Tuberculosis (TB) remains a major cause of morbidity and mortality among children worldwide, with approximately one million new pediatric cases annually. The conventional treatment for drug-susceptible TB has long relied on a 6-month multidrug regimen, which is highly effective but associated with challenges in adherence, toxicity, and healthcare burden. Objectives: To evaluate whether short-course therapy is an appropriate regimen for children and young adolescents with drug-susceptible TB, with particular focus on its efficacy, safety, and applicability in different clinical contexts. Methods: A structured narrative review of the literature was conducted, including randomized controlled trials, observational studies, and international guidelines addressing treatment duration in children and young adolescents with drug-susceptible TB. Evidence was synthesized focusing on children and young adolescents < 16 years with drug-susceptible TB treated with short-course regimens compared to standard therapy. Results: A shorter treatment regimen, particularly 4-month courses, has been investigated as an alternative to standard therapy in the pediatric population with drug-susceptible TB. Children often present with paucibacillary and non-severe forms of TB, providing a biological rationale for treatment shortening. Evidence from a randomized controlled trial has demonstrated that a 4-month regimen is non-inferior to the standard 6-month therapy in children and young adolescents with non-severe, drug-susceptible TB. These findings have informed recent international guideline updates, which now recommend short therapy in carefully selected patients. However, a short regimen is not appropriate for infants younger than 3 months, children with severe or complicated TB, extrapulmonary disease such as central nervous system involvement, or those with drug-resistant TB. The overall quality of evidence remains moderate, and long-term relapse data are still emerging. Conclusions: Short-course therapy represents a promising but selective strategy in pediatric drug-susceptible TB management. It offers potential advantages, including improved adherence, reduced drug toxicity, and lower healthcare costs. However, its safe implementation requires accurate patient selection, access to appropriate diagnostic tools, and structured follow-up. Careful application within clearly defined clinical criteria is essential to ensure optimal outcomes.

Is Short Therapy an Appropriate Regimen for Children and Young Adolescents with Drug-Susceptible Tuberculosis? / Esposito, Susanna; Fainardi, Valentina; Campana, Beatrice Rita; Arnesano, Gaia Giorgia; Principi, Nicola. - In: PHARMACEUTICALS. - ISSN 1424-8247. - (2026). [10.3390/ph19050721]

Is Short Therapy an Appropriate Regimen for Children and Young Adolescents with Drug-Susceptible Tuberculosis?

Susanna Esposito
;
Valentina Fainardi;Beatrice Rita Campana;Gaia Giorgia Arnesano;
2026-01-01

Abstract

Tuberculosis (TB) remains a major cause of morbidity and mortality among children worldwide, with approximately one million new pediatric cases annually. The conventional treatment for drug-susceptible TB has long relied on a 6-month multidrug regimen, which is highly effective but associated with challenges in adherence, toxicity, and healthcare burden. Objectives: To evaluate whether short-course therapy is an appropriate regimen for children and young adolescents with drug-susceptible TB, with particular focus on its efficacy, safety, and applicability in different clinical contexts. Methods: A structured narrative review of the literature was conducted, including randomized controlled trials, observational studies, and international guidelines addressing treatment duration in children and young adolescents with drug-susceptible TB. Evidence was synthesized focusing on children and young adolescents < 16 years with drug-susceptible TB treated with short-course regimens compared to standard therapy. Results: A shorter treatment regimen, particularly 4-month courses, has been investigated as an alternative to standard therapy in the pediatric population with drug-susceptible TB. Children often present with paucibacillary and non-severe forms of TB, providing a biological rationale for treatment shortening. Evidence from a randomized controlled trial has demonstrated that a 4-month regimen is non-inferior to the standard 6-month therapy in children and young adolescents with non-severe, drug-susceptible TB. These findings have informed recent international guideline updates, which now recommend short therapy in carefully selected patients. However, a short regimen is not appropriate for infants younger than 3 months, children with severe or complicated TB, extrapulmonary disease such as central nervous system involvement, or those with drug-resistant TB. The overall quality of evidence remains moderate, and long-term relapse data are still emerging. Conclusions: Short-course therapy represents a promising but selective strategy in pediatric drug-susceptible TB management. It offers potential advantages, including improved adherence, reduced drug toxicity, and lower healthcare costs. However, its safe implementation requires accurate patient selection, access to appropriate diagnostic tools, and structured follow-up. Careful application within clearly defined clinical criteria is essential to ensure optimal outcomes.
2026
Is Short Therapy an Appropriate Regimen for Children and Young Adolescents with Drug-Susceptible Tuberculosis? / Esposito, Susanna; Fainardi, Valentina; Campana, Beatrice Rita; Arnesano, Gaia Giorgia; Principi, Nicola. - In: PHARMACEUTICALS. - ISSN 1424-8247. - (2026). [10.3390/ph19050721]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11381/3055873
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