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231225s2021 xx |||||o 00| ||eng c |
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|a 10.1016/j.clim.2021.108779
|2 doi
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|a pubmed24n1088.xml
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|a (DE-627)NLM326641599
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|a (NLM)34116213
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|a (PII)S1521-6616(21)00116-9
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|a DE-627
|b ger
|c DE-627
|e rakwb
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| 041 |
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|a eng
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| 100 |
1 |
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|a Lanz, Anna-Lisa
|e verfasserin
|4 aut
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| 245 |
1 |
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|a Abatacept for treatment-refractory pediatric CTLA4-haploinsufficiency
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| 264 |
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|c 2021
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| 336 |
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|a Text
|b txt
|2 rdacontent
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| 337 |
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|a ƒaComputermedien
|b c
|2 rdamedia
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| 338 |
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|a ƒa Online-Ressource
|b cr
|2 rdacarrier
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| 500 |
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|a Date Completed 16.09.2021
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|a Date Revised 16.09.2021
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|a published: Print-Electronic
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|a Citation Status MEDLINE
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|a Copyright © 2021 Elsevier Inc. All rights reserved.
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|a CTLA4-haploinsufficiency is a complex disease of immune dysregulation presenting with a broad spectrum of clinical manifestations. CTLA4-Fc fusion proteins such as abatacept have been described to alleviate immune dysregulation in several adult cases of CTLA4-haploinsufficiency. However, until now only few cases of pediatric CTLA4-haploinsufficiency treated with abatacept have been described. Here we present two pediatric cases of severe CTLA4-haploinsufficiency refractory to conventional immunosuppressive therapies that responded rapidly to treatment with abatacept. No side effects were observed during a follow-up period of 7-15 months. While one patient has successfully undergone HSCT the second patient continues to receive abatacept. Our cases demonstrate safe medium-term use of abatacept in the pediatric population
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| 650 |
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4 |
|a Case Reports
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|a Journal Article
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|a Research Support, Non-U.S. Gov't
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| 650 |
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4 |
|a Abatacept
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| 650 |
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4 |
|a CTLA4-fc fusion protein
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| 650 |
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4 |
|a CTLA4-haploinsufficiency
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| 650 |
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4 |
|a Immune dysregulation
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| 650 |
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4 |
|a Inborn error of immunity
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| 650 |
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7 |
|a CTLA-4 Antigen
|2 NLM
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| 650 |
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7 |
|a CTLA4 protein, human
|2 NLM
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| 650 |
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7 |
|a Immunosuppressive Agents
|2 NLM
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| 650 |
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7 |
|a Abatacept
|2 NLM
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| 650 |
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7 |
|a 7D0YB67S97
|2 NLM
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| 700 |
1 |
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|a Riester, Martin
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Peters, Philipp
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Schwerd, Tobias
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Lurz, Eberhard
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Hajji, Mohammad Samer
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Rohlfs, Meino
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Ley-Zaporozhan, Julia
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Walz, Christoph
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Kotlarz, Daniel
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Klein, Christoph
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Albert, Michael H
|e verfasserin
|4 aut
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| 700 |
1 |
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|a Hauck, Fabian
|e verfasserin
|4 aut
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| 773 |
0 |
8 |
|i Enthalten in
|t Clinical immunology (Orlando, Fla.)
|d 1999
|g 229(2021) vom: 10. Aug., Seite 108779
|w (DE-627)NLM098196855
|x 1521-7035
|7 nnns
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| 773 |
1 |
8 |
|g volume:229
|g year:2021
|g day:10
|g month:08
|g pages:108779
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| 856 |
4 |
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|u http://dx.doi.org/10.1016/j.clim.2021.108779
|3 Volltext
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|a AR
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|d 229
|j 2021
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|c 08
|h 108779
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