Efficacy and safety of granulocyte, monocyte/macrophage adsorptive in pediatric ulcerative colitis

Table 1 ADAPT trial key inclusion criteria

Children and adolescents < 18 yr and with a body weight ≥ 30 kg

Ulcerative colitis documented by clinical symptoms, endoscopic findings and histology since at least 3 mo prior to inclusion

Moderate active ulcerative colitis at baseline, defined as a PUCAI score between 35 and 64

Pancolitis or left-sided colitis

Receiving or having received one or more of the following medicinal products before screening:

Sulfasalazine, mesalamine and other 5-aminosalicylic acid agents for 4 wk or more with a stable dose for the last 2 wk

0.5 mg/kg per body weight with a maximum of 20 mg per day of prednisone with a stable dose for the last 2 wk, or

6-mercaptopurine or azathioprine for 12 wk or more with a stable dose for the last 4 wk

PUCAI: Pediatric ulcerative colitis activity index.

Table 2 ADAPT schedule of assessments

Visit	01	02	03	04	05	06	07	08	09	10
Day	-07	00	07	14	21	28
Week	-1	0	1	2	3	4	6	7	8	12
Apheresis		▲	▲	▲	▲	▲	(▲)	(▲)	(▲)
Physical examination	●									●
Endoscopy/EAI	●4									(●)5
PUCAI	●	●	●	●	●	●	●	●	●	●
Clin. Chemistry	●	●		●		●	●2	●2	●2	●
ESR	●	●		●		●	●2	●2	●2	●
Urinalysis	●	●		●		●	●2	●2	●2	●
Fecal sample	●
Coagulation	●					●	●2	●2	●2	●
Vital signs	●	●	●	●	●	●	●1	●1	●1	●
Concomitant medication	●	●	●	●	●	●	●	●	●	●
Adverse events	●3	●	●	●	●	●	●	●	●	●

¹Only for patients with treatment;

²Only for patients with last treatment;

³Only if an endoscopy was performed at post screening;

⁴The endoscopy should be done in the period between six weeks prior to screening and 5 d post screening visit;

⁵The timeframe for the optional endoscopy is 7 d prior and 7 d after Visit 10. PUCAI: Pediatric ulcerative colitis activity index.

Table 3 Categories of pediatric ulcerative colitis activity index changes at week 12

Significant improvement	≥ 35
Moderate improvement	≥ 20
Small improvement	≥ 10
No change	< 10

Table 4 Patient demography

Characteristic	mean ± SD [range]
Age (yr)	13.5 ± 2.6 [8.1-17.8]
Weight (kg)	47.7 ± 11.3 [31.0-72.2]
Height (cm)	157.3 ± 12.3 [132.0-175.0]
Duration of disease	3.1 ± 3.2 [0.2-14]
Smoking status	No patient ever smoked
Male	n = 13	52.0%
Female	n = 12	48.0%
Caucasian	n = 22	88.0%
Oriental (near East)	n = 2	8.0%
Other	n = 1	4.0%
Pancolitis	n = 18	72%
Left sided	n = 7	28%
Entry PUCAI score	mean = 42.6	median = 40
Entry EAI score (median)	mean = 7.0	median = 7.5

Table 5 Improvement in PUCAI at week 12, patients with post-baseline scores

Analysis set	n	mean	SD	95%CI
ITT	20	22.3	19.9	12.9-31.6
PP	8	36.3	5.8	31.4-41.1

ITT: Intention-to-treat; PP: Per-protocol.

Table 6 Pediatric ulcerative colitis activity index category improvement, week 12 - intention-to-treat set

Category	n	%
Significant improvement	9	45.0
Moderate improvement	5	25.0
Small improvement	1	5.0
No change	5	25.0

Table 7 Changes in endoscopy activity index

Analysis set	mean	SD	95%CI	n
ITT	-3.0	2.5	-4.8-(-1.2)	10
PP	-2.8	2.5	-5.4-(-0.2)	6

ITT: Intention-to-treat; PP: Per-protocol.

Table 8 Adverse events, n

Relation	Severity	AEs;
		patients
		(safety set)
None	Mild	35; 12 (48%)
	Moderate	7; 4 (16%)
	Severe	0; 0 (0%)
	Total	42; 13 (52%)
Possibly or definitely related	Mild	15; 6 (24%)
	Moderate	6; 5 (20%)
	Severe	0; 0 (0%)
	Total	21; 8 (32%)