Hope

Hello all,

We often ask ourselves that due to increased blood transfusion regimen, controlled iron chelation protocol and increased knowledge about thalassemia, are we at the verge of normal life span for patients. Is the management of thalassemia at its peak.

With the advent of fetal haemoglobin inducton techniques, stem cell research, cord blood transplantation method and advancement in BMT, is the cure only 10-15 years away.

We probably seems too optimistic but would be helpful if other members provide their feedback about what they feel after all these developments. In the end we are clinging to hope for survival and this is another way to expedite our hopes.

Regards.

Definitely! Things have become much better now compared to several years ago.

Treatment for Thalassemia can now be provided in countries like Pakistan thanks to the advances and development in medical science. Costs have been reduced thus more people can get treatment. As a result survival rate has increased and doctors no monger term Thalassemia as "Fatal disease"

Advances in medical science in general are occurring at a rapid rate, and specifically, the genetic science that will lead to a cure for thalassemia and many other diseases and disorders is advancing at an incredible rate. In fact, if we can somehow put political leaders in office throughout the world, who believe in peacefully co-existing with the rest of the world and make the decisions to spend public funds on peaceful objectives rather than on the weapons of war, we will see a great transformation in the quality of life for people across our planet. I realize that this is a very big if. Regardless, we will continue to see many major medical breakthroughs in the coming years, partly because there are many dedicated people working on research and partly because there is money to be made developing new treatments.

If you ask the parents of most thals over the age of 20, you will find that almost all were told their child would never live past their early 20's. In most of the world, this is no longer the case because treatment has advanced so much in the past two decades, that thal is now considered to be a manageable condition. The changes in the understanding of thalassemia and its treatment have led us to the point where thals do live and have productive lives. This will only continue to change for the better. We will see cures for genetic disorders in the coming years but trials and perfecting techniques and the subsequent approval will take time. However, I do think in the meantime we will see many changes in thal cure with an emphasis being placed on finding ways to increase one's own blood supply without transfusions, be it with fetal hemoglobin inducers or processes such as retrodifferentiation. We will also see new chelators that are safer and more effective. We may even see some new understanding that creates new approaches, such as the research our own member, Mustho is involved in, in Canada. We are already seeing a revolution in care with the comprehensive care programs offered in some areas. This will continue to spread and more thal centers will adopt this approach.

I know when a parent finds out that their child is thal that is can be a devastating revelation, but parents today will hear a much more positive prognosis than parents back in the 1980's heard. Things are far better and the direction of research is very encouraging. The younger patients today have so much better care than ever before and so much to look forward to in terms of new developments that optimism is indeed warranted. And even for older patients there is reason for hope. When Shilpa, Camillo and I spoke to Dr Sadelain in Dubai, he told us something that gives hope to even the older patients. There is nothing about gene therapy that will exclude older patients the way bone marrow transplants do. Once this is approved, older patients will also be eligible for gene therapy. We can only hope and pray that the trials move quickly and smoothly.

 That's true Andy..... I agree before Thalassemia is such a big scary  blood disorder, that seems no one is aware of it ,but now with the help of associations and groups like this a lot of peple are now aware some way and somehow............. and lot of physician now have encountered a patient with thalassemia...... and soon not now maybe in the near future medical breakthroughs will just surprise us.

That's a relief for sure. I agree with Andy that it would take years before perfecting any cure technique but lets keep our fingers crossed.

Thanks

Lyanne makes a very good point here. In the past, thalassemia was unknown by most people, but as people have migrated to new countries in recent decades, more doctors have been made aware of thal and in recent years, some doctors like Dr Vichinsky, have begun to realize the scope of the world wide thal problem and made huge efforts to expand awareness and design treatment programs. In the US, the number of known thal majors has almost doubled in the past 10 years. In 42 of the 50 US states, testing for hemoglobin disorders is mandatory at birth. Awareness about thal is growing rapidly among medical professionals.

