Improved Health-Related Quality of Life in a Phase 3 Islet Transplantation Trial in Type 1 Diabetes Complicated by Severe Hypoglycemia
Authors: Eric D. Foster, Nancy D. Bridges, Irene D. Feurer, Thomas L. Eggerman, Lawrence G. Hunsicker, Rodolfo Alejandro, Beth Begley, Jose Cano, Sallie Carpentier, Jennifer Hutchinson, Christian P. Larsen, Johanna Moreno, Marti Sears, Nicole A. Turgeon, Dasia Webster, James F. Markmann, Patrice Al-saden, Xioajuan Chen, Angela Hecyk, Xunrong Luo, Mark Molitch, Natalie Monson, Elyse Stuart, Amisha Wallia, Lingjia Wang, Shusen Wang, Xiaomin Zhang, Christine W. Czarniecki, Julia S. Goldstein, Allison Priore, Mark A. Robien, Elizabeth Schneider, Guillermo Arreaza-Rubin, Neal Green, David L. Bigam, Patricia Campbell, Parastoo Dinyari, Tatsuya Kin, Norman M. Kneteman, James Lyon, Andrew Malcolm, Doug O’Gorman, Chris Onderka, Richard Owen, Rena Pawlick, Brad Richer, Shawn Rosichuk, Donna Sarman, Adam Schroeder, Peter A. Senior, A. M. James Shapiro, Lana Toth, Vali Toth, Wendy Zhai, Kristina Johnson, Joan McElroy, Andrew M. Posselt, Marissa Ramos, Tara Rojas, Peter G. Stock, Gregory Szot, Barbara Barbaro, Joan Martellotto, Jose Oberholzer, Meirigeng Qi, Yong Wang, Levent Bayman, Kathryn Chaloner, William R. Clarke, Joseph S. Dillon, Cynthia Diltz, Gregory C. Doelle, Dixie Ecklund, Deb Feddersen, Carol Jasperson, David-Erick Lafontant, Tina Neill-Hudson, Deb Nollen, Julie Qidwai, Holly Riss, Traci Schwieger, Jamie Willits, Jon Yankey, Andrea Curry Corrales, Raquel Faradji, Tatiana Froud, Ana Alvarez Gil, Eva Herrada, Luca Inverardi, Norma Kenyon, Aisha Khan, Elina Linetsky, Eduardo Peixoto, Camillo Ricordi, Muhamad H. Abdulla, A. N. Balamurugan, Melena D. Bellin, Mary Brandenburg, James V. Harmon, Bernhard J. Hering, Raja Kandaswamy, Gopal Loganathan, Kate Mueller, Klearchos K. Papas, Jayne Pedersen, Joshua J. Wilhelm, Jean Witson, Cornelia Dalton-Bakes, Hongxing Fu, Malek Kamoun, Jane Kearns, Yanjing Li, Chengyang Liu, Eline Luning-Prak, Yanping Luo, Eileen Markmann, Zaw Min, Ali Naji, Maral Palanjian, Michael R. Rickels, Richard Shlansky-Goldberg, Kumar Vivek, Amin Sam Ziaie, Dixon B. Kaufman, Olle Korsgren
Journal: Diabetes Care (2018)

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 P ≤ 0.0013) and in the HFS total score and its two subscales (all P < 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 P < 0.0001).In CIT-07, 87.5% of the subjects achieved the primary end point of freedom from SHE along with glycemic control (HbA1c <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.