Advancement of pediatric blood and marrow transplantation research in North America: priorities of the Pediatric Blood and Marrow Transplant Consortium.
Advances in pediatric bone marrow transplantation (BMT) are slowed by the small number of patients with a given disease who undergo transplantation, a lack of sufficient infrastructure to run early-phase oncology protocols and studies of rare nonmalignant disorders, and challenges associated with funding multi-institutional trials. Leadership of the Pediatric Blood and Marrow Transplant Consortium (PBMTC), a large pediatric BMT clinical trials network representing 77 active and 45 affiliated centers worldwide, met in April 2009 to develop strategic plans to address these issues. Key barriers, including infrastructure development and funding, along with scientific initiatives in malignant and nonmalignant disorders, cellular therapeutics, graft-versus-host disease, and supportive care were discussed. The PBMTC's agenda for approaching these issues will result in infrastructure and trials specific to pediatrics that will run through the PBMTC or its partners, the Blood and Marrow Transplant Clinical Trials Network and the Children's Oncology Group.
Pulsipher
Michael A
MA
Primary Children's Medical Center, University of Utah School of Medicine, Salt Lake City, UT 84132, USA. michael.pulsipher@hsc.utah.edu
Horwitz
Edwin M
EM
Haight
Ann E
AE
Kadota
Richard
R
Chen
Allen R
AR
Frangoul
Haydar
H
Cooper
Laurence J N
LJ
Jacobsohn
David A
DA
Goyal
Rakesh K
RK
Mitchell
David
D
Nieder
Michael L
ML
Yanik
Gregory
G
Cowan
Morton J
MJ
Soni
Sandeep
S
Gardner
Sharon
S
Shenoy
Shalini
S
Taylor
Douglas
D
Cairo
Mitchell
M
Schultz
Kirk R
KR
D001803:Blood Transfusion / Q000379:methods* / Q000592:standards; D016026:Bone Marrow Transplantation / Q000379:methods* / Q000592:standards; D002648:Child; D006801:Humans; D009656:North America; D010372:Pediatrics / Q000379:methods / Q000592:standards; D017410:Practice Guidelines as Topic; D014184:Transplantation, Homologous