(5)EACCME® participant's evaluation form

Early HCT Following Newborn Screening for SCID: Results and Outcomes

Below you will find several questions about the contents and other aspects of the webinar. Once the webinar ends, you have up to 2 weeks to submit your answers and get ECMEC®s accreditation certificate.

Please enter your full name as it appears on the registration form:

1) According to the Catalan SCID NBS algorithm, does a TREC result below the critical threshold for urgent referral confirm a diagnosis of SCID?

Yes — a TREC below the critical threshold is itself diagnostic of SCID, and HCT can be planned without further testing

No — it triggers urgent referral to the SCID-CRU for confirmatory immunophenotyping and genetic testing, since SCID, non-SCID T-cell lymphopenia, and false-positive results can all present with very low TRECs

No — TREC values below the critical threshold are usually transient laboratory artefacts that resolve without referral

Yes, but only if confirmed on a second dried blood spot sample

2) What was the overall survival (OS) across all treatment modalities (HCT, gene therapy, thymus transplantation) in the Catalan cohort?

3) What is the current evidence regarding kidneys from HBcAb-positive donors?

Genetic diagnosis (genotype)

Donor type (MSD vs UCB vs haploidentical)

Absence of active infection at time of HCT

Conditioning regimen intensity

4) Why is establishing a precise genetic diagnosis essential before deciding on definitive treatment in an infant identified by NBS with severe T-cell lymphopenia?

To confirm eligibility for umbilical cord blood as a donor source

It determines whether the defect is intrinsic to haematopoietic stem cells (treatable with HCT or gene therapy) or due to a primary thymic defect such as congenital athymia (e.g., FOXN1, FOXI3), which requires thymus transplantation instead of HCT

It is used only to estimate the risk of post-HCT viral reactivation

All genotypes identified through NBS follow an identical treatment algorithm regardless of diagnosis

5) In 2024, how many European countries had a national or regional TREC-based NBS programme for SCID, according to Blom et al. (JACI 2025)?

6) Quality of the event

How useful for your professional activity did you find this event?

If this activity was not useful, please explain why:

What was your overall impression of this event in terms of the programme?

What was your overall impression of this event in terms of the organisation?

What was the best aspect of this event?

What was the worst aspect of this event?

7) Relevance of the event

Did the event fulfil your educational goals and expected learning outcomes?

Was the presented information well balanced and consistently supported by a valid scientific evidence base?

How useful to you personally was the session?

8) Suitability of formats used during the event

Was there adequate time available for discussions, questions & answers and learner engagement?

Always/Almost Always

Can you indicate any innovative elements during the activity?

9) Ways the event affects clinical practice

Will the information your learnt be implemented in your practice?

Can you provide ONE example how this event will influence your future practice?

Did all the speakers provide their potential conflict of interest declaration with the sponsor(s) as a second slide of their presentations?

Yes, but only a small part

Yes, for the majority

Can you provide an example of biased presentation in this activity?

Do you agree that the information was overall free of commercial and other bias?

Thank you very much for your time!