Two-Year Outcomes After Minimally Invasive Surfactant Therapy in Preterm Infants Follow-Up of the OPTIMIST-A Randomized Clinical Trial

被引：13

作者：

Dargaville, Peter A. ^{[1
,2
]}

Kamlin, C. Omar F. ^{[3
,4
]}

Orsini, Francesca ^{[5
]}

Wang, Xiaofang ^{[5
]}

De Paoli, Antonio G. ^{[2
]}

Kanmaz Kutman, H. Gozde ^{[6
]}

Cetinkaya, Merih ^{[7
]}

Kornhauser-Cerar, Lilijana ^{[8
]}

Derrick, Matthew ^{[9
]}

Ozkan, Hilal ^{[10
]}

Hulzebos, Christian V. ^{[11
]}

Schmoelzer, Georg M. ^{[12
]}

Aiyappan, Ajit ^{[13
]}

Lemyre, Brigitte ^{[14
]}

Kuo, Sheree ^{[15
]}

Rajadurai, Victor S. ^{[16
]}

O'Shea, Joyce ^{[17
]}

Biniwale, Manoj ^{[18
,19
]}

Ramanathan, Rangasamy ^{[18
,19
]}

Kushnir, Alla ^{[20
]}

Bader, David ^{[21
]}

Thomas, Mark R. ^{[22
]}

Chakraborty, Mallinath ^{[23
]}

Buksh, Mariam J. ^{[24
]}

Bhatia, Risha ^{[25
]}

Sullivan, Carol L. ^{[26
]}

Shinwell, Eric S. ^{[27
]}

Dyson, Amanda ^{[28
]}

Barker, David P. ^{[29
]}

Kugelman, Amir ^{[30
]}

Donovan, Tim J. ^{[31
]}

Goss, Kevin C. W. ^{[32
]}

Tauscher, Markus K. ^{[33
]}

Murthy, Vadivelam ^{[34
]}

Ali, Sanoj K. M. ^{[35
]}

Clark, Howard W. ^{[36
]}

Soll, Roger F. ^{[37
]}

Johnson, Samantha ^{[38
]}

Cheong, Jeanie L. Y. ^{[3
,4
,39
]}

Carlin, John B. ^{[5
,40
]}

Davis, Peter G. ^{[3
,4
]}

机构：

[1] Univ Tasmania, Menzies Inst Med Res, Hobart, Tas, Australia

[2] Royal Hobart Hosp, Dept Paediat, Hobart, Tas, Australia

[3] Royal Hosp Women, Neonatal Serv, Melbourne, Vic, Australia

[4] Univ Melbourne, Dept Obstet & Gynaecol, Melbourne, Vic, Australia

[5] Murdoch Childrens Res Inst, Clin Epidemiol & Biostat Unit, Melbourne, Vic, Australia

[6] Zekai Tahir Burak Matern Teaching Hosp, Dept Neonatol, Ankara, Turkiye

[7] Istanbul Kanuni Sultan Suleyman Training & Res Ho, Div Neonatol, Dept Pediat, Istanbul, Turkiye

[8] Univ Med Ctr, Div Gynaecol & Obstet, Dept Perinatol, Ljubljana, Slovenia

[9] Northshore Univ Hlth Syst, Div Neonatol, Evanston, IL USA

[10] Uludag Univ, Div Neonatol, Dept Pediat, Fac Med, Bursa, Turkiye

[11] Univ Med Ctr Groningen, Div Neonatol, Beatrix Childrens Hosp, Groningen, Netherlands

[12] Univ Alberta, Div Neonatol, Dept Pediat, Edmonton, AB, Canada

[13] Mercy Hosp Women, Neonatal Serv, Heidelberg, Vic, Australia

[14] Ottawa Hosp, Dept Obstet Gynecol & Newborn Care, Ottawa, ON, Canada

[15] Kapiolani Med Ctr Women & Children, Dept Pediat, Honolulu, HI USA

[16] KK Womens & Childrens Hosp, Duke NUS Med Sch, Dept Neonatol, Singapore, Singapore

[17] Royal Hosp Children, Neonatal Unit, Glasgow, Lanark, Scotland

[18] Los Angeles Cty USC Med Ctr, Div Neonatol, Dept Pediat, Los Angeles, CA USA

[19] USC, Good Samaritan Hosp, Keck Sch Med, Los Angeles, CA USA

[20] Cooper Univ Hlth Care, Dept Pediat, Childrens Reg Hosp, Camden, NJ USA

[21] Technion, Dept Neonatol, Rappaport Fac Med, Bnai Zion Med Ctr, Haifa, Israel

[22] Chelsea & Westminster Hosp NHS Fdn Trust, Dept Neonatal Med, London, England

