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Hereditary anemias (beta-thalassemia major and congenital
Blackfan-Diamond aplastic anemia) are fairly common among Russian children,
especially in southern regions of our country (Dagestan, Ingushetia, Stavropol krai, Krasnodar
krai), and less frequent in other regions. Without donor RBC transfusions, the life
expectancy of such patients is about three to seven years.
A complication of life-saving treatment of these diseases is iron overload, which
significantly deteriorates the condition of all organs and systems, leading to
early death of such patients at an age from 10 to 17.
To cope with this dangerous complications, so-called iron chelators are used. These
drugs remove the excess iron from the organism. Two of such medications
have been registered in Russia: Desferal in the form of solution for injections
and Exjade in the form of dispersible tablets (Novartis, Switzerland). Iron chelators
should be administered throughout any patient's life. Therefore, the drug in
tablets (Exjade) is significantly more advantageous for children.
None of these drugs are presently included in the list of vitally important medications
to be provided for free in Russia, and the parents have to buy them for their
own money. As a result, children receive the necessary drugs very rarely and their
treatment becomes a great deal less efficient; children suffer from early disabilies
and die at a young age.
Presently it has been proposed to include this drug in the list of vitally important
drugs provided to Russian citizens for free, but so far it has not been done.
Exjade is manufactured in the form of 250-mg dispersible tablets, 84 tablets per
pack. Each pack costs about 43,000 roubles ($1680).
Specialists of the Russian Children's Clinical Hospital regularly examine 17 children
of different ages with grave hereditary anemias. These patients receive treatment at the
Out-Patient Department and Department No. 19 of our hospital in the form of
monthly RBC transfusions.
Hematologist, Head of Out-Patient Department, Russian Children's Clinical Hospital
N.S. Smetanina
I am Nurullah Burtiev's mother. I want my child to live
a normal, happy life. But he has a very serious disease, beta-thalassemia major,
and he needs a very expensive drug,
Exjade, for this treatment. We cannot buy it by ourselves. We have two children: Nurullah is 2.5 years old,
and his younger sister is 18 months old. My husband's earnings are barely sufficient to cover
our daily expenses, and I have no job myself, because I have to take care of my ill son
and little daughter. Please help us if you can! We'll be whole-heartedly grateful to all
of you!
Yours respectfully,
Nurullah's mother
Sevil
The doctor's comment
Nurullal Burtiev, born Aug 30, 2007, has been under observation at the Russian Children's Clinical
Hospital since December 2009. His diagnosis is beta-thalassemia major (diagnosed in April 2009),
secondary iron overload.
Since the child still gets regular transfusions and iron overload persists, therapy with drugs for iron
removal should be continued. Exjade is preferable, since this drug has proved to be efficient
and the child tolerates it well.
The total cost of the drug is about 370,000 roubles ($11,935).
Head of Out-Patient Department N.S. Smetanina
Hematologist M.V. Krasilnikova
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My son Renat Gamzaev is five years old now,
and he has been ill almost since birth. His disease requires blood transfusions every month, and
now there is too much iron in his body. The only drug that can help him is Exjade, but it is
a very expensive drug. We cannot buy it ourselves. We will be grateful for any financial help.
Thank you.
The boy's father
Ulvi Gamzaev
The doctor's comment
Renat Gamzaev, born Feb 11, 2004, has been under observation
at the Russian Children's Clinical Hospital since February 2006. His diagnosis is
β-thalassemia major, secondary iron overload. The boy has been getting regular
packed RBC transfusions since February 2005. A total of 50 transfusions have been made.
Presently there are signs of cardiomyopathy and toxic hepatopathy, which are due to
secondary iron overload. Chelator therapy with Exjade has lead to improvement.
During the last examination (Nov 15, 2009), ferritin level was 1700 ng/mL.
Conclusion: Since the child has developed secondary iron overload and associated
complications due to regular transfusions, chelator therapy to remove excess iron
should be performed.
Recommended: Exjade 250 mg/day, 370 tab (250 mg) per course of therapy. This
amount of the drug costs a total of 215,000 roubles (about $7165).
Head of Out-Patient Department
Prof. N.S. Smetanina
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The doctor's comment
Gleb Kilin, born on Dec 9, 1993, has been under observation
at the Russian Children's Clinical Hospital since November 2008.
Diagnosis: Blackfan-Diamond congenital aplastic anemia (diagnosed in Jul 1994).
