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Published on: November 4, 2010
Current therapy of bronchopulmonary dysplasia
1Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee.
Insights
Bronchopulmonary dysplasia (BPD) treatment has seen little progress, with no convincing trials supporting long-term therapies. Continuous oxygen therapy remains the safest and most effective intervention for infants with BPD.
Area of Science:
- Neonatology
- Pediatric Pulmonology
- Respiratory Medicine
Background:
- Bronchopulmonary dysplasia (BPD) remains a significant challenge in neonatal care, with limited therapeutic advancements in recent years.
- Clinical research in BPD has been hampered by budget constraints and a shift towards molecular biology approaches.
- Current evidence does not convincingly support the long-term efficacy of diuretics, bronchodilators, vasodilators, or antioxidants for chronic BPD.
Purpose of the Study:
- To review the current state of BPD treatment and explore future therapeutic directions.
- To evaluate the effectiveness and safety of existing and emerging BPD therapies.
- To emphasize the importance of evidence-based clinical trial design and the continued role of oxygen therapy.
Main Methods:
- Review of existing clinical trial data and literature on BPD treatments.
- Discussion of ongoing research into novel therapeutic strategies, including enzyme, gene, and cytokine therapies.
- Exploration of potential non-specific treatments and advanced interventions like lung transplantation.
Main Results:
- Short-term corticosteroid use can facilitate extubation but does not improve long-term outcomes and raises safety concerns.
- No long-term therapies for chronic BPD have demonstrated convincing efficacy in clinical trials.
- Continuous oxygen therapy, alongside avoidance of environmental hazards, is highlighted as the most rational and safest treatment approach.
Conclusions:
- Future BPD treatment strategies should be informed by a deeper understanding of lung repair and inflammation mechanisms.
- Multicenter clinical trials are crucial for validating the efficacy of various therapies, with careful stratification for risk factors.
- Despite the development of novel agents, continuous oxygen therapy remains a cornerstone of BPD management due to its proven benefits and safety profile.
Abstract:
In summary, little progress has been made in the past several years with respect to the treatment of the baby with BPD. The conduct of convincing clinical research seems to be a casualty of budget cuts and a rush to learn the tools of molecular biology. To date, there are no clinical trials that have convincingly demonstrated that long-term diuretic, bronchodilator, vasodilator, or antioxidant therapy is effective in the treatment of chronic BPD. Short-term corticosteroid therapy hastens extubation, but long-term outcome is unaffected and serious questions remain about its safety. Multicenter clinical trials should be carefully designed and implemented to address the values of these therapies. In the design of these trials, care should be taken to stratify treatment groups for known risk factors for BPD. What are the future directions for the treatment of BPD? It is hoped that new BPD treatment strategies will be based on an improved understanding of mechanisms of lung repair and inflammation. Enzyme, gene, cytokine, antioxidant, and antiprotease therapies are being developed in animal models of lung injury. In addition, the use of lung transplantation has begun to be explored for severe cases of BPD. It is also possible, as has occurred in many chronic idiopathic diseases, that nonspecific treatment may prove beneficial. Perhaps it is only a matter of time before intravenous immunoglobulin, cyclosporine, methotrexate, or "biological response modifiers" will be administered to infants with severe BPD. For example, there is anecdotal evidence that recombinant human growth hormone may improve respiratory muscle function in adults with chronic obstructive pulmonary diseases. In the absence of convincing clinical trials, the clinician should reserve existing therapies for the ventilator-dependent infant or infants whose high oxygen requirement is prohibiting discharge or resulting in complex home care or frequent rehospitalizations. It should be emphasized that continuous oxygen therapy combined with avoidance of environmental inhalant and infectious hazards have the strongest rationale and widest margin of safety for treatment of the infant with BPD. Ironically, oxygen therapy is frequently underutilized and discontinued too rapidly. Early discontinuation of oxygen therapy with alveolar hypoxia results in feeding difficulty, slow growth, nutrient malabsorption, bronchoconstriction, and pulmonary hypertension. Oxygen therapy, although more cumbersome and certainly less glamorous than other pharmacologic agents, remains the essential element of BPD care.
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