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Pretermlerde respiratuvar distres sendromu tedavisinde kullanılan üç farklı doğal surfaktan preparatının maliyet etkinliğinin karşılaştırılması

Year 2025, Volume: 50 Issue: 1, 73 - 82, 31.03.2025
https://doi.org/10.17826/cumj.1589006

Abstract

Amaç: Bu çalışma, üç doğal surfaktanın maliyet-etkinliğini, respiratuvar ve kan gazı değişkenleri üzerindeki başlangıç etkileri ve ilişkili maliyetleri analiz ederek değerlendirmeyi amaçlamıştır.
Gereç ve Yöntem: Bu çalışmaya, Eylül 2019 ile Aralık 2022 tarihleri arasında üçüncü basamak yenidoğan yoğun bakım ünitesinde (YYBÜ) eksojen surfaktan replasman tedavisi almış, 32. gebelik haftasından önce doğmuş respiratuvar distres sendromu (RDS) tanılı preterm bebekler dahil edilmiştir. 985 hastaya ait veriler tıbbi kayıtlardan retrospektif olarak elde edilmiştir.
Bulgular: Çalışmaya dahil edilen hastaların %58’i (n=575) Poraktant Alfa, %35’i (n=343) Beraktant ve %7’si (n=67) Kalfaktant almıştır. Surfaktan grupları arasında kısa dönem pulmoner iyileşme, komplikasyonlar, klinik sonuçlar veya maliyetler açısından anlamlı bir fark gözlenmemiştir. Ancak, doğum ağırlıklarına göre yapılan alt grup analizinde, 750-999 gram ağırlığındaki bebeklerde Kalfaktant’ın (331,57±162,54 $), Beraktant (507,50±175,50 $) ve Poraktant Alfa’ya (472,44±93,73 $) göre anlamlı şekilde daha maliyet-etkin olduğu bulunmuştur. Ayrıca, 1000-1499 gram ağırlığındaki bebeklerde Beraktant (497,47±168,55 $) ve Kalfaktant’ın (531,54±293,20 $) Poraktant Alfa’ya (669,36±265,23 $) göre anlamlı şekilde daha düşük maliyetli olduğu görülmüştür.
Sonuç: Bu çalışma, surfaktan grupları arasında benzer kısa dönem respiratuvar iyileşme, komplikasyonlar ve klinik sonuçlar gözlemlendiğini, ancak maliyetin surfaktan seçiminde potansiyel olarak önemli bir rol oynayabileceğini göstermiştir. Doğum ağırlıklarına dayalı surfaktan seçimi için yeni bir model önerilmiştir. Buna göre, 750-999 gram ağırlığındaki bebekler için Kalfaktant’ın, 1000-1499 gram ağırlığındaki bebekler için ise Poraktant Alfa’nın tercih edilmesi önerilmektedir.

