My 5 year old qualifies for some new state of the art meds

[presence]

As many of you know my son has severe hemophilia B.
http://en.wikipedia.org/wiki/Haemophilia_B

I start an IV on him every 3 days and infuse Benefix. Even with "prophylaxis" treatments he bleeds internally ocassionally; he's been in a wheelchair all week w/ an ankle bleed from kicking a rubber ball with grammy. Tomorrow should be his first day back on his feet.

There is a new FDA approved med on the market called Alprolix. Apparently, he would need an IV every 7-10 days instead of every 3.

On the other side of the country, his cousin is 18 and just signed up for a Phase 3 trial of an even more long lasting clotting factor. One dose supposedly should last one whole year.

So I'm faced with this choice... keep on with the meds I know have worked. Or take a risk on something new. Worst case scenarios; thrombosis or inhibitors. Thrombosis would be immediately life threatening. Inhibitors would mean his body would reject any meds and require immuno suppression/tolerance therapy; essentially he'd be hooked up to an IV daily for a long time and healthcare costs would triple. Best case, half or 1/3 as many needle pokes, fewer bleed throughs between doses, and less damage to his already well marked veins.

This is the new new stuff:

http://www.biogenidec.com/therapies_....aspx?ID=19723

ALPROLIX™ [Coagulation Factor IX (Recombinant), Fc Fusion Protein] is the first recombinant, clotting factor IX therapy with prolonged circulation. In the United States, it is indicated for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia B. ALPROLIX is not indicated for immune tolerance induction therapy, which is a treatment for people with inhibitors. ALPROLIX is shown to reduce bleeding episodes with prophylactic (protective) infusions starting at least a week apart.


ALPROLIX is developed by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG₁ (a protein commonly found in the body). It is believed that this enables ALPROLIX to use a naturally occurring pathway to prolong the time therapy remains in the body. Biogen Idec is the only company to apply Fc fusion to the treatment of hemophilia.


ALPROLIX is contraindicated in individuals who have a known history of hypersensitivity reactions, including anaphylaxis, to ALPROLIX or its components.
Common adverse reactions (incidence of greater than or equal to 1 percent) from the clinical study were headache and oral paresthesia (an abnormal sensation in the mouth).

ALPROLIX is also approved in Canada, Australia and Japan.


For complete U.S. prescribing information, please visit ALPROLIX.com.

This is where we're at now, its GMO clotting factor that comes from hamsters:

http://www.benefix.com/learning-abou...hat-is-benefix

BeneFix® [Coagulation Factor IX (recombinant)] is a recombinant clotting factor IX that temporarily replaces the factor IX that is missing or at very low levels.


How it works

Inside the blood vessel are special proteins that become active when they are needed to form a clot. Let’s take a closer look at how these proteins called "clotting factors" work. The process starts when clotting factors are turned on or "activated." Each factor in the chain activates another, like falling dominoes. In people who don’t have hemophilia, this quickly leads to the activation of 2 very important clotting factors—factor VIII and factor IX. Factors VIII and IX bind together to activate factor X, which has a critical role in clotting. In people who have hemophilia B, factor IX is either missing or at very low levels. The level of factor IX is so low that it cannot bind with factor VIII to activate factor X.


Without factor IX, the rest of the clot*ting process cannot continue. After using BeneFix, clotting factor VIII binds with BeneFix and they work together to activate clotting factor X. The clotting process begins to work normally, resulting in the formation of a blood clot. Once the bleeding is stopped, the clotting proteins return to normal, al*lowing the blood to flow normally and the body to heal.

These are the meds he started on when he was a baby, its derived from human blood, carrying the risks of blood borne disease:

http://www.drugs.com/cons/alphanine-sd.html

Coagulation Factor IX (Human)
AlphaNine® SD
Solvent Detergent Treated/Virus Filtered
DESCRIPTION
Coagulation Factor IX (Human), AlphaNine® SD, is a purified, solvent detergent treated, virus filtered preparation
of Factor IX derived from human plasma.1 It contains a minimum of 150 IU Factor IX/mg protein; levels of
Factor VII (proconvertin), Factor II (prothrombin) and Factor X (Stuart-Prower Factor) which are below the limit
of detection (less than 0.04 Factor VII unit, less than 0.05 Factor II unit, and less than 0.05 Factor X unit per IU
Factor IX). AlphaNine SD is a sterile, lyophilized preparation intended for intravenous administration only. Each vial is a single dose container.

Cost is not an "issue" as they're all insurance covered and all so stupid expensive its in that scale of national debt; I think my kid is worth a million dollars a few times over already.

I'm interested in your thoughts on exposing kids with special needs to "new drugs". At first my wife and I were really hesitant to try out the benefix because relative to the alphanine it was "new" and untested. Our concerns of hepatitis from human plasma derived products finally made us concede to try the benefix and we've been generally happy with the results.

[donnay]

Wow that is a incredibly tough call. I will keep you all in my prayers.

[heavenlyboy34]

Wow that is a incredibly tough call. I will keep you all in my prayers.

+1 ~hugs presence~