Cryo-Save Facilitates the First Transplantation in Spain to Treat Blackfan-Diamond Anaemia Thanks to the Cost-Free Donation

      Cryo-Save Facilitates the First Transplantation in Spain to Treat
      Blackfan-Diamond Anaemia Thanks to the Cost-Free Donation Program

  PR Newswire

  ZURICH, April 29, 2013

ZURICH, April 29, 2013 /PRNewswire/ --

At no charge whatsoever to the family, this Cryo-Save program has made it
possible to treat this congenital anaemia, classified as a rare disease, using
cord blood stem cells.

A 4-year-old boy was treated for Blackfan-Diamond anaemia (BDA) with a stem
cell transplant from his sister's umbilical cord blood. The transplant was
performed on April 25 at the Hospital del Niño Jesús in Madrid, one of the
most important paediatric hospitals in Spain. It is the first transplant
performed in this country to treat BDA with a cryo-preserved sample from a
family bank, in this case, the leading Family Stem Cell Bank in Spain,
Crio-Cord, a subsidiary of the Cryo-Save Group. This treatment was provided
thanks to the Cryo-Save Cost Free Donation Program, which provides free
storage of the umbilical cord blood and cord tissue stem cells for families
who have a family member diagnosed with a disease treatable with stem cells.

The transplantation was successful, and the child is expected to make a normal
recovery. This pioneering treatment in Spain may herald a radical improvement
in the child's quality of life, as since birth he has required regular red
blood cell transfusions. "Up until the transplantation our life was bound to
these transfusions . We could hardly be happier, because now our son will be
able to lead a completely normal life ", said  Elena , the boy's mother. The
parents had only found out about the possibility of this type of treatment
when Elena was pregnant with her daughter, now the cord blood donor. They did
not hesitate for an instant. "We had the opportunity to heal our son, if the
sample w as  compatible, and we could not forego it",  she said . "Our
daughter will be very happy when she learns that her brother got better thanks
to her", she added.

" Blackfan-Diamond  anaemia is a congenital disease that prevents the bone
marrow from producing the red blood cells needed to carry oxygen throughout
the body," explained Dr. Luis Madero , Head of the Oncohaematology Department
of the Hospital Niño Jesús in Madrid, and the physician who performed the
transplantation. Until recently, the main treatments were periodical red blood
cell transfusions, which clearly affects the patient's quality of life, and
long-term corticosteroid therapy, which is not tolerated in all cases.

"The transplantation of umbilical cord blood stem cells, especially between
siblings, is now a recommended treatment option for certain types of
haematological diseases, such as this anaemia. The benefits versus other
treatments for this disease are clear", asserted Dr. Madero. This is confirmed
by some studies ^[ ^i] ^, ^[ ^ii] .

Cost Free Donation Program

As soon as they were informed by the family bank that there was a possibility
of treatment, the child's family opted into the Crio-Cord "Cost-Free Donation"
program. "Crio-Cord told us about the existence of this programme when we
called to enquire about the  storage  of our daughter's umbilical cord 
blood . As we met the requirements, we initiated the different procedures and,
once we were accepted on the program, we got the great news that our children
were compatible," said Elena. "Moreover, thanks to the programme we confirmed
the diagnosis of the disease, which was not clear up until then," she added.
All the tests performed on the patient and his sister, were coordinated by
Dr.Luis Madero.

The Cost-Free Donation Program also covers the whole process required for the
transplant to take place: the analyses required on the sample to guarantee
that it is compatible with the patient to be treated, the coordination of the
treatment and the transportation of the sample from the Cryo-Save Group's
Storage Facility in Belgium, to the hospital where the treatment will take
place, all free of charge within the European Union.

To qualify for the Program, a family member must have been diagnosed with a
disease that is currently treatable with stem cells . They must be a first
degree relative to the newborn child whose stem cells will be collected and
have the opinion of an oncologist or haematologist recommending stem cell
treatment. If participation in the Program is approved, the family will
receive the collection kit. At he time of the birth, the sample will be
collected, processed and stored until it is needed, totally free of charge.

"We are very proud and pleased to have contributed to alleviating this
family's suffering. Cryo-Save wants to demonstrate its commitment to the
development and welfare of society, not only through collaborative programs
with researchers and universities, but also by directly supporting families in
need,  through  this program," said Arnoud van Tulder , Cryo-Save's CEO.

Blackfan-Diamond  Anaemia

The annual incidence of BDA in the European general population is
approximately 1/150,000. This rare disease, which affects both sexes equally,
is usually detected in the first two years of life. The main symptoms are
pallor and dyspnoea, especially during feeding or breastfeeding.

Half of all patients with BDA are short and have congenital anomalies, the
most frequent ones being craniofacial, genitourinary and thumb anomalies. BDA
patients have a high risk of getting leukaemia and cancer. BDA is inherited in
an autosomal dominant pattern with variable penetrance. Currently, the
mutations that cause the disease have been identified in 40%-45% of patients.

Diagnosis can be confirmed by family history, associated malformations and
elevated levels of adenosine deaminase (ADA) in erythrocytes. In familial
cases, the risk of recurrence is 50%. In all cases careful ultrasound
monitoring is advisable during pregnancy, as a pregnancy where the baby is
affected by BDA is regarded as high-risk pregnancy for mother and child.
Disease severity depends on the quality of care received and the response to
treatment.

About Cryo-Save

Cryo-Save, the leading Stem Cell Family Bank, holds more than 225,000 blood
and umbilical cord tissue samples. Since the first successful cord blood
transplant in 1988, cord blood has been successful in the treatment of more
than 70 life-threatening diseases including leukemia, lymphoma, sickle cell
anemia, aplastic anemia and other blood related disorders. Transplants with
umbilical cord stem cells are recognized as an alternative to bone marrow
transplantation, and to date more than 25,000 umbilical cord stem cell
transplantations have been performed worldwide utilizing these stem cells for
unrelated recipients, family members and in some cases for the patient
themselves. Cryo-Save has cryopreserved samples from more than 70 countries on
six continents, with state-of-the-art processing and storage facilities in
Belgium, Germany, Dubai, India and South Africa.
http://www.cryo-save.com/group 

i. Renata Bizzetto et al. Outcomes after related and unrelated umbilical cord
blood transplantation for hereditary bone marrow failure syndromes other than
Fanconi anemia. Haematologica. 2011 January; 96(1): 134-141.

ii. Roy V et al. Bone Marrow Transplantation for Diamond-Blackfan Anemia.
Biology of Blood and Marrow Transplantation 11:600-608 (2005)_ 2005 American
Society for Blood and Marrow Transplantation

Contact: More information: For more information about the press release,
contact Cherie Daly, MD, Medical Advisor Cryo-Save - cherie.daly@cryo-save.com
(+41-55-222-02-56).
 
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