Department of Paediatrics, Christchurch School of Medicine and Health Sciences, University of Otago Christchurch, PO Box 4345, Christchurch 8140, New Zealand.
Arch Dis Child Fetal Neonatal Ed. 2013 Mar;98(2):F170-4. doi: 10.1136/archdischild-2011-301148. Epub 2011 Dec 30.
The publication of the BEAT-ROP study of bevacizumab (Avastin) treatment for Zone I and II retinopathy of prematurity (ROP) has raised hopes that there might now be a simpler, cheaper and more effective treatment than laser therapy, the current standard of care. However, we would urge caution at this point in time. We review the scientific background to the use of intravitreal anti-vascular endothelial growth factor for ROP, highlight a number of design issues in the BEAT-ROP study and problems with interpretation of the results. For example, no visual outcomes were reported and the study was underpowered to assess longer term safety. Intravitreal bevacizumab leaks into the systemic circulation in animals and adult humans and there are real concerns of potential harm to the developing preterm infant because vascular growth factors play a critical role in organogenesis. We conclude that bevacizumab should be reserved for exceptional circumstances and compassionate use pending further studies. Laser remains the proven effective therapy for first line treatment of all forms of ROP with little systemic morbidity. Neonatology and ophthalmology have an impressive record of conducting collaborative multicentre studies and we urgently need further rigorously designed, adequately powered randomised trials of anti-VEGF agents that evaluate visual outcomes as well as short and long term ocular and systemic safety.
BEAT-ROP 研究发表了贝伐单抗（阿瓦斯汀）治疗 I 区和 II 区早产儿视网膜病变（ROP）的结果，这给人带来了新的希望，可能会有比激光治疗更简单、更便宜、更有效的治疗方法，成为目前的标准治疗。然而，在这个时候我们还是需要谨慎。我们回顾了眼内抗血管内皮生长因子治疗 ROP 的科学背景，强调了 BEAT-ROP 研究中的一些设计问题，以及对结果的解释存在问题。例如，该研究没有报告视力结果，并且该研究在评估长期安全性方面的效力不足。贝伐单抗在动物和成年人体内会渗漏到全身循环中，因为血管生长因子在器官发生中起着关键作用，因此人们真正担心对发育中的早产儿可能会造成潜在的伤害。我们的结论是，贝伐单抗应保留用于特殊情况和同情使用，等待进一步的研究。激光仍然是所有形式的 ROP 的一线治疗方法，具有可靠的疗效，全身发病率低。新生儿学和眼科学在进行合作的多中心研究方面有着令人印象深刻的记录，我们迫切需要进一步进行设计严格、充分有力的抗 VEGF 药物随机试验，评估视觉结果以及短期和长期的眼部和全身安全性。
