New to this forum - Seek Andy's advice

  • 11 Replies
  • 11480 Views
*

Offline SYT

  • *
  • 7
New to this forum - Seek Andy's advice
« on: February 22, 2012, 04:05:04 PM »
Dear Andy,

My daughter is 11 months old with thal major.  Up to the moment, she is not required tranfusion as she can keep her HB level at 8-9 and it increases further to 9.5 after having fobic acid recently.  As her HB has not dropped lower, I wonder whether she is an intermedia. Following is the DNA report on her cord blood sample collected when she born :

HbA : 0
HbA2 : <1.0  L%
HbF : >90.0 H%
Hamoglobin H Bodies : Negative %
Codons 41/42 (-CTTT) Beta-zero Thalassaemia mutation

She also did 2nd DNA test when she was 7 months.  The doctor confirm again she is major without alpha deletion.

Andy, could you give me some advice :

1.  For major, why her HB did not drop lower ?
2.  Will she require regular transfusion later (maybe 2 or 3 years old) as advised by the doctor?  
3.  Any chance becoming intermedia ?
4.  Other than fobric acid, what medicine or supplement can help her ?
5.  If she keep no transfusion, how we can avoid the change of her face stucture ?  

 




 

Re: New to this forum - Seek Andy's advice
« Reply #1 on: February 22, 2012, 05:26:19 PM »
hi

Welcome!

Do u have any electrophoresis report. (current, not at the age of 6 month or prior)
thal mojor means no HB A
HbA2 should be more than 5-8% aprox
and rest is HBF

During  the birth to 6month aprox the main portion is HBF  as report shows also.
If homozygous beta deletion detected then she must be a thal mojor but what is her current HBA2?
Generally thal Major need Blood transfusion at the age of 6-8 months, so I beleive you should wait and see.

level of Hb seems to be intermedia though I m not expert or profesional this are only my net experience.

Hope Andy will through some light.

All the best
Dip


*

Offline SYT

  • *
  • 7
Re: New to this forum - Seek Andy's advice
« Reply #2 on: February 23, 2012, 01:56:19 AM »
Dear Dip,

Thanks for your advice.

I do not have her current report on hand.  I will try to ask her doctor when we meet him again in March.

Yes, her doctor also said her HB drop status is slower than their experienced for major.  So we can only wait and see.  I am confused if there is variant of the severity level for individual major. Anyone has similar experience ? (HB level drops and need transfusion later i.e. 1 year old or later).

Thanks again and have a good day.

SYT   

*

Offline Andy Battaglia

  • *****
  • 8793
  • Gender: Male
  • Will thal rule you or will you rule thal?
Re: New to this forum - Seek Andy's advice
« Reply #3 on: February 23, 2012, 03:37:54 PM »
Hi SYT,

I have been researching the gene deletion, but have found nothing that would indicate that anything other than thal major can be expected if both gene mutations are codons 41/42. However, there seem to be few case studies. I will reply more fully later.
Andy

All we are saying is give thals a chance.

*

Offline SYT

  • *
  • 7
Re: New to this forum - Seek Andy's advice
« Reply #4 on: February 23, 2012, 04:03:06 PM »
Dear Andy,

Please let me know more once you have full information.

Thanks,
SYT

*

Offline Andy Battaglia

  • *****
  • 8793
  • Gender: Male
  • Will thal rule you or will you rule thal?
Re: New to this forum - Seek Andy's advice
« Reply #5 on: February 25, 2012, 04:18:04 AM »
Hi SYT,

