1904 - Treosulfan for BMFD

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  • Protocol Short Name and Number: Treo for BMFD, 1904
  • Protocol Long Name: Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases
  • Protocol Synopsis and/or Protocol (PDF)
  • ICFs (PDF)
  • FAQs (PDF)
  • Key Personnel
    • BMT CTN # 1904 Co-Chair: Lauri Burroughs, MD
    • BMT CTN # 1904 Co-Chair: Margaret MacMillan, MD, MSc
    • BMT CTN # 1904 Protocol Officer: Mehdi Hamadani, MD
    • BMT CTN # 1904 Protocol Coordinator: Megan Scott; bmtctn1904@emmes.com
  • Keywords: Treosulfan, Bone Marrow Failure Disease, BMFD, Diamond Blackfan Anemia, DBA, Shwachman-Diamond Syndrome, SDS, Congenital Sideroblastic anemia, CSA, GATA2, SAMD9, SAMD9L, Bone Marrow Failure, Marrow Failure, Congenital amegakaryocytic thrombocytopenia, Paroxysmal nocturnal hemoglobinuria, PNH, Hematopoietic Cell Transplant, HCT, HSCT, Allogeneic Transplant, Allo Transplant, Allo, Unrelated, Unrelated Donor, Quality of Life, QOL, Patient Reported Outcomes, PROs, Pediatric, Child, Children, Teen, Adolescent