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Patient and family expectations of beta-cell replacement therapies in type 1 diabetes.
Islets ( IF 2.2 ) Pub Date : 2018-08-17 , DOI: 10.1080/19382014.2018.1503518
Akitsu Kawabe 1 , Shinichi Matsumoto 1, 2 , Masayuki Shimoda 1
Affiliation  

Background: New methods of beta-cell replacement have been developed to maintain excellent glycemic control, improve quality of life, and even eliminate insulin injections in patients with type 1 diabetes mellitus (T1DM). Previously, we demonstrated that being insulin-free is the strongest motivation for accepting a newly developed therapy. Multiple allogeneic islet transplantations with immunosuppression using a human donor is the best option to be insulin-free, but the necessity for immunosuppression and donor shortage are major issues. However, these issues have been improved with scientific progress. The aim of this study was to investigate the opinions of patients and their families about the current progress. Methods: We conducted a questionnaire survey of T1DM patients (n = 47) and their family members (n = 49) about newly developed therapies: single and multiple allogeneic islet transplantation, single and multiple encapsulated allogeneic islet transplantation, single and multiple xenogeneic islet transplantation, and induced pluripotent stem cell therapy. Results: More than 90% of respondents wished to be insulin-free and have stable glycemic control. More than 90% of respondents accepted at least one of the new therapies. The current standard treatment multiple allogeneic islet transplantation was not well accepted or favored. Conclusions: The next generation of treatments, including xenotransplantation and induced pluripotent stem cell therapy, were more acceptable and favorable. Even though the majority of patients wish to become insulin-free, it is not sufficiently strong motivation for accepting newly developed treatments.



中文翻译:

患者和家人对1型糖尿病患者的β细胞替代疗法的期望。

背景:已经开发出新的β细胞替换方法,以维持出色的血糖控制,改善生活质量,甚至消除1型糖尿病(T1DM)患者的胰岛素注射。以前,我们证明不含胰岛素是接受新开发疗法的最强动机。多次同种异体胰岛移植使用人类供体进行免疫抑制是不含胰岛素的最佳选择,但免疫抑制和供体短缺的必要性是主要问题。但是,随着科学的进步,这些问题已经得到改善。这项研究的目的是调查患者及其家属对当前进展的看法。方法:我们对T1DM患者(n = 47)及其家人(n = 49)进行了有关新近开发的治疗方法的问卷调查:单次和多次异基因胰岛移植,单次和多次封装异体胰岛移植,单次和多次异种胰岛移植,并诱导了多能干细胞疗法。结果:超过90%的受访者希望不含胰岛素并具有稳定的血糖控制。超过90%的受访者接受了至少一种新疗法。当前的标准治疗多异体胰岛移植尚未得到很好的接受或青睐。结论:包括异种移植和诱导性多能干细胞疗法在内的下一代治疗更加可接受和有利。即使大多数患者希望无胰岛素,接受新开发的治疗方法的动机也不足。

更新日期:2018-08-17
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