Outcome of haematopoietic cell transplantation in children with lysosomal acid lipase deficiency: a study on behalf of the EBMT Inborn Errors Working Party

被引:4
|
作者
Lum, Su Han [1 ,2 ]
Minkov, Milen [3 ]
Jones, Simon A. [4 ]
Hazelaar, Sheree [5 ]
Sirait, Tiarlan [5 ]
Potter, Jane E. [6 ]
Stepensky, Polina [7 ]
Garban, Frederic [8 ]
Pichler, Herbert [9 ]
Stein, Jerry [10 ]
Kaya, Zuhre [11 ]
Schulz, Ansgar [12 ]
Mellgren, Karin [13 ]
de Heredia, Cristina Diaz [14 ]
Pochon, Cecile [15 ]
Riesco, Susana [16 ]
Diaz, Miguel Angel [17 ]
Michel, Gerard [18 ,19 ]
Lindemans, Caroline [20 ,21 ]
Gruhn, Bernd [22 ]
Albert, Michael H. [23 ]
Lankester, Arjan C. [24 ]
Neven, Benedicte
Wynn, Robert [6 ]
机构
[1] Great North Childrens Hosp, Childrens Hematopoiet Stem Cell Transplant Unit, Newcastle Upon Tyne, England
[2] Newcastle Univ, Translat & Clin Res Inst, Newcastle Upon Tyne, England
[3] Sigmund Freud Univ, Vienna, Austria
[4] St Marys Hosp, Willink Unit, Manchester, England
[5] EBMT Leiden Study Unit, Leiden, Netherlands
[6] Royal Manchester Childrens Hosp, Manchester, England
[7] Hebrew Univ Jerusalem, Hadassah Hosp, Fac Med, Dept Bone Marrow Transplantat, Jerusalem, Israel
[8] Univ Grenoble Alpes, CHU Grenoble Alpes, Grenoble, France
[9] Med Univ Vienna, St Anna Childrens Hosp, Dept Paediat, Vienna, Austria
[10] Schneider Childrens Med Ctr Israel, Assistance Publ Hop Paris APHP, Pediat Hematol Immunol & Rheumatol Unit, Petah Tiqwa, Israel
[11] Gazi Univ, Unit Pediat Hematol & Pediat BMT, Sch Med, Ankara, Turkiye
[12] Univ Med Ctr Ulm, Dept Pediat, Ulm, Germany
[13] Sahlgrens Univ Hosp, Dept Pediat Oncol, Gothenburg, Sweden
[14] Hosp Univ Vall Hebron, Dept Pediat Hematol & Oncol, Barcelona, Spain
[15] Univ Lorraine, Nancy Univ Hosp, Pediat Onco Hematol Dept, Nancy, France
[16] Hosp Univ Salamanca, Dept Pediat, Salamanca, Spain
[17] Nino Jesus Childrens Hosp, Madrid, Spain
[18] La Timone Childrens Hosp, Marseille, France
[19] Aix Marseille Univ, Marseille, France
[20] Univ Med Ctr, Dept Pediat, Utrecht, Netherlands
[21] Princess Maxima Ctr Pediat Oncol, Utrecht, Netherlands
[22] Jena Univ Hosp, Dept Pediat, Jena, Germany
[23] Ludwig Maximilians Univ Munchen, Dr von Hauner Univ Childrens Hosp, Munich, Germany
[24] Leiden Univ, Willem Alexander Childrens Hosp, Pediat Stem Cell Transplantat program, Med Ctr, Leiden, Netherlands
关键词
WOLMANS-DISEASE;
D O I
10.1038/s41409-023-01918-4
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
引用
收藏
页码:594 / 596
页数:3
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