The boy has been getting RBC transfusions since Jan 18, 1995. The total number
of transfusions already received exceeds 110. There are manifestations of cardiomyopathy
and toxic hepatopathy due to secondary iron overload. Recently, increasing transfusion
dependence was found. The boy is admitted to hospital for RBC transfusions once
in 1-1.5 months; during each admission, he gets 3 or 4 transfusions (total volume up to
1000 mL). The highest ferritin level attained is 1368 ug/L (Jun 25, 2009).
The boy was included in Exjade clinical trials for treatment of posttransfusion iron overload.
Satisfactory tolerance of therapy, improvement of condition. However, the supply
of this drug within the aforementioned protocol ended on Nov 25, 2009.
Since the child still gets regular transfusions and iron overload persists,
chelator therapy with Exjade should be continued. This drug is efficient
and tolerated well by the child.
Recommended:
Exjade 1000 mg/day, 730 tab. (x 500 mg) per year.
Head of Out-Patient Department Prof. N.V. Smetanina
Hematologist M.V. Krasilnikova
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I am asking for financial help in buying Exjade for my son Andrei Chipilev,
five years old. He has been suffering from Blackfan-Diamond anemia since birth. He
needs regular transfusions of packed RBC, which lead to iron overload, and this extra iron should
be removed with Exjade. Otherwise, the outcome may be fatal. This drug is available both
abroad and in Russia, but it costs a lot.
Unfortunately, our family is far from rich. I am a single mother (Andrei's father left
us as soon as he learned our son's diagnosis and understood how difficult further
treatment will be), and, besides, I cannot get a permanent job, because I often
have to accompany my son to hospitals. And now we must somehow find
370,000 roubles ($12,420) to buy Exjade.
When we live at home with Andrei, we cannot go anywhere either, because it is very
dangerous for him to fall ill. So this hospital for him is like preschool for healthy
kids: there is a playroom and other children, with whom he can talk and play.
My son and I, we both hope that your help will come in time!
Valentina Chipileva
21.11.2009
The doctor's comment
Patient Andrei Chipilev, 5 years old. Diagnosis: congenital Blackfan-Diamond
aplastic anemia. The boy has been getting RBC transfusions twice a month since 2004.
For this reason, the child has developed iron overload, which is harmful for his organs.
Since Jun 19, 2009, the child has been getting Exjade 500 mg/day within a clinical study
of this drug in treatment of iron overload. Tolerates the therapy well; treatment effect
positive. Therapy within the present protocol ends on Nov 18, 2009.
The boy needs 364 tablets of Exjade 500 mg.
The total cost is 370,000 roubles ($12,420).
Hematologist N.S. Smetanina
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I am Tamara Bizyaeva, mother of an ill child.
My son Sergei Bizyaev, born in 1998, has been suffering from beta-thalassemia
for 11 years. He receives washed RBC transfusions once a month, and
excess iron has accumulated in his body. He has leg pains now and
even walks with difficulty. Sergei is still optimistic and tries
to convince me that he will be all right. But, to feel all right, he
needs Exjade, and this drug is very expensive. He has been getting
therapy with this drug for a year, but this single year was not enough
to remove the entire amount of excess iron accumulated during 10 years.
Our family has no extra money: we have two more children, and
their father is dead. I'll never raise enough money to buy this drug by myself.
Please help us buy Exjade if you can. My child wants to live and to be happy,
just like all other children. Thank to this therapy, he can now walk and
live a normal life. And, moreover, he wants to live and struggle for his life.
Sergei's mother
Tamara Bizyaeva
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Please help me save my child! My daugher Alina Maltseva has been ill since
birth. Her diagnosis is hereditary nonspherocytic hemolytic anemia (RBC enzymopathy),
severe form, chronic hemolysis. She used to get blood transfusions once a month, but
now they are required more frequently. Because of these regular transfusions,
she has developed many additional diseases: cardiopathy, gallstones,
and a serious iron overload, which affects her organs (including heart and liver) and can
become fatal. To prolong her life and to lessen the symptoms of this disease,
Alina must take Exjade. We cannot buy this expensive drug ourselves, and we will
be very happy if somebody could help provide my girl with this drug. Thank you
very much.
Alina's mother N.M. Kovaleva
24.11.09 |
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News
22.11.2009
In 2008, your help allowed to provide Exjade
to 12 hospital patients with hereditary anemias. Since January
2009 , the firm producing Exjade provided this drug to children free of charge, within its own charitable program.
Now, unfortunately, Novartis has ended this program, and the patients will have to search for money to
buy Exjade since December 2009. Therefore, we are resuming the fundraising to purchase it.
They have helped
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