Overview
Patient-Centric Approaches to Improve Timely Diagnosis and Personalized Management of Primary Immunodeficiency (PID)
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Although clinical presentation is variable, most primary immunodeficiencies (PI/PID), also referred to as inborn errors of immunity, are associated with increased susceptibility to infections and may remain undiagnosed for many years -- usually detected only after a patient has experienced recurrent or severe infections, which may cause permanent organ damage.
Infections related to PIDs because of delayed diagnosis and treatment can become chronic and produce long-lasting health concerns or, in severe cases, may be fatal, making a timely diagnosis and initiation of treatment vital. And once diagnosed, specialists must work collaboratively with patients to personalize therapy selection from the wide and growing array of IVIG and SCIG formulations available, each with specific clinical advantages and disadvantages that must be considered, along with patient preference, satisfaction, and quality of life.
To prevent the occurrence of unnecessary comorbidities and infections, enhance patient quality of life, and reduce the cost of care burden, expert faculty will review real-world cases and discuss opportunities to facilitate earlier diagnosis and ensure optimal implementation of individualized treatment in patients with PID.
This activity is intended for immunologists, primary care providers, pediatricians, APPs in immunology practice and other clinicians who participate in the collaborative diagnosis and management of patients with PID.
Primary immunodeficiencies (PI/PID), also referred to as inborn errors of immunity (IEIs), are genetic disorders caused by inherited mutations that directly affect the immune system. There are more than 500 different genetic causes of PID that render children and adults more susceptible to infections. PIDs are usually present at birth, but commonly and unfortunately for patients, are not diagnosed until much later. PID cases often do not follow a typical pattern; instead, these cases are usually complex and challenging to diagnose due to high individual variability. It is estimated that over 70% of patients with PID remain undiagnosed. In patients where PID manifests in late childhood or adolescence, there is often a significant lag time between the onset of symptoms and diagnosis, often with increased morbidity following chronic infections. Early diagnosis and adequate implementation of appropriate treatment prevent the occurrence of unnecessary comorbidities and infections, enhancing the quality of life for patients while simultaneously reducing the overall cost burden of care. Guidelines offer recommendations to guide Ig therapy relative to dose, frequency, and routes of administration, but ultimately therapeutic success with Ig replacement therapy requires a personalized approach, formulated, and modified as needed considerate each patient’s characteristics, response to therapy and individualized treatment goals. Healthcare providers must employ personalized clinical strategies to improve PID care.
Upon completion of this activity, learners will be able to:
• Outline the most common PID manifestations in pediatric and adult patients
• Implement steps to improve screening and diagnostic practices for PID to decrease diagnostic delays
• Evaluate clinical benefits and limitations of intravenous and subcutaneous Ig replacement therapies to optimize treatment selection
• Discuss the role of SDM in Ig therapy selection to enhance patient care and treatment satisfaction
• Outline the most common PID manifestations in pediatric and adult patients
• Implement steps to improve screening and diagnostic practices for PID to decrease diagnostic delays
• Evaluate clinical benefits and limitations of intravenous and subcutaneous Ig replacement therapies to optimize treatment selection
• Discuss the role of SDM in Ig therapy selection to enhance patient care and treatment satisfaction
Provided by the Academy for Continued Healthcare Learning (ACHL).
Supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.
Richard L. Wasserman, MD, PhD (Chair)
Medical Director of Pediatric Allergy and Immunology
Medical City Children’s Hospital
Dallas, TX
Medical Director of Pediatric Allergy and Immunology
Medical City Children’s Hospital
Dallas, TX
Kenneth Paris, MD (Faculty)
Professor of Pediatrics
LSU Health Sciences Center New Orleans
Division Head and Clinical Service Line Chief
Manning Family Children’s
New Orleans, LA
Niraj Patel, MD, MS (Faculty)
Associate Professor of Pediatrics
Division of Allergy and Immunology
Duke University
Durham, NC
The Academy for Continued Healthcare Learning (ACHL) requires that the faculty participating in an accredited continuing education activity disclose all affiliations or other financial relationships within 24 months (1) with the manufacturers of any commercial product(s) and/or provider(s) of commercial services discussed in an educational presentation and (2) with all ineligible companies. All relevant financial relationships have been mitigated prior to this activity.
The following financial relationships have been provided:
Richard L. Wasserman, MD, PhD (Chair)
Consulting Agreements: Evolve Biologicals, GC Pharma, Grifols, Pfizer
Speakers' Bureau: CSL Behring, Grifols
Kenneth Paris, MD (Faculty)
Advisor: Biocryst, CSL Behring, Takeda
Clinical Trials Researcher: Takeda
Speakers’ Bureau: Takeda
Niraj Patel, MD, MS (Faculty)
Consulting Agreements: Takeda
Data Safety Monitoring Board: Janssen
Grants/Research Support: Takeda
Speakers' Bureau: Amgen, Pharming, Takeda, X4 Pharmaceuticals
ACHL staff members and others involved with the planning, development, and review of the content for this activity have no relevant affiliations or financial relationships to disclose.
The content for this activity was developed independently of any ineligible company. All materials are included with permission. The opinions expressed are those of the faculty and are not to be construed as those of the publisher or grantor(s).
This educational activity was planned and produced in accordance with the ACCME Standards for Integrity and Independence in Accredited Continuing Education. Recommendations involving clinical medicine in a continuing medical education (CME/CE) activity must be based on evidence that is accepted within the profession of medicine as adequate justification for their indications and contraindications in the care of patients. All scientific research referred to, reported, or used in CME/CE in support or justification of a patient care recommendation must conform to the generally accepted standards of experimental design, data collection, and analysis.
This CME/CE activity might describe the off-label, investigational, or experimental use of medications and/or devices that may exceed their FDA-approved labeling. Physicians should consult the current manufacturers’ prescribing information for these products. ACHL requires the speaker to disclose that a product is not labeled for the use under discussion.
Discussion of scientific information on unapproved uses (SIUU), off-label, investigational, or experimental drug/device use: None
This activity will take approximately 60 minutes to complete. To receive credit, learners are required to complete the pretest, view the online activity, and complete the posttest and evaluation. To receive credit, 80% must be achieved on the posttest. A certificate will be immediately available. There is no fee to participate in the activity or for the generation of the certificate.
The Academy for Continued Healthcare Learning is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
The Academy for Continued Healthcare Learning designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Physician assistants, nurse practitioners, and nurses may participate in this educational activity and earn a certificate of completion as AAPA, AANP, and ANCC accept AMA PRA Category 1 Credits™ through their reciprocity agreements.
