A 5-year randomized trial on the safety and efficacy of pimecrolimus in atopic dermatitis: a critical appraisal.

AIM: The PETITE study (Sigurgeirsson et al.) aimed to compare safety and efficacy of pimecrolimus 1% cream (PIM) and low-to-medium-potency topical corticosteroids (TCS) in children with mild-to-moderate atopic dermatitis (AD).

SETTING AND DESIGN: Participants of this 5-year drug-company sponsored multicentre, open-label, parallel-group trial were recruited between April 2004 and October 2005. No details are reported regarding the study sites.

STUDY EXPOSURE: Infants aged ≥ 3 to < 12 months with mild-to-moderate AD were randomly assigned in a 1 : 1 ratio to receive either PIM or a low- or medium-potency TCS cream/ointment for 5 years. No information on specific TCS products used was provided. The topical treatment was applied twice daily 'until complete AD clearance or for as long as allowed by the label of the specific TCS', and was reinitiated at the occurrence of first signs and symptoms of AD flares. In the PIM group, exacerbations not controlled by PIM were treated with short-term TCSs.

OUTCOMES: Adverse events (AEs) and serious AEs (SAEs) were recorded 'during clinic visits'. In a proportion of the patients, various immunological assessments including antibody titres to common vaccine antigens, immunoglobulin levels, B and T lymphocyte cell counts, and T-cell proliferation tests were performed. The children's growth was assessed by measuring height and weight. AD severity was measured using the Investigator Global Assessment (IGA) score and the percentage of the total body surface area affected. No specific information was provided on the number and scheduling of study visits. Primary outcomes were the incidence of AEs 'of primary clinical interest' and those with a crude incidence of ≥ 5% in either treatment group. Secondary outcome was 'long-term efficacy' defined as IGA ≤ 1 at week 3 and year 5.

RESULTS: Patients in the PIM group experienced significantly more AEs [bronchitis (P = 0·02), infected eczema (P ≤ 0·001), impetigo (P = 0·045), nasopharyngitis (P = 0·04)]. No significant differences were seen for the other AEs. The overall incidence of SAEs was slightly higher for PIM (20·5% vs. 17·3%; P = 0·046). The proportion of participants with IGA ≤ 1 at year 5 was 88·7% for PIM and 92·3% for TCS, a success rate difference of 3·6% (95% confidence interval 0·8-6·4) favouring TCS.

CONCLUSIONS: Sigurgeirsson et al. conclude that the long-term management of mild-to-moderate AD in children with both TCS and PIM is safe, and that PIM has similar efficacy to TCS. Further, they conclude that their data support the use of PIM as a first-line treatment of mild-to-moderate AD in children.