A meta-analysis of the efficacy and safety of islet transplantation and combined islet-stem cell transplantation for type 1 diabetes mellitus

doi:10.3969/j.issn.1004-583X.2026.07.001

Abstract

Abstract:

Objective To systematically evaluate the efficacy and safety of islet transplantation (ITA) and combined islet-stem cell transplantation (ISCT) in the treatment of type 1 diabetes mellitus (T1DM). Methods CNKI, Wanfang Data, VIP, the Cochrane Library, PubMed, and Embase were searched to identify clinical studies assessing the efficacy of ITA and ISCT for T1DM, from database inception to August 2024. Two reviewers independently screened the literature and extracted data. Study quality was assessed using the MINORS scale, and meta-analysis was performed using Stata 11. Results A total of 13 studies were included, comprising 8 retrospective studies and 5 prospective studies, with 222 patients with T1DM. The meta-analysis showed that both ITA and ISCT significantly reduced glycated hemoglobin (HbA₁c), increased fasting C-peptide (C-P) levels, decreased insulin requirements, and improved insulin independence. Subgroup analysis indicated that ISCT was more effective than ITA in improving HbA₁c and C-P. In terms of safety, the main adverse events were immunosuppressant-related reactions, such as leukopenia; however, these risks were generally manageable. Conclusion Both ITA and ISCT have favorable therapeutic effects in patients with T1DM, and ISCT appears to be superior in improving transplantation outcomes and maintaining long-term function.

Key words: type 1 diabetes mellitus, meta-analysis, islet-stem cell transplantation, islet transplantation

CLC Number:

R587.1

Cao Huanzheng, Ren Qianyu, Zhang Kaiqian, Wang Zihan, Li Haoru, Jin Yangyu, Zhang Lihui, Wang Jing. A meta-analysis of the efficacy and safety of islet transplantation and combined islet-stem cell transplantation for type 1 diabetes mellitus[J]. Clinical Focus, 2026, 41(7): 581-589.

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URL: https://www.lchc.cn/EN/10.3969/j.issn.1004-583X.2026.07.001

https://www.lchc.cn/EN/Y2026/V41/I7/581

Figures/Tables 15

References 22

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作者	样本量	地址	干预措施	试验设计	单中心/ 多中心	平均年龄	女性占比	文献质量	胰岛细胞移植数量	干细胞移植数量
Takita 2012^[13]	9	美国	ITA	RC	单中心	43.9岁	77.80%	9/16	>4 000 IEQ/kg
Maffi 2011^[4]	33	意大利	ITA	RC	单中心	36岁	45.40%	17.5/24	未明确指出
Nakamura 2020^[3]	7	日本	ITA	RC	多中心	39.0岁	71.40%	18/24	中位数:408 000 IEQ
O'Connell 2013^[14]	17	澳大利亚	ITA	RC	多中心	49.8岁	未提及	11/16	平均值(96.17±44.58)×10⁴ IEQ
Rickels 2015^[15]	12	美国	ITA	PC	单中心	45岁	71.43%	19/24	平均值9 648±666 IEQ/kg
Holmes-Walker 2017^[16]	10	澳大利亚	ITA	RC	多中心	49.8岁	60%	12/16	未明确指出
Stanley 2024^[1]	60	英国	ITA	RC	单中心	49岁	57.60%	13/16	中位数4 300 IEQ/kg
姚豫桐2022^[9]	5	中国	ITA	RC	单中心	32.6岁	40%	20/24	平均值(50.60±2.51)×10⁴ IEQ
姚豫桐2022^[9]	5	中国	ISCT	RC	单中心	35岁	40%	20/24	平均值(48.70±4.76)×10⁴ IEQ	平均值(9.2±2.6)×10⁷个
Rydzon 2018^[6]	22	美国	ITA	PC	单中心	45.3岁	81.80%	13/16	中位数14 309 IEQ/kg
Brooks 2015^[17]	12	英国	ITA	PC	单中心	51岁	83.30%	13/16	平均值11 232 IEQ/kg
姚豫桐2016^[5]	10	中国	ITA	RC	单中心	28.6岁	30%	13/16	平均值501 740 IEQ
Forbes 2015^[18]	14	英国	ITA	PC	单中心	46岁	57%	12/16	平均值9 355 IEQ/kg
Mineo 2008^[19]	6	美国	ISCT	PC	单中心	39岁	66.70%	11/16	平均值(57.45±14.16)×10⁴ IEQ	平均值4.3±1.9×10⁵HSC/kg

