Ready Reckoner on Severe Asthma and Omalizumab

Study Name

Objective

Reference

N (No. of patients)

Duration

Key findings

NHLBI SARP (Severe Asthma Research Program), USA¹

To improve the understanding of severe asthma to gain a better understanding of asthma and its endotypes, in children and adults, by defining the disease at the molecular and cellular levels, to develop better treatments.

Am J Respir Crit Care Med, 2012

N=1644

10 years

SARP studies revealed that severe asthma is a heterogeneous disease with varying molecular, biochemical, and cellular inflammatory features and unique structure–function abnormalities.

TENOR

(The Epidemiology and Natural History of Asthma: Outcomes and Treatment Regimens study), USA²

To characterize the natural history of disease in adults and children with severe or difficult-to-treat asthma.

J Allergy Clin Immunol,

2012

N= 4756

3 years

High rates of health care use and substantial asthma burden despite receiving multiple long-term controller medications.

IgE and allergen sensitization played a role in majority of severe or difficult-to-treat asthmatic patients.

Recent exacerbation history was the strongest predictor of future asthma exacerbations.

U-BIOPRED (Unbiased Biomarkers for the Prediction of Respiratory Disease Outcomes), a part of the IMI, EU³

To bring about a better understanding of the different types of severe asthma, with the long-term goal of developing more effective treatments for people with the condition.

Thorax,

2011

N=1025

5 years

The U-BIOPRED programme not only reached international consensus on the definition and diagnosis of severe asthma but, more importantly, produced for the first time a stepwise algorithm by which the patient with truly severe refractory asthma may be identified.

ENFUMOSA (European Network for Understanding the Mechanisms of Severe Asthma)⁴

To study of the clinical phenotype of chronic severe asthma.

Eur Respir

J, 2003

N=163

Retrospective study

The ENFUMOSA study identified features of Severe Asthma that are distinct from those described for mild-moderate disease. Persistent symptoms and abnormal lung function, despite high-dose regular therapy, accompanied by irreversible airflow obstruction, neutrophilic inflammation, ongoing mediator release and reduced association with atopy.

Study Name

Objective

Reference

N (No. of patients)

Duration

Key findings

The eXpeRience Registry: Real world effectiveness of Omalizumab in allergic asthma ⁵

To evaluate real-world effectiveness, safety and use of Omalizumab therapy in patients with uncontrolled persistent allergic asthma

Respir Med,

2013

N=943

2 years

↓ exacerbations; ↑ asthma control; ↓ symptoms; ↑ lung function (FEV₁ ,PEF); ↑ QoL; ↓ healthcare utilization; ↓ use of OCS and rescue medication; 70% with good/excellent response by physician’s GETE

The PERSIST Study: Real-life effectiveness of Omalizumab in patients with severe persistent allergic asthma⁶

To evaluate the 16 – and 52-week effectiveness of add-on Omalizumab treatment under real-life heterogeneity in patients, settings and physicians in patients with severe allergic asthma in Belgium.

Respir Med,

2009

N=158

52 weeks

↓ exacerbations; ↓ symptoms; ↑ FEV₁; ↑ QoL; ↓ healthcare utilization; >72% with good/excellent response by physician’s  GETE

APEX II Study: Impact of Omalizumab on treatment of Severe Allergic Asthma in UK clinical practice⁷

To describe the impact of Omalizumab on asthma management in adults (16 years and over) treated with Omalizumab as a part of clinical practice within the UK NHS.

BMJ Open,

2016

N=258

12-month pre- omalizumab and 12-month post- omalizumab

↓ exacerbations; ↓ mean daily dose of OCS; ↓ hospital visits due to exacerbations; ↑ FEV₁; ↑ asthma- specific patient-reported outcomes

EXCEL Study: Evaluating Clinical Effectiveness and Long-Term Safety in

Patients with Moderate to Severe Asthma (EXCELS)⁸

To describe longitudinal changes of asthma control and asthma-related work, school, and activity impairment for patients with moderate-to- severe asthma treated with Omalizumab and those not treated with Omalizumab in a real-world setting.

