Inborn Errors Working Party Live Forum Sessions on IEI

IEIs are rare and ultra-rare conditions, many of which can be cured through HCT. However, because of their rarity, individual centres often encounter only a small number of cases over many years. As a result, the sharing of collective experience, expert opinion, and practical insight is essential for improving patient care and advancing the field.

These live sessions provide a structured and interactive forum in which clinicians can present challenging cases and engage in focused, expert-led discussion on HCT indication, timing, conditioning strategies, donor selection, and other procedural considerations. The series also serves as an ongoing educational resource for the IEI community, fostering shared learning and collaboration across centres and countries.

All advice provided during the forum is non-binding, and responsibility for final clinical decisions remains with the local treating team. Presenting physicians must ensure that all patient information is fully anonymised and that appropriate patient and/or caregiver consent has been obtained and documented.

Together, these sessions aim to strengthen collective expertise, support clinicians managing rare and complex cases, and ultimately improve outcomes for patients with IEIs.

After participating in these live sessions, participants will be able to:
 

• Critically evaluate the indications and optimal timing for HCT in patients with inborn errors of immunity (IEI), including rare and ultra-rare conditions, by integrating disease biology, clinical presentation, and available evidence with insights from expert consensus discussions.
• Apply shared expert experience to complex, real-world clinical scenarios, improving decision-making around HSCT strategies (including procedural considerations), while recognising the non-binding, advisory nature of the forum and the responsibility of local treating teams for final clinical decisions.
• Strengthen multidisciplinary and international collaboration within the IEI community, fostering continuous professional development through case-based learning, peer discussion, and the engagement of both senior experts and junior clinicians, in line with EBMT educational principles.

Submissions will open on 30 March and remain open until the conclusion of the series.

• Case Submission
  • Applications must be submitted via the designated submission form.
  • Upon clicking the “Submit your Case” button, applicants will be required to upload (all documents in pdf format):
    • The clinical case
    • A motivation letter (maximum 350 words)
    • A concise CV
• Case Selection
  • In accordance with the schedule, two cases from the IEWP will be selected at least 14 days prior to each session.
• Notification and Confirmation
  • Selected case presenters will be notified on the same day the selection is made.
  • They will receive an invitation to confirm their participation.
  • Confirmation will require:
    • Providing any additional requested information
    • Accepting the key dates
• Presentation Format
  • Each case will be presented during the live forum session.
  • The presentation will last approximately 10 minutes.
  • This will be followed by a 20-minute discussion.
  • An IEWP-appointed moderator will join the presenter and lead the discussion.

We are particularly interested in cases that meet the following criteria:

• Paediatric or young adult patients are preferred.
• Cases in which bone marrow transplantation (BMT) is being considered, or where it could reasonably be explored as a potential treatment option.

Case Submissions Open on 30 March 2026