Assessing Treatment Goals for Patients with MDS


Drs Zeidan and Fazal share their opinions on setting appropriate goals for treatment of patients with MDS.

Ryan Haumschild, PharmD, MS, MBA: Let’s talk a little more about the management of myelodysplastic syndrome [MDS]. What are some of the treatment paradigms we’re experiencing? What are some of the standard-of-care therapies for different patient types? Before we start with those therapies, we have to understand the goals. We’ve talked about what we’re looking for. Dr Zeidan, you’re probably an expert. What are some of the goals of therapy for patients with MDS from your perspective? You stratify patients by low risk vs high risk. What are the goals of therapy between those 2 patient populations?

Amer Zeidan, MBBS, MHS: These are good data to emphasize, because sometimes in the middle of the details of the treatment, people can lose focus of the big goal. It’s very important to emphasize to the patient what you’re trying to achieve. For patients who have lower-risk MDS, we don’t have treatments that were proved in a randomized fashion to improve survival. Because many of those patients can live multiple years—sometimes even a decade—and they’re in their 70s, the goal of the treatment is generally to try to improve their quality of life, minimize the complications of the MDS, especially issues related to anemia, infection, bleeding, and to try to keep them out of the hospital. We’re cautious about not trying to do therapies that might compromise the quality of life of patients. This is a very important thing to consider when you’re thinking about treatments for patients.

On the other hand, patients with high-risk MDS have a significantly compromised survival without treatment. If you look at the different prognostic scores we discussed earlier, the median survival can be less than a year for untreated patients. For those patients, we’re trying to improve their survival. In some cases, you can go for a cure. However, you can’t cure a patient with MDS without a bone marrow transplant, which doesn’t apply to the vast majority of patients with MDS. It applies only to the occasional younger and healthier patients. Your goal most of the time becomes improvement and survival, and ideally improvement in quality of life. The only way to achieve that is with hypomethylating agents. In addition, it’s important to consider other factors, such as the patient goals and their comorbidity situation, because those also are affecting how you recommend treatment for your patients.

Ryan Haumschild, PharmD, MS, MBA: I like how you establish goals clinically, such as survival and quality of life, and how you incorporated taking patient input into the decision. That’s sometimes so important, especially as we’ve learned in this disease state that a key component is trying to preserve quality of life and making sure their activities of daily living are OK, along with functional life assessments. When we think about those and we recognize that quality of life is a huge portion of the goals of patients with MDS, I’m always curious how clinicians are evaluating this. How do they look forward in the literature within their patients? I’m going to start with you, Dr Fazal, and get your thoughts. Are quality-of-life assessments valuable to you in MDS? Is it something that you look for in the literature? How are you using those assessments and applying them to your clinical practice?

Salman Fazal, MD: Patients with myelodysplastic syndrome experience a lot of symptoms related to low blood counts. They experience fatigue, shortness of breath, insomnia, and anxiety. On top of that, these patients with a median age of 70 [years] have several comorbidities that also add to quite a lot of symptoms that they’re experiencing. Because of the low blood counts, significant cardiac comorbidity, or pulmonary comorbidities, they’re going to experience worse symptoms.

In terms of the quality-of-life assessment, there have been several attempts made in terms of assessing the quality of life in myelodysplastic syndrome. The major comprehensive attempt was developed by Dana-Farber [Cancer Institute]. They have developed Quality of Life in Myelodysplasia Scale, which has 38-item scoring and a score of 0 through 100. They identified patients who had anemia with a hemoglobin of less than 8 g/dL. They had worse symptoms compared with people who had hemoglobin above 10 g/dL. Similarly, they saw that patients who were transfusion dependent had worse scores compared with patients who weren’t transfusion dependent. However, in clinical practice, these scores haven’t been very well established or used.

We believe that these patients are experiencing a lot of these symptoms. In certain other diseases, a lot of the clinical trials have also included quality of life to assess the response to therapy in these patients. For patients with low-risk MDS, as Dr Zeidan mentioned, our goals of care are somewhat different. We have to make sure that the therapies we’re offering these patients are also improving their quality of life while they improve their hematologic parameters.

One thing I always found intriguing by this study for high-risk MDS was that patients’ self-reported fatigue had a very significant impact on prognosis, and these patients had worse prognosis compared with patients who didn’t report fatigue. As a clinician, I always make sure that these assessments are done from time to time to assess how our therapies are impacting their quality of life. Unfortunately, we should be paying more attention in terms of including quality of life in our daily assessment of these patients.

Ryan Haumschild, PharmD, MS, MBA: It sounds like quality of life is important and something providers are aware of, but how do we keep putting that to the forefront as we’re measuring the impacts on the patients? Fatigue is a great example. It impacts the patient, their ability, and their absenteeism if they’re working, if they aren’t in that aging area of their life. Those are important assessments. Sometimes we manage that through supportive care. It’s one way to keep up with patients and to make sure we’re preserving their functionality.

Transcript edited for clarity.

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