A recent study (W Faubion et al. Inflamm Bowel Dis 2017; 23: 453-60) reports on the long-term safety/effectiveness of Adalimumab in pediatric patients entering the IMAgINE 2 trial (& who completed the 52 week IMAgINE 1 trial).
Patients with a PCDAI <10 were considered to be in remission and those who had a drop in PCDAI of 15 or more were considered to have had a treatment response.
- Of the 100 patients enrolled in IMAgINE 2, 41% achieved remission and 48% had a treatment response at week 240.
- >80% of patients were “able to discontinue use of corticosteroids.”
- Adalimumab treatment was associated with growth normalization.
- No new safety signals were identified.
While this study provides some reassurance regarding long-term adalimumab use, it should be noted that the instruments used to assess efficacy in this trial (& many others) are suboptimal.
A recent study (D Turner et al. JPGN 2017; 64: 254-60) showed that PCDAI (and several similar versions) had “poor correlation with calprotectin” and none of the PCDAI versions “can give a valid assessment of mucosal healing.” This study had used prospectively collected data from the ImageKids study of 100 children with Crohn’s disease. For the weighted PCDAI, the “best cut-off to identify endoscopic mucosal healing was <12.5 points” with a sensitivity of 58% and specificity of 84%.\
History: (recall 1 week):
- Abdominal Pain 0=None, 10=Mild (does not interfere with activities, brief), 20=Moderate/Severe
- Patient functioning 0=No limitations, 10=Occn difficulty with activities (below par), 20=frequent limitations
- Stools per day 0=0-1 liquid stools, no blood, 7.5=up to 2 semiformed stools with blood or 2-5 liquid nonblood, 15=Gross bleeding or ≥6 liquid stools or nocturnal diarrhea
- ESR 0 points if <20, 7.5 points if 20-50, 15 points if >50
- Albumin 0 points if ≥3.5 g/dL, 10 points if 3.1-3.4 g/dL, and 20 points if ≤3.0 g/dL
- Weight 0= Weight gain or stable or voluntary weight loss, 5=involuntary weight loss 1-9% or involuntary weight stable, 10= weight loss ≥10%
- Perirectal Disease 0=None or asymptomatic tags, 7.5= 1-2 indolent fistula, scant drainage, no tenderness, 15=active fistula, drainage, tenderness or abscess
Extraintestinal Manifestatons: Fever for 3 days (≥38.5), definite arthritis, uveitis, erythema nodosum, or pyoderma gangrenosum
- Points: 0=None, 10 ≥1
Total Score 0-125: ______________________
As compared with PCDAI, the weighted PCDAI drops height velocity, abdominal examination, and hematocrit. Turner et al note “their exclusion does not mean that they have no role in reflecting disease activity, but that the other included items, as a whole, are inclusive of the contribution of the 3 items.” Also, the weighted PCDAI simplifies the “extraintestinal manifestation” into a simple choice; overall, this affects few scores due to the low frequency of these manifestations.
Related blog posts:
- Adalimumab for children with Crohn’s disease | gutsandgrowth
- More on Adalimumab (Humira) in Pediatrics | gutsandgrowth
- What We Know Now: Therapeutic Drug Monitoring for …
- More NASPGHAN Meeting Notes: IBD Hot Topics | gutsandgrowth