Long-term Follow-up Study With Darvadstrocel in the Treatment of Complex Perianal Fistula

An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered an investigational medicinal product; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product.

TEAE is defined as: any adverse event emerging/manifesting at or after the initiation of treatment with a study intervention/medicinal product or any existing event that worsens in either intensity/frequency following exposure to the study intervention/medicinal product. Serious adverse event (SAE) is an untoward medical occurrence, significant hazard, contraindication, side effect/precaution that at any dose: results in death, is life-threatening, required in-patient hospitalization/prolongation of existing hospitalization, results in persistent/significant disability/incapacity, is a congenital anomaly/birth defect or is medically significant.

AESIs are AEs that are not solicited local or systemic AEs, they are predefined AEs that require close monitoring and prompt reporting to the sponsor. Protocol pre-specified AESIs included immunogenicity/allo-immunoreactions, tumorigenicity, ectopic tissue formation and fistula/abscess. In addition, ad hoc AESIs of anaphylactic reaction, hypersensitivity, and malignancy.

Clinical remission is defined as closure of all treated external fistula openings that were draining at baseline of ADMIRE-CD II despite gentle finger compression. Percentages were rounded off to the nearest second decimal place.

Clinical response is defined as closure of at least 50% of all treated external fistula openings that were draining at baseline of ADMIRE-CD II despite gentle finger compression. Percentages were rounded off to the nearest second decimal place.

Relapse is defined as participants who were in combined remission at Week 52 of ADMIRE-CD II and who have either reopening of any of the treated fistula(s) external openings with active drainage as clinically assessed or, the development of a perianal fluid collection >2 cm of the treated perianal fistulas confirmed by centrally read magnetic resonance imaging (MRI) assessment. Combined remission at Week 52 was defined as clinically assessed closure of all treated external openings that were draining at baseline of ADMIRE-CD II, despite gentle finger compression, and absence of collection(s) >2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by blinded central MRI assessment. Percentages were rounded off to the nearest second decimal place.

Combined remission of complex perianal fistula(s) is defined as the clinical assessment of closure of all treated external openings that were draining at baseline of ADMIRE-CD II, despite gentle finger compression, and absence of collection(s) >2 cm (in at least 2 dimensions) of the treated perianal fistula(s) confirmed by centrally read blinded MRI assessment. Percentages were rounded off to the nearest second decimal place.

Percentages were rounded off to the nearest second decimal place.

The PDAI is a scoring system to evaluate the severity of perianal CD. From the 5-item instrument, only 'discharge' was used for this outcome measure. Each category is graded on a 5-point Likert scale ranging from no symptoms (score of 0) to severe symptoms (score of 4); a higher score indicates more severe disease.

The PDAI is a scoring system to evaluate the severity of perianal CD. From the 5-item instrument, only 'pain' was used for this outcome measure. Each category is graded on a 5-point Likert scale ranging from no symptoms (score of 0) to severe symptoms (score of 4); a higher score indicates more severe disease.