Since 1992, the Pediatric Blood and Marrow Transplant Consortium (PBMTC) has been meeting the challenge of designing safe and effective treatment protocols for children receiving stem cell* transplants. The PBMTC provides a venue for investigator-initiated and managed clinical trials to be performed among multiple institutions. Whereas the relatively small number of blood and marrow transplants required by children prevents meaningful comparison to prior or other BMT methods, the combined efforts of multiple programs provides a large enough population of patients treated similarly to draw meaningful conclusions from how well the children do with the therapy under investigation. By drawing on the combined expertise and experience of the membership, consortium members design new treatment protocols. Once developed, the protocols are reviewed by peer physicians and scientists for scientific and safety considerations. After careful analysis, new studies are shared among member institutions.
With its membership of over 70 hospitals in the US, Canada, Australia and New Zealand, serving children with cancer and other blood disorders, the research efforts of the consortium are well served. Through its cooperative work, enrollment on protocols is expanded, results of treatment are systematically collected and analyzed, and patient safety has organized oversight. Researchers and practitioners also benefit from the cooperative sharing among members.
Presently, PBMTC offers clinical trials in the areas of cancer treatment, immunological and metabolic diseases, hematological disorders, graft versus host disease, stem cell sources, and supportive care. The consortium is also a member of the recently formed Bone Marrow Transplant Clinical Trials Network (BMT) which is an NIH effort to create and institute transplant research. This is specifically funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute of the NIH.
Children’s Mercy Hospital currently serves as the Operations Center of the PBMTC. Led by Alan Gamis, MD, the current group chair, the PBMTC holds semi-annual meetings for group members. Over 500 active members, including physicians, pharmacists, nurses, and research/data staff attend and share in research efforts. Recent studies led by our local physicians, Dr. Gamis and Dr. Andrew Gilman, have investigated the use of new transplant techniques for recurrent solid tumors and pediatric brain tumors, respectively. A new study which has much of its origin and oversight at Children’s Mercy is looking at a potential new treatment for graft versus host disease.
The membership of the PBMTC continues to make strides in fulfilling its stated mission of improving the safety and efficacy of blood and marrow transplantation in children for all its current and future indications.
* Bone marrow transplants (BMT) is an all encompassing term with many synonyms including blood and marrow transplant and hematopoietic stem cell transplant. For all types of BMT, the intent is to transplant cells into a person that will grow into a functioning bone marrow that produces the blood and immune systems. The site from which these cells are obtained often divides subtype categories of BMT. These include: bone marrow transplant, peripheral blood stem cell transplant (also known as peripheral blood progenitor cell transplant), and cord blood transplant. These are the same cells, just obtained (harvested) from a different location of the body in each circumstance. BMT differs from all other types of transplants (kidney, liver, etc) because the transplanted cells must grow into a functioning organ, whereas the others are functioning from the moment they are transplanted. The cells that are transplanted are immature bone marrow cells.