Two Iowa researchers—third-year hematology and oncology fellow Kittika Poonsombudlert, MD, and Christopher Strouse, MD, clinical assistant professor in Hematology, Oncology, and Blood & Marrow Transplantation—will examine potential cardiovascular risks from a drug in a recently approved study. The proposal, “Post-Transplant Cyclophosphamide Related Cardiomyopathy; incidence, risk factors, and outcome,” was approved by the Center for International Blood and Marrow Transplant Research (CIBMTR), granting the researchers access to the center’s database in order to conduct their retrospective study. CIBMTR is a unique resource, collecting federally mandated data from all US transplant centers as well as those in more than 30 countries. The center’s large and high-quality pool of data provides information that would otherwise be unavailable, making this a crucial opportunity for the duo’s large-scale question.
Poonsombudlert and Strouse’s proposal was only one of a few among the 380 submissions to be approved by CIBMTR. They developed this question when they encountered a patient who had an acute heart failure following the use of cyclophosphamide in the course of treatment. Strouse noted that they look to CIBMTR resources when faced with questions they don’t have satisfactory answers to while caring for patients. Poonsombudlert credits Strouse’s guidance in shaping the question. Strouse stresses that the development was collaborative, noting, “CIBMTR has recognized the importance of mentorship and supporting young investigators.”
Cyclophosphamide is used to prevent graft-versus-host disease, in which the transplanted donor’s immune system attacks the recipient patient’s organs. Its use allows for a larger donor pool for the recipient because the matches do not need to be as precise. But, as in the case observed by Poonsombudlert and Strouse, cyclophosphamide also may cause severe cardiomyopathy in certain patients. Knowing what factors increase that risk is a critical piece of information for doctors and their transplant patients so they can make better informed decisions about treatment. Poonsombudlert said, “So that is why we went back and looked through the literature, and other centers seem to have known this is a problem too.” She explained that getting access to data held by CIBMTR, gathered from so many institutions, could allow them to confirm the link, define the incidence, how often it is occurring and to whom. It will provide a much more thorough understanding of the risk factors involved. Mitigating those risks, Poonsombudlert said, is the long-term goal.
When asked about the scope of the data they will be granted access to for their study Strouse explained, “We have a protocol writing team which will include Kit, myself, some collaborators from other institutions, and also representatives from the CIBMTR. We will define what population of patients will be best to answer our question. Once the protocol is finalized, data from the patients from the whole CIBMTR database will be drawn for the analysis.” Because this is Poonsombudlert’s first project working with CIBMTR, she sees a double value in the work, getting to answer an important question but also to learn more about how the whole process works.