‘Hereditary anemia is very common in countries where iron chelators are limited. With antacids you can therefore still offer an effective treatment there. They are cheap means that are no longer patented. The implementation requires an adjustment of the guidelines, because a doctor does not check every day whether a new study has been published. An article is therefore never enough, even if – as in our study – it has been published in a renowned journal such as the American Journal of Hematology (see below, ed.). We have good contacts with the Thalassaemia International Federation (TIF) in Eurobloodnet, where I am the Research and Trials Coordinator. So far we have only informed the doctors about our results. It’s also an idea to approach the TIF patient representative.”
And the implementation in the Netherlands?
‘As an expert, I am part of groups that look at the guidelines. In it I present our study. Bringing study results to the attention requires lobbying, including within the Dutch Association of Hematology. It is not for nothing that pharmaceutical companies have such a huge marketing budget. An innovation in the field of medicines never sells itself.’
Is there any further research to be done?
‘Our study was about patients who are not dependent on blood transfusions. It would be nice if we could also confirm our results in transfusion-dependent patients. This is particularly relevant because we have found a biomarker in the form of a protein. In patients who have a high level of it – and this is more often the case in transfusion-dependent people – antacids have much less effect. By investigating the influence of the biomarker profile, we could better predict for that group in whom antacids are likely to work well. The good thing is that it is precisely the transfusion-dependent patients who already have a good picture, because they regularly visit the centers of expertise. Then you can do a pragmatic trial, without a placebo. Such a follow-up study does not have to cost that much.’
Any tips for fellow researchers?
‘Take inclusion very seriously and do a good one beforehand feasibility– check. So that you get a clear picture of how many patients you can motivate to participate. We really overestimated that in advance and were fortunately able to solve it with an extra subsidy. Many scientists are dreamers. Very important, but you also have to keep looking businesslike.’
2023-07-17 07:54:50
#Iron #overload #reduced #antacids
