20.30-21.30 | X4 Pharmaceuticals Symposium

Title: “Understanding Chronic Neutropenic Disorders Including WHIM Syndrome: 2023 and Beyond”

Scientific Rationale: WHIM syndrome is a rare immunodeficiency disease with a great unmet need. The presentation will aim to:

– Improve disease awareness of chronic neutropenic disorders, including WHIM syndrome.
– Present the most recent literature about these disease states.
– Highlight the experience of people with chronic neutropenic disorders

Chair: Raffaele Badolato, MD, PhD

  • Overview of WHIM Syndrome – Raffaele Badolato, MD, Full Professor of Pediatrics; Chair of Pediatrics, ASST Spedali civili di Brescia; Chairman Post-graduate School of Pediatrics, University of Brescia
  • Incidence of Serious Infections in People with Chronic Neutropenic Disorders – Kylle Tollefsen, PhD Senior Medical Director, X4 Pharmaceuticals
  • Patient Experience of Neutropenia – Anna Ellis, Associate Director, Patient Affairs & Advocacy, X4 Pharmaceuticals

07.30-08.30 | CSL Behring Symposium

Title: Personalisation In Disease: diagnosing, managing and empowering patients with primary immunodeficiency.

Scientific Rationale: Primary immunodeficiency (PID) is a group of heterogeneous disorders of the immune system that, if not properly diagnosed and treated, result in chronic, serious and often life threatening infections. This symposium will share the perspectives of PID of a clinician, nurse and patient, with the aim to increase awareness of the overlap between primary and secondary immunodeficiency, facilitate the scientific exchange regarding the evolving PID landscape, and also discuss managing and personalising immunoglobulin replacement therapy in PID

Chair: Martin van Hagen

  • Lecture 1: “Diagnosing immunodeficiency diseases with impaired antibody production: a clinician’s perspective” –  Prof. Silvia Sánchez-Ramon
  • Lecture 2: “Personalising the management of primary immunodeficiency with impaired antibody production using immunoglobulin pre-filled syringes: a nurse’s perspective” – Dr. Emily Carne
  • Lecture 3: “Personalising immunoglobulin administration: a patient’s perspective” – Ayesha Ilahi

12.00-13-00 | Sumitomo Pharma Inc Symposium

Title: Newborn Screening, Diagnosis and Care of Patients with Congenital Athymia

Scientific Rationale: Universal newborn screening has not been implemented worldwide and new genetic mutations leading to congenital athymia are being uncovered. Raising awareness of difficult to diagnose patients will ultimately aid in identifying best practices for diagnosis. Given the ultra-rare nature of congenital athymia, a need has been identified to answer standard of care questions from those who have clinical experience before implantation. 

  • Speakers: Vivian Hernandez-Trujillo, MD and Keith Sacco, MD

13.00-14.00 | Symposium organised by PeerVoice. Supported by an educational grant from Takeda.

Title: Patients Receiving Immunoglobulin Therapy – Emerging Technology for Patient-Integrated Care

Scientific Rationale: Overall Educational Goal: Clinicians are aware of device and digital technologies in development to support patient-integrated care in those requiring Ig therapy, and how these may be implemented in the future.

  • Lecture 1: Technology in the World of Disease Management – Nicholas L. Rider, DO
  • Lecture 2: Maximising The Value of Integrated Health Technology for Chronic Disease Management: What Have We Learnt So Far? – Prof. Martin van Hagen, MD, PhD; Nicholas L. Rider, DO; Dorothea Grosse-Kreul RN, BSc (Hon)
  • Lecture 3: Achieving Optimised Patient-Integrated Care: The Specific Challenges of Immunoglobulin Therapy – Prof. Martin van Hagen, MD, PhD; Nicholas L. Rider, DO; Dorothea Grosse-Kreul RN, BSc (Hon)
  • What Smart Moves Are Being Made for Simplifying Home Management of Immunoglobulin Therapy? – Prof. Martin van Hagen, MD, PhD; Nicholas L. Rider, DO; Dorothea Grosse-Kreul RN, BSc (Hon)

Session accredited by EBAC.
Organised by PeerVoice. Supported by an educational grant from Takeda Development Center Americas, Inc.

17.30-18.30 | Pharming Symposium

Title: Disease awareness symposium: an update on activated PI3K delta syndrome (APDS)

Scientific Rationale: To understand the patient burden of living with IEIs and APDS. To examine the clinical manifestations and burden of activated PI3K delta syndrome (APDS), using data from the ESID APDS registry

Chair: Dr V. Koneti Rao, MD, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.