Allergy Asthma Proc, 2015

N=4930

5 years

↓ asthma-related work, school and activity impairment; ↑ Asthma control  (ACT Scores)

Study Name

Objective

Reference

N (No. of patients)

Duration

Key findings

INNOVATE⁹

To evaluate the effect of add-on Omalizumab on asthma exacerbations in patients with severe persistent asthma who are inadequately controlled despite GINA step 4 therapy.

Allergy,

2005

N=419

28 weeks

↓ rate of clinically significant exacerbations; ↓ rate of emergency visits; ↑ FEV₁; ↑ improved  QOL

Effectiveness of Omalizumab in patients with inadequately controlled severe persistent allergic asthma: An open- label study¹⁰

To assess of the effectiveness of Omalizumab in a subgroup of patients with inadequately controlled severe persistent allergic (IgE-mediated) asthma despite receiving high- dose ICS plus a LABA.

Repir Med,

2008

N=164

1 year

↓ annual exacerbation rate; ↓ annual asthma deterioration- related incident; ↑ % predicted FEV₁ ↑asthma symptom scores; ↑ mini AQLQ scores.

Efficacy and tolerability of anti-immunoglobulin  E therapy with Omalizumab in patients with poorly controlled (moderate-to- severe) allergic asthma¹¹

To evaluate the efficacy and tolerability of Omalizumab in patients with poorly controlled, moderate-to-severe allergic asthma.

Allergy,

2004

N=312

1 year

↓ annual asthma deterioration- related incident; ↓ rescue medication use; ↑ FEV1; ↑asthma symptom scores.

Study Name

Objective

Reference

N (No. of patients)

Duration

Key findings

After 6 years of Xolair; a 3-year withdrawal follow-up¹²

To determine the clinical and immunological state of patients 3 years after a 6-year period of Xolair treatment for severe allergic asthma.

Allergy,

2010

N=18

6 years

Most patients in the study had, still 3 years after closing of 6-year Xolair treatment, showed mild and stable asthma.

Long-term efficacy and safety of Omalizumab in patients with persistent uncontrolled allergic asthma: a systematic review and meta- analysis¹³

To systematically review the evidence regarding (i) long- term efficacy of Omalizumab in patients with persistent uncontrolled allergic asthma (ii) discuss the cost-effectiveness evidence published for Omalizumab in this patient population.

Sci Rep, 2015

6 studies; N=2749

52 weeks

After 52 weeks of treatment with Omalizumab in patients with persistent uncontrolled allergic asthma was accompanied by an acceptable safety profile, but lacked effect on asthma exacerbations.

Long term clinical outcomes of Omalizumab therapy in severe allergic asthma: Study of efficacy and safety¹⁴

To assess the effectiveness and safety of Omalizumab in a responder population with mean treatment duration of 5 years (range 2-11 years).

Respir Med, 2017

N=45

Retrospective study

↓annual acute asthma- related hospitalization rate; ↓ daily mean maintenance OCS dose; improvement in ACQ score; ↑ FEV₁; ↓ no. of work/school missed days.

Study Name

Objective

Reference

N (No. of patients)

Duration

Key findings

Long-term safety, efficacy, pharmacokinetics and pharmacodynamics of Omalizumab in children with severe uncontrolled asthma¹⁵

To evaluate the long- term safety, efficacy, pharmacokinetics and pharmacodynamic of Omalizumab in children with uncontrolled severe asthma.

Allergology International, 2017

N=35

16 weeks+ Treatment till approval for paediatric indication

↑QOL scores; ↑ annual rate of hospitalization and ER visits; well-tolerated with no new safety risks in children.

Add-on Omalizumab in children with severe allergic asthma: a 1-year real life survey¹⁶

To evaluate Omalizumab efficacy and safety in a real- life setting in severe asthmatic children.

Eur Respir J, 2013

N=104

 1 year

↑ Asthma control; ↓exacerbation and hospitalization rate; ↓ ICS dose; ↑ FEV₁; well-tolerated.

Study Name

Objective

Reference

N (No. of patients)

Duration

Key findings

Evaluation of long-term safety and efficacy of Omalizumab in elderly patients with uncontrolled allergic asthma¹⁷

To evaluate the long-term safety and efficacy of the anti-IgE antibody Omalizumab in elderly (aged ≥65 years) patients with uncontrolled allergic asthma.

Ann Allergy Asthma Immunol, 2016

N=19

Retrospective study

No significantly important adverse event experienced; safe and effective for elderly population.