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XHIM BMTs

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F,

I think did a great job answering your BMT questions, but since you

wanted to know about XHIM (X-linked Hyper IgM), I thought I'd respond

also. XHIM is caused by a defect on the CD40 ligand, which affects both T

& B cell production, which in turn affects the antibody production. As

expected, before transplant, my boys were able to produce their own IgM,

but not any other antibodies. Some XHIM patients produce too much IgM,

hence the name " hyper IgM. " Neutropenia is also a problem with some XHIM

patients. Both our boys had chronic neutropenia and had to be on GCSF. At

first, our oldest son was thought to have SCID, then CVID, then FINALLY

diagnosed with XHIM after his brother was born. I do know that we are

expecting to be totally cured...both T & B cell function fixed! It just

takes time to get all your immune function up and running after

transplant (usually at least a year), so in the meantime, GCSF and IVIG

is used when you're low. We are suppose to be the 4th & 5th XHIM BMTs at

Duke. The other 3 were successful, although we've only met and spoken

with one of those.

Hope this info was helpful. I've heard of XLA, but can't remember the

details....care to give me a refresher?

Wishing you the best,

T.~ Mom to Kaitlyn,13 (healthy, double BMT donor- our hero!)

,10 (XHIM, 8 mo. post BMT)

, 5 (XHIM, 8 mo. post BMT)

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T....

How wonderful that Kaitlyn has the distinction of " hero " in your family----it

really makes her an indispensable part of her brothers' medical care! I presume

that she was a great match and close relative.....which should help the

transplant succeed???

F

mom of Calvin, 4yo, XLA

T.~ Mom to Kaitlyn,13 (healthy, double BMT donor- our hero!)

---------------------------------

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Hi, F~

Yes, Kaitlyn definitely is a special part of our medical care for the

boys. She was a " perfect match " (6/6) for and a good match for

(5/6). Funny thing is, has actually done better, faster on

his immune studies since transplant, although he had the mismatch. And

yes, a matched, related donor is a BIG advantage in having a successful

outcome, we're told. If we hadn't had a related match, the risks of a

BMT weighed against the risks of living with XHIM, were too high....in

other words, the doctors wouldn't have recommended a transplant.

I still am confused about what XLA is....might have to get my IDF

handbook out. I hope it's listed...XHIM wasn't for quite a while, which

was frustrating!

Hope things are going well with Calvin.

~ T.

Mom of Kaitlyn,13 (healthy donor for double BMT)

,10 (XHIM, 8 months postBMT)

, 5 (XHIM, 8 months postBMT)

Message: 4

Date: Wed, 14 Dec 2005 07:51:54 -0800 (PST)

From: lisa fisher <blessd8@...>

Subject: Re: XHIM BMTs

T....

How wonderful that Kaitlyn has the distinction of " hero " in your

family----it really makes her an indispensable part of her brothers'

medical care! I presume that she was a great match and close

relative.....which should help the transplant succeed???

F

mom of Calvin, 4yo, XLA

T.~ Mom to Kaitlyn,13 (healthy, double BMT donor- our hero!)

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T

I have meant to get back to you about XLA....but life is busy here these days.

I intend to write an email and then about three days fly by when I am not

looking:-).

XLA stands for X-linked Agammaglobulinemia. It is also known as Bruton's

Agammaglobulinemia. It is X-linked like XLHM. It is basically completely

confined to the population of boys-----though there are girls that have B-cell

defects that may act like XLA.

XLA boys make B-cells of the immature kind, but have no mature B-cells. This

leads researchers to theorize that there is a breakdown in the maturation

process. The most obvious result is the almost complete absence of Igs of any

kind. Calvin has no IgG, IgA, IgM, IgE or any other Igs.

They have identified the genetic location of XLA: a gene known as Bruton's

Tyrosine Kinase (or BTK). I think that XLA was the first PID for which they

located the genetic basis. They do not routinely do BMTs for XLA since the IG

treatment is SO compatible that most XLA boys are relatively healthy with IG

treatment. Calvin also uses prophylactic abx to help address the absince of

IgA----which is not replaced by IG products. Matter of fact, XLA boys commonly

require a very low IgA product like Gammaguard, since they are prone to develop

resistance to the IgA in IG products. This happens when there is a complete

absence of IgA in their system. This does vary from patient to patient.

Gene therapy for XLA may be a possibility in the not-so-distant future, since

the genetic basis of XLA is well-known. I think that everyone is waiting and

watching the long-term outcomes of patients with more severe disease who have

had genetic therapy. Again, the risk-benefit ratio does not warrant

transplant/gene therapy since XLA patients are relatively healthy with IG

replacement.

That's all the info I have off of the top of my head. I think that the IG

treatment " fits " the deficiency very well and that is why most of us XLA folks

live relatively quiet medical lives. That being said, I have also heard that

there is a mortality of 10% for every 10 years of life for XLA boys...and life

for us before Calvin's diagnosis was not easy at all. It is only by God's

providence and care that my son did not die, or become severely handicapped

because of illness. Calvin was dxed at 18 mos.

I can also mention that Calvin presented first with neutropenia(cyclic) and

sepsis. It is not unusual to have neutropenia along with XLA----though it is

not present in a majority of patients. For calvin, his neutrophil counts have

stablized somewhat with treatment, though they are not completely stable. His

ANC has not been " clinical " since IG treatment.

HOpe that helps.

Fisher

mom of Calvin, 4yo, XLA

mptatem@... wrote:

Hi, F~

Yes, Kaitlyn definitely is a special part of our medical care for the

boys. She was a " perfect match " (6/6) for and a good match for

(5/6). Funny thing is, has actually done better, faster on

his immune studies since transplant, although he had the mismatch. And

yes, a matched, related donor is a BIG advantage in having a successful

outcome, we're told. If we hadn't had a related match, the risks of a

BMT weighed against the risks of living with XHIM, were too high....in

other words, the doctors wouldn't have recommended a transplant.

I still am confused about what XLA is....might have to get my IDF

handbook out. I hope it's listed...XHIM wasn't for quite a while, which

was frustrating!

Hope things are going well with Calvin.

~ T.

Mom of Kaitlyn,13 (healthy donor for double BMT)

,10 (XHIM, 8 months postBMT)

, 5 (XHIM, 8 months postBMT)

Message: 4

Date: Wed, 14 Dec 2005 07:51:54 -0800 (PST)

From: lisa fisher <blessd8@...>

Subject: Re: XHIM BMTs

T....

How wonderful that Kaitlyn has the distinction of " hero " in your

family----it really makes her an indispensable part of her brothers'

medical care! I presume that she was a great match and close

relative.....which should help the transplant succeed???

F

mom of Calvin, 4yo, XLA

T.~ Mom to Kaitlyn,13 (healthy, double BMT donor- our hero!)

This forum is open to parents and caregivers of children diagnosed with a

Primary Immune Deficiency. Opinions or medical advice stated here are the sole

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