Historically, clinical trials have played an immense role in collecting patient data, understanding disease progression, and crafting more targeted treatments. This is especially true in the oncology realm. But according to Healio, enrollment in cancer-related clinical trials is often woefully low. In fact, around 86% of all oncological trials do not enroll enough participants, and about 1/5 of all trials close early due to this. Clinical trials examining myelodysplastic syndromes (MDS) often grapple with low enrollment. Because of this, it is difficult to develop or test potential treatments.

Clinical Trial Difficulties

So why is it so tough to hold adequate clinical trials around MDS? According to Dr. Charlotte K. Brierly, MD, there are a few reasons for this. First, MDS typically affects older individuals. When examining data from 1,919 patients with MDS treated over a 26-year period, they found that age played a distinct role in whether or not patients might pursue clinical trial enrollment. Of the 1,919 patients, only 449 patients participated in any form of clinical trial. Brierly and other researchers determined that those who participated were often males and younger than the median age (70s) of patients with MDS. Older patients might find it difficult to travel, particularly if they are dealing with other health concerns. Additionally, traveling requires some financial burden. More affluent patients are also more likely to participate than those in lower socioeconomic positions.

Next, clinical trials often see an overrepresentation of certain types of MDS, with an underrepresentation of others. This makes it harder for researchers to study MDS or develop adequate therapies. Says Dr. David P. Steensma, MD:

“There might not be trials open for a particular disease, because many of the disease subtypes are rare.”

Finally, Dr. Brierly explains that enrollment criteria may exclude certain groups. In many cases, patients with MDS may have comorbidities or a history of other cancers. This excludes them from certain clinical trials. By reducing various restrictions, Dr. Brierly believes that clinical trials can become more helpful and inclusive.

Myelodysplastic Syndromes (MDS)

Researchers aren’t quite sure what causes myelodysplastic syndromes (MDS), progressive conditions which prevent the formation of healthy blood cells (red and white blood cells, platelets) in bone marrow. However, some believe the conditions result from a mixture of genetics, chemotherapy, or chemical exposure. Overall, there are five subsets of MDS: refractory anemia, refractory anemia with sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia. In all cases, the cells either have short lives or stay in the bone marrow. However, they never mature. In about 50% of all cases, MDS progresses to become acute myeloid leukemia (AML).

Generally, MDS affects patients over the age of 60. It impacts males more than females. At the early stages, there are few symptoms. However, as the condition progresses, patients may experience:

• Frequent lung, sinus, urinary tract, or skin infections
• Fatigue
• Chest pain
• Shortness of breath / difficulty breathing
• Neutropenia (low white blood cell count)
• Anemia (low red blood cell count)
• Heart palpitations
• Thrombocytopenia (low platelet count)
• Easy bruising and bleeding

Learn more about MDS.