Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach

被引：75

作者：

Halter, J. ^{[1
]}

Schuepbach, W. M. M. ^{[2
,3
,4
]}

Casali, C. ^{[5
]}

Elhasid, R. ^{[6
]}

Fay, K. ^{[7
]}

Hammans, S. ^{[8
]}

Illa, I. ^{[9
]}

Kappeler, L. ^{[4
]}

Kraehenbuehl, S.

Lehmann, T. ^{[1
]}

Mandel, H. ^{[6
]}

Marti, R. ^{[10
,11
]}

Mattle, H. ^{[2
,4
]}

Orchard, K. ^{[12
]}

Savage, D. ^{[13
]}

Sue, C. M. ^{[14
]}

Valcarcel, D. ^{[15
]}

Gratwohl, A. ^{[1
]}

Hirano, M. ^{[16
]}

机构：

[1] Univ Basel Hosp, Dept Hematol, CH-4031 Basel, Switzerland

[2] Ctr Invest Clin, Paris, France

[3] INSERM, UMR 679, Pitie Salpxetriere Grp, Paris, France

[4] Univ Hosp Bern, Dept Neurol, Inselspital Bern, CH-3010 Bern, Switzerland

[5] Univ Roma La Sapienza, Univ Hosp, Rome, Italy

[6] Rambam Med Ctr, Haifa, Israel

[7] St Vincents Hosp, Dept Hematol, Sydney, NSW 2010, Australia

[8] Southampton Univ Hosp Trust, Wessex Neurol Ctr, Southampton, Hants, England

[9] Univ Autonoma Barcelona, Hosp Santa Creu & St Pau, E-08193 Barcelona, Spain

[10] CIBERER, Barcelona, Spain

[11] Univ Hosp Vall Hebron, Inst Recerca, Barcelona, Spain

[12] Univ Southampton, Dept Haematol, Southampton, Hants, England

[13] Columbia Univ, Med Ctr, Dept Haematol, New York, NY USA

[14] Univ Sydney, Dept Neurogenet, Royal N Shore Hosp, Kolling Inst Med Res, Sydney, NSW 2006, Australia

[15] Santa Creu & San Pau, Dept Hematol, Barcelona, Spain

[16] Columbia Univ, Dept Neurol, Med Ctr, New York, NY USA

来源：

BONE MARROW TRANSPLANTATION | 2011年 / 46卷 / 03期

基金：

美国国家卫生研究院;

关键词：

allogeneic hematopoietic SCT; mitochondrial neurogastrointestinal encephalomyopathy; MNGIE; consensus; recommendation; STEM-CELL TRANSPLANTATION; LACTIC-ACIDOSIS; CYCLOSPORINE-A; DISEASE; ENCEPHALOPATHY; INSTABILITY; DYSFUNCTION; TACROLIMUS; MUTATIONS; TOXICITY;

D O I：

10.1038/bmt.2010.100

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

Allogeneic hematopoietic SCT (HSCT) has been proposed as a treatment for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). HSCT has been performed in nine patients using different protocols with varying success. Based on this preliminary experience, participants of the first consensus conference propose a common approach to allogeneic HSCT in MNGIE. Standardization of the transplant protocol and the clinical and biochemical assessments will allow evaluation of the safety and efficacy of HSCT as well as optimization of therapy for patients with MNGIE. Bone Marrow Transplantation (2011) 46, 330-337; doi:10.1038/bmt.2010.100; published online 3 May 2010

引用

页码：330 / 337

页数：8

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