Update on Lil A

Thanks Carisma,

I have been thinking of you and lil Ari as well    The children's school year is wrapping up and we are moving to a new home in a few days.  I will call you as soon as we get settled.  I hope that everything is going well with you  .

Sharmin

Dear friends,

Lil A has been out of his cast for 3 weeks now.  We leave for Oakland on Sunday.  He will be seen by the group there and then we will drive to LA where he will be seen by Dr. Wood.  We have enrolled him in a pancreas and pituitary study. I hope all of the results will be fine. 

I will bring back any information Dr. Vichinsky and Dr. Wood have to impart during our visit. 

We will miss you all and look forward to getting back to you soon,

Sharmin

Sharmin,

Bon Voyage my friend,i am glad that Lil A is out of his cast,i hope he is feeling better too,i am sure the results will be fine  .Will miss you here  .

Zaini.

Thank you:)  I'll miss you too!  We're gone 2 1/2 weeks and I start school once we return.  It's been a busy time!

I wish you all the best. 

Sharmin

I hope you all are doing well.  Lil A's appt went well.  His SQUID result was 700.  Pretty good considering that we had to take away exjade for two months (due to extreme stomach issues - I would not recommend it under any other circumstances).  We hope the next result is closer to 100 or below. 

Dr. Vichinsky was very very caring and helpful as always.  Lil A's vitamin D is better than before - but Dr. V wants it higher he put us on 50,000 IU a week for 8 weeks.  He also wants lil A to have more zinc.  Although his bone density is in an acceptable range he wants him to build more bone density right now to avoid problems when he is older. 

He was very very optimistic this time around about the future of thals.  About them living full lives, having children and being cured in the near future.  He was optimistic about gene therapy and told us that a new oral chelator will be available next year.  He said that combination chelation will be used a lot more now and that it is most effective and produces the least amount of side effects. 

He insisted that all thal patients and families look into consulting counselling services as children grow and the responsibility shifts from the family to the patient.  He also insisted that doctors talk to the patient rather than the parents at this age. 

I am at a hotel and will write more once I get home. 

I hope this is helpful to all of you.

Best,

Sharmin

Thanks for an update Sharmin,

Good to know everything is fine,regarding Vitamin D ,Few days ago i was talking to my cousin who is a family physician and when i told her that me and my daughter we both take 1000 IU of vitamin D daily,she was like you don't need that much and you shouldn't be taking this much,neither you nor your daughter,i reminded her that me and Z both were deficient according to our blood reports,but she said that body requires only 400 IU daily,not more then that.To be honest,i did become a lil bit concerned when she said this so confidently,but then i remembered how much D Andy takes and has been taking for a long time,so i went on with 1000 IU,and now i am happy that i did,seeing what dose Dr Vichinsky has put Lil A on.

Will wait for your another update

Zaini.

Dr Vichinsky told me exactly the same thing at the NYC conference. They do not see good results turning around vitamin D deficiency on low dose and doses of 50,000 IU were being used to get some real progress. The research on vitamin D is progressing rapidly and what was considered a proper dose just a few years ago is now considered to be useless. Dr Vichinsky's program will ensure that little A's bones are strong during his developmental years and will prevent many problems in his adult years. As thals are growing older, many new problems have come to light, and preventing osteoporosis has become an important goal. With thals, an ounce of prevention is worth a ton of treatment.

Hi Sharni,

so  nice to hear about LilA doing well & his SQUID number is great. Your post is so encouraging. Thank you. Today I was feeling low and came to this site and already feeling uplifted.

I want to take this opportunity to remind everyone why I am so convinced that an annual evaluation is necessary. These small steps being taken with little A will have huge rewards over the years. Making any necessary changes to the treatment program are the result of this annual evaluation and this has long reaching importance. An adjustment here or there may seem like simple things with minor meaning, but they are extremely important for the long term. Do we want patients who are constantly trying to correct situations as adults, or do we want patients who are able to avoid so many of these problems as adults, because their treatment has been modified as needed throughout their younger years.

Never hesitate to get those annual evaluations.

Hi Sharmin and dear friends

I am so happy to hear about the good news of little A, congratulation. Both of you are doing an excellent job and i am happy too that you will be meeting Dr. Wood, he is a wonderful doctor.

I am too very interested in this issue of bone density. On what basis or reference did Dr. Vinchinsky decided that he wanted the the bone density to increase. Is it the DEXA???

Is taking zinc,  the way to increase bone density???

Concerning DEXA, in the last conference i attended, a doctor mentioned in his lecture that DEXA is not reliable for children and that even there are ''normal ranges'' for every ethnic group. He mentioned too that most of the comparison references have to do with the diagnosis of osteoprosis in women in the 50s

Actually i can not recall all his point of view, but what i am sure of is that he said that the clinical observation, is more important and if the child has to do the DEXA, it will be compared with the closest ethnic group and then each time you do a DEXA, you will take the previous one as a reference for the new one. Hope you can understand what i mean

manal

Hi Manal,

Lil A had a Dexa one year ago and he had one during this visit.  We don't have his most recent results yet, but the DEXA done one year ago was normal for a normal child of his age.  I don't know if they took into account his ethnicity.  Dr. Vichinsky felt that it would benefit Lil A to get his bone density higher so that he does not have problems over time. 

In order to increase bone density his clinic has done several studies.  Zinc on its own has had significant affects on increasing bone density.  His clinical nurse told me to put Lil A on 20 to 25mg of Zinc a day. 

Another factor in increasing bone density is vitamin D - vitamin D is very helpful for many many aspects of thalassemia.  Calcium, magnesium and trace minerals are also very important.  They stress milk and milk products a lot in Oakland.  We were given many pamphlets on the importance of milk.  They suggest to take lactaid with milk if milk upsets your stomach.  They find that milk and milk products significantly increase your bone density. 

Also, very importantly keeping up with transfusions - keeping a hemoglobin near 10 (at least above 90) in transfused thals is very important as it prevents the marrow from becoming unnecessarily active and making retics.  This process weakens the bone from within as the marrow expands. 

I hope this information helps, we are still in California.  I will write more once we get home. 

Best,

Sharmin

We are still waiting to get results for the pancrease and pituitary study.  The purpose of this study is to learn about iron deposit in these organs and the effect on their size and function.  Hopefully Lil A's participation in this study will be helpful to others. 

As Andy has said many times, getting the iron levels as low as possible is best to prevent impact on the function of these organs and possibly reverse the tissue damage by revoming iron and restoring function.  Getting the iron levels as low low low as possible is very important.  As Lena has told us - combination chelation is key - iron levels as low and close to normal as possible are best in order to avoid any iron related problems. 

As for this new chelation medcation: This doctor means that this new medicine will enter the lastest phaes of approval? Next year we can expect trials of a new medicine?

This brings me by Novatis newest application for Iphone.It is made for professionals to calculate how much mg Exjade a patient should get. At first I liked it untill I typed '500' as ferritin level. There was no advice because of the low level. A level of 500 was seen as healthy.

I also have heard that Dexa's by children are not reliable. As for vit D, there are more and more voices which are saying that the standards of vit D should be review.

I am glad to hear there is such study because I am very interested in that field of study. Please, share more details when you got them. I am glad to hear little A is doing well. Did you have a nice time there as well? (outside hosp.)

I am sorry, but I do not believe in milk. The most part of our world population is allergic for it.Here in Netherlands it was the industry who paid all the campagnes to let us consume more dairy products.

Sharmin,
i'm glad to see his latest results ... i wish himm all the best of everything ....

Best Regards
Take Care
Umair

Thanks Sharmin, this was very helpful. Hope you are all doing great

manal