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Immunodeficiency Diseases

Immunodeficiency Diseases

Immunodeficiency disorders are conditions in which the immune system's ability to fight infectious disease and cancer is compromised or entirely absent. The immune system comprises a complex network of cells, tissues, and organs that work together to protect the body from potentially harmful invaders. When this system is deficient or not functioning properly, it leaves the body susceptible to infections, diseases, or other complications.
There are three main causes of immunodeficiency...

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Primary Immune Deficiency Treatment Consortium (PIDTC) report.

Linda M Griffith¹, Morton J Cowan², Luigi D Notarangelo³

¹Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Md.

The Journal of Allergy and Clinical Immunology

|October 22, 2013

Summary

The Primary Immune Deficiency Treatment Consortium (PIDTC) advances rare disease research through multi-center studies and training. It focuses on severe combined immunodeficiency (SCID) and other primary immunodeficiencies, aiming for optimal treatment strategies.

Keywords:

ADA Adenosine deaminase Allogeneic hematopoietic cell transplantation CGD CIBMTR Center for International Blood and Marrow Transplant Research Chronic granulomatous disease EBMT ESID European Group for Blood and Marrow Transplantation European Society for Immunodeficiencies GT GVHD Gene therapy Graft-versus-host disease HCT Hematopoietic cell transplantation IEWP Inborn Errors Working Party MAC MUD Matched unrelated donor Myeloablative conditioning NBS NIAID NIH NK National Institute of Allergy and Infectious Diseases National Institutes of Health Natural killer Newborn screening for SCID PID PIDTC Primary Immune Deficiency Treatment Consortium Primary immunodeficiency RIC Reduced-intensity conditioning SCETIDE SCID Severe combined immunodeficiency Stem Cell Transplantation for Immunodeficiencies in Europe USIDNET United States Immunodeficiency Network WAS Wiskott-Aldrich syndrome clinical trial gene therapy primary immunodeficiency

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Area of Science:

Immunology
Genetics
Clinical Research

Background:

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a North American network of 33 centers.
It focuses on rare and severe primary immunodeficiency diseases.

Purpose of the Study:

To report the progress of the PIDTC to date.
To highlight the first two PIDTC workshops.
To consider future consortium objectives.

Main Methods:

Retrospective, prospective, and cross-sectional studies.
Pilot demonstration projects.
Collaborations with international colleagues and patient advocacy groups.

Main Results:

Funded 2 pilot projects: SCID newborn screening in Navajo Native Americans and B-cell reconstitution post-hematopoietic stem cell transplantation.
Awarded grants to 10 junior investigators.
Held annual workshops fostering collaboration and discussion on research progress and future directions.

Conclusions:

The PIDTC is actively progressing in studying and treating primary immunodeficiencies.
Future goals include prospective treatment studies to determine optimal therapies.
The consortium fosters research, training, and community awareness for rare immune disorders.