References

  • Whitsett JA, Weaver TE. Hydrophobic surfactant proteins in lung function and disease. N Engl J Med. 2002;347:2141-8.
  • Dargaville PA, Herting E, Soll RF. Neonatal surfactant therapy beyond respiratory distress syndrome. Semin Fetal Neonatal Med. 2023;28:101501.
  • Trembath A, Hornik CP, Clark R, Smith PB, Daniels J, Laughon M; Best pharmaceuticals for children act pediatric trials network. Comparative effectiveness of surfactant preparations in premature infants. J Pediatr. 2013;163:955-60.e1.
  • Ardell S, Pfister RH, Soll R. Animal derived surfactant extract versus protein-free synthetic surfactant for the prevention and treatment of respiratory distress syndrome. Cochrane Database Syst Rev. 2015;8:CD000144.
  • Fenton TR. A new growth chart for preterm babies: Babson and Benda's chart updated with recent data and a new format. BMC Pediatr. 2003;3:13.
  • Satar M, Arısoy AE, Çelik İH. Turkish Neonatal Society guideline on neonatal infections-diagnosis and treatment. Turk Pediatri Ars. 2018;53:S88-S100.
  • Cakir U, Tayman C. What is the first-line treatment for closure of hemodynamically significant patent ductus arteriosus in premature infants? Arq Bras Cardiol. 2022;118:548-55.
  • Volpe JJ. Impaired neurodevelopmental outcomes after mild germinal matrix-intraventricular hemorrhage. Pediatrics. 2015;136:1185-7.
  • Kinsella JP, Greenough A, Abman SH. Bronchopulmonary dysplasia. Lancet 2006;367:1421-31.
  • Chiang MF, Quinn GE, Fielder AR, Ostmo SR, Paul Chan RV, Berrocal A et al. International Classification of Retinopathy of Prematurity, Third Edition. Ophthalmology. 2021;128:e51-68.
  • Ozkan H, Erdeve O, Kutman HGK. Turkish Neonatal Society guidelines on the management of respiratory distress syndrome and surfactant treatment. Turk Pediatri Ars. 2018;53(Suppl 1):S45-54.
  • Pawale D, D S, Anne RP, Oleti T, Murki S, Fursule A. A survey of less invasive surfactant administration in India. Indian J Pediatr. 2023;90:181-3.
  • Zayek MM, Eyal FG, Smith RC. Comparison of the pharmacoeconomics of calfactant and poractant alfa in surfactant replacement therapy. J Pediatr Pharmacol Ther. 2018;23:146-51.
  • Bissinger R, Carlson C, Michel Y, Dooley C, Hulsey T, Jenkins D. Secondary surfactant administration in neonates with respiratory decompensation. J Perinatol. 2008;28:192-8.
  • Jeon GW, Oh M, Sin JB. Efficacy of surfactant-TA, calfactant, and poractant alfa in preterm infants with respiratory distress syndrome: A retrospective study. Yonsei Med J. 2015;56:433-9.
  • Yılmaz FH, Tarakçı N, Gültekin ND, Yücel M, Keçeci R, Öztürk ENY et al. Comparison of the efficacy of three natural surfactants in preterm Turkish newborns with respiratory distress syndrome. J Pediatr Res. 2021;8:433-9.
  • Sánchez Luna M, Bacher P, Unnebrink K, Martinez-Tristani M, Ramos Navarro C. Beractant and poractant alfa in premature neonates with respiratory distress syndrome: A systematic review of real-world evidence studies and randomized controlled trials. J Perinatol. 2020;40:1121-34.
  • Tugcu AU, Ince DA, Turan O, Ecevit A. Comparison of two surfactant preparates derived from the same animal for the treatment of respiratory distress syndrome. Ann of Med Res. 2019;26:2948–51.
  • Ramanathan R, Bhatia JJ, Sekar K, Ernst FR. Mortality in preterm infants with respiratory distress syndrome treated with poractant alfa, calfactant, or beractant: A retrospective study. J Perinatol. 2013;33:119-25.
  • Qiu C, Ma C, Fan N, Zhang X, Zheng G. Comparative efficacy of pulmonary surfactant in respiratory distress syndrome in preterm infants: a Bayesian network meta-analysis. Arch Med Sci. 2023;19:1446-53.
  • Marsh W, Smeeding J, York JM, Ramanathan R, Sekar K. A cost minimization comparison of two surfactants-beractant and poractant alfa-based upon prospectively designed, comparative clinical trial data. J Pediatr Pharmacol Ther. 2004;9:117-25.
  • Brown S, Hurren J, Sartori H. Poractant alfa versus beractant for neonatal respiratory distress syndrome: A retrospective cost analysis. J Pediatr Pharmacol Ther. 2018;23:367-71.
  • Sekar K, Fuentes D, Krukas-Hampel MR, Ernst FR. Health economics and outcomes of surfactant treatments for respiratory distress syndrome among preterm infants in US level III/IV neonatal intensive care units. J Pediatr Pharmacol Ther. 2019;24:117-27.
  • Magni T, Ragni C, Pelizzi N, Sharma S, Perez-Kempner L, Turkstra E et al. Health economic studies of surfactant replacement therapy in neonates with respiratory distress syndrome: A systematic literature review. Pharmacoecon Open. 2023;7:359-71.

