Blood journal to publish Soliris study results in Paroxysmal Nocturnal Haemoglobinuria

The Canadian Association of PNH Patients welcomes the upcoming publication of a study (Long term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival, Kelly et al.) that resoundingly proves the efficacy of Soliris (eculizumab), the first and only life-saving treatment for the ultra-rare blood disease known as Paroxysmal Nocturnal Haemoglobinuria (PNH). The results of the study, which show that long-term treatment with Soliris allows a patient's life expectancy to return to that of a healthy person, were prepublished online in Blood, the authoritative peer-reviewed journal of the American Society of Hematology, and will appear in an upcoming print and online issue.

Currently, the provinces do not fund Soliris under their public drug plans, and Canada remains one of the last jurisdictions in the developed world that doesn't ensure access to the drug for its citizens. The issue of inequitable access to Soliris in Canada has raised a question of ethics, where those without private insurance cannot receive the treatment through their provincial drug plan, while others have access to the drug through private coverage. Since it was approved by Health Canada in 2009, the Canadian Association of PNH Patients has been pleading with the Ontario government to cover Soliris for all patients who require it. However, no decisive action has been taken to provide a long-term funding solution.

"The acceptance of these study results by the publication shows that the global medical community stands solidly behind this treatment, and supports mounting evidence that Soliris saves lives," said Barry Katsof, President of the Canadian Association of PNH Patients. "It is our understanding that Ontario is negotiating on behalf of all provinces to provide access to Soliris, but by needlessly delaying its decision, Ontario is putting the lives of PNH patients in B.C. at risk."

PNH is a very rare, progressive and fatal acquired disease that affects the blood and major organs. It develops without warning, usually when patients are in the prime of their lives. It is characterized by haemolysis (the destruction of red blood cells) which can lead to life-threatening blood clots, kidney disease, pulmonary hypertension and other catastrophic consequences.  Without treatment, approximately one‐third of PNH patients do not survive more than five years and about half die within 10 years from the time of diagnosis.

The study notes that Soliris "dramatically alters the natural course of PNH, reducing symptoms and disease complications as well as improving survival to the extent that it is equivalent to that of the general population." Numerous studies have shown that patients with PNH who received Soliris experienced a number of benefits, including an immediate and sustained reduction in chronic haemolysis. The medication also reduces blood clots, improves fatigue levels and overall quality of life while significantly reducing the need for transfusions.

"These study results add to the existing and robust body of clinical evidence for Soliris that have prompted governments worldwide to ensure that their patients with PNH do not suffer or die needlessly without access to this treatment," says Prof. Peter Hillmen, Chairman of the International PNH Interest Group and co-author of the study. "There is no doubt that the failure to provide Soliris for patients with haemolytic PNH, which I believe is the case for provincial governments in Canada, will lead to unnecessary suffering and premature deaths in patients with this devastating disease."

In Canada, there are fewer than 90 people living with PNH, and only four patients in B.C. who are awaiting publicly-funded access to Soliris.

"Without Soliris, my quality of life has suffered immensely and my major organs are beginning to fail," said Garrett Shakespeare, a 22-year-old North Vancouver resident who has lived with PNH for 11 years. "It's a horrible feeling, knowing that this amazing treatment exists, but my province will not provide me or other patients with access to it. It's like the government is telling us our lives don't matter, because we know we will die from this disease."