Stem cell match saved life: Man

Stem cell match saved life: Man.  Vancouver native pushing campaign for Chinese donors


Our love goes out to the family and friends of Valerie Niles

January 2, 2009 at 12:00 AM EST

A 36-year-old leukemia patient who fought the Ontario government to have a stem-cell transplant funded in the United States won her case – but died before she was able to obtain the treatment, prompting calls for an investigation.

Valerie Niles’s last few hours of life were spent in an Ottawa hospital bed with her mother’s hand on her heart until its final beat on the clear, cold morning of Dec. 22.

Her death marked the end of an 11-month battle with acute myeloid leukemia, but also with government. Ms. Niles and her oncologists garnered a coveted spot for a special stem-cell transplant treatment in Seattle; she was accepted, but the Ontario Health Insurance Plan refused to fund it in August.

Although Ms. Niles, who spent much of her life in Toronto, eventually won her case on an appeal, by then seven weeks had passed and the earliest treatment opportunity was in November. Shortly before she was to leave for Seattle, she developed a complication, rendering her ineligible for the treatment, her best shot at survival gone.

“If I had gone in August, I would be in recovery now. It was OHIP’s refusal to fund our case that caused the delay,” Ms. Niles said in a telephone interview from her Ottawa hospital room five days before her death. “… I feel they did it just to save money. They do it to weed out the weak.”

Ms. Niles’s case raises disturbing questions about Ontario’s out-of-country process, which has already undergone one review after cancer patient Suzanne Aucoin of St. Catharines, Ont., was denied funding for treatment she received in the United States.

Although that external review is complete, problems persist, not only for Ms. Niles but another patient, Susan Caiger-Watson. Like Ms. Niles, she was denied similar treatment in Seattle, won on appeal but then developed a complication, making it impossible for her to obtain treatment. She died on Aug. 24, leaving behind husband Chris Fraser and daughter Sophie, 11.

“It’s one thing to fight a deadly disease but when you have to fight the system at the same time, that’s doubly deadly,” Mr. Fraser said. “You would think that after one sacrifice the lesson would be learned.”

Perry Brodkin, a health lawyer in private practice who was the sole lawyer for OHIP from 1973 to 1991, read Ms. Niles’s case and said evidence from her oncologists was overwhelmingly in her favour.

“This case cries out for a brand new investigation of OHIP’s prior approval process,” Mr. Brodkin said. “It’s not working if they deny funding in a case like this. Are we going to put another patient’s life on the line with this OHIP approval process?”

He also called on the coroner to investigate whether the government’s denial of funding and the subsequent delay was a contributing factor in Ms. Niles’s death, which he labelled a travesty.

Ontario’s out-of-country pre-approval process is seen as a medical safety valve for patients. For treatment to be approved, the procedure must be unavailable in Ontario, cannot be experimental and should be deemed medically appropriate. However, patients can have out-of-country treatment funded even if it is available in the province if there is a delay that would cause irreversible tissue damage or death. The patient’s physician must fill out part of the form.

Ontario’s Health Ministry says it has increased the number of patients approved for out-of-country funding.

In the 2002-03 fiscal year, 2,083 patients were approved for out-of-country care. From April 1, 2007, to March 11, 2008, a total of 6,132 patients had their treatment approved, according to government figures. (Those numbers do not include the referrals of emergency neurosurgery and cardiac patients sent to the United States for care.)

The number of people denied out-of-country treatments has also increased, although not nearly as much. In fiscal 2002-03, 225 patients were denied funding through the program, compared with 388 patients who were denied from April 1, 2007, to March 11, 2008.

After four rounds of chemotherapy failed to provide a remission for Ms. Niles, oncologists recommended she go to the Fred Hutchinson Cancer Research Center in Seattle, where a cancer-busting regimen of chemotherapy, total body irradiation and a radioactive monoclonal antibody that targets leukemia cells were provided.

Without this treatment, it was undisputed that Ms. Niles would die within a year, according to the decision of Ontario’s Health Services Appeal and Review Board, a panel that hears from patients who have been denied funding for out-of-country treatment.

In refusing her case on Aug. 19 last year, OHIP said the treatment is not accepted in Ontario as appropriate for a person in the same medical circumstances.

Ms. Niles saw the denial as the government “playing the odds and that’s disgusting.”

Even obtaining public documents on Ms. Niles case was riddled with obstacles. Although Ms. Niles did not ask for her name or documents to be restricted at the review board public hearing, the exhibits and efforts to contact her were achieved only after a lawyer became involved on behalf of The Globe and Mail.

Lorri Puil, an Ottawa-based physician scientist who also does medical advocacy, spent two months researching the case for Ms. Caiger-Watson’s appeal at no cost and was also involved with Ms. Niles’s case.

“I’ve seen patients who are dealing with a life-threatening disease, they’re trying to do the best they can with it and all of a sudden they are hit with the fact that their provincial government won’t pay for their treatment,” Dr. Puil said. “And so they get into this bureaucracy they shouldn’t have to deal with and are put through an extraordinarily painful process.”

Indeed, Mark Minden, medical oncologist and senior scientist at Princess Margaret Hospital and the Ontario Cancer Institute, who spent a full day of his own time testifying at the appeal of Ms. Niles, said the legislation to obtain out-of-country treatment can be prohibitive, especially when it involves cancer cases.

“I saw the legislation as not being very fair,” said Dr. Minden, who holds the Philip S. Orsino Chair in Leukemia Research at Princess Margaret. “I think it was denying people treatment that was potentially lifesaving.”

Dr. Minden said that if Ms. Niles had gone to Seattle, she had a 20- to 25-per-cent chance of survival at three years, compared with 10 to 15 per cent if she received treatment in Ottawa. The Seattle treatment would cost roughly $500,000 (U.S.).

He stressed that novel therapies to treat patients like Ms. Niles are expected to be available in Ontario this year.

With the Seattle treatment no longer possible, Ms. Niles went to the Ottawa Hospital, where she was to have a stem-cell transplant; her brother, Judd, a perfect match, was her donor. However, after two days of intensive chemotherapy, she developed complications and stopped breathing.

A newlywed, she leaves husband Jean-Marc Salvagno of Belleville, who owns a French bistro. “She was so full of life,” he said.

Yesterday, he was retrieving her ashes.

“Bureaucracy seems to have a lot to do this,” said mother Marianne Niles, 66, who is holding a private memorial of family and friends today. “She was good to go in the summertime. The doctors tried so hard; they all worked so hard for her.”

Click here for your local Registry’s link and REGISTER TODAY to help Carolyn and Others.