Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

被引：281

作者：

Angelucci, Emanuele ^{[1
]}

Matthes-Martin, Susanne ^{[2
]}

Baronciani, Donatella ^{[1
]}

Bernaudin, Francoise ^{[3
]}

Bonanomi, Sonia ^{[4
]}

Cappellini, Maria Domenica ^{[5
]}

Dalle, Jean-Hugues ^{[6
]}

Di Bartolomeo, Paolo ^{[7
]}

Diaz de Heredia, Cristina ^{[8
]}

Dickerhoff, Roswitha ^{[9
]}

Giardini, Claudio ^{[10
]}

Gluckman, Eliane ^{[11
]}

Hussein, Ayad Achmed ^{[12
]}

Kamani, Naynesh ^{[13
]}

Minkov, Milen ^{[2
]}

Locatelli, Franco ^{[14
]}

Rocha, Vanderson ^{[15
]}

Sedlacek, Petr ^{[16
]}

Smiers, Frans ^{[17
]}

Thuret, Isabelle ^{[18
]}

Yaniv, Isaac ^{[19
]}

Cavazzana, Marina ^{[20
,21
,22
,23
]}

Peters, Christina ^{[2
]}

机构：

[1] Osped Oncol Riferimento Reg Armando Businco, Cagliari, Italy

[2] Med Univ, Dept Pediat, St Anna Childrens Hosp, Vienna, Austria

[3] CHI Creteil, Ctr Reference Drepanocytose, Creteil, France

[4] Hosp San Gerardo, Monza, Italy

[5] Policlin Hosp, IRCCS Fdn, Dipartimento Sci Clin & Comunita, Milan, Italy

[6] Hop Robert Debre, Serv Hematoimmunol, F-75019 Paris, France

[7] Osped Civile, BMT Ctr, Pescara, Italy

[8] Hop Maternoinfantil Val Hebron, Barcelona, Spain

[9] Univ Dusseldorf, Paediat Unit Haematol Oncol & Immunol, Dusseldorf, Germany

[10] Hematol & Stem Cell Transplant Ctr, Pesaro, Italy

[11] Hop St Louis, Paris, France

[12] King Hussein Canc Ctr, Amman, Jordan

[13] Childrens Natl Med Ctr, Div BMT Immunol, Washington, DC 20010 USA

[14] IRCCS Osped Pediat Bambino Gesu, Rome, Italy

[15] Hop St Louis, Eurocord, Paris, France

[16] Charles Univ Motol Prague, Sch Med 2, Dept Pediat Hematol & Oncol Teaching Hosp Motol, Prague, Czech Republic

[17] Pediat Hematol Oncol & Bone Marrow Transplantat W, Leiden, Netherlands

[18] Pediat Hop Enfants Timone, Serv Hematol, Marseille, France

[19] Schneider Childrens Med Ctr Israel, Petah Tiqwa, Israel

[20] INSERM, U768, Paris, France

[21] Hop Univ Necker Enfants Malades, AP HP, Dept Biotherapie, Paris, France

[22] Univ Paris 05, Sorbonne Paris Cite, IMAGINE Inst, Paris, France

[23] AP HP, INSERM, CIC Biotherapie GHU Ouest, Paris, France

来源：

HAEMATOLOGICA | 2014年 / 99卷 / 05期

关键词：

BONE-MARROW-TRANSPLANTATION; CORD BLOOD TRANSPLANTATION; BETA-THALASSEMIA; PERIPHERAL-BLOOD; LONG-TERM; ALLOGENEIC TRANSPLANTATION; CONDITIONING REGIMEN; MIXED CHIMERISM; PREPARATIVE REGIMEN; IRON OVERLOAD;

D O I：

10.3324/haematol.2013.099747

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Thalassemia major and sickle cell disease are the two most widely disseminated hereditary hemoglobinopathies in the world. The outlook for affected individuals has improved in recent years due to advances in medical management in the prevention and treatment of complications. However, hematopoietic stem cell transplantation is still the only available curative option. The use of hematopoietic stem cell transplantation has been increasing, and outcomes today have substantially improved compared with the past three decades. Current experience world-wide is that more than 90% of patients now survive hematopoietic stem cell transplantation and disease-free survival is around 80%. However, only a few controlled trials have been reported, and decisions on patient selection for hematopoietic stem cell transplantation and transplant management remain principally dependent on data from retrospective analyses and on the clinical experience of the transplant centers. This consensus document from the European Blood and Marrow Transplantation Inborn Error Working Party and the Paediatric Diseases Working Party aims to report new data and provide consensus-based recommendations on indications for hematopoietic stem cell transplantation and transplant management.

引用

页码：811 / 820

页数：10

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