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

Danielle E Arnold; Deepak Chellapandian; Jennifer W Leiding

doi:10.3389/fimmu.2021.692219

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

Front Immunol. 2021 Jun 24:12:692219. doi: 10.3389/fimmu.2021.692219. eCollection 2021.

Authors

Danielle E Arnold¹, Deepak Chellapandian², Jennifer W Leiding^{2

3}

Affiliations

¹ National Cancer Institute, National Institutes of Health, Bethesda, MD, United States.
² 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.
³ Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, St. Petersburg, FL, United States.

Abstract

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.

Keywords: Abatacept; CTLA4; Emapalumab; Jakinib; PIRD; Pi3Kinase; STAT1; STAT3.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Biological Products / therapeutic use*
Hematopoietic Stem Cell Transplantation*
Humans
Immune System Diseases / drug therapy*
Immune System Diseases / genetics
STAT1 Transcription Factor / genetics
STAT3 Transcription Factor / genetics

Substances

Biological Products
STAT1 Transcription Factor
STAT1 protein, human
STAT3 Transcription Factor
STAT3 protein, human