title: Advancement of pediatric blood and marrow transplantation research in North America: priorities of the Pediatric Blood and Marrow Transplant Consortium.
abstract: 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.
authors.affiliation: Primary Children's Medical Center, University of Utah School of Medicine, Salt Lake City, UT 84132, USA. michael.pulsipher@hsc.utah.edu
authors.forename: Michael A
authors.initials: MA
authors.lastname: Pulsipher
authors.forename: Edwin M
authors.initials: EM
authors.lastname: Horwitz
authors.forename: Ann E
authors.initials: AE
authors.lastname: Haight
authors.forename: Richard
authors.initials: R
authors.lastname: Kadota
authors.forename: Allen R
authors.initials: AR
authors.lastname: Chen
authors.forename: Haydar
authors.initials: H
authors.lastname: Frangoul
authors.forename: Laurence J N
authors.initials: LJ
authors.lastname: Cooper
authors.forename: David A
authors.initials: DA
authors.lastname: Jacobsohn
authors.forename: Rakesh K
authors.initials: RK
authors.lastname: Goyal
authors.forename: David
authors.initials: D
authors.lastname: Mitchell
authors.forename: Michael L
authors.initials: ML
authors.lastname: Nieder
authors.forename: Gregory
authors.initials: G
authors.lastname: Yanik
authors.forename: Morton J
authors.initials: MJ
authors.lastname: Cowan
authors.forename: Sandeep
authors.initials: S
authors.lastname: Soni
authors.forename: Sharon
authors.initials: S
authors.lastname: Gardner
authors.forename: Shalini
authors.initials: S
authors.lastname: Shenoy
authors.forename: Douglas
authors.initials: D
authors.lastname: Taylor
authors.forename: Mitchell
authors.initials: M
authors.lastname: Cairo
authors.forename: Kirk R
authors.initials: KR
authors.lastname: Schultz
mesh_terms: 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