Studies investigating omalizumab in allergic bronchopulmonary aspergillosis (ABPA) treatment in patients with cystic fibrosis (CF)

Author [ref.]Type of studyPatients nSex (M:F)Age (years)CF (yes/no)ABPA (yes/no)Reason for omalizumab administrationObjectivesOmalizumab dosageTreatment durationFollow-upResultsBenefit (yes/no)
van der Ent [20]Case report10:112YesYesCSS dependence
CSS side-effects
ABPA exacerbations
NA300 mg every 2 weeks6 weeksNoSymptoms resolution
CSS suspension
↑ FEV1 (transient)
= total IgE
Nové-Josserand [19]Case series321:211–59YesYesCSS dependence or CSS side-effectsBMI, FEV1, FVC, total IgE levels, concomitant treatment for ABPA450 mg (150–1200) every 2, 3 or 4 weeksMedian 18 months (3–50)No↓ (50%) or stop (27.5%) in CSS use
No change in FEV1, but wide dispersion of values
9% stopped omalizumab after 20–24 months without exacerbations
22% stopped omalizumab because not effective
12.5% mild AEs
Zirbes [21]Case series33:012.8–17YesYesCSS dependence
ABPA exacerbations
CSS side-effects
No response to itraconazole
NA300–375 mg s.c. every 2 weeks8–18 monthsNo↓ exacerbation rate
↓ hospitalisation
Oral CSS suspension
=/↑ FEV1 (−4–+19%)
Randhawa [22]Case report11:014YesYesCSS dependence
CSS side-effects (hyperglycaemia)
ABPA exacerbations
NA375 mg s.c.
every 2 weeks
6 months11 monthsCSS suspension
Radiological resolution of pulmonary infiltrates
↑ FEV1
Elmallah [23]Case report22:012–16YesYesCSS dependence
ABPA exacerbations
NA450 mg s.c. every 2 or 4 weeks12–18 monthsNo↓ symptoms
↓ exacerbation rate
↓ oral CSS use
↑ FEV1 (+22%)
↓ free IgE (−88– −96%)
Wong [24]Case report22:014–15YesYesCSS dependence
CSS side-effects
ABPA exacerbations
NA300 mg s.c. every 4 weeks24–32 monthsNo↓ and stop oral CSS use
ABPA exacerbation when omalizumab administrated every 6 weeks in patient 1
= FEV1
↓ free IgE
Lehmann [25]Case series61:25–33,
15.6 (±7.1)
YesYesCSS side-effects or standard treatment failureNADosage individually adapted to body weight and IgE level at the beginning up to 600 mg every 2 weeks19.6 months (±19.1); 2 patients had a 2nd treatment course 10 and 25 months after discontinuation7–56 monthsNo ABPA relapses during treatment
ABPA relapses after treatment (2 patients) (33%)
↓ or stop CSS use
↑ FEV1 (+2.3%)
Zicari [26]Case report10:113YesYesCSS side-effects (IGT)NA300 mg every 2 weeks, calculated on the weight and total IgE level12 monthsNo↓ and stop oral CSS use
↓ antibiotic use
↑ FEV1 (+21%)
Delgado Pecellín [27]Case series31:214–29YesYesCSS dependenceNA300 mg every 4 weeks to 600 mg every 2 weeks5–18 monthsNo↓ and stop oral CSS use
No exacerbation
↑/= FEV1
Perisson [28]Case series187:1117.1±5.2YesYesTreatment-refractory ABPA, CSS dependence and CSS side-effects
Nontuberculous mycobacteria infection
Evaluation at T−3, T0, T3, T6, T12 of: FEV1, BMI, AEC, total IgE levels and A. fumigatus-specific antibody levels, side-effects489.7 mg (300–600) every 2 weeks12 monthsNo↓ exacerbation rate
↓ or stop CSS use (83%)
= FEV1 (p=0.6)
= total IgE and AEC
↑ BMI (p=0.01)
Ashkenazi [29]Case series retrospective study92:123±9YesYesCSS side-effects
Contraindication for CSS
Failure of antifungal treatment
Number of pulmonary exacerbations
CSS dosage
