54:247-54|
|Gemmill, Julie Anne L; Brown, Rebecca J; Nandagopal, Radha et al. (2011) Clinical trials in youth with type 2 diabetes. Pediatr Diabetes 12:50-7|