Science Stories

What if we could pinpoint biomarkers to detect deadly transplant complications before symptoms occur?

Those who have recently heard the words ’you have cancer’ or ’we need to start you on a treatment regimen right away’ may have experienced overwhelming feelings around the extensive list of possible side effects from treatment. Chemotherapy can seem especially scary for hematopoietic stem cell transplant (HSCT) patients, due to its potentially damaging effects on the bone marrow and, consequently, the production of new blood cells. HSCT can provide an important option in improving outcomes for appropriate patients because it restores bone marrow and blood cells while high-dose chemotherapy works to eliminate the cancer cells for cancer patients and prepare the marrow to accept new donor cells.1 HSCT can also be applied to help improve quality of life for those living with a variety of genetic diseases such as metabolic conditions, immune deficiencies, and red cell and platelet disorders, among others.2

“Many patients who suffer from advanced or refractory disease look to HSCT as their last hope for prolonged survival, yet those who undergo these transplants may encounter a life-threatening complication called veno-occlusive disease, or VOD,” said Beth Hamilton, Senior Director of Hematology/Oncology Marketing at Jazz Pharmaceuticals. “VOD has been reported to occur in approximately 13.7 percent of adult and pediatric patients who undergo stem cell transplants, with risk being greater for those with preexisting conditions or previous medications. That’s why Jazz is using its expertise to focus on specific patient populations to treat VOD successfully.”

While some people do recover from VOD without treatment, it can cause various symptoms including pain and tenderness in the abdomen, especially on the upper right side due to swelling of the liver; rapid weight gain; and yellowing of the skin, known as jaundice. VOD can also quickly affect the function of some of the most important organs in the body in addition to the liver, most notably the kidneys and lungs, which may become life-threatening.3-4

“Research has shown that early treatment intervention for VOD can improve survival outcomes; however, there is limited medical literature or pharmaceutical attention on biomarker research for this rare disorder,” said Missy Stricherz, Associate Medical Director for Hematology/Oncology Medical Affairs at Jazz. “If we can pinpoint biomarkers that reliably detect VOD prior to the onset of clinical symptoms, it may lead to better outcomes.”

Because an HSCT patient’s outcome is significantly altered by VOD – which typically occurs within three weeks following the HSCT infusion and where mortality rate can be more than 80 percent in patients with renal or pulmonary dysfunction,5 early detection is critical. The ability to intervene early-on in VOD also can minimize additional organ complications which may require supportive care, medications and longer time spent in the hospital. Finally, for patients undergoing HSCT, identifying those who are suspected to be at high risk of developing VOD allows healthcare professionals to make the most impactful use of existing interventional treatments.

In partnership with leading institutional and academic blood disorder experts, Jazz is undertaking research to identify a novel biomarker signature that appears early after HSCT. Detection of VOD prior to clinical manifestations could substantially improve clinical management and patient outcomes.6

“By focusing our research on VOD detection as part of a multisectoral collaboration, we are connecting with some of the brightest minds in biomarker research to ultimately improve clinical management and outcomes for transplant patients in an area that few others are focused on,” said Stricherz. “Our R&D teams are dedicated to strengthening relationships with fellow researchers and HCPs to redefine possibilities for patients where unmet medical need is the highest.”

Jazz aims to continue its R&D efforts in collaboration with institutional and academic partners to advance novel scientific discoveries that go beyond the use, or increase the efficacy of, its existing medicines. By promoting research and education that can be implemented in clinical practice for the transplantation field, Jazz hopes to increase survival post-HSCT – which we know has a lasting, meaningful impact for patients and their families.

Stem cell transplants are an intensive and complex approach that can cause life-threatening side effects. If you’re considering a stem cell transplant, be sure to speak with your doctor about complications related to VOD that may occur following transplant. Your doctor can also help to determine which type of HSCT works best for you, as options differ depending on diagnosis.1

1American Cancer Society. Stem cell transplant for acute lymphocytic leukemia (ALL). Updated October 17, 2018. Accessed January 12, 2021.
2Steward CG, Jarisch A. Haemopoietic stem cell transplantation for genetic disorders. Arch Dis Child. 2005;90:1259-1263.
3Canadian Cancer Society. Veno-occlusive disease (VOD). Accessed January 12, 2021. Signs and symptoms of VOD. Accessed February 5, 2021.
5Coppell JA, Richardson PG, Soiffer R, et al. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010 Feb;16(2):157-68.
6Putta S, Young B, Ho VT, et al. Prognostic biomarker signature for hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) in recipients of myeloablative (MA) allogeneic hematopoietic cell transplantation (HCT). Accessed February 3, 2021.