TY  - JOUR
AU  - Tesch, Victoria Katharina
AU  - Abolhassani, Hassan
AU  - Shadur, Bella
AU  - Zobel, Joachim
AU  - Mareika, Yuliya
AU  - Sharapova, Svetlana
AU  - Karakoc-Aydiner, Elif
AU  - Rivière, Jacques G
AU  - Garcia-Prat, Marina
AU  - Moes, Nicolette
AU  - Haerynck, Filomeen
AU  - Gonzales-Granado, Luis I
AU  - Santos Pérez, Juan Luis
AU  - Mukhina, Anna
AU  - Shcherbina, Anna
AU  - Aghamohammadi, Asghar
AU  - Hammarström, Lennart
AU  - Dogu, Figen
AU  - Haskologlu, Sule
AU  - İkincioğulları, Aydan I
AU  - Köstel Bal, Sevgi
AU  - Baris, Safa
AU  - Kilic, Sara Sebnem
AU  - Karaca, Neslihan Edeer
AU  - Kutukculer, Necil
AU  - Girschick, Hermann
AU  - Kolios, Antonios
AU  - Keles, Sevgi
AU  - Uygun, Vedat
AU  - Stepensky, Polina
AU  - Worth, Austen
AU  - van Montfrans, Joris M
AU  - Peters, Anke M J
AU  - Meyts, Isabelle
AU  - Adeli, Mehdi
AU  - Marzollo, Antonio
AU  - Padem, Nurcicek
AU  - Khojah, Amer M
AU  - Chavoshzadeh, Zahra
AU  - Avbelj Stefanija, Magdalena
AU  - Bakhtiar, Shahrzad
AU  - Florkin, Benoit
AU  - Meeths, Marie
AU  - Gamez, Laura
AU  - Grimbacher, Bodo
AU  - Seppänen, Mikko R J
AU  - Lankester, Arjan
AU  - Gennery, Andrew R
AU  - Seidel, Markus G
AU  - Inborn Errors, Clinical, and Registry Working Parties of the European Society for Blood and Marrow Transplantation and the European Society for Immunodeficiencies
PY  - 2019
DO  - 10.1016/j.jaci.2019.12.896
UR  - http://hdl.handle.net/10668/14904
T2  - The Journal of allergy and clinical immunology
AB  - Recent findings strongly support hematopoietic stem cell transplantation (HSCT) in patients with severe presentation of LPS-responsive beige-like anchor protein (LRBA) deficiency, but long-term follow-up and survival data beyond previous patient...
LA  - en
KW  - CTLA4
KW  - Inborn error of immunity
KW  - abatacept
KW  - clinical score
KW  - combined immunodeficiency
KW  - hematopoietic stem cell transplantation
KW  - immune dysregulation
KW  - performance scale
KW  - primary immunodeficiency disorder
KW  - sirolimus
KW  - Adaptor Proteins, Signal Transducing
KW  - Adolescent
KW  - Adult
KW  - Aged
KW  - Child
KW  - Child, Preschool
KW  - Female
KW  - Hematopoietic Stem Cell Transplantation
KW  - Humans
KW  - Immunologic Deficiency Syndromes
KW  - Male
KW  - Middle Aged
KW  - Survival Analysis
KW  - Treatment Outcome
KW  - Young Adult
TI  - Long-term outcome of LRBA deficiency in 76 patients after various treatment modalities as evaluated by the immune deficiency and dysregulation activity (IDDA) score.
TY  - research article
VL  - 145
ER  -