Bone Marrow Transplant of Shaheera Faheem

I am so sorry to hear that. On one moment you know that you are cured and the next moment you are told that your child has developed thal again...really no words to express the grief.

But I agree with everybody. It is better not to put your precious baby in danger again. Shaheera is close to under 3 years or so, correct? So even if you wait 5-10 years, before she turns adult, I am very confident now with the trials that are going these days, a GREAT cure would be found.

I don't want you to go through the pain and "Oh if or only I wished..." again. A relative of mine, I was very small. He even went the BMT successfully of great sibling match donor. But he developed extreme fever and other symptoms and doctors couldn't save him. Today when I meet his mother, she always tells me that only if she didn't went for that BMT.

This was like 20-25 years ago. Things have improved today and you went for BMT nicely and that all is great. But really, risking 2nd time is something I would consider to a very larger scale.

Quality of life of thals has improved today and by the time she steps in the important years of her life, with God's grace hopefully, we will have a rock solid treatments like gene therapy.

Stay strong!

-P.

Dear Friends,

Thanks for encouragement and support.

I have discussed the case of my daughter with Dr. Pietro Sodani and Professor Guido Lucarelli (from Mediterranean Institute of Hematology Italy) and they are pretty hopeful and optimistic for the 2nd Transplant.

Not decided anything yet but for sure will fight till last breath of my life.

They are the most experienced doctors at doing BMT's. Their expertise at post BMT care is especially important.

Hi,

I'd like to add some thoughts about doing or not doing BMT in general (regardless of this second transplant).
Some people in this post have written that is better not to do BMT because Gene Therapy might be a better option in the next years.
 
I've discussed it with Dr. Pietro Sodani and here is point of view:

1) BMT is the only cure, with 30 years of experience and data available
2) BMT is more effective in young children
3) Gene Therapy is still a trial, it will take some years (nobody knows how many, it could be 5-10) before we will know the results and then the long term effects
4) In Southeast Asia thalassemia patients live on average up to 20-25 years, while in western countries it is 40 years or more.
5) We don't know about the cost of Gene Therapy, it could be much higher than BMT.
6) So if today in Southeast Asia (or in the Middle East) you're a parent of a thalassemia child and you don't want to do BMT waiting for Gene Therapy, it might happen that after 5-10 years Gene Therapy is not yet available and for BMT is too late (which means that the risk of death is too high).

So it is anyway a though choice, we just wanted to give some food for thoughts. 

Agreed with most of the statement but point 4 and 5. The concerned child is too young (if I am not mistaken, less that 5 years old). The parents have plenty of time to decide. Even in worst case scenario, the gene therapy fails (5-10 years) the parents can still opt for second BMT in future, why not wait for safer option till then.

I strongly object to your statement "Risk of death is too high". Thalassemia is not considered a fatal condition anymore, it is manageable. I would even not considered it a disease, it is a condition.

I do not agree with your estimation of mortality rates. We have seen people living in thier 50's and in some cases 60's in the west, the children are living much longer in East due to awareness and better management of thal. We are talking about a generation of children who begin their chelation at age of two (unlike those who started when they were in teens or older). Thal children today are expected to live normal life span in future.

However, in the interest of child, I hope the parents will find your comments helpful.

Canadian Family...I could not agree more! Thalassemia is not considered a disease anymore, its more of a disorder. Also, EugenioLaMesa, I am not a Doctor but a parent and those ages you gave (in my opinion) were, with all do respect, way off. Thal patients, not just in North America, are living normal lives due to early treatment and implementation of chelation at an early stage. BMT is starting to become a thing of the past if thal is diagnosed and treated properly. In 2014 time is on the side of the patient and waiting for Gene Therapy is a luxury patients now have.

James

Shareeha maine apki khabr akhbaar main parhi, umeed abhi baqi hai dost plz hamen updated rakhen is forum par ye hamare liye boht important hai...Allah kamyab kare inshAllah.

In english,

I read about your case in newspaper that you want to go for 2nd BMT with Dr. Sodani, hope is still alive and please keep us updated on this forum because it is very very important for us .. May Allah bless us All.

Hi EugenioLaMesa,

  Things have really changed. I'm a thal patient who had 150+ transfusion, experienced with kelfer, desirox and another chelation injection which is directly given in tummy after transfusion. My spleen is not removed.
I'm 26 years old from India, south Asia and I'm leading a normal life except this hip bone joint problem which I'm gonna operate by this year end. And I'm working too for the past 3+ years. Everything is normal. I hope I'll live for 50+. Thal is 100% manageable even in developing countries. Due to my lazyness & lack of knowledge during early 2000 I developed this bone degradattion in my both hips.

