Following the publication of results from a phase 2 study of eribulin in two vascular sarcomas, angiosarcoma and EHE, Dr. Tom Chen of National Taiwan University College of Medicine shared commentary in Clinical Cancer Research. He praised the investigators for their collaborative study while also pointing to important considerations for future research. Although angiosarcoma and EHE share certain features as vascular sarcomas, they are distinct diseases and should be evaluated accordingly. Dr. Chen emphasized the need for innovative, disease-relevant assessments and endpoints, as well as careful, histology-specific (disease-specific) interpretation of response data.

Dr. Chen proposes a three-pronged approach to developing endpoints for ultra-rare sarcomas such as EHE.

First, interpretation of results from clinical trials such as the eribulin study, which include patients with multiple types of sarcoma, must be done in a “histology-specific” manner. This means that conclusions about EHE in this case may not necessarily be applied to other sarcomas, and vice versa. 

Second, Dr. Chen argues that assessment methods, the tools that researchers and doctors use to determine if the cancer has responded to the treatment, must also be tailored to the unique features of the cancer being studied. This means that the ways researchers and scientists measure success for a patient with EHE might be different than the ways they measure success for a patient with another sarcoma. 

Finally, Dr. Chen emphasizes that clinicians and scientists, patient advocates, and regulatory agencies (such as the FDA in the United States and the EMA in Europe) must all be part of the conversation when designing and conducting trials like this. This collaboration will help ensure that results are not only interpretable but also usable in the effort to develop new therapies for patients around the world.

Why this matters:
This commentary highlights that clinical trials in ultra-rare cancers like EHE are more beneficial when designed and interpreted with consideration of a disease’s unique characteristics.

For additional insight, read our summary of the publication of the Eribulin Phase 2 study.