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

来源：

BONE MARROW TRANSPLANTATION | 2023年 / 58卷 / 05期

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

页码：594 / 596

页数：3

共 50 条

[1] Outcome of Haematopoietic Cell Transplantation in Children with Lysosomal Acid Lipase Deficiency: A Study On Behalf of The Ebmt Inborn Errors Working Party
Lum, Su Han
Minkov, Millen
Jones, Simon
Hazelaar, Sheree
Potter, Jane
Stepensky, Polina
Garban, Frederic
Holter, Wolfgang
Stein, Jerry
Kocak, Ulker
Schulz, Ansgar
Mellgren, Karin
Diaz de Heredia, Cristina
Rubio, Marie Therese
Carballero, Dolores
Diaz, Miguel Angel
Neven, Benedicte
Michel, Gerard
Lindemans, Caroline
Gruhn, Bernd
Albert, Michael H.
Lankester, Arjan
Wynn, Robert
BONE MARROW TRANSPLANTATION, 2021, 56 (SUPPL 1) : 260 - 261
[2] Outcome of haematopoietic cell transplantation in children with lysosomal acid lipase deficiency: a study on behalf of the EBMT Inborn Errors Working Party
Su Han Lum
Milen Minkov
Simon A. Jones
Sheree Hazelaar
Tiarlan Sirait
Jane E. Potter
Polina Stepensky
Frederic Garban
Herbert Pichler
Jerry Stein
Zuhre Kaya
Ansgar Schulz
Karin Mellgren
Cristina Diaz de Heredia
Cecile Pochon
Susana Riesco
Miguel Angel Diaz
Gérard Michel
Caroline Lindemans
Bernd Gruhn
Michael H. Albert
Arjan C. Lankester
Bénédicte Neven
Robert Wynn
Bone Marrow Transplantation, 2023, 58 : 594 - 596
[3] Hematopoietic Stem Cell Transplantation for C1q Deficiency: A Study on Behalf of the EBMT Inborn Errors Working Party
Buso, Helena
Adam, Etai
Arkwright, Peter D.
Bhattad, Sagar
Hamidieh, Amir Ali
Behfar, Maryam
Belot, Alexandre
Benezech, Sarah
Chan, Alice Y.
Crow, Yanick J.
Dvorak, Christopher C.
Flinn, Aisling M.
Kapoor, Urvi
Lankester, Arjan
Kobayashi, Masao
Matsumura, Risa
Mottaghipisheh, Hadi
Okada, Satoshi
Ouachee, Marie
Parvaneh, Nima
Ramprakash, Stalin
Satwani, Prakash
Sharafian, Samin
Triaille, Clement
Wynn, Robert F.
Movahedi, Nasim
Ziaee, Vahid
Williams, Eleri
Slatter, Mary
Gennery, Andrew R.
JOURNAL OF CLINICAL IMMUNOLOGY, 2025, 45 (01)
[4] HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CTLA-4 INSUFFICIENCY: AN EBMT INBORN ERRORS WORKING PARTY STUDY
Tsilifis, Christo
Speckmann, Carsten
Lum, Su Han
Gennery, Andrew R.
Lankester, Arjan C.
Tjon, Jennifer
Buddingh, Emilie P.
Sedlacek, Petr
Laberko, Alexandra
Elcombe, Suzanne
Bigley, Venetia
Lougaris, Vassilios
Malagola, Michele
Wynn, Robert
Hauck, Fabian
Wehr, Claudia
Grimbacher, Bodo
Fox, Thomas
Morris, Emma C.
Margarit-Soler, Adriana
Mozo, Yasmina
Corral, Dolores
Ewins, Anna-Maria
Hague, Rosie
Oikonomopoulou, Christina
Kalwak, Krzysztof
Drabko, Katarzyna
Albert, Michael H.
Neven, Benedicte
Slatter, Mary A.
BONE MARROW TRANSPLANTATION, 2024, 59 : 89 - 90
[5] Haematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT Inborn Errors Working Party (IEWP) study
