Thalassemia Patients and Friends
Discussion Forums => General Chatter => Topic started by: Sharmin on January 07, 2009, 10:19:14 PM
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Little A has an appointment in Oakland mid February. Once again I am making a list of questions and concerns. If any of you have questions I will do my best to get answers for you.
Manal, you had a question last time which I didn't get until I got back - was it about pulmonary hypertension?
Sharmin
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Sharmin,
i have a question that is abt combination therapy of Exjade + desferal ,
when the combination therapy of exjade+desferal will be proved to secure ??..
when trials report will be available of exjade+desferal combination therapy ??..
wat is his opinion abt gene therapy ??
take care
Best regards
umair
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Is there any possibility or any trial (even individual),for ferriprox and exjade going on?
Zaini.
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Yes Zaini,
in my Blood Transfusion Unit, there is a little girl of about 8 years old who is on a trial of combination ferriprox and exjade but it is too recent to publish any results.
If I learn something I ll let you know.
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Sharmin
Thanks a lot dear, yes it was about PHT but i can not recall the nature of the question, i will search for the old post and let you know, thanks my friend :hugfriend
Zaini
I remember that Dr. Piga in Singapore didn't recommend at all combining ferriprox and exjade togather since they both have their side effects in the same area and this put a big negative load on the body
manal
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Lena,
Please post any details you find,how long has she been taking them both?
Manal,
Yes that's what we have heard till now,but like we have seen people doing desferal and exjae together,may be ferriprox and exjade will become a possibility too,and it will be very helpful for people with very high iron overload.
Zaini.
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Hi Zaini,
I will definitely ask the question. My personal opinion is that since both L1 and exjade remove iron at the tissue and organ level - using desferal in combination can chelate the iron removed from the tissue by either L1 or exjade. (Desferal does not have the ability to easily get inside tissue to bind an remove iron - but it is very effective at binding iron in the plasma for its removal). In this way the effect of desferal with either exjade or L1 is synergestic. L1 or exjade remove iron from tissue - making it available for desferal to remove. Desferal makes L1 or exjade therapy more effective by quickly removing iron that they have removed from tissue.
On the surface, it does not appear to me that L1/exjade combination therapy would have synergestic effects since they both remove iron from tissue - and not is as effective as desferal at removing it from the plasma. The possible benefits that I see are 1) They are both oral - meaning no needles!! 2) L1 removes iron very effectively from the heart, exjade is believed to remove iron from the liver and other organs and together they could possibly remove a lot more iron. 3) L1 does not cause the same spike in SF when one first starts to use it therefore I am assuming that either it removes iron less quickly than exjade or L1 is more capable of excreting iron from the plasma then exjade and in this case it would work well together with exjade. For these reasons the two may work well in combination.
My reasons for not thinking that they should be used in combination are: 1) desferal is known to have very few side effects while exjade and L1 are both considered to pose more threat - therefore using them in combination with each other (as compared to using either in combination with desferal) may pose of a risk 2) exjade may in fact be good at removing iron from the heart as well as other organs - in this case L1 may not be necessary while using exjade to remove cardiac iron overload 3) Desferal requires very few tests to ensure that it is not causing side effects - exjade requires testing every 3 weeks - and L1 requires many tests as well (because of its effect on immune system, joints etc) - therefore if you were using both imagine the number of tests that one would have to have done (which also shows you how many side effects one is risking).
These are my thoughts for why one may or may not consider the drugs in combination - it is a great idea - why not use to effective oral drugs together - remove iron and avoid needles! However the synergestic effects may not be high enough to risk the numerous side effects. I would like to hear what the experience is for the one patient taking the two drugs in combination - because who knows maybe the two would great together. I am excited about the possibility of someday using starched desferal with exjade.
Oakland is not keen on giving L1 to children - because when I asked them about it they had said that they were concerned about its effects on children. By the way Zaini - this is a great question and provokes a lot of thought - I will be honored to ask it :hugfriend
Andy, what do you think?
Thank you everyone for your questions - I will take note and please keep asking and praying that little A's tests go well. We will be repeating the SQUID, T2* and we will be doing a dexa scan for the first time. I am quite nervous but hopeful that the results will be favorable for my son and show that exjade/desferal combination is safe and effective so that it can benefit everyone else.
