On Feb. 12, Larisa Broglie, MD, MS, will give an overview of a study being developed as part of the Blood & Marrow Transplant Clinical Trials Network (BMT-CTN), which aims to build a biorepository of samples from patients with non-malignant diseases who will undergo hematopoietic cell transplantation and gene therapy for select diseases.

BMT CTN Non-Malignant Biorepository will begin at 8:55 a.m. in Room 316 ABC as a way to inform coordinators who will be helping participating centers enroll patients in the BMT-CTN and National Heart, Lung, and Blood Institute (NHLBI)-supported study when it opens later this year.
“Instead of being an intervention protocol where people are enrolled in a study and get a certain course of treatment, this study will collect patient samples — possibly blood or stool — and store them for researchers to come back later and perform different types of research,” said Broglie, a pediatric transplant physician at the Children’s Hospital of Wisconsin, the scientific director of the CIBMTR’s Pediatric Cancer and Non-Malignant Diseases Working Committees, and a protocol officer for the CIBMTR Clinical Research Organization (CRO) and BMT-CTN.
She will detail the intent of the upcoming study, how it will differ from other BMT-CTN studies, how to prepare each site to screen patients for enrollment, and how to integrate possible inclusion in the study into the evaluation of patients at the center.
“Transplants for non-malignant diseases require a different approach and considerations than those for patients who have cancer and malignancies,” Broglie said. “Having this biorepository not only lends to new research on the underlying disease and its response to transplant and gene therapy but also helps inform specific outcomes and how to achieve better transplant outcomes for these patients.”
Study coordinators are integral to the development of the biorepository, as a critical mass of enrolled patients and samples is needed for its success and utility to future medical advances on behalf of patients with non-malignant diseases.
“We hope researchers in the future can use this valuable resource of patient samples to gain valuable information on different non-malignant diseases to better outcomes for patients,” Broglie said.
This and other sessions at the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR® will be available for on-demand viewing for registered attendees following the live presentation.
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