There appears to have been quite a bit of coverage lately on a recent journal article by Grabhorn et al (actually it’s not so recent anymore – took a while for me to investigate it). The Birth and Breastfeeding News blog, an article distributed by Reuters Health, and an update on the neonatalhemochromatosis.org all report on the findings of the paper. The favourable outcomes the paper refers to are all about increased successful outcomes when infants are treated with an antioxidant-chelator therapy and/or receive liver transplantations. The antioxidant-chelator therapy is essentially a cocktail of drugs that help mop up the damaging excess iron and free radicals in the body of babies born with NH.
I’ve attempted to summarise the results from the study as clearly as possible below.
- 4 infants received no treatment at all
- 3 died within 25d, a good outcome for the other
- 5 infants received antioxidant-chelator therapy only
- 1 died at6 weeks, the other four had good outcomes
- 5 infants received antioxidant-chelator therapy followed by liver transplantation
- 1 died at 26 months, the other four had good outcomes
- 2 infants received a liver transplantation
- both infants had good outcomes
This all sounds really good, but you have to bear in mind a few things. In our experience the doctors were not alert to the possibility of NH as a diagnosis (you can’t really blame them, this is a rare disease). In fact Ellen’s diagnosis was only confirmed after an autopsy, so the chances of getting this treatment if no-one is expecting it are low. But for Harry we were alert to the high chance of recurrence. As you can see above the odds are still not great, but they do seem to improve with the antioxidant-chelator therapy.
Liver transplantation has long been known to be a successful way of treating babies with NH, but our advice from doctors was that you would have to be pretty lucky to get one in time.
When we became pregnant with Harry, news of the antioxidant-chelator therapy was the first bit of hope that we might have a healthy baby. I think one of the Doctors gave us a copy of a paper by Flynn et al.
However, like so many things associated with NH, it’s not all black and white. Leonis & Balistreri point out that there are agents within the antioxidant-chelator cocktail that may be potentially dangerous to newborns. They also refer to a paper by Rodrigues et al who studied 19 infants and found outcomes of the 10 infants who received antioxidant-chelator therapy were nearly identicle to the 9 that did not.
But then again… Flynn concluded that:
…Early treatment with antioxidant cocktail is beneficial and may be curative in those who present with milder phenotype [Note: this seems to have been what happened with Harry]. Liver transplantation should always be considered at an early stage in non-responders and in children with more severe acute liver failure.
So what does it all mean? I don’t really know (which is not surprising considering my completely non-extensive medical experience!). Like everything else it just means you need to ask lots of questions… and that there’s always something more behind the quick media releases (but you knew that already).
Finally, the treatment does not appear to have anywhere near the success associated with the IVIg treatment (that Mary had with Harry) which results in babies who present with a much milder form of NH. For the record, Harry received Vitamin E (an antioxidant), but not the complete chelator cocktail, after he was born as part of the standard treatment when his ferritin levels were found to be high.
I understand Dr Whitington will be publishing a new paper updating the progress of the IVIg treatment in the coming months, so I’ll report back then.
- Grabhorn, E., Richter, A., Burdelski, M., Rogiers, X., and Ganschow, R., 2006, “Neonatal Hemochromatosis: Long-term Experience With Favorable Outcome” PEDIATRICS, Vol. 118 No. 5 November 2006, pp. 2060-2065 (doi:10.1542/peds.2006-0908)
- Leonis, M & Balistreri, W, 2005, “Neonatal hemochromatosis: It’s OK to say ‘NO’ to antioxidant-chelator therapy”, Liver Transplantation, Vol 11, Issue 11, pp1323-1325 (doi:10.1002/lt.20541)
- Rodrigues F, Kallas M, Nash R, Cheeseman P, D’Antiga L, Rela M, Heaton N, Mieli-Vergani G, 2005, “Neonatal hemochromatosis-medical treatment vs. transplantation: the Kings experience” Liver Transplantation, Vol 11: pp1417-1424
- Flynn D, Mohan N, McKiernan P, Beath S, Buckels J, Mayer D, Kelly D, 2002, “Progress in treatment and outcome for children with neonatal haemochromatosis”, Archives of Disease in Childhood Fetal and Neonatal Edition, 88:F124