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Registry Could be ‘Invaluable’ for Tracking Long-term CAR T-cell Therapy Outcomes
The Center for International Blood and Marrow Transplant Research, through an alliance with Novartis, aims to track the long-term outcomes of patients treated with chimeric antigen receptor T-cell therapies.
The center will collect data from children and adults treated with tisagenlecleucel (Kymriah, Novartis) who agree to participate in the center’s cellular therapy registry.
“We are excited to announce this collaboration with Novartis, a pioneer in the field of immunocellular therapy,” Marcelo C. Pasquini, MD, MS, senior scientific director of Center for International Blood and Marrow Transplant Research (CIBMTR) and associate professor in the department of medicine at Medical College of Wisconsin, said in a press release. “This alliance will leverage CIBMTR’s deep experience collecting and analyzing data on both short- and long-term outcomes of complex cellular therapies.”
Cell Therapy Next spoke with Pasquini about how this collaboration came about, how the outcomes data tracked through this registry can be used to guide or inform patient treatment, and whether the research center plans to incorporate data related to the other CAR T-cell therapies, such as FDA-approved axicabtagene ciloleucel (Yescarta; Kite Pharma/Gilead).
Q: How did this collaboration come about?
A: The CIBMTR has a long history of data collection on hematopoietic stem cell transplantation and, with increased use of cellular therapies for treatment of different indications, the outcomes database was expanded to capture information on recipients of these therapies. The increased use of genetically modified cells accents the importance of the outcomes database, given the FDA requires that these patients be followed for 15 years. The CIBMTR created a mechanism to capture data on any cellular therapy for several indications. The cellular therapy registry was launched in 2016 and, since then, transplant centers voluntarily have reported data on cellular therapies. The CIBMTR reached out to Novartis and Kite, among other stakeholders in the cellular therapy field, early on in the development of this cellular therapy registry. We have been working ever since to make sure all data being collected comply with regulatory requirements.
Q: Why is this registry important and necessary?
A: Initial data utilized to approve these therapies demonstrated a significant therapeutic effect. However, the number of patients treated was small, and follow-up was short. Tying the approval to a requirement to follow recipients of these agents was necessary to understand the effect of these therapies in a larger cohort of patients. Additionally, following these patients for many years can uncover important safety outcomes. Focusing on a single, standardized registry maximizes its usefulness for research.
Q: What short-term and long-term outcomes will be tracked in this registry?
A: CAR T-cell infusion has been associated with early toxicities, particularly in the first 100 days after initiating treatment. The cellular therapy registry captures all the important early safety endpoints, including cytokine release syndrome, neurotoxicities, organ toxicities, cytopenias, infections, hypogammaglobulinemia, tumor lysis syndrome and other nonhematologic organ toxicities. Long-term safety outcomes include the development of subsequent neoplasms and potential pregnancy outcomes, if the patient or partner becomes pregnant. Efficacy outcomes are also included in the follow-up to understand disease-specific best responses, and whether or when patients experience disease progression.
Q: Does CIBMTR have plans to create a similar registry for the other FDA-approved CAR T-cell therapy, axicabtagene ciloleucel?
A: The CIBMTR has a similar relationship with Kite to capture data on recipients of axicabtagene ciloleucel. The principle is the same and utilizes the same mechanism, with which centers are already familiar. This is an enormous advantage for centers, because it avoids the need to report to different databases for each CAR T-cell product. Having a single, standardized database that applies to multiple CAR T-cell products is ideal and facilitates data collection. Additionally, patients can potentially receive different CAR T cells and an HSCT in the course of their care, so having a database that can track all of these therapies is invaluable.
Q: How will word be spread through the clinical community to promote patient participation in these registries?
A: All centers that are registered with Novartis to infuse Kymriah are being trained on this registry. The same approach was done with centers registered with Kite. To date, more than 60 centers have been trained on CIBMTR data collection. All centers using CAR T cells have existing agreements with CIBMTR, as these therapies are being administered at centers with established and [Foundation for the Accreditation of Cellular Therapy]-accredited HSCT programs. New centers can easily come onboard.
Q: What is the importance of high participation rates?
A: High participation in this project will provide better understanding of outcomes. Consecutive reporting on treated patients minimizes selection bias and improves the quality of the data to support robust research in the future. The CIBMTR is reimbursing centers for data reporting. The CIBMTR also is working on developing mechanisms to capture data elements directly from the patient’s electronic medical record, which will further facilitate data-sharing. All data that are shared with the CIBMTR by centers are then accessible through an application, which allows centers to visualize their data using tables and figures or download data for other uses.
Q: How can the outcomes data tracked through this registry be used to guide or inform patient treatment?
A: The CIBMTR has mechanisms for proposal and conduct of clinical research on the outcomes database. The CIBMTR was awarded funding to serve as the Cellular Immunotherapy Data Resource, an NCI-funded project that is a part of the Cancer Moonshot Initiative with the sole purpose to establish a database with innovative approaches for data collection, dissemination and connection with other cancer data repositories within, but not limited to, NCI. – by Jennifer Southall
- For more information:
- Marcelo C. Pasquini, MD, MS, can be reached at CIBMTR, Milwaukee Campus, 9200 W. Wisconsin Ave., Suite C5500, Milwaukee, WI 53226; email: mpasquini@mcw.edu.
Disclosure: Pasquini reports no relevant financial disclosures.