Typically, beta° thal majors need transfusions at an early age, and once diagnosed at or before birth, the usual response is to begin transfusions as soon as the Hb drops significantly, because there is no advantage in delaying transfusion, as low Hb will impair normal development. For a beta° major to have an Hb above 9 at 11 months is unusual, unless some moderating factor such as coinciding alpha thal trait is also present. Since alpha trait has been ruled out, unless the Hb does suddenly drop, as would be expected with beta°, some other moderators might be investigated. These include Xmn I polymorphism and variants in the  BCL11A, and HBS1L-MYB genes. These all can cause an increase in fetal hemoglobin to the point where there are known cases of beta° thals who do not transfuse and manifest as thal intermedia. I have no way of knowing if any of these may be present, but the fact is that by this age, most infants have levels of HbF below 10%, which by itself would represent a total Hb of around 1.5% if no other type of hemoglobin was present. Perhaps there is another reason for the persistence of fetal hemoglobin at this age. Over the next few months it will be apparent if any major changes take place and transfusions will begin, but if things continue as is an explanation in terms of a fetal hemoglobin moderator should be explored.

1. I cannot tell you at this point why the Hb hasn't dropped much lower. I have suggested possibilities above.
2. She may require transfusions and if so, it may happen within a couple months. If not, a fetal hemoglobin moderator may be present and transfusions may be delayed longer as with thal intermedia, or, as seen with some recorded cases involving these moderators, transfusions may be only necessary in a transient manner, only when Hb has dropped due to illness. My first reaction on reading your post was that there was no way this child can be phenotyped as anything other than major, but after more reading on the subject, I will say there is some small chance that the current Hb level may be the result of a fetal hemoglobin moderating factor. Because historically, most of these cases have not been reported or analyzed because they had no reason to suspect a genetic thal major, there is no way to know how common these factors appear in thal majors.
3. Possible intermedia? It is possible but under current levels of understanding, it is not common. As I said, because these cases don't manifest physically as major, they have not been recorded for the most part, so I don't think anyone can give a percentage chance. Right now, the child's Hb is what one might expect for a thal intermedia.
4. You have already seen the value of folic acid and this should be continued. My suggestions at this age are few. I would suggest 50-100 IU daily of natural vitamin E complex. I would also suggest monitoring of vitamin D levels and intervention if needed. Both D and E are commonly found to be deficient in thals. Follow a good diet. Avoid high iron foods as long as the child is not transfusing, as the body will try to absorb more iron from diet than is needed. Establish good eating habits at a young age, with emphasis on nutrition and avoidance of highly processed and junk foods. Wheatgrass has also been shown to aid fetal hemoglobin production in some thals, so this might also be considered in some form. As she gets a bit older, I would suggest adding a daily B complex vitamin.
5. In cases of non-transfusing intermedias, changes in facial features are one of the signs that transfusion is needed. The only way to avoid this is by keeping the Hb up. Some patients can manage with Hb levels of just above 7, while others will have more symptoms and changes in bone development unless their Hb is kept higher through transfusions.

If the saying time will tell was ever relevant, it is here. She is at an age where the lack of a drop in hemoglobin level gives some hope, but does not rule out the expected thal major. I would prepare mentally for transfusions, as it is most likely, but be aware of the small possibility of a manifestation of intermedia. Keep in mind that many intermedias also do have to begin transfusions at some point. 

This little note got me digging deeper and I do wonder why the word "usually" is used. It is unusual to see beta° phenotype to be expressed as usually. It is normally expressed as "Severe transfusion-dependent beta-thal."
http://globin.bx.psu.edu/cgi-bin/hbvar/query_vars3?mode=output&display_format=page&i=849
Quote
Codons 41/42
Homozygote   
Usually transfusion-dependent
Andy

All we are saying is give thals a chance.

*

Offline SYT

  • *
  • 7
Re: New to this forum - Seek Andy's advice
« Reply #6 on: February 25, 2012, 02:54:51 PM »
Dear Andy,

Thank you so much for your useful information and your explanation on these possibilities.  Actually, I have not heard before about the moderators other than alpha such as Xmn I polymorphism and variants in the  BCL11A, and HBS1L-MYB genes.

Yes, my thought is as you advised, to make well preparation for transfusion.  I hope there is still an exception in my daughter's case. 