Cost-effectiveness comparison of three different natural surfactant preparations in the treatment of preterm infants with respiratory distress syndrome

Year 2025, Volume: 50 Issue: 1, 73 - 82, 31.03.2025
https://doi.org/10.17826/cumj.1589006

Abstract

Purpose: This study aimed to assess the cost-effectiveness of three natural surfactants by analyzing their initial impact on respiratory and blood gas variables and their associated costs.
Materials and Methods: This study included preterm infants with respiratory distress syndrome (RDS) born before the 32nd gestational week who received exogenous surfactant replacement therapy in a tertiary neonatal intensive care unit (NICU) between September 2019 and December 2022. Data from 985 patients were retrospectively obtained from medical records.
Results: Among the enrolled patients, 575 received Poractant Alfa (58%), 343 received Beractant (35%), and 67 received Calfactant (7%). No significant differences were observed between the surfactant groups in terms of short-term pulmonary improvement, complications, clinical outcomes, or costs. However, in subgroup analysis based on birth weights, Calfactant (331.57±162.54 $) was found to be significantly more cost-effective than Beractant (507.50±175.50 $) and Poractant Alfa (472.44±93.73 $) for infants weighing 750-999 grams. Furthermore, for infants weighing 1000-1499 grams, both Beractant (497.47±168.55 $) and Calfactant (531.54±293.20 $) were significantly less costly than Poractant Alfa (669.36±265.23 $).
Conclusions: This study demonstrated comparable short-term respiratory improvements, complications, and clinical outcomes among surfactant groups, emphasizing the potential influence of cost on surfactant choice. A novel model for selecting surfactant preparations based on birth weights was proposed, suggesting the use of Calfactant for infants weighing 750-999 grams and considering the use of Poractant Alfa for infants weighing 1000-1499 grams.

