CFDB - Cystic Fibrosis DataBase

primary studies published RCT

A double-blind comparison of RU-41740 ('Biostim') and placebo in recurrent upper respiratory tract infections in children with cystic fibrosis.

Study design (if review, criteria of inclusion for studies)

RCT

Participants

49 CF children aged 2 to 8 years were followed for 12 months. The RU-41740 patients (n = 25) were older, taller and more ill (lower Shwachmann score) than the control group (n = 24)

Interventions

RU-41740 ('Biostim') and placebo used against recurrent respiratory infections

Outcome measures

URT infections, infection time, duration of antibiotic treatment, clinical and laboratory tolerability

Main results

the RU-41740 group had significantly fewer URT infections (2.16) than the placebo group (3.63) over the 12 months (p = 0.05). The total infection time was shorter (17.12 vs 25.54 days, p = 0.12) and the duration of antibiotic treatment significantly shorter (12.28 vs 23.5 days, p = 0.03). The clinical (particularly gastrointestinal) and laboratory tolerability of RU-41740 were good. RU-41740 provided prophylactic treatment against URT infections in these cystic fibrosis patients in whom infection is a serious threat.

Authors' conclusions

Further investigations with this drug are warranted.

Keywords: Child; placebo; RU-41740 -Biostim-; Respiratory Tract Infections; Respiratory Tract Diseases; Infection; Immunoregulatory; pharmacological_intervention;