Abatacept for treatment-refractory pediatric CTLA4-haploinsufficiency

Clin Immunol. 2021 Jun 8:108779. doi: 10.1016/j.clim.2021.108779. Online ahead of print.ABSTRACTCTLA4-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.PMID:34116213 | DOI:10.1016/j.clim.2021.108779

Source: Clinical Immunology - June 11, 2021 Category: Allergy & Immunology Authors: Anna-Lisa Lanz Martin Riester Philipp Peters Tobias Schwerd Eberhard Lurz Mohammad Samer Hajji Meino Rohlfs Julia Ley-Zaporozhan Christoph Walz Daniel Kotlarz Christoph Klein Michael H Albert Fabian Hauck Source Type: research