Thalassemia is a much larger problem throughout the world than has previously been recognized. There are tens of thousands of thal major children born in the world each year who get no treatment and die within a few years of birth. As countries modernize, these problems become far more apparent and governments and health organizations begin to realize that much will be needed in the future, as modern societies will not accept the death toll that thal takes on the population. The importance of treatment and a cure will be a given. I believe we will see major changes in awareness about thal and treatment options over the next 25 years. The hemoglobinopathies are the most common genetic disorders on earth and there will be no choice but to address the issues. The era of "thal isn't a big enough problem to get the attention it needs" will soon be over and we all do our part with our efforts in this group and in our personal lives to raise awareness about thalassemia.

I think people out there in the world need knowledge on thalassemia.Most people
i come across have never heard of it,which is understandable as these days there
are so many illnesses out there.
I think people all around the world need to know what it is & thal needs to be "promoted".

Advances in medical science in general are occurring at a rapid rate, and specifically, the genetic science that will lead to a cure for thalassemia and many other diseases and disorders is advancing at an incredible rate. In fact, if we can somehow put political leaders in office throughout the world, who believe in peacefully co-existing with the rest of the world and make the decisions to spend public funds on peaceful objectives rather than on the weapons of war, we will see a great transformation in the quality of life for people across our planet. I realize that this is a very big if.

Yes Andy,

It's a huge if,i wish we can make political leaders understand that world would be a much better place without deadly weapons and with healthy people,anyways,conditions for thals now a days are much hopeful,then 20 years back,i had a cousin who died in her early twenties about 20 years ago,and to be honest when my daughter was diagnosed people told me that it would be the same case,and at that time ,devastated i thought,yes ,may be, who knows,and you can imagine how big the blow was for me, being a mother,i was unable to stop looking at my daughter like she was going to vanish in thin air any time,but then time passed,we saw elder patients,though not in very good state,but the real spirit to fight thal was given to me through this group,through all of you,it was like,entering into a new era of knowledge about thal,and hope also,so i am really thankful to you guys for being there.

HATS OFF TO ALL OF YOU,

LOVE ALWAYS,

ZAINI.

Hello,

The awareness of thalassemia as a manageable condition needs to be in the fore front. When our daughter was first diagnosed we were told by a doctor that she may not survive twenties. We were devastated until we met the haematologist who said forget what others say, your daughter will grow normally and will go to college like other children. Also, when we find this group in MSN and start reading and posting the posts we realized how much advancement has been made in managing thalassemia.

As a matter of fact we still remember Miaki’s first email some two years ago with very powerful words and Andy’s first word of wisdom which put us into great respect for him.

Thanks for your hardwork.

Regards

Thank you Andy for acknowledging my point.   :smile

Canadian Family,

What you said about being told your daughter's early prognosis is still all too common. Far too many medical professionals have a rudimentary understanding of thalassemia at best. It is often left to the patient to educate their own doctor about thal. When Lisa was in the hospital, her general practicioner was coming to our MSN site to learn about thal. I was happy to hear that he found value in our site, but dismayed that a doctor would know little about thal in the first place.

Mass education and awareness programs are needed in many countries in the world, and it may have reached the point where this would be advisable in the US and Canada, due to the amount of Asian immigration that has taken place in the past few decades. Many of these people who are carriers, are not aware they are and often second generation Asian-Americans/Canadians marry other carriers, resulting in the possibility of children who are thal majors. This is exactly the same pattern previously established in the Greek and Italian American populations. Unfortunately, these populations were never aware of thalassemia either A few years ago the number of thal major patients in the US was around 1000. Today it is estimated at 1800. This is a substantial increase in just a few years and will undoubtedly get worse as the second generation children grow and marry. At the very least, we should be targeting these populations with awareness programs and a message that everyone should be tested. It will be far cheaper to educate the public rather than lifetime treatment that majors require. It should be worth it to governments to promote these programs and help prevent the thal population from booming. If nothing else, this cost effective view might get their attention and the attention of the insurance companies.

So much more needs to be done and I feel that eventually, we will see education and awareness programs developed, simply because no one is going to be able to afford the huge medical expenses that will be incurred by a large jump in the number of thals being treated worldwide. In the meantime, we have to nudge and push in that direction as much as possible. The public needs to know about thal and the importance of testing. Testing at birth for all hemoglobinopathies should be mandatory worldwide.

