Childhood: a window of opportunity to change the natural history of asthma?

Persistent barriers to the early management of asthma

The burden of pediatric asthma

Asthma is the most common chronic disease in children¹VonMutius E. Le fardeau de l’asthme infantile. Arch Dis Child. 2000;82 Suppl. Although current treatment options allow most children to maintain preserved lung function, the medical and economic burden of asthma remains high. This is reflected in emergency room visits, hospital admissions, and absenteeism—from school for the child and from work for the parents. As a result, children with asthma miss roughly 1.5 times more school days per year²Sullivan PW, Ghushchyan V, Navaratnam P, Friedman HS, Kavati A, Ortiz B, Lanier B. The national burden of poorly controlled asthma, school absence and parental work loss among school-aged children in the United States. J Asthma. 2018 Jun;55(6):659-667. doi: 10.1080/02770903.2017.1350972.

Asthma in children is therefore associated with:

• increased school absenteeism
• reduced participation in extracurricular activities,
• lower academic performance,
• more frequent sleep problems,
• increased anxiety and a higher risk of depression.

A diagnosis that remains complex in very young children

Despite progress in understanding the disease, diagnosing asthma in children remains difficult. In Europe, the prevalence of pediatric asthma at age 4 varies widely from country to country, ranging from less than 2% in Germany to more than 13% in the United Kingdom³Variations in the prevalence of childhood asthma and wheeze in MeDALL cohorts in Europe, Eleonora P. Uphoff et al, 2017, https://doi.org/10.1183/23120541.00150-2016, despite comparable socioeconomic contexts.

These discrepancies highlight how challenging it is to diagnose the pediatric population. In young children, symptoms can be mistaken for viral respiratory infections or episodes of bronchiolitis. This uncertainty sometimes delays the identification of the children most at risk and limits opportunities for early intervention.

More complicated treatment adherence in children

In pediatric asthma, adherence is a major challenge. In children, management relies heavily on the parents, whose understanding of the disease determines how well treatment is followed. As a result, reliever medications are often favored over inhaled corticosteroids, even though the latter are essential for lasting asthma control.

Adolescence then marks a turning point. As responsibility shifts to the young patient, treatment adherence tends to decline, driven by reduced parental supervision and a growing desire for independence.

On top of these issues comes the challenge of mastering inhalation devices. Despite a wide range of options, usage errors remain common. Differences in handling between pressurized metered-dose inhalers (MDIs) and dry powder inhalers (DPIs) can cause confusion, particularly when switching treatments, thereby undermining therapeutic effectiveness.

Solutions are emerging to improve adherence, such as connected sensors fitted to inhalation devices, or digital adherence-monitoring tools like the Drago app, which aims to “put serious gaming at the service of adherence”. The APHP is currently conducting a pilot study exploring the role of digital tools in improving asthma management in children.

Read also: How Medtech is revolutionizing remote medical monitoring

Delayed access to therapeutic innovations

Historically, clinical development in pediatrics has lagged several years behind that in adults. Indication extensions, particularly for very young children (under 6), often come several years after the initial marketing authorizations.

This delay stems in part from the complexity of pediatric clinical trials, recruitment difficulties, and concerns about the long-term safety of treatments that modulate the immune system during a period of growth and maturation.

This situation is especially apparent for biologic therapies used in severe asthma. While nearly 80% of pediatric asthma cases begin before the age of 6⁴Martinez F.D. et al. Pediatrics. 2002; 109(2 Suppl), 362–367, most biologic therapies are only available from the age of 6 or even 12, depending on the molecule.

To limit children’s exposure to clinical trials, the EMA encourages extrapolation from adult data, provided it is based on robust biological, pharmacokinetic, and pharmacodynamic assumptions. The goal is no longer to systematically replicate adult trials, but rather to demonstrate the validity of the extrapolation rationale through specific data (particularly PK/PD). Complementary real-world studies can then be conducted post-marketing authorization to address any remaining uncertainties, especially for immunomodulatory treatments or new therapeutic classes, where long-term effects (e.g., growth, immune development) have yet to be characterized.