Thanks guys,
Sharmin
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I hope that the tests in Oakland reveal that little A's iron levels are low enough such that he can also stop using desferal - or at the very least such that his desferal dose can be decreased to 2 or 3 times a week. He only lets us put it on his stomach - and his stomach is quite bruised now. Because he plays ice hockey (he is skating all of the time) and soccer - his thighs have only muscle and no fat therefore we can't put it on his legs. His arms are too skinny, and he won't let us put it on his hips. I am praying that he will get a break from the needles too.
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My all prayers are with Little A :hugfriend With the strict regimen you have followed i am sure his iron levels must be down. :pray
Little A,
Lets find out your results and then we'll celebrate together :wink
Zaini.
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hey Lil A ,
when ever i start writing a post to you , a thought blink's in my mind and remind me the movie in whic young blood team rule over the game ( ice hockey ) against the cheater ones ( cheater use's all that they have to hit the not the strike but the opponent ) thats wh i remember them as cheaters ..... wish you all the best for your Tests especialy SF .. you are such a brave boy buddy ... and brave boy never fails ... take care of your self and keep playing ice hockey ...
Best Regards
Take Care
Umair
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his thighs have only muscle and no fat therefore we can't put it on his legs. His arms are too skinny, and he won't let us put it on his hips. I am praying that he will get a break from the needles too.
I just pray that all the children of the world and especially children will have an alternative for desferal and have a break from the needles, Ameen :pray :pray :pray :pray :pray :pray
manal
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:pray AMEEN :pray
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Thank you very much my friends - I love you all :grouphug
My prayers are the same - for all thals to be well without needles.
Sharmin
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I have been hearing that the other patients in the trial are continuing to do well and are being allowed to get off of the desferal - how wonderful it would be to not have to put desferal on anymore! Dr. Vichinsky told us that the ultimate goal is to get rid of desferal - but another doctor said that eventually we will use mostly exjade but desferal 2 or 3 times a week....I wonder what they will decide when we get there. I think that if his iron levels are below 600 we can get off of desferal, however I have also heard that one whose iron levels are below 1000 should not be using desferal.... ??? ??? ???
Sharmin
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Isn't level of 1000 enough to get rid of desferal? :-\ Exjade will remove the rest?
Zaini.
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that is what i had thought...they did mention 1000 as a mark but I don't know if it mean taking away desferal completely or just reducing it.
3 more weeks until we will know for sure. I hope that the transfusion frequency remains low because if it does then the rest will fall into place.
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List of Questions
1. is fetal hg less efficient for growing children than adult hg?
2. do fetal hg inducers trigger unwanted functions?
3. L-carnitine - what is the opinion on its usage
4. pulmonary hypertension - who is at risk, how to manage risk - NO inducers? L-carnitine, citrilline
5. vitamin C with exjade, vitamin C when not using desferal
6. what is treatment for post splenectomy non transfusing alpha/beta thals? anything other than anticoagulants required?
7. have exjade/L1 been used in combination?
others??
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Good list of questions sis ,
Way to go :tumbsup
Best Regards
Take Care
Umair
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Sharmin, thanks a lot for your effort dear :hugfriend
If it would be possible, can he tell us what are the suitable anticoagulants that can be given to children rather than asprin,since it shouldn't e given to children and teens.
http://www.orthop.washington.edu/uw/medications/tabID__3376/ItemID__72/PageID__34/Articles/Default.aspx
Effects on children
Recent reports have said there could be a link between the use of aspirin and the development of Reye's syndrome. Reye's syndrome is a rare but possibly fatal disease seen most often in children and teenagers. It usually affects those recovering from chicken pox or a viral illness such as the flu. These reports have raised concern in pediatricians (doctors who specialize in treating children) and parents of children with arthritis who need to take large doses of aspirin to control their disease.
Vitamin E is mainly an antioxidant, but also has the ability to thinner the blood, so would this be enough or there would be a better anticogulante????????
manal
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Hi Manal,
I will ask about anticoagulants.
Andy, should I ask about magnesium, bilirubin and gall bladder stones as well?
