Reema Garegrat, Atul Londhe, Swati Manerkar, Sudhindrashayana Fattepur, Laxmikant Deshmukh, Amol Joshi, Savitha Chandriah, Mallesh Kariyappa, Sahana Devadas, Theranirajan Ethirajan, Kalaivani Srivasan, Chinnathambi Kamalarathnam, Anitha Balachandran, Elango Krishnan, Deepthy Sahayaraj, Prathik Bandiya, Niranjan Shivanna, Constance Burgod, Ashwini Thayyil, Annie Alocious, Marianna Lanza, Pallavi Muraleedharan, Stuti Pant, Harini Venkateswaran, Maria Moreno Morales, Paolo Montaldo, Vaisakh Krishnan, Thaslima Kalathingal, Anagha Rajeev Joshi, Ajay Vare, G C Patil, Babu Peter Satyanathan, Pavan Hapat, Abhishek Deshmukh, Indramma Shivarudhrappa, Manjesh Kurupalya Annayappa, Mythili Baburaj, Christina Muradi, Esprance Fernandes, Nishad Thale, Ismat Jahan, Mohammed Shahidullah, Sadeka Moni Choudhury, Sanjoy Kumer Dey, Sutapa B Neogi, Rupsa Banerjee, Vanessa Rameh, Farah Alobeidi, Ellen Grant, Sandra E Juul, Martin Wilson, Enrico De Vita, Ronit Pressler, Paul Bassett, Seetha Shankaran, Sudhin Thayyil
{"title":"缺氧性缺血性脑病后早期和延长的促红细胞生成素单药治疗：多中心双盲先导随机对照试验。","authors":"Reema Garegrat, Atul Londhe, Swati Manerkar, Sudhindrashayana Fattepur, Laxmikant Deshmukh, Amol Joshi, Savitha Chandriah, Mallesh Kariyappa, Sahana Devadas, Theranirajan Ethirajan, Kalaivani Srivasan, Chinnathambi Kamalarathnam, Anitha Balachandran, Elango Krishnan, Deepthy Sahayaraj, Prathik Bandiya, Niranjan Shivanna, Constance Burgod, Ashwini Thayyil, Annie Alocious, Marianna Lanza, Pallavi Muraleedharan, Stuti Pant, Harini Venkateswaran, Maria Moreno Morales, Paolo Montaldo, Vaisakh Krishnan, Thaslima Kalathingal, Anagha Rajeev Joshi, Ajay Vare, G C Patil, Babu Peter Satyanathan, Pavan Hapat, Abhishek Deshmukh, Indramma Shivarudhrappa, Manjesh Kurupalya Annayappa, Mythili Baburaj, Christina Muradi, Esprance Fernandes, Nishad Thale, Ismat Jahan, Mohammed Shahidullah, Sadeka Moni Choudhury, Sanjoy Kumer Dey, Sutapa B Neogi, Rupsa Banerjee, Vanessa Rameh, Farah Alobeidi, Ellen Grant, Sandra E Juul, Martin Wilson, Enrico De Vita, Ronit Pressler, Paul Bassett, Seetha Shankaran, Sudhin Thayyil","doi":"10.1136/archdischild-2024-327107","DOIUrl":null,"url":null,"abstract":"<p><strong>Objective: </strong>To examine the feasibility of early and extended erythropoietin monotherapy after hypoxic ischaemic encephalopathy (HIE).</p><p><strong>Design: </strong>Double-blind pilot randomised controlled trial.</p><p><strong>Setting: </strong>Eight neonatal units in South Asia.</p><p><strong>Patients: </strong>Neonates (≥36 weeks) with moderate or severe HIE admitted between 31 December 2022 and 3 May 2023.</p><p><strong>Interventions: </strong>Erythropoietin (500 U/kg daily) or to the placebo (sham injections using a screen) within 6 hours of birth and continued for 9 days. MRI at 2 weeks of age.</p><p><strong>Main outcomes and measures: </strong>Feasibility of randomisation, drug administration and assessment of brain injury using MRI.</p><p><strong>Results: </strong>Of the 154 neonates screened, 56 were eligible; 6 declined consent and 50 were recruited; 43 (86%) were inborn. Mean (SD) age at first dose was 4.4 (1.2) hours in erythropoietin and 4.1 (1.0) hours in placebo. Overall mortality at hospital discharge occurred in 5 (19%) vs 11 (46%) (p=0.06), and 3 (13%) vs 9 (40.9%) (p=0.04) among those with moderate encephalopathy in the erythropoietin and placebo groups. Moderate or severe injury to basal ganglia, white matter and cortex occurred in 5 (25%) vs 5 (38.5%); 14 (70%) vs 11 (85%); and 6 (30%) vs 2 (15.4%) in the erythropoietin and placebo group, respectively. Sinus venous thrombosis was seen in two (10%) neonates in the erythropoietin group and none in the control group.</p><p><strong>Conclusions: </strong>Brain injury and mortality after moderate or severe HIE are high in South Asia. 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引用次数: 0
