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

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 cl...

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Main Authors: Danielle E. Arnold, Deepak Chellapandian, Jennifer W. Leiding
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-06-01
Series:Frontiers in Immunology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2021.692219/full
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author Danielle E. Arnold
Deepak Chellapandian
Jennifer W. Leiding
Jennifer W. Leiding
author_facet Danielle E. Arnold
Deepak Chellapandian
Jennifer W. Leiding
Jennifer W. Leiding
author_sort Danielle E. Arnold
collection DOAJ
description 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.
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spelling doaj.art-a7144cf5961d4bdcacc95d4f96ab7f8e2022-12-21T22:36:23ZengFrontiers Media S.A.Frontiers in Immunology1664-32242021-06-011210.3389/fimmu.2021.692219692219The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory DisordersDanielle E. Arnold0Deepak Chellapandian1Jennifer W. Leiding2Jennifer W. Leiding3National Cancer Institute, National Institutes of Health, Bethesda, MD, United StatesCenter for Cell and Gene Therapy for Non-Malignant Conditions, Cancer and Blood Disorders Institute, Johns Hopkins All Children’s Hospital, St. Petersburg, FL, United StatesCenter for Cell and Gene Therapy for Non-Malignant Conditions, Cancer and Blood Disorders Institute, Johns Hopkins All Children’s Hospital, St. Petersburg, FL, United StatesDivision of Allergy and Immunology, Department of Pediatrics, University of South Florida, St. Petersburg, FL, United StatesRecently, 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.https://www.frontiersin.org/articles/10.3389/fimmu.2021.692219/fullPIRDCTLA4Pi3KinaseSTAT1STAT3Jakinib
spellingShingle Danielle E. Arnold
Deepak Chellapandian
Jennifer W. Leiding
Jennifer W. Leiding
The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
Frontiers in Immunology
PIRD
CTLA4
Pi3Kinase
STAT1
STAT3
Jakinib
title The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
title_full The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
title_fullStr The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
title_full_unstemmed The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
title_short The Use of Biologic Modifiers as a Bridge to Hematopoietic Cell Transplantation in Primary Immune Regulatory Disorders
title_sort use of biologic modifiers as a bridge to hematopoietic cell transplantation in primary immune regulatory disorders
topic PIRD
CTLA4
Pi3Kinase
STAT1
STAT3
Jakinib
url https://www.frontiersin.org/articles/10.3389/fimmu.2021.692219/full
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