Abatacept for treatment-refractory pediatric CTLA4-haploinsufficiency.

Clinical Immunology : the Official Journal of the Clinical Immunology Society
Anna-Lisa LanzFabian Hauck

Abstract

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.

References

Nov 14, 1997·Immunity·C B Thompson, J P Allison
Oct 21, 2014·Nature Medicine·Desirée SchubertBodo Grimbacher
Oct 20, 2015·The Journal of Allergy and Clinical Immunology·Sangmoon LeeMurim Choi
Apr 23, 2016·The Journal of Allergy and Clinical Immunology·Mary A SlatterTroy R Torgerson
Dec 3, 2016·The Journal of Allergy and Clinical Immunology·Alexander A NavariniMike Recher
Jun 14, 2017·Drugs·Hannah A Blair, Emma D Deeks
May 8, 2018·The Journal of Allergy and Clinical Immunology·Charlotte SchwabBodo Grimbacher
Sep 27, 2018·Frontiers in Immunology·David EggBodo Grimbacher
Oct 30, 2018·Nucleic Acids Research·Philipp RentzschMartin Kircher
Aug 10, 2019·Frontiers in Immunology·Owen M SiggsChristopher C Goodnow
Jan 30, 2020·International Journal of Hematology·Hiroshi UreshinoShinya Kimura

❮ Previous
Next ❯

Related Concepts

Related Feeds

Allogenic & Autologous Therapies

Allogenic therapies are generated in large batches from unrelated donor tissues such as bone marrow. In contrast, autologous therapies are manufactures as a single lot from the patient being treated. Here is the latest research on allogenic and autologous therapies.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Related Papers

Nature Reviews. Drug Discovery
Larry W MorelandPeter Kirkpatrick
Revue de l'infirmière
Corinne Taéron
British Journal of Clinical Pharmacology
Eline A Dubois, Adam F Cohen
© 2021 Meta ULC. All rights reserved