Interviews with the Experts Pioneering Quality of Life Research for MDS Patients | Aplastic Anemia and MDS International Foundation

Interviews with the Experts Pioneering Quality of Life Research for MDS Patients

Dr. Gregory Abel received his MD and MPH from Columbia University in 2000. He completed his postgraduate training in internal medicine at Massachusetts General Hospital and his hematology/oncology fellowship at the Dana-Farber Cancer Institute. In 2007, he joined Dana-Farber as a member of the Hematologic Oncology program, as well as a researcher in the Division of Population Sciences.

Dr. Abel is a 2010-2012 AA&MDSIF grant recipient for his research project, “Developing a Disease-Specifi c Measure for Quality of Life in Patients with Myelodysplastic Syndromes (MDS).” In this interview, he speaks about the origin, process, outcomes, and possible future applications from what was learned in his research.

How did your interest in quality of life issues for MDS patients begin?

It began by taking care of patients. As a fellow, I was lucky to be mentored by physicians such as Richard Stone and Daniel DeAngelo who have large practices of MDS patients. Most of these patients had the issues and concerns you would expect–for example, wondering how long they would live–but I noticed they also had many quality of life concerns.  For example, in addition to fatigue, they often experienced uncertainty and anxiety, changes in their day-to-day functioning, inability to care
for others, and financial issues, to name a few. In discussing these issues with my clinical mentors, we thought it would be important to measure MDS-related quality of life in a rigorous way..

How was your general interest in this area refined to the specific subject for your AA&MDSIF-funded project?

I’m a health services researcher, which means I focus on how blood cancers affect populations at large. While I was learning how to take care of patients with MDS, I also was learning how to perform research under the mentorship of the late Jane Weeks, who was a pioneer in this area for cancer. I wanted to focus on quality of life for a defined group patients. Developing a quality of life measure for MDS seemed liked a good match, since I wanted to devote my clinical time to taking care of patients with MDS.

Most of the measures that already existed only focused on general quality of life, or quality of life for patients with cancer, and while MDS has similarities to cancer, it’s not the same as other cancers like pancreatic or breast cancer. We felt that a new MDS-specific quality of life measure was needed. It was a good match because I had also learned about the program that AA&MDSIF has to help young researchers. So I applied for funding, was delighted to be awarded the grant, and that’s how I was able to develop this new instrument.

What specific activities were part of the research project?

The part that AA&MDSIF funded was the development of the tool. We first held a series of focus groups. One included patients with MDS and their partners and families. Another group included MDS physicians, and a third group contained nurses, physician assistants, social workers, and a representative from the A&MDSIF. Our mission was the same for each group: to elicit the most important ways in which MDS affects the quality of life of affected patients.

We categorized the information that we gathered from these three groups into broad domains and more specific question topics. We then refined those questions topics into actual questions and created our draft instrument, known as the Quality of Life in Myelodysplasia Scale or QUALMS-1. We next piloted it on another set of 20 MDS patients, which we interviewed one-on-one, adjusting the measure after each interview. We went through the instrument with each patient in detail, asking if the questions made sense to them and were relevant to them. We also asked if any of the questions were seen as offensive or intrusive. When we were done, we felt confident that our instrument captured many of the main concerns of patients with MDS.

The next step is to take our instrument and compare it to other ones that are not MDS-focused, but are concerned with quality of life, using a new multi-institutional group of patients (Canada, US, and Europe) to assess its performance. This is our current validation project which is being funded by the Canadian Cancer Society. This project is observational in that we are gathering information about patient’s quality of life but not intervening on the disease. We also have an interventional validation in the works which is going to be undertaken with the Clinical Trials Network that is funded by the AA&MDSIF Clinical Research Consortium.

What were the general findings, and were any of them surprising to you?

The primary observation was that around 40% of patients on the NIH trial who were refractory to immunosuppression did have a response to eltrombopag, whether platelets, white or red blood cells or any combination. Some actually were tri-lineage, meaning responses in all three cell types, some had two cell lines improve, and others were just one of the three. But 40% had a response of some kind that was clinically meaningful, for instance being able to discontinue red cell or platelet transfusions.

What are the next steps for building on the results of your research?

We’re in the process of validating the measure and figuring out the best way to score it. My hope is that once we have a validated measure, and once we understand the psychometric properties of our measure, we will see it incorporated into clinical trials. That’s one thing we will be doing through the AA&MDSIF Clinical Research Consortium. Another idea is that this measure can eventually be included in routine patient care. Some of my future work will focus on how we collect this information in the “real world” of patient care outside of clinical trials. Additionally, it might inform doctors about how patients are doing with treatments, or even if they are ready to have treatment because their quality of life is adversely affected by MDS.

What would you most like MDS patients and caregivers to know about your quality-of-life findings? Is there anything they can currently apply to their daily lives and long-term outlook?

    Through the process of developing this measure and the validation study, I have been privileged to interact with many of the MDS thought leaders in the country. I am pleased to say they really understand what we are trying to do. They care about patients’ quality of life, and they understand that this is an important outcome in addition to helping patients live longer. There is a real enthusiasm about this measure and understanding of its importance for this disease.

    So patients should feel comfortable talking to their doctors about their quality of life – for example, if they feel tired, anxious, or uninformed. These are some of the things that came out in our measure. Doctors who take care of MDS patients really do want to know about these things. So until the QUALMS-1 is ready for use in the clinic (and even after), I encourage patients can talk to their doctors about these issues. They will be surprised that the doctors are receptive to discussing issues like this in addition to disease and treatment-related topics.

    Interviewee: 

    Gregory A. Abel, MD, MPH

    Position / Title: 
    Assistant Professor in Medicine, Harvard Medical School
    Institution: 
    Dana-Faber Cancer Institute

    Dr. Abel received his MD and MPH from Columbia University in 2000. He completed his postgraduate training in internal medicine at Massachusetts General Hospital and his hematology/oncology fellowship at Dana-Farber Cancer Institute. In 2007, he joined Dana-Farber and is a member of the Hematologic Malignancies staff, as well as the Center for Outcomes and Policy Research.