-International study published in leading medical journal underscores
efficacy of Soliris® -
TORONTO, July 25, 2013 /CNW/ - Only a few months ago, a very small group
of Canadians affected by atypical Hemolytic Uremic Syndrome (aHUS)
rejoiced upon hearing the news that the drug Soliris® (eculizumab) had
been approved for a second ultra-rare disorder - the one that threatens
their lives. Today, these same patients - approximately 60 children and
adults in Canada - fear access to this life-saving treatment may be
delayed or restricted due to a recommendation made by the Common Drug
Review (CDR) to provincial and territorial drug programs not to fund
Soliris is the first and only pharmaceutical treatment approved for aHUS
in children and adults, and is hailed by experts worldwide as a
critical breakthrough in altering the course of the disease. It has
been shown to significantly improve patients' health and quality of
lifei. In clinical trials, Soliris was proven effective in preventing blood
vessel damage and abnormal blood clotting,ii,iii leading to remission and significant improvement in kidney function.iv,v Soliris has also allowed patients to discontinue dialysis and plasma
"Before starting Soliris, I experienced extreme weakness, constant
fatigue, heart problems, and life-threatening reactions to frequent
plasma exchange treatments that prevented me from working and providing
for my young family - a very difficult reality for anyone to face,"
says Brian Tjepkema of Abbotsford, B.C., who was diagnosed with aHUS in
2010. "I have been taking Soliris for a year now, I no longer live in
fear of suffering a deadly stroke, and my health has almost returned to
normal. I am grateful that I can once again be the husband and father
I've always known I can be."
Recently-published international evidence supports efficacy
The results of a recently-published clinical trial co-led by one of the
leading international aHUS experts, Dr. Christoph Licht, coupled with
the existing body of evidence for Soliris show that the treatment not
only has the potential to save the lives of children and adults
affected by the disease, but also to improve health-related quality of
life. According to the international study,vi which appeared in the New England Journal of Medicine in June 2013,
Soliris was able to restore control of part of the patient's immune
system known as the complement system. Whereas, the traditional
treatment of plasma infusion/exchange only achieved partial control.
Patients in the study treated with eculizumab were able to discontinue
plasma infusion/exchange and dialysis therapies. Results also showed
that Soliris improved kidney function, reduced blood vessel damage and
decreased the risk of blood clots.
"For the first time, we have a demonstrated treatment for aHUS that
directly and effectively targets the underlying cause of this
devastating disease, bringing a life-altering benefit to patients,"
says Dr. Christoph Licht, Associate Professor of Pediatrics, Division
of Nephrology at The Hospital for Sick Children, Toronto, and Medical
Advisor to aHUS Canada. "My international colleagues and I concur that
Soliris is a critical tool that can have a positive impact on patients'
Families hope funders look beyond CDR
In 2009, Soliris was first approved in Canada for another ultra-rare
blood disease called paroxysmal nocturnal hemoglobinuria (PNH), and
despite receiving a negative CDR recommendation, it is now accessible
to patients across the country through private and public drug plans.
Families like the DeBortolis from Vaughan, Ont. who see the benefits of
Soliris in their 11-year-old son Joshua, can only hope that provincial
and territorial drug plans will once again base their funding decisions
on the advice of aHUS experts and the strength of clinical evidence,
rather than a review process ill-designed to get medications for rare
disorders to patients quickly.
"We are fearful that any delay in funding could mean that patients'
lives will remain needlessly at risk. We know how fortunate our son was
to receive Soliris when he did, it's heartbreaking to think that other
children, or adults, may not be so lucky," says Sonia DeBortoli, a
member of the aHUS Canada Board of Directors. "Still, we have to be
optimistic that governments will look beyond CDR and act quickly to
provide publicly-funded access to this life-saving treatment for aHUS
patients, before any more damage is done to life, or limb."
aHUS is a very rare, chronic and life-threatening genetic condition,
which leaves a part of the immune system (known as the complement
system) uncontrolled and always active. As a result, the immune system
attacks the body's unhealthy and healthy cells which can cause blood
vessel damage, abnormal blood clottingvii,viii and progressive damage to the major organs, leading to heart attack,
stroke, kidney failure and death.iv
The management of aHUS has relied on plasma infusion and plasma exchange
therapies with variable results.v These lifelong therapies are costly, painful and time-consuming, and
have not been studied or approved for the treatment of aHUS. If kidney
failure has already occurred as a result of aHUS, dialysis is required,
though it is not a curative treatment. Within a year of diagnosis, over
half of patients will need dialysis, will have irreversible kidney
damage, or will not survive.ix The majority of aHUS patients progress to end-stage kidney failure
within three years of diagnosis.xi,xii
About aHUS Canada
aHUS Canada was formed in November 2012 to support Canadian patients and
families living with aHUS. In addition to establishing a Canadian aHUS
community, the group is committed to building public awareness and
understanding of aHUS and advocating for the best possible care and
treatment for patients. For more information, please visit www.ahuscanada.org.
i Kim J, Waller S and Reid C. Clinical Report: Eculizumab in atypical
haemolytic-uraemic syndrome allows cessation of plasma exchange and
dialysis. Clin Kidney J. 2012;0:1-3.
ii Legendre C, Babu S, Furman R, et al. Safety and Efficacy of Eculizumab
in aHUS Patients Resistant to Plasma Therapy: Interim Analysis from a
Phase 2 Trial. Abstract presented at the 43rd annual meeting of the
American Society of Nephrology, Denver, CO, USA, 16-21 November 2010.
iii Muus P, Legendre C, Douglas K et al. Safety and Efficacy of Eculizumab
in aHUS Patients on Chronic Plasma Therapy: Interim Analysis of a Phase
2 Trial. Abstract presented at the 43rd annual meeting of the American
Society of Nephrology, Denver, CO, USA, 16-21 November 2010.
iv Noris M and Remuzzi G. Review Article: Atypical Hemolytic-Uremic
Syndrome. N Engl J Med 2009;361:1676-87.
v Mache C, Acham-Roschitz B, Frémeaux-Bacchi V, et al. Complement
Inhibitor Eculizumab in Atypical Hemolytic Uremic Syndrome. Clin J Am
Soc Nephrol. 2009;4:1312-1316.
vi Legendre C.M., Licht, C., Muus P. et al. Terminal Complement Inhibitor
Eculizumab in Atypical Hemolytic-Uremic Syndrome. N Engl J Med
vii Benz K and Amann K. Thrombotic microangiopathy: new insights. Curr Opin
Nephrol Hypertens. 2010;19(3):242-247.
viii Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney
Int 2006 Jul;70(1):16-23.
ix Caprioli J, Noris M, Brioschi S, et al. Genetics of HUS: the impact of
MCP, CFH, and IF mutations on clinical presentation, response to
treatment, and outcome. Blood. 2006;108:1267-1279.
x The Kidney Foundation of Canada. Dialysis. Accessed on March 1, 2013.
Available at http://www.kidney.ca/page.aspx?pid=337
xi Noris M, Caprioli J, Bresin E, et al. Relative role of genetic
complement abnormalities in sporadic and familial aHUS and their impact
on clinical phenotype. Clin J Am Soc Nephrol. 2010;5:1844-1859.
xii Kavanagh D and Goodship T. Atypical Hemolytic Uremic Syndrome, Genetic
Clinical Manifestations. Acquired Hematopoietic Disordrers:
Complement-Mediated Blood Disorders. 2011:15-20.
SOURCE: aHUS Canada
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