We agree Andy, infact we have seen increased burden on thalassemia clinic at sick kids. New cases comes every now and then. Eight years ago I was told by a doctor that I might have a thalassemia minor and he ordered electrophesis test, completely ignorant about thalassemia or its repercussions I never did that test. I consider myself healthy at that time and thought this may be a normal thing and the doctor is just being too cautious about small blood cell size. Our gynecologist did all sorts of tests on us but he never did thalassemia test. After our daughter case we met with the Gynecologist and told him about the slip off, he is doing the thal test on others (most of his patients are asian and south asian).

The liberal government is now testing all new borns in Ontario for genetic disorders including thalassemia and other hemoglobinopathies but this testing is after the fact, voice needs to be raised to test before the birth. We can do our part by spreading the word but than privacy issues kicks in and people will feel they are unfairly treated or why they should be tested. It’s a difficult issue and needs careful consideration, perhaps we could talk to our MP and bring some facts to him to raise this issue in the parliament.

Atleast we can try.

Regards.

Testing for thal trait at birth is an important tool in the battle but should not be the only tool. It should also be done before high risk groups conceive children, so that they can make informed decisions about having children. Canadian Family, I am glad that you have brought a positive change to your own doctor's practice. This is a wonderful idea and should be practiced universally.

I do support testing at birth because it gives each person access to knowledge about their own genetic makeup, at least in theory. My question about testing at birth is, does this information mean anything in a patient's file if the doctor doesn't understand the implications of being a carrier. If a carrier is never educated to the facts about thal, does it do any good to test at birth? I think we need a constant emphasis on creating awareness and educating the medical professionals. As with Canadian family, what is the point of knowing you're a carrier if it is never explained what this means in terms of your own children's fate? Knowing that both parents should be tested could prevent so many thal major births. I am not saying that a couple should not be allowed to take their chances, but they should at least be aware of the implications and the possibilities so that they can be the ones to decide how they will proceed regarding children. If they choose to accept the risk and the ultimate responsibility of caring for a thal child, it is much better if this is an informed choice. Carrier's not only need to know that they carry the gene, but also what that can mean.

As far as privacy issues, couples are already tested for certain diseases before marriage. This is mandatory and adding thal to the list should not become a privacy issue. People need to know the facts about themselves before making decisions on having children. Working to change things should always be looked at as useful regardless of what the initial outcome may be, for someone new will always be educated about thal as a result.

Andy,

We see your point regarding the genetic testing at birth but the purpose of such testing cannot be accomplished until the results are explained to the person. A great deal of efforts are needed to educate people and urge them to consider genetic testing before having children.

I do support testing at birth because it gives each person access to knowledge about their own genetic makeup, at least in theory. My question about testing at birth is, does this information mean anything in a patient's file if the doctor doesn't understand the implications of being a carrier. If a carrier is never educated to the facts about thal, does it do any good to test at birth?

We are still thinking to meet with our MP and take our chances.

Regards

My grandson was born on Oct 24th this year and he was not tested at birth for blood disorders and we live in Ontario??? At one week old he was admitted to hospital severly dehydrated and now, at 7 weeks old he is now in with ? menengitis? His hgb is 89 and I mentioned to the peds doc that I'm Thal intermedia and so they referred him to hemotologist, but he had not been tested at birth , which city in ontario do these routine tests??

We agree Andy, infact we have seen increased burden on thalassemia clinic at sick kids. New cases comes every now and then. Eight years ago I was told by a doctor that I might have a thalassemia minor and he ordered electrophesis test, completely ignorant about thalassemia or its repercussions I never did that test. I consider myself healthy at that time and thought this may be a normal thing and the doctor is just being too cautious about small blood cell size. Our gynecologist did all sorts of tests on us but he never did thalassemia test. After our daughter case we met with the Gynecologist and told him about the slip off, he is doing the thal test on others (most of his patients are asian and south asian).

The liberal government is now testing all new borns in Ontario for genetic disorders including thalassemia and other hemoglobinopathies but this testing is after the fact, voice needs to be raised to test before the birth. We can do our part by spreading the word but than privacy issues kicks in and people will feel they are unfairly treated or why they should be tested. It’s a difficult issue and needs careful consideration, perhaps we could talk to our MP and bring some facts to him to raise this issue in the parliament.

Atleast we can try.

Regards.