Meanwhile, Andy, if I want to let my daughter try wheatgrass juice later.  What is the age and dose per day you recommend ?  What brand of wheatgrass juice is most effective for thal and the taste is okay  ?  If it is not convenient to tell me the brand here, could you please e-mail to me ?

Thank you again for all your kind advices.

SYT

   

*

Offline Andy Battaglia

  • *****
  • 8793
  • Gender: Male
  • Will thal rule you or will you rule thal?
Re: New to this forum - Seek Andy's advice
« Reply #7 on: February 25, 2012, 06:58:52 PM »
SYT,

At a young age, wheatgrass shots, which are an extract, would be the only way to give wheatgrass. Check the www.drwheatgrass.com website. One bottle will last two months for a child.
Andy

All we are saying is give thals a chance.

*

Offline SYT

  • *
  • 7
Re: New to this forum - Seek Andy's advice
« Reply #8 on: February 26, 2012, 07:30:35 AM »
Thank you so much, Andy.   

I will update you my daughter's status few months later.

SYT


*

Offline SYT

  • *
  • 7
Re: New to this forum - Seek Andy's advice
« Reply #9 on: March 30, 2012, 04:23:48 PM »
Dear Andy,

My daughter's hb level was dropped a bit to 8 & 7.5 recently.  Doctor suggests her to start transfusion as earliest as possible as it is benefitical to her and whatever the DNA or electrophoresis test done in her 7 months old showing she is a major (HBA : 0%, HBF : 98%). 

He also explained to me that maybe there is still some fetal hemoglobin is still functioning so that she can delay her transfusion till this moment (1 yr old).  However, it will be used up one day!!!

Andy, how do you think to start transfusion at this moment ?  Should I wait and see ?   Meanwhile, if the fetal hemoglobin moderator as you advised is explored, will it be persist for long time therefore no adult HB is required ?  What checking can be done to find out the fetal hemoglobin moderators ?   

Many thanks again for your kind advice.

SYT
     

*

Offline Andy Battaglia

  • *****
  • 8793
  • Gender: Male
  • Will thal rule you or will you rule thal?
Re: New to this forum - Seek Andy's advice
« Reply #10 on: March 31, 2012, 12:25:58 AM »
SYT,

There has been a continuing drop in Hb and it has dropped almost to the point where you will expect transfusions to begin. I am concerned about failure to thrive if transfusions don't start soon. Once the pattern of constantly dropping Hb is established, there is no point in holding out, as not transfusing will begin to affect development. Once this occurs, there is no longer any point to refusing transfusion.

I will continue to keep the group updated about new advancements, including the progress of trials for fetal hemoglobin inducing drugs. There are other novel approaches to treating thalassemia that are being researched that may eventually lead to completely different methods of treatment, so that transfusions will no longer be required. We have to be very patient because proving safety and effectiveness and getting FDA approval do take years, but there will be huge changes in the next 10-20 years. The important thing for all thals today is to take good care of themselves so that they can take advantage of the rapid advances in treatment for thalassemia.
Andy

All we are saying is give thals a chance.

*

Offline SYT

  • *
  • 7
Re: New to this forum - Seek Andy's advice
« Reply #11 on: March 31, 2012, 07:34:39 AM »
Dear Andy,

Thank you for your valueable advice.  I will let my daughter to begin transfusion soon.  Also, my daughter has started to drink DrWheatgrass Supershot your recommended for a week and find the taste is acceptable.

In Hong Kong, it is quite frustrated that our government would rather provide the cheaper drug as the 1st line medicine. Even for Exjade, only the thal patients at age 2-6 are allowed for free usage since year 2011.   But actually, we only have 400 thal major in Hong Kong.  So for us, we are not only wait for 10-20 years but should be more longer time. 

Thanks anyway.   

SYT   

           



 

 

SMF spam blocked by CleanTalk