Dupilumab for Eosinophilic Esophagitis: Characterizing Symptoms and Clinical Outcomes in a Real-World Pediatric Population

1 Patient Characteristics

Characteristic	N	N = 117¹
Age at first fill	117
Mean (SD)		12.4 (3.8)
Median (IQR)		13.0 (9.5, 15.7)
Range		3.6, 18.7
Sex	117
Male		90 (76.9%)
Female		27 (23.1%)
Race	117
White		95 (81.2%)
Black or African American		13 (11.1%)
Hispanic/Latino/a		5 (4.27%)
Middle Eastern or North African		2 (1.71%)
American Indian or Alaska Native		1 (0.85%)
None of these		1 (0.85%)
Ethnicity	117
Not Hispanic, Latino/a, or Spanish origin		81 (69.2%)
Other		16 (13.7%)
None of these		13 (11.1%)
Other Hispanic, Latino/a, or Spanish origin		6 (5.13%)
Decline to Answer/Prefer not to answer		1 (0.85%)
Pharmacy insurance	117
Commercial		56 (47.9%)
Medicaid		52 (44.4%)
Tricare		9 (7.69%)
Height-age-Z score at baseline as of clinic date	116
Mean (SD)		0.10 (1.25)
Median (IQR)		0.15 (-0.71, 0.93)
Range		-4.73, 3.10
Missing		1
Weight-age Z score at basline as of clinic date	117
Mean (SD)		0.33 (1.35)
Median (IQR)		0.24 (-0.64, 1.29)
Range		-3.30, 3.07
Does the patient have a diagnosis of autism?	117
No		100 (85.5%)
Yes		17 (14.5%)
Diagnosis of GERD at initiation	117
No		40 (34.2%)
Yes		77 (65.8%)
On a dietary therapy at initiation?	117
No		76 (65.0%)
Yes		41 (35.0%)
Time from date of diagnosis to start of therapy (years)	116
Mean (SD)		3.79 (3.35)
Median (IQR)		3.07 (1.09, 5.02)
Range		0.08, 16.17
Missing		1
Turnaround time (days)	117
Mean (SD)		12 (36)
Median (IQR)		5 (2, 10)
Range		0, 373
¹ n (%)

Characteristic	N = 117¹
Dysphagia outcome
Dysphagia	59 (50.4%)
No dysphagia and no compensatory mechanisms	50 (42.7%)
Compensatory mechanisms without dysphagia	8 (6.84%)
¹ n (%)

Histological findings patient had at baseline EGD	Total (N = 116)
Distal Esophagus	115 (99%)
Mid Esophagus	75 (65%)
Proximal Esophagus	44 (38%)

Characteristic	N	N = 117¹
Peak esophageal intraepithelial eosinophil count	116
<15		10 (8.62%)
>=15		106 (91.4%)
N/A		1
¹ n (%)

Endoscopic findings at baseline	Total (N = 101)
Furrows	83 (82%)
Edema	66 (65%)
Exudates	50 (50%)
Rings	15 (15%)
Unavailable	7 (7%)
Strictures	3 (3%)

Characteristic	N = 117¹
Number of dilations in 6 months prior to dupilumab initiation
0	109 (93.2%)
1	6 (5.13%)
2	1 (0.85%)
Unknown	1 (0.85%)
¹ n (%)

Concomitant medications at baseline	Total (N = 117)
PPI	82 (70%)
Swallowed topical corticosteroids	76 (65%)
None	13 (11%)
Dupilumab for other condition or EoE	6 (5%)

Reason no longer taking PPI (not taking at initiation)	Total (N = 29)
Not effective	15 (52%)
Nonahderence	6 (21%)
Unknown	5 (17%)
Affordability	1 (3%)
Adverse effects experienced	1 (3%)
Adverse effect concerns (not yet experienced)	1 (3%)

Reason no longer taking swallowed steroids (not taking at initiation)	Total (N = 27)
Not effective	8 (30%)
Nonahderence	7 (26%)
Adverse effects experienced	6 (22%)
Unknown	3 (11%)
De-escalated due to prior improvement	3 (11%)
Adverse effect concerns (not yet experienced)	1 (4%)

Reason for starting	Total (N = 117)
Failure of PPI and steroid	62 (53%)
Multiple Type 2 comorbidities	23 (20%)
Failure of previous PPI (only)	20 (17%)
Nonadherence to previous therapies	17 (15%)
Not able to taper off other therapies	15 (13%)
Side effects (experienced or concerns for)	12 (10%)
Other	9 (8%)
Failure of previous steroids (only)	8 (7%)
Not able to tolerate (spit out, vomit up, etc)	5 (4%)

Baseline Type 2 inflammatory conditions	Total (N = 117)
Allergic rhinitis	70 (60%)
Food allergies	59 (50%)
Asthma	58 (50%)
Atopic dermatitis	51 (44%)
None	23 (20%)

Reasons for 3rd party denials	Total (N = 40)
Below FDA-approved age-weight (appropriate)	18 (45%)
Frequency and/or severity of dysphagia	15 (38%)
Below FDA-approved age-weight (inappropriate)	6 (15%)
Have not failed topical steroids	5 (12%)
In remission on current therapies	3 (8%)
Have not tried elimination diet	1 (2%)
Have not failed PPI	1 (2%)
Exceeds quantity limit	1 (2%)

Table 2.3: Table 2.4: Cox Proportional Hazards Model Summary
	HR	95% CI- Lower	95% CI- Upper	P-value
Dysphagia	1.457	1.001	2.120	0.049
Government insurance	1.061	0.724	1.554	0.762
Weight z-score	1.028	0.878	1.204	0.728