Stem cell transplantation for primary immunodeficiencies

被引：0
作者：
F Porta
C Forino
D De Martiis
E Soncini
L Notarangelo
K Tettoni
C D'Ippolito
R Soresina
K Shiha
S Berta
R Baffelli
F Bolda
A Bosi
F R Schumacher
A Lanfranchi
E Mazzolari
机构：
[1] Oncology-Haematology and BMT Unit,
[2] Ospedale dei Bambini,undefined
来源：
Bone Marrow Transplantation | 2008年 / 41卷
关键词：
primary immunodeficiencies; matched unrelated donor;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
BMT is curative in almost 75% of children affected by severe primary immunodeficiencies (PIDs). Recently, the chance of cure has increased thanks to the availability of matched unrelated donors (MUDs). Nevertheless, besides the conventional indications to BMT (profound or absent T-cell function, profound or absent natural killer function, known syndromes with T-cell deficiencies), indications to BMT for PIDs affecting the quality of life or having an expectation of life that does not exceed the third-fourth decade remain unclear. Infact, if it is evident that the survival rate in an infant grafted for a PID with a MUD is expected to be more than 80%, alternative treatments such as gene therapy are now available.
引用
收藏
页码：S83 / S86
相关论文
共 56 条
[1]  
Porta F(1998)Bone marrow transplantation in congenital immunodeficiency diseases Bone Marrow Transplant 21 S21-S23
[2]  
Friedrich W(1999)Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for blood and marrow transplantation and the European society for immunodeficiency J Pediatr 134 740-748
[3]  
Bertand Y(2000)Haploidentical peripheral blood and marrow stem cell transplantation in nine cases of primary immunodeficiency Haematologica 85 41-46
[4]  
Landais P(2002)Treatment of chronic granulomatous disease with myeloablative conditioning and an unmodified hemopoietic allograft: a survey of the European experience, 1985–2000 Blood 100 4344-4350
[5]  
Friedrich W(2003)Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report Bone Marrow Transplant 32 657-663
[6]  
Gerritsen B(2002)B cell function after haploidentical in utero bone marrow transplantation in a patient with severe combined immunodeficiency Bone Marrow Transplant 29 625-628
[7]  
Morgan G(2004)Reconstitution of T-cell compartment after in utero stem cell transplantation: analysis of T-cell repertoire and thymic output Haematologica 89 450-461
[8]  
Fasth A(2005)Hematopoietic stem cell transplantation in Omenn syndrome: a single-center experience Bone Marrow Transplant 36 107-114
[9]  
Lanfranchi A(2006)Bone marrow transplantation for severe combined immune deficiency JAMA 295 508-518
[10]  
Verardi R(2006)Stem cell transplantation for the Wiskott–Aldrich Syndrome: a single-centre experience confirms efficacy of matched unrelated transplantation Bone Marrow Transplant 38 671-679
123456