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Lanz, Anna-Lisa; Riester, Martin; Peters, Philipp; Schwerd, Tobias; Lurz, Eberhard; Hajji, Mohammad Samer; Rohlfs, Meino; Ley-Zaporozhan, Julia; Walz, Christoph; Kotlarz, Daniel; Klein, Christoph; Albert, Michael H. and Hauck, Fabian (2021): Abatacept for treatment-refractory pediatric CTLA4-haploinsufficiency. In: Clinical Immunology, Vol. 229, 108779

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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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