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(1)EACCME® participant's evaluation form
Changing the Course of Metabolic Disease: Advances in Bone Marrow Transplantation
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:
*
QUESTIONS
1) Which of the following statements best reflects the current role of hematopoietic stem cell transplantation (HSCT) in inherited metabolic diseases?
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A
HSCT is curative for all organ systems in most metabolic diseases.
B
HSCT is primarily effective in selected metabolic diseases where donor-derived cells can provide enzyme replacement.
C
HSCT has largely been replaced by gene therapy in clinical practice.
D
HSCT outcomes are independent of timing and patient age at transplantation.
2) What is a major advantage of newborn screening for metabolic diseases in the context of HSCT?
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A
It eliminates the need for conditioning therapy.
B
It allows treatment before significant disease progression has occured.
C
It ensures that all patients are eligible for gene therapy.
D
It removes variability in drug pharmacokinetics.
3) What is the rationale for model-based dosing of conditioning agents in HSCT for non-malignant diseases?
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A
Conditioning intensity is less relevant in non-malignant diseases, so precise dosing is not required.
B
Higher drug exposure is always preferred to ensure engraftment in non-malignant diseases.
C
Optimal drug exposure is required to balance engraftment and toxicity, as both under- and overexposure impact outcomes.
D
Model-based dosing is mainly relevant in malignant diseases where tumor control is needed.
4) What is the most appropriate approach to patient selection for HSCT or gene therapy in inherited metabolic diseases?
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A
Treatment should be offered to all diagnosed patients to prevent disease progression.
B
Patient or caregiver preference should be the primary determinant for proceeding with treatment.
C
HSCT and gene therapy should be considered only when the expected benefit outweighs the risk of treatment-related harm, including potential disease acceleration.
D
Gene therapy should be preferred over HSCT in all eligible patients due to lower toxicity.
5) What have long-term follow-up studies shown about the effects of HSCT in patients with metabolic diseases?
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A
HSCT improves survival and stabilizes disease, but residual disease may persist
B
HSCT fully normalizes disease processes in all tissues and organ systems.
C
HSCT leads to complete resolution of systemic inflammation and disease burden.
D
Long-term outcomes after HSCT are primarily determined by donor type rather than disease biology.
6) Quality of the event
How useful for your professional activity did you find this event?
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Not useful
Fairly useful
Useful
Extremely useful
If this activity was not useful, please explain why:
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What was your overall impression of this event in terms of the
programme
?
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Very poor
Poor
Good
Excellent
What was your overall impression of this event in terms of the
organisation
?
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Very poor
Poor
Good
Excellent
What was the
best
aspect of this event?
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What was the
worst
aspect of this event?
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7) Relevance of the event
Did the event fulfil your educational goals and expected learning outcomes?
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Not at all
Not much
Somewhat
Very much
Was the presented information well balanced and consistently supported by a valid scientific evidence base?
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Not at all
Not much
Somewhat
Very much
How useful to you personally was the session?
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Not useful
Fairly useful
Useful
Extremely useful
8) Suitability of formats used during the event
Was there adequate time available for discussions, questions & answers and learner engagement?
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Never
Only rarely
Sometimes
Always/Almost Always
Can you indicate any innovative elements during the activity?
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9) Ways the event affects clinical practice
Will the information your learnt be implemented in your practice?
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Not at all
Not much
Somewhat
Very much
Can you provide ONE example how this event will influence your future practice?
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10) Commercial bias
Did all the speakers provide their potential conflict of interest declaration with the sponsor(s) as a second slide of their presentations?
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No
Yes, but only a small part
Yes, for the majority
Yes, all
Can you provide an example of biased presentation in this activity?
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Do you agree that the information was overall free of commercial and other bias?
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Strongly disagree
Rather disagree
Rather agree
Strongly agree
Thank you very much for your time!
Submit