As the appointment is getting closer I am beginning to get nervous. I hope that his iron levels have come down. I hope that the results of his SQUID, T2* and DEXA scan are good. He has never had a DEXA scan before - he bone age test was normal for his age - but the DEXA provides more information. The last appointment was rough because we realized that his iron levels were higher than we thought - we were about to embark on some very difficult treatments. I hope that this appointment concludes that things are working. I also want to cut down his desferal if possible.
I think what Andy said about reducing iron overload and restoring pituitary function may apply here too - a few months after the intense chelation my son grew 5 cm in a very short period of time.
We will need your prayers, I am hopeful that things are going the way that we had hoped.
Sharmin
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Which date is the appointment?
My fingers and toes are crossed for his tests!!!
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Thanks Dore :hugfriend :hugfriend
We are flying out on February 11th. His appointments are February 12th and 13th.
Sharmin
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I am very hopeful that every thing will be OK Sharmin :hugfriend don't worry,our prayers are with you and Little A :hugfriend.
Zaini.
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Sharmin & Lil A ,
Wish you all the best for your apointment and...and....and.. hummmm everything .....
i m always praying for you Little A , and dont forget to update me that how many goals you scored okiey ..... Take Care
Best Regards
Umair
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It might be interesting.
Thalassemia Standards of Care Available from CHO
January 20, 2009 - The Children’s Hospital & Research Center Oakland has published its new Standards of Care Guidelines for Thalassemia. The guidelines, which have been authored by a team of knowledgeable thalassemia care experts lead by Elliott Vichinsky, MD, and Laurice Levine, MA, CCLS, provide valuable information about the proper care of individuals with thalassemia.
Pdf copies of the Standards of Care can be downloaded by clicking here. Additional copies can also be ordered by contacting Laurice Levine at LLevine@mail.cho.orgThis e-mail address is being protected from spambots. You need JavaScript enabled to view it or (510) 428-3885, ext. 5427.
CAF congratulates the dedicated thalassemia team at Children's Hospital Oakland for their hard work on this important publication.
http://www.cooleysanemia.org/index.php?option=com_content&view=article&id=88:cho-standards-of-care-available&catid=1:latest-news
This will be my new homework :wink
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This can also be downloaded in our documents section at http://www.thalassemiapatientsandfriends.com/index.php?topic=1762.msg14639#msg14639
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Ok, shall I delete those posts?
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Nope. Anywhere people can find the link is helpful.
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Actually, I hope people mention this document often and link to it. These guidelines should be seen by every doctor who treats thalassemia, so please do mention Standards of Care Guidelines for Thalassemia often.
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The list of questions is below. I will do my best to get answers.
1. is fetal hg less efficient for growing children than adult hg?
2. do fetal hg inducers trigger unwanted functions?
3. L-carnitine - what is the opinion on its usage
4. pulmonary hypertension - who is at risk, how to manage risk - NO inducers? L-carnitine, citrilline
5. vitamin C with exjade, vitamin C when not using desferal
6. what is treatment for post splenectomy non transfusing alpha/beta thals? anything other than anticoagulants required?
7. have exjade/L1 been used in combination?
8. magnesium, bilirubin and gall bladder stones
9. Patients with PKU
Sharmin
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Sharmin, if possible ask about the
1- effiency of DEXA at young age
2- suitable anticoagulants that can be given to children rather than asprin,since it shouldn't e given to children and teens. Vitamin E is mainly an antioxidant, but also has the ability to thinner the blood, so would this be enough or there would be a better anticogulante
Thanks dear
manal
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The list of questions is below. I will do my best to get answers.
1. is fetal hg less efficient for growing children than adult hg?
2. do fetal hg inducers trigger unwanted functions?
3. L-carnitine - what is the opinion on its usage
4. pulmonary hypertension - who is at risk, how to manage risk - NO inducers? L-carnitine, citrilline
5. vitamin C with exjade, vitamin C when not using desferal
6. what is treatment for post splenectomy non transfusing alpha/beta thals? anything other than anticoagulants required?
7. have exjade/L1 been used in combination?
8. magnesium, bilirubin and gall bladder stones
9. Patients with PKU
10. effiency of DEXA at young age
11. suitable anticoagulants that can be given to children rather than asprin,since it shouldn't e given to children and teens. Vitamin E is mainly an antioxidant, but also has the ability to thinner the blood, so would this be enough or there would be a better anticogulante
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9. Patients with PKU
Sharmin
If it was my suggestion: PKD (PK def.)
