The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders

Frontiers in Immunology

Deepak Chellapandian¹  Jennifer W. Leiding²  Danielle E. Arnold³ 

[1] Center for Cell and Gene Therapy for Non-Malignant Conditions, Cancer and Blood Disorders Institute, Johns Hopkins All Children’s Hospital, St. Petersburg, FL, United States;Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, St. Petersburg, FL, United States;National Cancer Institute, National Institutes of Health, Bethesda, MD, United States;

关键词: PIRD;    CTLA4;    Pi3Kinase;    STAT1;    STAT3;    Jakinib;   

DOI  :  10.3389/fimmu.2021.692219

来源: DOAJ

【 摘 要 】

Recently, primary immune regulatory disorders have been described as a subset of inborn errors of immunity that are dominated by immune mediated pathology. As the pathophysiology of disease is elucidated, use of biologic modifiers have been increasingly used successfully to treat disease mediated clinical manifestations. Hematopoietic cell transplant (HCT) has also provided definitive therapy in several PIRDs. Although biologic modifiers have been largely successful at treating disease related manifestations, data are lacking regarding long term efficacy, safety, and their use as a bridge to HCT. This review highlights biologic modifiers in the treatment of several PIRDs and there use as a therapeutic bridge to HCT.

【 授权许可】

Unknown