Key messages . |
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1. Differences between childhood- and adult-onset type 1 diabetes should be part of the design of studies of disease-modifying therapies. |
2. Studying disease-modifying agents in children should not require efficacy data from adults. |
3. Children may benefit more from disease-modifying therapies due to the more rapid loss of insulin secretion before and after their diagnosis, the unique burdens on them and their families, and the greater vulnerability of young children’s neurocognitive development. |
4. Investigators should work with regulatory agencies early in the study design process and leverage the pediatric expertise of the agencies. |
5. A practical approach to assessing risk-to-benefit ratio is to consider the following: Given what is known about the intervention and the limited alternatives, would an independent and expert clinician regard enrolling children with type 1 diabetes as promoting the children’s clinical interests? |
Open research questions |
1. What is the long-term effect of age at onset and early glycemic control on complication risk both within childhood and adolescence (i.e., pre- vs. peri- or postpuberty) and in comparison with adult-onset type 1 diabetes? |
2. What are the biological mechanisms underlying the varying pathways to type 1 diabetes? Which mechanisms are seen in all individuals and which are age dependent? |
3. Given the high frequency of residual β-cell function in adults, how should type 1 diabetes in adults be defined? What is the incidence and prevalence of the disease in adults? |
4. Are there differences in loss of residual insulin secretion rates in young adults compared with older adults? |
5. How is QOL affected by disease-modifying therapies? Do effects differ between adults and children? |
6. Are there biomarkers of responses to immune therapies that can discriminate responses in children and adults? |
Key messages . |
---|
1. Differences between childhood- and adult-onset type 1 diabetes should be part of the design of studies of disease-modifying therapies. |
2. Studying disease-modifying agents in children should not require efficacy data from adults. |
3. Children may benefit more from disease-modifying therapies due to the more rapid loss of insulin secretion before and after their diagnosis, the unique burdens on them and their families, and the greater vulnerability of young children’s neurocognitive development. |
4. Investigators should work with regulatory agencies early in the study design process and leverage the pediatric expertise of the agencies. |
5. A practical approach to assessing risk-to-benefit ratio is to consider the following: Given what is known about the intervention and the limited alternatives, would an independent and expert clinician regard enrolling children with type 1 diabetes as promoting the children’s clinical interests? |
Open research questions |
1. What is the long-term effect of age at onset and early glycemic control on complication risk both within childhood and adolescence (i.e., pre- vs. peri- or postpuberty) and in comparison with adult-onset type 1 diabetes? |
2. What are the biological mechanisms underlying the varying pathways to type 1 diabetes? Which mechanisms are seen in all individuals and which are age dependent? |
3. Given the high frequency of residual β-cell function in adults, how should type 1 diabetes in adults be defined? What is the incidence and prevalence of the disease in adults? |
4. Are there differences in loss of residual insulin secretion rates in young adults compared with older adults? |
5. How is QOL affected by disease-modifying therapies? Do effects differ between adults and children? |
6. Are there biomarkers of responses to immune therapies that can discriminate responses in children and adults? |