[23] Univ Hosp Wales, Reg Neonatal Intens Care Unit, Cardiff, Wales

[24] Auckland Hosp, Newborn Serv, Starship Child Hlth, Auckland, New Zealand

[25] Monash Childrens Hosp, Monash Newborn, Clayton, Vic, Australia

[26] Singleton Hosp, Dept Neonatol, Swansea, W Glam, Wales

[27] Bar Ilan Univ, Ziv Med Ctr, Dept Neonatol, Fac Med, Safed, Israel

[28] Canberra Hosp, Dept Neonatol, Centenary Hosp Women & Children, Woden, ACT, Australia

[29] Dunedin Publ Hosp, Neonatal Intens Care Unit, Dunedin, New Zealand

[30] Technion, Rappaport Fac Med, Dept Neonatol, Rambam Med Ctr, Haifa, Israel

[31] Royal Brisbane & Womens Hosp, Div Neonatol, Brisbane, Qld, Australia

[32] Princess Anne Hosp, Neonatal Intens Care Unit, Southampton, Hants, England

[33] Ascens St Vincent, Div Neonatol, Peyton Manning Childrens Hosp, Indianapolis, IN USA

[34] Royal London Hosp Barts Hlth NHS Fdn Trust, Neonatal Intens Care Ctr, London, England

[35] Sidra Med, Div Neonatol, Doha, Qatar

[36] UCL, Fac Populat Hlth Sci, Neonatol, EGA Inst Womens Hlth, London, England

[37] Univ Vermont, Larner Coll Med, Div Neonatal Perinatal Med, Burlington, VT 05405 USA

[38] Univ Leicester, Infant Mortal & Morbid Studies Res Grp, Dept Populat Hlth Sci, Leicester, Leics, England

[39] Murdoch Childrens Res Inst, Clin Sci, Melbourne, Vic, Australia

[40] Univ Melbourne, Dept Paediat, Melbourne, Vic, Australia

来源：

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION | 2023年 / 330卷 / 11期

基金：

英国医学研究理事会;

关键词：

CHRONIC LUNG-DISEASE; RESPIRATORY OUTCOMES; VENTILATION; CHILDREN; RATES; AGE;

D O I：

10.1001/jama.2023.15694

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

IMPORTANCE The long-term effects of surfactant administration via a thin catheter (minimally invasive surfactant therapy [MIST]) in preterm infants with respiratory distress syndrome remain to be definitively clarified. OBJECTIVE To examine the effect of MIST on death or neurodevelopmental disability (NDD) at 2 years' corrected age. DESIGN, SETTING, AND PARTICIPANTS Follow-up study of a randomized clinical trial with blinding of clinicians and outcome assessors conducted in 33 tertiary-level neonatal intensive care units in 11 countries. The trial included 486 infants with a gestational age of 25 to 28 weeks supported with continuous positive airway pressure (CPAP). Collection of follow-up data at 2 years' corrected age was completed on December 9, 2022. INTERVENTIONS Infants assigned to MIST (n = 242) received exogenous surfactant (200 mg/kg poractant alfa) via a thin catheter; those assigned to the control group (n = 244) received sham treatment. MAIN OUTCOMES AND MEASURES The key secondary outcome of death or moderate to severe NDD was assessed at 2 years' corrected age. Other secondary outcomes included components of this composite outcome, as well as hospitalizations for respiratory illness and parent-reported wheezing or breathing difficulty in the first 2 years. RESULTS Among the 486 infants randomized, 453 had follow-up data available (median gestation, 27.3 weeks; 228 females [50.3%]); data on the key secondary outcome were available in 434 infants. Death or NDD occurred in 78 infants (36.3%) in the MIST group and 79 (36.1%) in the control group (risk difference, 0%[95% CI, -7.6% to 7.7%]; relative risk [RR], 1.0 [95% CI, 0.81-1.24]); components of this outcome did not differ significantly between groups. Secondary respiratory outcomes favored the MIST group. Hospitalization with respiratory illness occurred in 49 infants (25.1%) in the MIST group vs 78 (38.2%) in the control group (RR, 0.66 [95% CI, 0.54-0.81]) and parent-reported wheezing or breathing difficulty in 73 (40.6%) vs 104 (53.6%), respectively (RR, 0.76 [95% CI, 0.63-0.90]). CONCLUSIONS AND RELEVANCE In this follow-up study of a randomized clinical trial of preterm infants with respiratory distress syndrome supported with CPAP, MIST compared with sham treatment did not reduce the incidence of death or NDD by 2 years of age. However, infants who received MIST had lower rates of adverse respiratory outcomes during their first 2 years of life.

引用

页码：1054 / 1063

页数：10

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