Abstract Body

Some patients living with type 1 diabetes (T1D) are participating in clinical trials of cell therapy. As these trials advance, Physician Investigators and clinical trial Sponsors are learning more about outcomes desired and experienced by patients.

Three themes that emerge in this setting are fear of hypoglycemia, a reduction of the burden of daily management of T1D, and a desire for better glycemic control in order to reduce the risk for micro- and macro-vascular complications. Hypoglycemia has a major impact on patients and their behavior,¹ and a history and fear of severe hypoglycemia may be a prominent concern driving interest in cell therapy; these issues are even more pronounced for parents and their offspring. For others, cell therapy may be attractive because it does not require daily monitoring. HbA1C control is recognized as important by patients, yet its improvement has to be tempered by reducing hypoglycemia risk and improving glucose time in range, in the context of simplifying the burden of T1D management.

Recent experience suggests that patients with T1D and hypoglycemia unawareness in particular have a strong interest in cell therapy. The interest exists despite the availability of continuous glucose monitoring and advancements in diabetes technological tools. Patients suggest that cell therapy can be successful in several dimensions other than A1C control, and it can be attractive without offering complete insulin independence. Patient personal experiences may not always be related to changes in traditional clinical indicators² including A1C. Questionnaires used in clinical trials help capture real-time feedback from patients with T1D and can help illuminate both benefits and burdens associated with these interventions. Given the diversity of perspectives and the lack of PRO questionnaires specific to diabetes cell therapy, additional research should include qualitative interviews of patients receiving cell therapy and validation of these instruments. Interviews in the clinical trial setting may be a good supplement for capturing clinically meaningful patient-reported outcomes.

References:

1. K. Khunti, S. Alsifri, R. Aronson, M. Cigrovski Berković, C. Enters-Weijnen, T. Forsén, G. Galstyan, P. Geelhoed-Duijvestijn, M. Goldfracht, H. Gydesen, R. Kapur, N. Lalic, B. Ludvik, E. Moberg, U. Pedersen-Bjergaard and A. Ramachandran. 2017. Impact of hypoglycaemia on patient-reported outcomes from a global, 24-country study of 27,585 people with type 1 and insulin-treated type 2 diabetes. Diabetes research and clinical practice, 130, 121-129.

2. L. Fisher, W. Polonsky, V. Bowyer and D. Hessler. 2020. When patient-reported experience does not match change in clinical outcomes: A perplexing view from the inside of a diabetes distress intervention. Journal of diabetes and its complications, 34(4), 107533.

3. S. N. DuBose, C. Bauza, A. Verdejo, R. W. Beck, R. M. Bergenstal, J. Sherr and H. S. Group. 2021. Real-World, Patient-Reported and Clinic Data from Individuals with Type 1 Diabetes Using the MiniMed 670G Hybrid Closed-Loop System. Diabetes technology & therapeutics, 23(12), 791-798.