Improved Health-Related Quality of Life in a Phase 3 Islet Transplantation Trial in Type 1 Diabetes Complicated by Severe Hypoglycemia

• Published in: Diabetes care Vol. 41; no. 5; pp. 1001 - 1008
• Main Authors: Foster, Eric D., Bridges, Nancy D., Feurer, Irene D., Eggerman, Thomas L., Hunsicker, Lawrence G., Alejandro, Rodolfo, Begley, Beth, Cano, Jose, Carpentier, Sallie, Hutchinson, Jennifer, Larsen, Christian P., Moreno, Johanna, Sears, Marti, Turgeon, Nicole A., Webster, Dasia, Markmann, James F., Al-saden, Patrice, Chen, Xioajuan, Hecyk, Angela, Luo, Xunrong, Molitch, Mark, Monson, Natalie, Stuart, Elyse, Wallia, Amisha, Wang, Lingjia, Wang, Shusen, Zhang, Xiaomin, Czarniecki, Christine W., Goldstein, Julia S., Priore, Allison, Robien, Mark A., Schneider, Elizabeth, Arreaza-Rubin, Guillermo, Green, Neal, Bigam, David L., Campbell, Patricia, Dinyari, Parastoo, Kin, Tatsuya, Kneteman, Norman M., Lyon, James, Malcolm, Andrew, O’Gorman, Doug, Onderka, Chris, Owen, Richard, Pawlick, Rena, Richer, Brad, Rosichuk, Shawn, Sarman, Donna, Schroeder, Adam, Senior, Peter A., Shapiro, A. M. James, Toth, Lana, Toth, Vali, Zhai, Wendy, Johnson, Kristina, McElroy, Joan, Posselt, Andrew M., Ramos, Marissa, Rojas, Tara, Stock, Peter G., Szot, Gregory, Barbaro, Barbara, Martellotto, Joan, Oberholzer, Jose, Qi, Meirigeng, Wang, Yong, Bayman, Levent, Chaloner, Kathryn, Clarke, William R., Dillon, Joseph S., Diltz, Cynthia, Doelle, Gregory C., Ecklund, Dixie, Feddersen, Deb, Jasperson, Carol, Lafontant, David-Erick, Neill-Hudson, Tina, Nollen, Deb, Qidwai, Julie, Riss, Holly, Schwieger, Traci, Willits, Jamie, Yankey, Jon, Corrales, Andrea Curry, Faradji, Raquel, Froud, Tatiana, Alvarez Gil, Ana, Herrada, Eva, Inverardi, Luca, Kenyon, Norma, Khan, Aisha, Linetsky, Elina, Peixoto, Eduardo, Ricordi, Camillo, Abdulla, Muhamad H., Balamurugan, A. N., Bellin, Melena D., Brandenburg, Mary, Harmon, James V., Hering, Bernhard J.
• Format: Journal Article
• Language: English
• Published: United States American Diabetes Association 01.05.2018
• Subjects: Adult; Blood Glucose - metabolism; Clinical Care/Education/Nutrition/Psychosocial Research; Consortia; Diabetes; Diabetes mellitus; Diabetes mellitus (insulin dependent); Diabetes Mellitus, Type 1 - blood; Diabetes Mellitus, Type 1 - complications; Diabetes Mellitus, Type 1 - therapy; Female; Follow-Up Studies; Glucose monitoring; Health; Health Status; Humans; Hypoglycemia; Hypoglycemia - chemically induced; Hypoglycemia - complications; Hypoglycemia - pathology; Hypoglycemia - therapy; Hypoglycemic Agents - therapeutic use; Islet cells; Islets of Langerhans Transplantation; Male; Middle Aged; Pancreas transplantation; Pancreatic islet transplantation; Polls & surveys; Quality of Life; Research design; Severity of Illness Index; Statistical analysis; Surveys and Questionnaires; Transplantation; Treatment Outcome; Visual observation; Young Adult
• ISSN: 0149-5992; 1935-5548; 1935-5548
• DOI: 10.2337/dc17-1779

Attaining glycemic targets without severe hypoglycemic events (SHEs) is a challenging treatment goal for patients with type 1 diabetes complicated by impaired awareness of hypoglycemia (IAH). The CIT Consortium Protocol 07 (CIT-07) trial showed islet transplantation to be an effective treatment for subjects with IAH and intractable SHEs. We evaluated health-related quality of life (HRQOL), functional health status, and health utility before and after pancreatic islet transplantation in CIT-07 trial participants. Four surveys, the Diabetes Distress Scale (DDS), the Hypoglycemic Fear Survey (HFS), the Short Form 36 Health Survey (SF-36), and the EuroQoL 5 Dimensions (EQ-5D), were administered repeatedly before and after islet transplantation. Summary statistics and longitudinal modeling were used to describe changes in survey scores from baseline and to characterize change in relation to a minimally important difference (MID) threshold of half an SD. Improvements in condition-specific HRQOL met the MID threshold. Reductions from baseline in the DDS total score and its four DDS subscales (all ≤ 0.0013) and in the HFS total score and its two subscales (all < 0.0001) were observed across all time points. Improvements were observed after both 1 and 2 years for the EQ-5D visual analog scale (both < 0.0001). In CIT-07, 87.5% of the subjects achieved the primary end point of freedom from SHE along with glycemic control (HbA <7% [<53 mmol/mol]) at 1 year post-initial islet transplantation. The same subjects reported consistent, statistically significant, and clinically meaningful improvements in condition-specific HRQOL as well as self-assessments of overall health.