Lena,
Thanks for sharing and yes, this is exactly what I mean about sharing your experience. Knowing what is truly possible is the best way for our group to be effective. It's easy to talk but when we can demonstrate the results of actions, it means so much to others who are dealing with these same challenges. What you have told us should be a wake up call for any adult thals who think that there is nothing that can be done to ward off the inevitable, and a statement of hope to all those struggling with being parents of thal children and who are uncertain about the future of their children. With dedication to compliance, a normal life can be achieved. Yes, there are many things that have to be done to ensure good health in thals but it can be done with work and this is the message everyone needs to learn from the experience of Lena and our older thal brothers and sisters.
What seems like a simple little post here actually touches on many subjects. In a few lines we see not only Lena's history, but a history of the treatment of thalassemia. When desferal first became available to Lena, who was a woman in her early twenties at that time, and who may have understood that her days were surely numbered, because non-chelating thals live only into their mid-twenties if they are lucky, Lena embraced that pump and the chance it gave her to live. Desferal and the pump gave Lena a chance to have a real life. What has always disturbed me since I first got involved with thal is the fact that so many thals choose a different path and pay for it with their lives. One of my earliest posts at our MSN site dealt with this. In an interview with Shilpa, this reality was addressed. Why do thals refuse the treatment that could save their lives? As it turns out, there are many reasons. Some of these I saw in Lisa. I think one of the things that bothers me most about what Lisa went through was that she really did not believe a thal could live longer. I wish I had known some of the older thals back then because I also didn't really know any better. Lisa's dad, who lived into his late 40's seemed an aberration and we surely could not expect this potential for thals in general. In addition, Lisa reacted badly to desferal. Her description of the pain and burning when using desferal made it easier for me to understand why one could neglect this life-saving drug. At the time, deferiprone had been tested in the US and Lisa had been in the trial, but this came to a quick halt and the only option was desferal and Lisa just could not stand taking it often enough to keep her ferritin in check. Shilpa, on the other hand, enlightened me about the host of cultural issues that can make it so hard for thals to gain acceptance in their cultures. Lena may have dealt with this in Greece, as thalassemia was actually known as the "stigma" and indeed held a stigma. Shilpa told me how thals are not accepted in India and I soon learned that the same situation exists in many countries, even in the Maldives where 18% of the population carry thal trait. I find it amazing that people can be so obtuse when they may even be carriers themselves, but this is the reality. Most parents in Maldives will not allow their child to even date a thal major, let alone marry one. This one issue is perhaps the most important outcome of the lack of social acceptance of thals that exists in many cultures around the globe. Why don't so many thals do what Lena has so faithfully followed for the past 25 years? Why won't they chelate? Of course we have the obvious reason that needles are painful and desferal does irritate. But is this enough reason to give one's life away? For most who don't chelate, the answer to that is no. The answer is more like "why poke a needle in your skin 5-6 days a week when life has nothing to offer a thal?" When your prospects in life are severely limited, what is the incentive to stay alive? When your options in life do not include relationships and marriage, education and careers and so forth, life can look pretty bleak and not worth the effort. I will say that oral chelation and the ease of using it, has somewhat changed this for many people, but the non-acceptance by society is still a major issue that does affect how many thals view their lives, leaving them with little reason to go on. This remains a major challenge and honestly, if the world is going to deal with this most prevalent genetic disorder, a change will have to come on a cultural level also. In a society such as what exists in India, marriage is so highly regarded that people often feel like their lives have no meaning unless they can marry. Living in the US, where so many people choose to either remain single or return to being single after failed marriages, this emphasis on the importance of marriage is not so easily understood as a life or death matter, but for a thal living in India, this is what it can become. In addition, thals are often seen as unemployable in India, leading to a situation where thals often have to hide their status if they hope to gain employment. For a woman like Gurleen Bedi, who was very small in stature and whose condition made itself obvious, it became a near impossibility to find employment in spite of stellar academic achievements. I have been told that doctors in India do not want to tell even minors about their status because it may lead to reduced opportunities for marriage. Obviously, we have a monumental task in the areas of education and awareness about thalassemia and creating acceptance of thals everywhere. But change does happen, just as it does in the area of treatment and I see some cracks in the status quo. When Maako and Akka, both from Maldives, married, we saw some evidence that for some in the Maldives, things are beginning to change, and it does give us hope that eventually, the world will recognize that thalassemia is both widespread and manageable. There is a lot of work to be done and we can all do our part in advancing understanding about thalassemia and creating more acceptance for thals everywhere. This can happen and it make take involvement on a huge scale by an entity like the Bill and Melinda Gates Foundation (keep writing them because the more we explain thalassemia to them, the more likelihood that it will eventually get some attention) but at some point the world will have to confront the reality that there are hundreds of millions of thal carriers in the world and that without awareness, we are in for a problem of tremendous proportions. As countries try to deal with this health issue, a realization that education and awareness are the keys will be made and as awareness is created, thals will become more accepted simply because it won't seem like an unknown to others. Creating awareness does bring acceptance.
Another thing I want people to think about is how much things change in regards to treatment and managing thalassemia. We often hear frustration about the slow pace of developments but think about this. 25 years ago, Lena started on desferal. Since then we have also seen the introduction of deferiprone and Exjade as chelators. We have also seen great strides made in the diagnostic tools used with thal. Things like Ferriscan and T2* are making it so much easier for doctors to identify and correct problems. Our understanding of the nutritional needs in thalassemia is growing by leaps and bounds. Yes, as far as a cure, BMT is still the only answer but there are so many new therapies on the horizon and even another potential cure with gene therapy, and we see that the complaint that it is taking so long for anything new to develop just does not stand up to reality. In those 25 years, things have changed drastically for thal treatment and in the next 25 years we will see far more. A thal born 40 years ago had no real prospect of living beyond 25 but that has all changed. Thals are living into their 40's and 50's and probably beyond that and no one really even knows now what we can expect given the treatment that is standard in today's world. This is simply amazing and when you think about all the diseases and disorders that exist in the world and how really slow progress is made in treating and curing these health problems, we see that the changes in treatment of thalassemia have indeed been great. Step back and be objective and patient and you have to agree that things have developed at a rapid pace and more is on its way. Thals born today have a much better outlook for their lives than when Lena was born, so let's acknowledge this and avoid the negative thoughts that do creep in. Let's not complain about the slowness of new developments because if you're honest you will have to admit that as far as thalassemia is concerned, developments in treatment have happened at a rapid pace. Let's not bog ourselves down with negativity when we should be looking forward to life. Let's celebrate Lena's achievements and also let us recognize that determination and hard work are keys to success. The enemy may be around the corner but that enemy can be kept at bay and rendered toothless with good management and compliance.