Improved Health-Related Quality of Life in a Phase 3 Islet Transplantation Trial in Type 1 Diabetes Complicated by Severe Hypoglycemia
Authors: Foster Eric D. , Bridges Nancy D. , Feurer Irene D. , Eggerman Thomas L. , Hunsicker Lawrence G. , Alejandro Rodolfo ,   Clinical Islet Transplantation Consortium, 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. , Kandaswamy Raja , Loganathan Gopal , Mueller Kate , Papas Klearchos K. , Pedersen Jayne , Wilhelm Joshua J. , Witson Jean , Dalton-Bakes Cornelia , Fu Hongxing , Kamoun Malek , Kearns Jane , Li Yanjing , Liu Chengyang , Luning-Prak Eline , Luo Yanping , Markmann Eileen , Min Zaw , Naji Ali , Palanjian Maral , Rickels Michael R. , Shlansky-Goldberg Richard , Vivek Kumar , Ziaie Amin Sam , Kaufman Dixon B. , Korsgren Olle 
Published: March 2018

OBJECTIVEAttaining 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.RESEARCH DESIGN AND METHODSFour 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.RESULTSImprovements 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 &lt; 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 &lt; 0.0001).CONCLUSIONSIn CIT-07, 87.5% of the subjects achieved the primary end point of freedom from SHE along with glycemic control (HbA1c &lt;7% [&lt;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.