For pediatric populations, the challenge then lies in collecting follow-up data, which is most often reported by parents. Engaging, playful digital solutions can be developed to make data collection easier and improve patient engagement. This is notably the case with the ePASD (electronic Pediatric Asthma Symptom Diary) project, a tablet application combining visuals, sounds, and text designed to minimize the need for reading skills in the ability to self-assess, developed for children aged 6–11 with mild to severe asthma, as well as Ludocare, which provides children with tailored, playful tools to manage their condition from a very young age by engaging both children and caregivers.

Given the limitations in diagnosing and managing pediatric asthma, attention is turning toward earlier interventions to address asthma risk factors. This approach draws in particular on the allergic continuum, according to which allergic manifestations of the upper airways, such as allergic rhinitis, may precede the onset of asthma and represent a first opportunity for action.

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Childhood: a still-underused window of opportunity to act on asthma risk factors

The allergic continuum as a first target for intervention

One of the strongest arguments in favor of an early approach lies in the close relationship between allergic rhinitis and asthma.

Patients with allergic rhinitis are two to seven times more likely to develop asthma than non-allergic populations⁵Batard T. et al., Clin Exp Allergy, 2025. This association is now explained by the concept of “unified airway disease⁶Batard T. et al., Clin Exp Allergy, 2025», according to which the upper airways (nose, sinuses) and lower airways (bronchi) share common inflammatory mechanisms.

In this context, managing respiratory allergies emerges as a first lever for influencing the course of the disease.

A particularly critical biological window with still poorly understood risk factors

Beyond respiratory allergies, asthma results from complex interactions between genetic and environmental factors, which are especially active during pregnancy and the first years of life.

While prevention strategies remain limited and lack consensus, several factors now appear to play an important role. International guidelines, such as those from GINA⁷GINA 2026, “Primary prevention of asthma”, p229, highlight in particular the importance of limiting exposure to tobacco during pregnancy and avoiding excessive use of broad-spectrum antibiotics during the first year of life.

This vigilance is all the more important given that each exacerbation contributes to increased inflammation and potentially to the progressive remodeling of the airways⁸Société de pneumologie de langue française, 2025, https://splf.fr/e-remodelage-bronchique-dans-lasthme/[/mfn].

Toward preventive medicine for pediatric asthma?

While childhood is a prime period for taking action, it still requires having the tools to intervene before asthma is fully established.

Allergen immunotherapy: a potential preventive strategy?

Allergen immunotherapy (AIT), or desensitization, involves administering precise doses of allergen extracts over a long period to “educate” the immune system and raise its tolerance thresholds.

Several studies⁸Société de pneumologie de langue française, 2025, https://splf.fr/e-remodelage-bronchique-dans-lasthme/ suggest in particular that allergen immunotherapy in patients under 18 could reduce the subsequent risk of developing asthma in allergic children, or at least delay it, reinforcing the hypothesis that early intervention can have a lasting impact on clinical progression. These findings have led scientific societies such as the EAACI to recognize the preventive potential of AIT in children and adolescents with allergic rhinitis caused by grass or birch pollen. The benefits have been demonstrated for up to two years after treatment discontinuation, with some studies suggesting an effect that may persist beyond that⁹Halken S, Larenas-Linnemann D, Roberts G, et al. EAACI guidelines on allergen immunotherapy: Prevention of allergy. Pediatr Allergy Immunol. 2017;28:728–745. https://doi.org/10.1111/pai.12807.

However, a gap persists between the potential of AIT and its use in routine practice. Worldwide, it is estimated that fewer than 10% of patients with allergic rhinitis or asthma are receiving allergen immunotherapy (AIT) treatment¹⁰Marek Jutel et al, International consensus on allergy immunotherapy, Journal of Allergy and Clinical Immmunology, 2015, https://doi.org/10.1016/j.jaci.2015.04.047. Practices vary from country to country, but, for example, in France, only 23% of allergic patients report that a physician offered them allergen immunotherapy¹¹Reccole, E. (2026, June 18). Enquête sur les allergies et leur impact sur la vie quotidienne – Ifop Group. Ifop Group. https://www.ifopgroup.com/article/enquete-sur-les-allergies-et-leur-impact-sur-la-vie-quotidienne/. These figures underscore the need to strengthen information and referral of eligible patients toward this type of care.