Ferrua, F.
Courteille, V.
Janda, A.
Slatter, M.
Albert, M. H.
Al-Mousa, H.
Al-Saud, B.
Balashov, D.
Bertrand, Y.
Booth, C.
Bordon, V.
Czogala, W.
Dogu, F.
Fasth, A.
Formankova, R.
Gozdzik, J.
Heilmann, C.
Hoenig, M.
Ikinciogullari, A.
Kalwak, K.
Keogh, S. J.
Krivan, G.
Lange, A.
Lankester, A. C.
Ljungman, P.
Meyts, I.
Neven, B.
Soncini, E.
Suarez, F.
Mahlaoui, N.
Fischer, A.
Davies, E. G.
Gennery, A. R.
BONE MARROW TRANSPLANTATION, 2015, 50 : S45 - S46
[6] Outcome of Haematopoietic Cell Transplantation in 813 Children with Fanconi Anaemia: A Study on Behalf of the EBMT Severe Aplastic Anaemia Working Party and Paediatric Disease Working Party
Lum, Su Han
Samarasinghe, Sujith
Eikema, Dirk-Jan
Piepenbroek, Brian
Dalissier, Arnaud
Ayas, Mouhab
Mousavi, Ashrafsadat
Hamladji, Rose-Marie
Yesilipek, Akif
Dalle, Jean-Hugues
Kansoy, Savas
Locatelli, Franco
Cetinkaya, Duygu
Bierings, Marc
Kupesiz, O. Alphan
Tbakhi, Abdelghani
Skorobogatova, Elena
Ozturk, Gulyuz
Faraci, Maura
Bertrand, Yves
Evans, Pamela
Carbacioglu, Selim
Dufour, Carlo
Risitano, Antonio
Wynn, Robert F.
de Latour, Regis Peffault
BLOOD, 2022, 140
[7] EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity
A. C. Lankester
M. H. Albert
C. Booth
A. R. Gennery
T. Güngör
M. Hönig
E. C. Morris
D. Moshous
B. Neven
A. Schulz
M. Slatter
P. Veys
Bone Marrow Transplantation, 2021, 56 : 2052 - 2062
[8] EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity
Lankester, A. C.
Albert, M. H.
Booth, C.
Gennery, A. R.
Guengoer, T.
Hoenig, M.
Morris, E. C.
Moshous, D.
Neven, B.
Schulz, A.
Slatter, M.
Veys, P.
BONE MARROW TRANSPLANTATION, 2021, 56 (09) : 2052 - 2062
[9] Outcome Of Unrelated Umbilical Cord Blood Transplantation For Children With Osteopetrosis: An Eurocord and Inborn Errors Working Party (IEWP)-EBMT Study
Chiesa, Robert
Ruggeri, Annalisa
Zecca, Marco
Locatelli, Franco
Gonzalez-Vincent, Marta
Bordon, Victoria
Yaniv, Isaac
Lawson, Sarah
Poire, Xavier
Dini, Giorgio
Abecasis, Manuel
Al-Seraihy, Amal
Kenzey, Chantal
Xavier, Erick
Bierings, Marc
Gluckman, Eliane
Schulz, Ansgar S.
Gennery, Andrew
Rocha, Vanderson
BLOOD, 2013, 122 (21)
[10] Outcome of Haematopoietic Cell Transplantation in Children with Fanconi Anaemia: A Study on Behalf of the EBMT SAAWP and PDWP
Lum, Su Han
Samarasinghe, Sujith
Wynn, Robert
Eikeman, Dirk-Jan
Bosman, Paul
Aksoylar, Serap
Ghavamzadeh, A.
Bierings, M.
Bertrand, Yves
Kitra-Roussou, V.
Smiers, F.
Strahm, Brigitte
Faraci, M.
Schulz, A.
Robinson, S.
Krivan, G.
Taskinen, M.
ALseraihy, Amal
Snowden, John
Itala-Remes, Maija
Bernard, Fanette
Kroger, N.
Bambi, F.
Riesco, Susana
Risitano, Antonio
Dufour, Carlo
Corbacioglu, Selim
de Latour, Regis Peffault
BONE MARROW TRANSPLANTATION, 2020, 55 (SUPPL 1) : 35 - 36

← 1 2 3 4 5 →