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

被引：9

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

共 50 条

[1] The OPTIMIST-A trial: evaluation of minimally-invasive surfactant therapy in preterm infants 25–28 weeks gestation
Peter A Dargaville
Camille Omar F Kamlin
Antonio G De Paoli
John B Carlin
Francesca Orsini
Roger F Soll
Peter G Davis
BMC Pediatrics, 14
[2] The OPTIMIST-A trial: evaluation of minimally-invasive surfactant therapy in preterm infants 25-28 weeks gestation
Dargaville, Peter A.
Kamlin, Camille Omar F.
De Paoli, Antonio G.
Carlin, John B.
Orsini, Francesca
Soll, Roger F.
Davis, Peter G.
BMC PEDIATRICS, 2014, 14
[3] Effect of Minimally Invasive Surfactant Therapy vs Sham Treatment on Death or Bronchopulmonary Dysplasia in Preterm Infants With Respiratory Distress Syndrome The OPTIMIST-A Randomized Clinical Trial
Dargaville, Peter A.
Kamlin, C. Omar F.
Orsini, Francesca
Wang, Xiaofang
De Paoli, Antonio G.
Kutman, H. Gozde Kanmaz
Cetinkaya, Merih
Kornhauser-Cerar, Lilijana
Derrick, Matthew
Ozkan, Hilal
Hulzebos, Christian, V
Schmolzer, Georg M.
Aiyappan, Ajit
Lemyre, Brigitte
Kuo, Sheree
Rajadurai, Victor S.
O'Shea, Joyce
Biniwale, Manoj
Ramanathan, Rangasamy
Kushnir, Alla
Bader, David
Thomas, Mark R.
Chakraborty, Mallinath
Buksh, Mariam J.
Bhatia, Risha
Sullivan, Carol L.
Shinwell, Eric S.
Dyson, Amanda
Barker, David P.
Kugelman, Amir
Donovan, Tim J.
Tauscher, Markus K.
Murthy, Vadivelam
Ali, Sanoj K. M.
Yossuck, Pete
Clark, Howard W.
Soll, Roger F.
Carlin, John B.
Davis, Peter G.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2021, 326 (24): : 2478 - 2487
[4] MINIMALLY INVASIVE SURFACTANT TREATMENT VERSUS STANDARD THERAPY IN PRETERM INFANTS AT BIRTH (OPTIMIST-A TRIAL): AN ANALYSIS OF INITIAL HOSPITALISATIONCOSTS AND RESOURCE CONSUMPTION
Cox, I
de Graaff, B.
Otahal, P.
Kamlin, O.
Orsini, F.
De Paoli, A.
Clark, H.
Soll, R.
Carlin, J.
Davis, P.
Dargaville, P.
Palmer, A. J.
VALUE IN HEALTH, 2023, 26 (06) : S69 - S69
[5] Outcomes After Minimally Invasive Surfactant Therapy in Preterm Infants
Ko, Shen
Tsai, Shin-Hwa
Hsu, Nin-Chieh
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2024, 331 (04):
[6] Less Invasive Surfactant Administration (LISA) Is Safe: Two-Year Follow-Up of 476 Infants
Herting, E.
Kribs, A.
Roth, B.
Haertel, C.
Goepel, W.
NEONATOLOGY, 2015, 107 (04) : 372 - 373
[7] Outcomes After Minimally Invasive Surfactant Therapy in Preterm Infants-Reply
Dargaville, Peter A.
Carlin, John B.
Davis, Peter G.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2024, 331 (04):
[8] BEVACIZUMAB FOR RETINOPATHY IN PRETERM INFANTS: TWO-YEAR DEVELOPMENTAL FOLLOW-UP
Parker, K.
Zayek, M.
Eyal, F.
Bhat, R.
Rifai, A.
JOURNAL OF INVESTIGATIVE MEDICINE, 2020, 68 (02) : 588 - 588
[9] Efficacy of minimally invasive surfactant therapy in moderate and late preterm infants: A multicentre randomized control trial
Olivier, Francois
Nadeau, Sophie
Belanger, Sylvie
Julien, Anne-Sophie
Masse, Edith
Ali, Nabeel
Caouette, Georges
Piedboeuf, Bruno
PAEDIATRICS & CHILD HEALTH, 2017, 22 (03) : 120 - 124
[10] 2-YEAR FOLLOW-UP OF INFANTS ENROLLED IN A RANDOMIZED TRIAL OF SURFACTANT SUPPLEMENTATION
DUNN, MS
HOSKINS, EM
ENHORNING, G
PEDIATRIC RESEARCH, 1987, 21 (04) : A359 - A359

← 1 2 3 4 5 →