Sponsored Programme

Wednesday, 27 April 2022

20:30 – 21:30

Pharming Symposium (not part of CME programme)

Title: “Diagnosis, management, and recent advances in the treatment of Activated phosphoinositide 3-kinase δ syndrome (APDS), a rare genetic primary immunodeficiency disorder”

Rationale: Activated phosphoinositide 3-kinase δ syndrome (APDS) is a rare, combined immune deficiency (CID), which presents clinicians with many diagnostic and management challenges. It has few signs and symptoms that clearly differentiate it from other CIDs and current management options are often aimed at treating symptoms as they present. In this symposium we will discuss the advances made in understanding this condition through the ESID APDS registry and the diagnostic and treatment challenges of APDS as experienced by an expert in the field. Following this we present the latest PhIII clinical data on leniolisib, a selective oral PI3Kδ inhibitor in development for APDS treatment. This symposium will be of interest to all those working in the field of primary immune deficiencies.

Chair: Prof Stephan Ehl, MD, Ph.D – Institute for Immunodeficiency, Freiburg

  • Challenges with diagnosis and management of APDS – Dr Anita Chandra, MD, Ph.D – Cambridge University Hospitals/University of Cambridge
  • Leniolisib: safety and efficacy results from Phase 3 clinical trial – Prof Anna Sediva, MD, Ph.D – Charles University, Prague

Thursday, 28 April 2022

07:30 – 8:30

X4 Pharmaceuticals Symposium (not part of CME programme)

Title: “Natural History and Diagnostic Challenges of WHIM Syndrome”

Rationale: An educational symposium discussing WHIM (Warts, Hypogammaglobulinemia, Infections, Myelokathexis) syndrome, a primary immunodeficiency/congenital neutropenia with a heterogenous presentation and potential long-term consequences. Learn about the natural history of the disease and new approaches that can aid in early and accurate diagnosis.

Symposia faculty:

  • Dr. Nizar Mahlaoui, MD, MPH, PhD; Pediatric Immuno-Hematology & Rheumatology, Necker-Enfants University Hospital, Paris, France
  • Dr. Raffaele Badolato, MD, PhD; Professor of Pediatrics, ASST Spedali Civili di Brescia, and Chairman Post-graduate School of Pediatrics, University di Brescia, Brescia, Italy

12:00 – 13:00

CSL Behring Company Symposium (not part of CME programme)

Title: “Improving primary immunodeficiency care: Following patient journeys from diagnosis to treatment”

Rationale: Primary immunodeficiency diseases (PID) are 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.

  • Welcome and PID Introduction: A group of disorders more common than you think – Stephen Jolles, MD, PhD, FRCP, FRCPath
  • Strategies for improving diagnosis in PID – Antonio Condino-Neto, MD, PhD, FAAAAI
  • Therapeutic management of PID and HRQoL – Susana Lopes da Silva, MD, PhD
  • PID patient journeys: Making it personal – all
  • Q&A – all

17:30 – 18:30

Takeda Symposium (not part of CME programme)

Title: “A synergistic vision: The importance of a collaborative multi-disciplinary approach when diagnosing and managing primary immunodeficiency”

Rationale: The journey of a person with Primary Immunodeficiency to diagnosis and beyond can be long and turbulent. Come join our expert faculty to learn more about different collaborative multi-disciplinary approaches across both local and regional levels that can continue our synergistic vision to improving the diagnosis and management of people living with primary immunodeficiency.

Moderator: Andrew Cant, Newcastle University, UK

Speakers:

  • Annick van de Ven, University Medical Center Groningen, The Netherlands
  • Jenny Coles, Royal Free London NHS Foundation Trust, UK
  • John Hurst, Royal Free London NHS Foundation Trust, UK
  • Joseph Jacob, University College London, UK
  • Kathryn Poad, The Newcastle upon Tyne NHS Hospitals Foundation Trust, UK
  • Klaus Warnatz, Center for Chronic Immunodeficiency, Medical Center – University of Freiburg, Germany
  • Mary Slatter, The Newcastle upon Tyne NHS Hospitals Foundation Trust, UK

Friday, 29 April 2022

08:00 – 09:00

Kedrion Symposium (not part of CME programme)

Title:“PID patients and scientific community medical need”

Rationale: Update the clinical and laboratory features of selected recently described PIDs associated with immune dysregulation and novel precision medicine strategies aimed to control the disease burden in affected patients. Provide clinical and immunological features able to unmask a PID in a SID. Present the results of a Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety and Pharmacokinetics of a new Intravenous Immunoglobulin 10% (KIg10) in Primary Immunodeficiency Disease (PID) Patients (Adults).

Moderator: Dr. Susana Lopes da Silva / Dr. Laura Marques

  • Immunodeficiency and disorders of immune dysregulation: new acquisitions – Ottavia Delmonte, US
  • When to suspect a PID in a SID – Viviana Moschese, IT
  • Kedrion’s commitment to the availability of new IVIgs for patients and the scientific community: KIg10 data – Richard L. Wasserman, US

13:45 – 14:45

Orchard Therapeutics Symposium (not part of CME programme)

Title: “Understanding the impact and unmet needs in Wiskott-Aldrich syndrome”

Rationale: An expert faculty will guide the audience through this symposium, which focusses on understanding the impact of disease and unmet needs in people affected by Wiskott‒Aldrich syndrome. The symposium will begin with an introduction to WAS, including an overview of the mechanism of disease, and its diagnosis and management. The burden of disease on patients and their families will then be discussed, based on the results of a caregiver-reported survey. Finally, the considerations and challenges associated with the current standard of care for WAS will be discussed from a nursing perspective. The audience will have the opportunity to ask questions to our expert faculty after the presentations.

  • An overview of Wiskott-Aldrich syndrome (WAS): from mechanism of disease to diagnosis and management – Sabina Cenciarelli (Italy)
  • The voice of the caregiver: results of a WAS caregiver survey – Susan Walsh (UK)
  • Supporting patients and families through the HSCT process: a nurse’s perspective – Federico Fraschetta (Italy)
  • Panel discussion – all speakers