Pulmonology Advisor: What are the remaining needs in this area?

Dr McGrath-Morrow: There is a need for randomized controlled trials of children, biomarker research, and long-term follow-up to determine the success of treatment approaches and long-term outcomes in pediatric PH, and how long-term outcomes can differ according to the underlying etiology of the pediatric PH.

Dr Avitabile: With the use of prostacyclins over the past few decades, survival rates of patients with pediatric PH have improved, but there is still significant work to be done. Much new research in the pediatric PH community will be devoted to in-depth phenotyping and genotyping of patients with various classifications of PH. Techniques such as whole exome sequencing may allow us to identify genetic mutations common in patients with certain forms of PH.6 We may be able to identify the proteins that are incorrectly formed as a result of these PH-causing mutations. These can be therapeutic targets for new drugs.


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Mechanical support will also continue to evolve in order to support patients who suffer right ventricular failure. Finally, we are always trying to improve the quality of life of pediatric patients with PH and hope to have advances in the areas of mental health support, educational support, and home exercise programs.

Dr Carl: Identification of patient biomarkers specific to pediatric disease processes may aid in the development of personalized therapeutics. Improved understanding of the balance of inflammatory and anti-inflammatory cytokines or oxidant vs antioxidant capacity may help with prognosis. These developments will allow for disease-specific randomized controlled trials.

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References

  1. Farhat N, Lador F, Beghetti M. Diagnosis and treatment of pediatric pulmonary arterial hypertension. Expert Rev Cardiovasc Ther. 2019;17(3):161-175.
  2. Berger RM, Beghetti M, Humpl T, et al. Clinical features of paediatric pulmonary hypertension: a registry study. Lancet. 2012;379(9815):537-546.
  3. Grady RM, Eghtesady P. Potts shunt and pediatric pulmonary hypertension: what we have learned. Ann Thorac Surg. 2016;101(4):1539-1543.
  4. Gorbachevsky SV, Shmalts AA, Barishnikova IY, Zaets SB. Potts shunt in children with pulmonary arterial hypertension: institutional experience. Interact Cardiovasc Thorac Surg. 2017;25(4):595-599.
  5. Stoecklin B, Simpson, SJ, Pillow JJ. Bronchopulmonary dysplasia: rationale for a pathophysiological rather than treatment based approach to diagnosis [published online December 19, 2018]. Paediatr Respir Rev. doi:10.1016/j.prrv.2018.12.002
  6. Zhu N, Gonzaga-Jauregui C, Welch CL, et al. Exome sequencing in children with pulmonary arterial hypertension demonstrates differences compared with adults. Circulation: Genomic and Precision Medicine. 2018;11(4).