From: Mary-Lisa Orth Subject: [FAPG] EPO before Spinal Surgery To: "FAPG" Date: Wednesday, January 14, 2009, 4:19 PM All - I sanitized the EPO discussion and sent it to Dr. Mark Payne, our FARA cardiologist. His reply is below. I also left the reference to the Cardiac Issues paper because I don't want any of our kids to have surgery without waving that paper in the doctors office, the hospital, and everyplace else. Read it, memorize it, paper your bathroom with it! ============================== Subject: EPO before Spinal Surgery? Mark, There has been some discussion on FAPG about EPO before Spinal Surgery. Can you share your thoughts on this matter? Thanks, Mary-Lisa P.S. I think everyone in the world has a copy of the cardiac issues paper. I know it has been translated into Spanish and French also. Thanks for your help on it. ml ==================================================================== Dear Mary-Lisa: Prospective clinical trials evaluating Epo in this setting are missing, of course. Small, phase II trials have been done (or are ongoing) but they do not provide survival or outcomes data because their duration and size are too small. The scientific rationale (increased FXN) has been demonstrated and provides justification for asking the question. Concurrent with this, increasing hematocrit prior to a surgery that takes a lot of blood is a reasonable approach. Is this a bad thing? Not always. The note below refers to 'thickening the blood', which really means increasing the hematocrit. Is this harmful? Most of the time, no, so long as it is short term in nature. Over the long term (years) it would result in an increase in cardiovascular events, such as stroke and heart attack, and cannot be recommended. But over the short course of a few months prior to surgery, the rise in hematocrit won't matter and a child's heart should tolerate this just fine...assuming it was not severely compromised by FA to begin with.. So, what to do? It would seem reasonable to use Epo to raise the hematocrit prior to surgery in this setting. It may even raise FXN levels sufficiently to make a difference in outcome from surgery, although this cannot be expected. But the point to make to the prescribing hematology doctor is that the child needs scoliosis surgery and will need blood. If transfusions can be avoided, or minimized, then the Epo will have been justified and may even help reduce the degree of 'fluid shifts' caused by administration of numerous fluids and blood transfusions following scoliosis surgery in a child with FA. mark RE: Cardiac issues paper - you are famous, and you deserve it. I see that paper as a perfect example of how such papers should be written. Namely, expertise from multiple disciplines with strong input from the parent. No single author will ever have the impact that multiple experts will have, and the parent or child will always be the strongest motivation for the paper. Nice work.

The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.

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Research projects currently being financed by BabelFAmily

Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:

1) Gene Therapy for Friedreich's Ataxia research project:

https://www.irbbarcelona.org/en/news/international-patient-advocates-partner-to-fund-spanish-gene-therapy-project-to-treat

The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.

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https://www.irbbarcelona.org/en/news/new-research-front-to-tackle-friedreichs-ataxia
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.

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