Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant

被引：107

作者：

Dufour, Carlo ^{[1
]}

Pillon, Marta ^{[2
]}

Socie, Gerard ^{[3
]}

Rovo, Alicia ^{[4
]}

Carraro, Elisa ^{[2
]}

Bacigalupo, Andrea ^{[5
]}

Oneto, Rosi ^{[5
]}

Passweg, Jakob ^{[4
]}

Risitano, Antonio ^{[6
]}

Tichelli, Andre ^{[4
]}

de Latour, Regis Peffault ^{[3
]}

Schrezenmeier, Hubert ^{[7
,8
]}

Hocshmann, Britta ^{[7
,8
]}

Peters, Christina ^{[9
]}

Kulasekararaj, Austin ^{[10
]}

Van Biezen, Anja ^{[11
]}

Samarasinghe, Sujith ^{[12
]}

Hussein, Ayad Ahmed ^{[13
]}

Ayas, Mouhab ^{[14
]}

Aljurf, Mahmoud ^{[14
]}

Marsh, Judith ^{[10
]}

机构：

[1] G Gaslini Childrens Hosp, Clin & Expt Haematol Unit, I-16147 Genoa, Italy

[2] Univ Padua, Paediat Haemato Oncol Clin, I-35100 Padua, Italy

[3] Hop St Louis, Dept Haematol, Paris, France

[4] Univ Basel Hosp, CH-4031 Basel, Switzerland

[5] San Martino Hosp, Div Haematol 2, Genoa, Italy

[6] Univ Naples Federico II, Dept Clin Med & Surg, Naples, Italy

[7] Med Univ Ulm, Inst Clin Transfus Med & Immunogenet, Ulm, Germany

[8] Med Univ Ulm, Dept Transfus, Ulm, Germany

[9] St Anna Childrens Hosp, Paediat Haemtopoiet Stem Cell Transplantat, Vienna, Austria

[10] Kings Coll Hosp London, Kings Coll, Dept Haematol Med, London, England

[11] Univ Med Ctr, EBMT Data Off, Leiden, Netherlands

[12] Great Ormond St Hosp Sick Children, London WC1N 3JH, England

[13] King Hussein Canc Ctr, Bone Marrow & Stem Cell Transplantat Program, Amman, Jordan

[14] King Faisal Specialist Hosp & Res Ctr, Riyadh 11211, Saudi Arabia

来源：

BRITISH JOURNAL OF HAEMATOLOGY | 2015年 / 169卷 / 04期

关键词：

aplastic anaemia; children; haematopoietic stem cell transplantation; immunosuppression; RABBIT ANTITHYMOCYTE GLOBULIN; STEM-CELL TRANSPLANTATION; UNRELATED DONOR TRANSPLANTATION; IMMUNOSUPPRESSIVE THERAPY; ALTERNATIVE DONOR; PERIPHERAL-BLOOD; CYCLOSPORINE; MANAGEMENT; SURVIVAL; HORSE;

D O I：

10.1111/bjh.13297

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

This study analysed the outcome of 563 Aplastic Anaemia (AA) children aged 0-12years reported to the Severe Aplastic Anaemia Working Party database of the European Society for Blood and Marrow Transplantation, according to treatment received. Overall survival (OS) after upfront human leucocyte antigen-matched family donor (MFD) haematopoietic stem cell transplantation (HSCT) or immunosuppressive treatment (IST) was 91% vs. 87% (P 018). Event-free survival (EFS) after upfront MFD HSCT or IST was 87% vs. 33% (P 0001). Ninety-one of 167 patients (55%) failed front-line IST and underwent rescue HSCT. The OS of this rescue group was 83% compared with 91% for upfront MFD HSCT patients and 97% for those who did not fail IST up-front (P 0017). Rejection was 2% for MFD HSCT and HSCT post-IST failure (P 073). Acute graft-versus-host disease (GVHD) grade II-IV was 8% in MFD graft vs. 25% for HSCT post-IST failure (P<00001). Chronic GVHD was 6% in MFD HSCT vs. 20% in HSCT post-IST failure (P<00001). MFD HSCT is an excellent therapy for children with AA. IST has a high failure rate, but remains a reasonable first-line choice if MFD HSCT is not available because high OS enables access to HSCT, which is a very good rescue option.

引用

页码：565 / 573

页数：9

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