  • Welcome and introductions

Chair: Dr V. Koneti Rao

  • Patient burden of living with IEIs/APDS – Dr Virgil A.S.H. Dalm, MD
  • APDS – clinical updates from the ESID registry – Dr Anita Chandra
  • Q&A – All
  • Summary and close – Chair: Dr V. Koneti Rao

08.00-09.00 | Grifols Symposium

Title: New tools for Primary Immunodeficiency diagnosis: from awareness to artificial intelligence

Scientific Rationale: Primary immunodeficiencies (PID) encompass a group of rare heterogeneous diseases with a wide range of symptoms. Clinical presentation includes infectious and autoimmune symptoms and higher risk of malignancy. Because of the rarity and the broad spectrum of signs and symptoms, diagnostic delays up to a median of 12.5 years, often due to lack of awareness. It is estimated that approximately 70% of PID patients remain undiagnosed. Early diagnosis and proper treatment are key to increase survival and overall quality of life of patients, which limits the high economic costs to the healthcare systems. Thus, there is an urgent need to raise PID awareness among different medical specialties and to gather better tools to identify PID patients. This symposium will review existing and future tools for PID identification.

  • Introduction and ASPIRE Award Ceremony
    Chair: Siobhán Burns, MD. PhD University College London (UK)
  • Lecture 1: New tools for primary immunodeficiency diagnosis – Pere Soler-Palacín, MD. PhD. Vall d’Hebron University Hospital, Barcelona (Spain)
  • Lecture 2: Artificial intelligence solutions for detecting primary immunodeficiencies – Nicholas Rider, DO. Liberty University, Lynchburg, VA (USA)
  • Q&A: all

12.30-13.30 | AstraZeneca Symposium

Title: Optimising COVID-19 prevention for vulnerable patients: leaving no patient behind

Scientific Rationale: While much of the world is rapidly moving beyond the COVID-19 pandemic, millions of people across the world remain at serious risk from complications of COVID-19. Vaccines and other measures are effective in preventing the most serious outcomes of COVID-19, however, they may not provide enough protection for those who are immunocompromised and at high-risk of severe illness.  Approximately 2% of the overall population are considered immunocompromised and are vulnerable to the most serious outcomes from COVID-19, including hospitalisation and death. An even broader population of people are considered ‘high-risk’ of complications from COVID-19 due to underlying health conditions or older age. These populations remain disproportionately affected by COVID-19. Studies have shown 40-44% of fully vaccinated hospitalized patients in the US were immunocompromised. Even when vaccinated, immunocompromised individuals are more likely to die in hospital, more likely to end up in ICU9 and more likely to need non-invasive ventilator support. Astrazeneca has pioneered novel approaches to develop highly targeted, long-lasting antibodies using our proprietary half-life extension technology, aiming to provide protection for those unable to mount their own immune response. This symposium aims to highlight the unmet need for additional protection in vulnerable populations.

Chair: Prof Alex Richter

  • Lecture 1: Are vaccines the solution for everyone? – Dr Virgil Dalm
  • Lecture 2: Stratifying risk for COVID-19 – Prof Alex Richter
  • Lecture 3: Monoclonal antibody use: Clinical and Real-World data – Prof David Strain

13.45-14.45 | Octapharma Symposium

Title: Clinical cases: Uncovering atypical manifestations of PID

Scientific Rationale: Patients with primary immune deficiency (PID) are susceptible to develop infections but in a significant percentage of cases autoimmunity, atopy or lymphoproliferation are the first signs of the immune dysregulation. The Octapharma symposium will consist of an interactive discussion of such complex PID cases, with atypical clinical manifestations beyond infections, between the invited experts and the audience.

Chair: Prof. Elie Haddad, Sainte-Justine University Hospital, Montreal, Canada

  • Welcome and Introduction – Prof. Elie Haddad, Sainte-Justine University Hospital, Montreal, Canada
  • Case 1: Unmasking PID in a SID patient with haematological malignancy – Prof. Stephen Jolles, University Hospital of Wales, Cardiff, UK
  • Case 2: A surprising diagnosis – Prof. Elie Haddad, Sainte-Justine University Hospital, Montreal, Canada
  • Case 3: Could neuropsychiatric disorders PANS/PANDAS be associated with PID? – Prof. Michael Daines, University of Arizona, Tucson, USA
  • Closing Remarks – Prof. Elie Haddad, Sainte-Justine University Hospital, Montreal, Canada