作者	样本量	地址	干预措施	试验设计	单中心/ 多中心	平均年龄	女性占比	文献质量	胰岛细胞移植数量	干细胞移植数量
Takita 2012^[13]	9	美国	ITA	RC	单中心	43.9岁	77.80%	9/16	>4 000 IEQ/kg
Maffi 2011^[4]	33	意大利	ITA	RC	单中心	36岁	45.40%	17.5/24	未明确指出
Nakamura 2020^[3]	7	日本	ITA	RC	多中心	39.0岁	71.40%	18/24	中位数:408 000 IEQ
O'Connell 2013^[14]	17	澳大利亚	ITA	RC	多中心	49.8岁	未提及	11/16	平均值(96.17±44.58)×10⁴ IEQ
Rickels 2015^[15]	12	美国	ITA	PC	单中心	45岁	71.43%	19/24	平均值9 648±666 IEQ/kg
Holmes-Walker 2017^[16]	10	澳大利亚	ITA	RC	多中心	49.8岁	60%	12/16	未明确指出
Stanley 2024^[1]	60	英国	ITA	RC	单中心	49岁	57.60%	13/16	中位数4 300 IEQ/kg
姚豫桐2022^[9]	5	中国	ITA	RC	单中心	32.6岁	40%	20/24	平均值(50.60±2.51)×10⁴ IEQ
姚豫桐2022^[9]	5	中国	ISCT	RC	单中心	35岁	40%	20/24	平均值(48.70±4.76)×10⁴ IEQ	平均值(9.2±2.6)×10⁷个
Rydzon 2018^[6]	22	美国	ITA	PC	单中心	45.3岁	81.80%	13/16	中位数14 309 IEQ/kg
Brooks 2015^[17]	12	英国	ITA	PC	单中心	51岁	83.30%	13/16	平均值11 232 IEQ/kg
姚豫桐2016^[5]	10	中国	ITA	RC	单中心	28.6岁	30%	13/16	平均值501 740 IEQ
Forbes 2015^[18]	14	英国	ITA	PC	单中心	46岁	57%	12/16	平均值9 355 IEQ/kg
Mineo 2008^[19]	6	美国	ISCT	PC	单中心	39岁	66.70%	11/16	平均值(57.45±14.16)×10⁴ IEQ	平均值4.3±1.9×10⁵HSC/kg

随访时间	组别	纳入研究数	效应模型	I²	SMD(95%CI)	P值	组间P值
6个月	ITA	4	随机	58.60%	-2.13(-3.04, -1.22)	0.065
	ISCT	1	固定		-2.56(-4.32, -0.79)
合计		5		48.10%	-2.16(-2.94, -1.38)	0.103	0.058
1年	ITA	7	随机	58.50%	-1.11(-1.61, -0.61)	0.025
	ISCT	2	随机	48.90%	-1.86(-3.46, -0.26)	0.162
合计		9		57.80%	-1.22(-1.70, -0.73)	0.015	0.003
2~5年	ITA	4	随机	0.00%	-1.08(-1.53, -0.63)	0.403
	ISCT	1	固定		-2.18(-3.81, -0.54)
合计		5		11.70%	-1.17(-1.65, -0.68)	0.339	0.438

随访时间	组别	纳入研究数	效应模型	I²	SMD(95%CI)	P值	组间P值
6个月	ITA	4	随机	58.60%	-2.13(-3.04, -1.22)	0.065
	ISCT	1	固定		-2.56(-4.32, -0.79)
合计		5		48.10%	-2.16(-2.94, -1.38)	0.103	0.058
1年	ITA	7	随机	58.50%	-1.11(-1.61, -0.61)	0.025
	ISCT	2	随机	48.90%	-1.86(-3.46, -0.26)	0.162
合计		9		57.80%	-1.22(-1.70, -0.73)	0.015	0.003
2~5年	ITA	4	随机	0.00%	-1.08(-1.53, -0.63)	0.403
	ISCT	1	固定		-2.18(-3.81, -0.54)
合计		5		11.70%	-1.17(-1.65, -0.68)	0.339	0.438

随访时间	组别	纳入研究数	效应模型	I²	SMD(95%CI)	P值	组间P值
3个月	ITA	2	随机	0.00%	3.33(2.16, 4.50)	0.317
	ISCT	2	随机	0.00%	9.38(6.20, 12.55)	0.978
合计		4		77.40%	5.91(2.91, 8.91)	0.004	0.062
6个月	ITA	2	随机	29.20%	3.80(2.12, 5.47)	0.235
	ISCT	1	固定		10.40(5.15, 15.64)
合计		3		73.30%	5.41(2.23, 8.59)	0.024	0.397
1年	ITA	2	随机	39.70%	4.41(2.31, 6.51)	0.198
	ISCT	2	随机	92.70%	16.97(-13.89, 47.84)	<0.01
合计		4		82.50%	4.87(1.68, 8.06)	0.001	0.429
3年	ITA	2	随机	0.00%	3.08(1.97, 4.19)	0.585
	ISCT	1	固定		19.92(10.08, 29.76)
合计		3		82.50%	5.09(1.36, 8.81)	0.003	<0.01