References

  • Whitsett JA, Weaver TE. Hydrophobic surfactant proteins in lung function and disease. N Engl J Med. 2002;347:2141-8.
  • Dargaville PA, Herting E, Soll RF. Neonatal surfactant therapy beyond respiratory distress syndrome. Semin Fetal Neonatal Med. 2023;28:101501.
  • Trembath A, Hornik CP, Clark R, Smith PB, Daniels J, Laughon M; Best pharmaceuticals for children act pediatric trials network. Comparative effectiveness of surfactant preparations in premature infants. J Pediatr. 2013;163:955-60.e1.
  • Ardell S, Pfister RH, Soll R. Animal derived surfactant extract versus protein-free synthetic surfactant for the prevention and treatment of respiratory distress syndrome. Cochrane Database Syst Rev. 2015;8:CD000144.
  • Fenton TR. A new growth chart for preterm babies: Babson and Benda's chart updated with recent data and a new format. BMC Pediatr. 2003;3:13.
  • Satar M, Arısoy AE, Çelik İH. Turkish Neonatal Society guideline on neonatal infections-diagnosis and treatment. Turk Pediatri Ars. 2018;53:S88-S100.
  • Cakir U, Tayman C. What is the first-line treatment for closure of hemodynamically significant patent ductus arteriosus in premature infants? Arq Bras Cardiol. 2022;118:548-55.
  • Volpe JJ. Impaired neurodevelopmental outcomes after mild germinal matrix-intraventricular hemorrhage. Pediatrics. 2015;136:1185-7.
  • Kinsella JP, Greenough A, Abman SH. Bronchopulmonary dysplasia. Lancet 2006;367:1421-31.
  • Chiang MF, Quinn GE, Fielder AR, Ostmo SR, Paul Chan RV, Berrocal A et al. International Classification of Retinopathy of Prematurity, Third Edition. Ophthalmology. 2021;128:e51-68.
  • Ozkan H, Erdeve O, Kutman HGK. Turkish Neonatal Society guidelines on the management of respiratory distress syndrome and surfactant treatment. Turk Pediatri Ars. 2018;53(Suppl 1):S45-54.
  • Pawale D, D S, Anne RP, Oleti T, Murki S, Fursule A. A survey of less invasive surfactant administration in India. Indian J Pediatr. 2023;90:181-3.
  • Zayek MM, Eyal FG, Smith RC. Comparison of the pharmacoeconomics of calfactant and poractant alfa in surfactant replacement therapy. J Pediatr Pharmacol Ther. 2018;23:146-51.
  • Bissinger R, Carlson C, Michel Y, Dooley C, Hulsey T, Jenkins D. Secondary surfactant administration in neonates with respiratory decompensation. J Perinatol. 2008;28:192-8.
  • Jeon GW, Oh M, Sin JB. Efficacy of surfactant-TA, calfactant, and poractant alfa in preterm infants with respiratory distress syndrome: A retrospective study. Yonsei Med J. 2015;56:433-9.
  • Yılmaz FH, Tarakçı N, Gültekin ND, Yücel M, Keçeci R, Öztürk ENY et al. Comparison of the efficacy of three natural surfactants in preterm Turkish newborns with respiratory distress syndrome. J Pediatr Res. 2021;8:433-9.
  • Sánchez Luna M, Bacher P, Unnebrink K, Martinez-Tristani M, Ramos Navarro C. Beractant and poractant alfa in premature neonates with respiratory distress syndrome: A systematic review of real-world evidence studies and randomized controlled trials. J Perinatol. 2020;40:1121-34.
  • Tugcu AU, Ince DA, Turan O, Ecevit A. Comparison of two surfactant preparates derived from the same animal for the treatment of respiratory distress syndrome. Ann of Med Res. 2019;26:2948–51.
  • Ramanathan R, Bhatia JJ, Sekar K, Ernst FR. Mortality in preterm infants with respiratory distress syndrome treated with poractant alfa, calfactant, or beractant: A retrospective study. J Perinatol. 2013;33:119-25.
  • Qiu C, Ma C, Fan N, Zhang X, Zheng G. Comparative efficacy of pulmonary surfactant in respiratory distress syndrome in preterm infants: a Bayesian network meta-analysis. Arch Med Sci. 2023;19:1446-53.
  • Marsh W, Smeeding J, York JM, Ramanathan R, Sekar K. A cost minimization comparison of two surfactants-beractant and poractant alfa-based upon prospectively designed, comparative clinical trial data. J Pediatr Pharmacol Ther. 2004;9:117-25.
  • Brown S, Hurren J, Sartori H. Poractant alfa versus beractant for neonatal respiratory distress syndrome: A retrospective cost analysis. J Pediatr Pharmacol Ther. 2018;23:367-71.
  • Sekar K, Fuentes D, Krukas-Hampel MR, Ernst FR. Health economics and outcomes of surfactant treatments for respiratory distress syndrome among preterm infants in US level III/IV neonatal intensive care units. J Pediatr Pharmacol Ther. 2019;24:117-27.
  • Magni T, Ragni C, Pelizzi N, Sharma S, Perez-Kempner L, Turkstra E et al. Health economic studies of surfactant replacement therapy in neonates with respiratory distress syndrome: A systematic literature review. Pharmacoecon Open. 2023;7:359-71.
There are 24 citations in total.

Details

Primary Language English
Subjects Neonatology
Journal Section Research
Authors

Mustafa Şenol Akın 0000-0003-0055-8277

Ufuk Çakır 0000-0002-9409-185X

Publication Date March 31, 2025
Submission Date November 21, 2024
Acceptance Date January 1, 2025
Published in Issue Year 2025 Volume: 50 Issue: 1

Cite

MLA Akın, Mustafa Şenol and Ufuk Çakır. “Cost-Effectiveness Comparison of Three Different Natural Surfactant Preparations in the Treatment of Preterm Infants With Respiratory Distress Syndrome”. Cukurova Medical Journal, vol. 50, no. 1, 2025, pp. 73-82, doi:10.17826/cumj.1589006.