Biologic therapies: beyond symptom control?

Biologics are attracting growing interest as tools for early intervention.

In adults, stopping them generally leads to a return of inflammatory activity. In children, however, lung growth and immune maturation could offer an opportunity to achieve more lasting effects.

Several studies are currently exploring this hypothesis and point to the possibility of a persistence of the therapeutic effect, and even a form of remission in some patients after treatment is discontinued¹²Remission in Children Receiving Biologic Therapy for Severe Asthma, 2025, https://doi.org/10.1002/ppul.71240¹³Clinical, functional, and laboratory course of children and adolescents with severe asthma after discontinuation of biologics, 2025, https://doi.org/10.3389/falgy.2025.1672424. While these findings still need to be confirmed, they hint at an opportunity to delay the onset of severe exacerbations in very young children.

Microbiome: a new prevention strategy?

At the same time, approaches targeting the microbiome hold promise for preventing the onset of asthma. Recent work from the Technical University of Denmark has shown that specific gut bacteria—bifidobacteria—produce a molecule capable of curbing allergic diseases from the very first days of life.

These findings support the hypothesis that the composition of the microbiota could influence the later risk of asthma. Research is now aimed at understanding why some children have higher or lower concentrations of this protective bacterium. In the long run, nutritional strategies—such as adding this bacterium to infant formulas—could help prevent the disease from developing in certain at-risk children.

Read also: Microbiome therapeutics: what are the current advances?

Biomarkers to identify at-risk children?

The early identification of children likely to develop asthma is also a major area of research.

For example, analysis of the respiratory volatilome—the volatile organic compounds found in exhaled air—has made it possible to identify a biological signature of pediatric asthma: the presence of the bacterium Eubacterium siraeum. Although still exploratory, these approaches could, in the future, make it easier to detect asthma early and to start treatment as soon as possible.

A vaccine against asthma?

Finally, among the most exploratory approaches is Kinoïde®, a so-called therapeutic vaccine (since the disease is already present), developed by French research teams and the biotech company Neovacs. It aims to induce the production of antibodies directed against interleukins 4 and 13, two cytokines that play a central role in the onset of allergic asthma. Early results in mouse models have notably shown a reduction in IgE levels (the antibodies responsible for triggering the allergic reaction), in bronchial hyperresponsiveness, and in mucus production¹⁴A vaccine targeting the cytokines IL-4 and IL-13 protects against allergic asthma in mice, 2022, https://doi.org/10.1051/medsci/2021232. These are effects that could improve asthma control and reduce the severity of symptoms. Although this program is still at the preclinical stage and is not currently the subject of any pediatric development, it illustrates a shift in therapeutic strategies toward treatments that could lastingly alter the natural course of the disease rather than simply ensuring its ongoing control. Childhood is therefore probably the period when it is most relevant to act in order to lastingly change the trajectory of asthma.

This shift means going beyond a mere adaptation of the strategies developed in adults. It calls for accelerating children’s access to therapeutic innovations, developing diagnostic and stratification tools tailored to this population, and investing in preventive approaches.

Long seen as a constraint of clinical development, pediatrics could thus become one of the main drivers for transforming asthma care. More than a specific population, it may today represent the best opportunity to intervene before the disease takes lasting hold.

Would you like to explore how to better integrate pediatric populations into your therapeutic developments or strengthen your innovation strategy in pediatric asthma? The Alcimed team regularly carries out projects on pediatric issues: developing new drugs, adapting medical devices for children, training and raising awareness about this population, or even optimizing care protocols. Don’t hesitate to contact our team!

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About the author,

Margot, Consultant within Alcimed’s Healthcare team in France