摘要
目的：研究缺氧缺血性脑病（HIE）后早期和延长红细胞生成素单一疗法的可行性：研究缺氧缺血性脑病（HIE）后早期和延长促红细胞生成素单药治疗的可行性：设计：双盲试验性随机对照试验：患者：新生儿（≥36 周）：患者：2022年12月31日至2023年5月3日期间入院的中度或重度HIE新生儿（≥36周）：干预措施：新生儿出生后 6 小时内注射促红细胞生成素（每天 500 U/kg）或安慰剂（使用筛网进行假注射），并持续 9 天。2周大时进行核磁共振成像：主要结果和测量方法：随机化的可行性、给药和使用核磁共振成像评估脑损伤：在筛选出的 154 名新生儿中，56 名符合条件；6 名拒绝同意，50 名被招募；43 名（86%）为先天性。首次服用促红细胞生成素的平均（标清）年龄为 4.4（1.2）小时，服用安慰剂的平均（标清）年龄为 4.1（1.0）小时。红细胞生成素组和安慰剂组的中度脑病患者出院时的总死亡率分别为 5 (19%) vs 11 (46%) (p=0.06)，3 (13%) vs 9 (40.9%) (p=0.04)。在促红细胞生成素组和安慰剂组中，基底节、白质和皮层出现中度或重度损伤的比例分别为：5（25%）vs 5（38.5%）；14（70%）vs 11（85%）；6（30%）vs 2（15.4%）。红细胞生成素组有两名（10%）新生儿出现窦静脉血栓，而对照组则没有：结论：在南亚，中度或重度 HIE 后的脑损伤和死亡率都很高。结论：在南亚，中度或重度 HIE 后的脑损伤和死亡率很高。在这种情况下，使用 MRI 评估促红细胞生成素单一疗法以检查治疗效果是可行的：NCT05395195.
Early and extended erythropoietin monotherapy after hypoxic ischaemic encephalopathy: a multicentre double-blind pilot randomised controlled trial.
Objective: To examine the feasibility of early and extended erythropoietin monotherapy after hypoxic ischaemic encephalopathy (HIE).
Design: Double-blind pilot randomised controlled trial.
Setting: Eight neonatal units in South Asia.
Patients: Neonates (≥36 weeks) with moderate or severe HIE admitted between 31 December 2022 and 3 May 2023.
Interventions: Erythropoietin (500 U/kg daily) or to the placebo (sham injections using a screen) within 6 hours of birth and continued for 9 days. MRI at 2 weeks of age.
Main outcomes and measures: Feasibility of randomisation, drug administration and assessment of brain injury using MRI.
Results: Of the 154 neonates screened, 56 were eligible; 6 declined consent and 50 were recruited; 43 (86%) were inborn. Mean (SD) age at first dose was 4.4 (1.2) hours in erythropoietin and 4.1 (1.0) hours in placebo. Overall mortality at hospital discharge occurred in 5 (19%) vs 11 (46%) (p=0.06), and 3 (13%) vs 9 (40.9%) (p=0.04) among those with moderate encephalopathy in the erythropoietin and placebo groups. Moderate or severe injury to basal ganglia, white matter and cortex occurred in 5 (25%) vs 5 (38.5%); 14 (70%) vs 11 (85%); and 6 (30%) vs 2 (15.4%) in the erythropoietin and placebo group, respectively. Sinus venous thrombosis was seen in two (10%) neonates in the erythropoietin group and none in the control group.
Conclusions: Brain injury and mortality after moderate or severe HIE are high in South Asia. Evaluation of erythropoietin monotherapy using MRI to examine treatment effects is feasible in these settings.
期刊介绍：
Archives of Disease in Childhood is an international peer review journal that aims to keep paediatricians and others up to date with advances in the diagnosis and treatment of childhood diseases as well as advocacy issues such as child protection. It focuses on all aspects of child health and disease from the perinatal period (in the Fetal and Neonatal edition) through to adolescence. ADC includes original research reports, commentaries, reviews of clinical and policy issues, and evidence reports. Areas covered include: community child health, public health, epidemiology, acute paediatrics, advocacy, and ethics.
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