Regards,
Tamizhan.

Thalspals,

It seems that some pals misread Mr.Eugenio's message. He did not mean thals could not live healthier lives, rather he pointed out risks and complications involved in Thals life generally. Please have some respect for the gentleman.

I totally agree with EugenioLaMesa at most respect. I have seen both worlds south asian and western. Although I am not at all agree with the risk level of unmatched or unrelated donor. At this moment what i think is gene therapy specifically may not cure me depending up on my multiple mutations where as BMT works irrespective of mutation/s. It hearts me when we think of cure more than prevention. I would like to get the lowest risk available at this moment towards cure. With introduction of gene therapy i except some lowering in price of the BMT. Matching sibling BMT is my choice and will remain until the last day of gene therapy available at nearest bmt hospital.

Jay,

It doesn't matter what the original mutations are. The point of gene therapy is to insert a new working beta globin gene that does the work your current genes cannot. The original gene becomes irrelevant.

thanks Andy it is very blur idea when i see some bluebird videos.

Hi EugenioLaMesa,

  Things have really changed. I'm a thal patient who had 150+ transfusion, experienced with kelfer, desirox and another chelation injection which is directly given in tummy after transfusion. My spleen is not removed.
I'm 26 years old from India, south Asia and I'm leading a normal life except this hip bone joint problem which I'm gonna operate by this year end. And I'm working too for the past 3+ years. Everything is normal. I hope I'll live for 50+. Thal is 100% manageable even in developing countries. Due to my lazyness & lack of knowledge during early 2000 I developed this bone degradattion in my both hips.

Regards,
Tamizhan.

Hi Tamizhan,

Great work! I want to know if you don't mind as to how that hip joint degradation happened. Asking so that we and all other thals can prevent and take extreme care of it. And more than that, be alarmed of early signs.

I really hope you feel better and live a much more awesome life. Don't worry, you'll kick ass for many many more years to come and we will be still chatting on Thalpal like this in 2060 and beyond!

Best,

-P

Dear Shaheera,

How are you and your family?Now how is your baby? You have so much patience which Allah gives you to manage the situation. May Allah always help you and keeps well and good health of your baby with all of ours also..

Best Regards

Hi Pratik,

Before having any problem related to bone, I was told by doctors that I would suffer from joint pain as I was about to start my first iron chelator kelfer in 1997. This affects me and my parents mentaly. And before its start paining I constrained myself from adequate walking, playing needed for that age. This is the first mistake I did. I shouldn't be lazy those days. I should ve adequate walk.
Besides kelfer also played his role. In couple of years after having kelfer, It become difficult for me to sit on the ground folding my legs. It was paining but we didn't take any action against it. It went on till 2009. Meanwhile kelfer was stopped and i was advised to take desifrox as it will cause less damage to bone when compared with kelfer.
Day by day my walking style differs but v didn't notice as there s no much difference to notice. Till now it was knee joint pain only. In 2009, I was supposed to stand for 3 hours for my engineering lab session. That time I felt my first hip joint pain. It was very severe and shocking pain.
I told this to my hematologist even then vitamin d or calcium is not tested and no supplement is advised. It was my 19th age, the age of growth. My bones grows with less bone density.
In June 2009, my transfusion was also stopped as I was re.diagnosed As thala intermedia. So it was like my bone has to grow with less vitamin d, calcium along with 7.4+ HB only. We took x ray in 2009 July and told it was starting stage of degradation and I was advised to do few strengthening exercise which I never did.
After this, my walking became rare. No physical exercise. Hip joint keep on degrading. Pain becoming severe even if I walk for less distance. And I went to orthopedic, they advised me to undergo hip bone joint replacement surgery. They told like avion on both hips. I thought hip joint replacement surgery is not needed but when I found Walking gesture changed, I convinced myself for operation. In 2014 only my hematologist advised me to take calcium supplement. For my past 25 years, I didn't take any supplement other than folic acid. I'm planning for my operation by this November.

Mistakes I did are
1.Before real pain starts I was afraid of pain. Mentally gave way for the pain. Started no to many activities in early age.
2.kelfer, which cause bone problem.
3.lack of exercise, physical activities.
4.living with low HB in my adolescent & teenage which cause low bine density but I'm tall enough.
5.Low supplement for bones. Have the habit of having soda rarely. Whenever I v coke or Pepsi I literally feel my teeth erosion due to it. So I'm having it very rarely.