"It’s important that doctors tweak out what the objectives are for each individual patient and try to facilitate these in terms of how they are managed, and what treatments options are available," Mr. Low said. Quality of life should be considered as much as clinical outcomes, he suggested.
RareConnect Gathers Patient-Reported Information
An initiative called RareConnect could help provide important information about the effects of treatment from the patient perspective. Run by the EURODIS (European Organization for Rare Diseases), and by the U.S.-based NORD (National Organization for Rare Disorders), RareConnect is a social network of rare-disease communities that offers users the opportunity to assess patient-reported quality of life via a series of Internet-based questionnaires.
"What we are trying to do is bring a certain sense of structure now to the way patients can report on outcomes, essentially," said Denis Costello, who is leading the RareConnect project on behalf of EURODIS.
A pilot project is underway in patients with myeloma, he added in an interview. The Internet-based tool involves a series of interactive questionnaires that patients answer at a minimum of once a week. The collected information will include when patients were diagnosed, what treatments they have used, and their sliding-scale ratings of any side effects they have experienced.
RareConnect also allows patients to see how they compare with the rest of the community, and they can discuss their condition with others who are in a similar situation.
To develop a patient-friendly tool with the right level of lightness has been challenging because physicians want it to follow the lines of the quality of life scales and currently available instruments, Mr. Costello said. Physicians "want to get statistically significant data, but patients, we know, are not going to fill in those kinds of questionnaires."
Ultimately, "we want to provide a tool to patients to help them better self-manage their own outcomes in conjunction with their [health care professional], because we feel that this tool can help the patient, and bring a new dynamism between patient and doctor," he explained.
"It’s about discussion, really," Mr. Costello added, noting that the tool could help patients be more aware of their treatment and how it may affect them. In myeloma, for example, it could help prevent patients’ stopping treatment too early and later suffering a relapse.
"We really want to empower patients to see that [perhaps] you need to stay on the treatment a bit longer, and sometimes you need to fight," he suggested. RareConnect and other similar initiatives could provide the starting evidence that patients need to discuss such issues with their doctor.
Measuring Quality of Life
The European Hematology Association’s Scientific Working Group on Quality of Life and Symptoms has just published guidelines on how to assess quality of life in patients with hematologic disorders. Aimed at the practicing hematologist, these guidelines cover hematological malignancies, as well as other blood conditions.
"As physicians, as researchers, we understand that we do have new treatment modalities; we are able to provide good quality of care, but it very important to have the patient perspective to know if we are doing everything in the right way," said Tatyana Ionova, Ph.D., professor of the Postgraduate Education Institute at the National Medical Surgical Center Northwestern Branch in St. Petersburg, Russian Federation.
"Sometimes we may have information about the success or benefit or risks of treatment only from the patient perspective. That is why we try to develop the instruments, [which are] standardized measures that are able to ask the patients about their physical, social, psychological well-being, about [their] symptoms," Dr. Ionova said.
The EHA guidelines on "Patient-Reported Outcomes in Hematology" recognize that there are differences between hematologic diseases in terms of the quality of life assessments that can be used, "because bone marrow transplantation is very different from ITP [immune thrombocytopenic purpura," Dr. Ionova noted.
So how should hematologists measure quality of life? "First, read the guidelines," she suggested. "Then, have some very simple training about the questionnaires and how to use them."
These questionnaires shouldn’t take very long to be completed by or with the patient, she added. The important thing is that quality of life is considered.
The Myeloma Euronet survey was made possible through an unrestricted grant from Ortho Biotech. All authors reported no relevant disclosures other than working for their respective organizations.