375 mg every 4 weeks13.9±8.6 monthsNoNo significant improvement of any outcome
↓ CSS use (4 patients) (44%)
3 (33%) patients with ↑ levels of IgE at the end of the treatment had better outcomes than the 6 (67%) patients with ↓ IgE post-treatment
Koutsokera [30]Retrospective observational study27 (11/27 with ABPA, 16/27 with asthma)4:526.9–42.7YesYes (11 ABPA, 16 asthma)Poor control despite 1st line treatment and/or contraindication or important secondary effects to CSS or antifungal treatment
Relapse of asthma or ABPA
CSS use
Days of hospitalisation
Intravenous antibiotics
Spirometry measures
150–750 mg every 4 weeks12 months0.1–9.9 yearsABPA group:
= FEV1
↓ FEV1 decline and variability
No significant change CSS dose (p>0.05) but 1/3 of patients ↓ CSS by 50%
Brinkmann [31]Case report10:115YesYesCSS dependence
CSS side-effects
(growth retardation)
NA300 mg every 4 weeks12 monthsNoInitial ↓ oral CSS use
CSS dependence
1 ABPA relapse
= FEV1
NCT00787917 [32]Randomised, double-blind, placebo controlled study, phase 414YesYesInclusion criteria: CF complicated by ABPA
Oral CSS use for ABPA flare
Age ≥12 years (except for Italy; ≥18 years)
Total serum IgE levels ≥500 IU·mL−1
Δ need of rescue CSS
Δ ABPA exacerbation rate
Time to CSS suspension
Δ% oral CSS dosage
Participants responding to omalizumab, as defined by a reduction in oral CSS dose use of ≥50% as compared with baseline
Up to 600 mg daily
+ Itraconazole
6 months12 monthsAborted due to difficulties in enrolling patients
No published data
Early termination of the study
Participants dropped out of the intervention group: 1 due to AEs; 1 due to lack of efficacy; and 3 due to administrative problems
6 patients had serious AEs versus 1 in the placebo
Emiralioglu [33]Case series61:211–32YesYesNo response or adverse effects of CSS treatmentMonthly evaluation for: symptoms, exacerbation, IgE, spirometry, CSS dosage300 mg s.c. every 4 weeks; dosage adapted to body weight and IgE level at the beginning of treatment6–18 monthsNoNo pulmonary exacerbations in 4/6 (66.6%)
No ABPA exacerbations in 6/6 (100%)
↓ or stop CSS use
↓ total IgE (p=0.028)
= FEV1 (+3%, p=0.91)
Yes, especially if early started
Lebecque [34]Case series21:114YesYesABPA exacerbation
Alternative to CSS treatment
NA375 mg every 2 weeks for 4 months then every 4 weeks for 3 months11 injectionsNo↓ symptoms
↑ FEV1
Stable up to 20 weeks following treatment withdrawal (FEV1 of 98% and 99% predicted)
Parisi [35]Case series33:011–28YesYesNo or poor improvement with CSS and antifungal treatmentNA375–600 mg every 2 weeks s.c.8 weeksNo↓ symptoms
↓ total IgE (↓ 60–70%)
↑ FEV1 (+28–50%)
Kanu [37]Case report10:113YesYesCSS side-effects (IGT)NA300 mg s.c. every 2 weeks11 weeksNo↓ symptoms
↑ FEV1 (+36%)
Thomas [38]Case report11:011YesYesCSS dependence
CSS side-effects (IGT)
NA375 mg every 2 weeks s.c.16 weeksNo↓ symptoms
↓ systemic CSS use
↑ FEV1
BMI improvement
12 months after, patient needed omalizumab again

M: male; F: female; CSS: corticosteroids; NA: not applicable; FEV1: forced expiratory volume in 1 s; BMI: body mass index; FVC: forced vital capacity; s.c.: subcutaneous; IGT: impaired glucose tolerance; AEC: absolute eosinophil count; AE: adverse event.