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Sorry Dore, I did mean to say PK def. :)
I will have my husband print off the list tomorrow.
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Sorry Dore, I did mean to say PK def. :)
I will have my husband print off the list tomorrow.
:thumbsup
PKD stands also for polycystic kidney disease...... Pretty confusing!
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Dear Sharmin, :hugfriend
(http://i46.photobucket.com/albums/f110/ahcooey/bonvoyage.gif)
Do post when you get time :biggrin
Zaini.
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Friends, we're in San Francisco. Our appts are tomorrow, your prayers are needed. I hope it will go well.
I am so glad that Umair is well, I have been really concerned. I hope Zainab is better too now - my wishes are with you all.
Thanks Andy for showing us the way. Any improvementsmy son makes, we owe it all to you.
Sharmin
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Hi Sis ,
(http://www.atmgreetings.com/everyday/happy_journey/mantle.gif)
wish you all the best ,, take care
Best Regards
Umair
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Sharmin,
Enjoy your trip,i hope all the results of Little A will come back ok,my prayers are with you. :hugfriend
Zaini.
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Good luck Sharmin, you are all in my prayers, will be waiting :hugfriend
manal
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Hi Sharmin,
Have a nice time there (despite the appts) and I hope all will go well. I got my appt with the hemo tomorrow as well. To use a Dutch saying "We're (almost) in the same boat". :hugfriend
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Good luck to you too Dore, hope we hear good news. Take care
manal
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Thank you. Firday is going to be a hard day. If you have suggestions what I should ask too (what has pop up in your head after reading my posts) then tell me please!!!!! I will also ask of he has already convinced the cardio to buy T2* software...
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Dore, best of luck with your appt!
We have completed our first day of tests and appts.
Good news - SQUID results - LIC = 1545 - down from 2880 July/08.
The docs were impressed.
Lots more to come later - we are exhausted and hungry!!
Sharmin
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I am also impressed! Way to go little A!
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Dore,
I think you have to check the iron overload in organ through ferriscan (MRI on the liver) or T2*.
Also revising your supplements is a good idea too
manal
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Sharmin, congratulations dear, you deserve the best. You and little A have worked so hard, Way to goooooo
manal
:congrats :congrats :congrats
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Hi Sis.
Congrats for you behind all this, there is a big effort/hand of you Well Done...
Lill A :congrats To you buddy you got alot of strenght to make anything possible ... Well Done buddy and keep it up , i want to more super news from you like this , i hope n pray that other reports will come better fine than this one ... dont forget to enjoy your staty budy okiey .
Best Regards
Take Care
Umair
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I have some answers to our questions. We will be in again tomorrow for cardiac MRI and DEXA so I will try to get more answers then.
1. Fetal hemoglobin is less efficient than adult hemoglobin because it delivers less O2.
2. Fetal hg inducers such as hydroxyurea are deamed to be quite safe with very little side effects.
3. and 4. L-carnitine - very good - Oakland is currently doing a study on L-carnitine and lipoec acid. We are starting Little A on 600mg of L-carnitine. L-carnitine protects heart tissue from iron damage.
5. only take vitamin C with desferal - usage with exjade is not the same and not recommeded
6. Treatment post spenectomy thals - baby aspirin, and cormadine is used to keep blood thin, antibiotics.
7. To avoid exjade related intestinal issues - can divide exjade into two doses - morning and evening. Can try taking it at night - on empty stomach.
8. exjade/L1 together - not studied.
9. magnesium bilirubin - sorry didn't get to this question
10. PK def - will ask tomorrow
11. DEXA scan - when done in a big pediatric setting is useful because they have the data for young children. We are doing one tomorrow. Boys more susceptible then girls to bone loss issues. We declined today's appt - but after speaking with doctor decided to go through with it.
We had a lot to deal with our tests etc. trying to devise future treatment and chelation schedule - but in the scheduled appt time I tried to get as far down the list as possible before time ran out. I will try to get remaining questions tomorrow.
