15 December 2010

Avastin Fever

In the last days I had two inquiries from Neonatologists at different level 3 Intensive Care Units enthusiastic about starting to use Avastin instead of laser for primary treatment of ROP.  This is a major topic and I will not adequately treat the controversy tonight, however, I do want to lay out the central question in m mind.  They both attended the Hot Topics in Neonatology conference December 6 - 8 2010 where Dr H. Mintz-Hittner gave a presentation on What's new in ROP.

Background: 1. Laser for ROP is a proven treatment with a 90% success as defined by structural outcome.  It was a prospective randomized trial with 6 years of follow-up and visual outcomes.
2. Avastin is a new molecule created to bind and counteract VEGF (vascular endothelial growth factor).  It is approved by the FDA for colon cancer in order to prevent the growth of blood vessels that nourish the cancer cells, this limits its growth.  In the eye the VEGF is the key player in the development of advanced ROP.

Avastin has a significant effect on ROP and is very promising.  I have used it mainly in cases that were failing standard treatment or had unusual risk factors that made failure likely.  I believe it has helped many of my patients.  However,  I have limited experience with use in primary cases.  I have used it only when laser was impossible.

The BEAT-ROP study was done to compare primary avastin with laser, but the results have not been published and data has not met public scrutiny.  The principle investigator was Dr. Mintz-Hittner and she is pleased with the findings and I gather gave a rather persuasive presentation at the recent meeting.  Still I cannot imagine abandoning a proven effective treatment for Avastin without reading the fine print of the clinical trial.  I must also mention that some Ophthalmologist feel that the anti-VEGF molecule may have a negative effect on development and this remains to be seen.  I suspect mild effects on development since the dose is small and transient, however, it will be difficult to detect this effect among the infants.

October 19 and 20, 2010 I hosted a conference and we discussed the use of Avastin for about 4 hours.  Dr. Mintz-Hittner was a participant and could not present the data because it was embargoed.  I think that the use should start after the report is published.  I hope Dr. Hittner will publish it ASAP.  The amount of effort that she dedicated to this study is an inspiration to me and I feel a little guilty about pushing her to publish.  Nonetheless, I need to see the numbers and fine print and we all need the evidence to replace a scientifically well-established successful treatment.

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