Andy, we saw a different doctor from the team today - she also stressed that the future of thalassemia treatment is antioxidants. She was impressed that I asked for L-carnitine and was very eager to help me guide me with it. Thank you so much for guiding me in asking for help in Little A's treatment - it made a world of difference. Thank you again.
Sharmin
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Sharmin you did a great job :hugfriend SQUID results are indeed a good news,after a long journey of chelation,you and Little A deserved it :hugfriend.
Thanks to Andy our kids are already on antioxidants,while doctors are researching on them.
What makes me feel sad is in countries like mine,how can we determine any deposition of iron in organs when SQUID or ferriscan is not available :( .
Zaini.
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Thanks dear for everything. You are doing an excellent job. Thanks for helping us all
manal
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I had to take a long walk to absorb everything that we learned today.
One thing I want to stress is that everyone should have their vitamin D tested - and be sure to take a good vitamin D supplement. Little A was extremely deficient last spring and the doctors here want him on mega doses to make up for the deficiency.
Secondly, we have to do rituximab again this coming summer. It was a nightmare last year, but we need to repeat the course once again because Little A is still receiving too much blood. This has been spinning around in my head since our appt. Ultimately, we have to do what is best for him - so that his tx requirement and chelation can be reduced.
Thank you all for your support. Thank god that Andy lead us down this path before our child sustained any damage from the iron. I am really glad that his iron levels are reduced at this time and that we have many options. We are in a much better position than we were 6 months ago.
I am realizing that the information which we are receiving here is lifesaving - as is the support :hugfriend :hugfriend
Sharmin
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Vitamin D is all the rage these days and for good reason. Our bodies are based on a system that assumed regular extended exposure to sunlight and most of us no longer get this. In addition, there may be a more hazardous situation now in terms of lengthy exposure to the sun due to destruction of the ozone layer which helps protects from the sun's damage. So, vitamin D supplementation often becomes necessary, especially for those living in climates with long periods of little sun. I feel great in the summer time when I can get sun but during the winter months I am a wreck if I don't take vitamin D daily. Its affect is noticeable almost immediately. Thalassemics have a great demand for vitamin D and just as with vitamin E, as more thals are being tested, an extremely high rate of severe deficiency is being found. This can be corrected and large doses are required, but it will work and it will greatly improve bone health along with just about every other aspect of health. The simplest thing our body needs and it has taken science until very recently to understand how very important the sunshine vitamin really is.
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I am realizing that the information which we are receiving here is lifesaving - as is the support
I couldn't agree more.
Secondly, we have to do rituximab again this coming summer. It was a nightmare last year, but we need to repeat the course once again because Little A is still receiving too much blood.
Does this mean antibodies are still active ???
Best of luck Sharmin,you are doing your best,rest is up to God.
My prayers are with you.
Zaini.
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:congrats with the iron thing & :goodluck for today!!!!
Adíos, Dore
p.s. dont worry I will start an own topic.
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We are home and ready to implement everything that we were advised. We were lucky to have taken all of the knowledge that we gained here because doctors volunteer more assistance when you ask more questions.
Thank you all for your support while we were there - being away from home in a hospital setting and receiving tons of information can become heavy. Thanks to the support of our friends here we got through it - and actually enjoyed our stay in San Francisco during the last few days :)
Love Sharmin :hugfriend
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Welcome back Sharmin :hugfriend I was just thinking about it the other day,that i know i can't go to Oakland myself,but i can get all information from this forum through you,Thanks for informing and acknowledging us :hugfriend.
Zaini.
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My pleasure Zaini,
All of you come to Oakland with us in my thoughts :) I try to ask questions that will help all of our members - so that all of our little A's to Z's can be healthy :)
Thanks again for your support Zaini :hugfriend
Sharmin
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Missed you a lot and welcome back my dear :hugfriend :hugfriend :hugfriend I am sure you need a deep sleep after the stress of the passing days. Take care and welcome back to ''Home sweet home'' :wink
manal
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:welcomewagon Back To Home Sis , And wish you and Little-A with best of the luck in starting new treatments those has recently advised to lil-A in this apointment ...
:goodluck and Take CAre'z
Best Regards
Umair
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Thank you friends for your wishes :) We are happy to be home and to know that all in all lil A is doing very well.
He sends his love to all of you,
Sharmin