ABSTRACT Busulfan (BU) has a narrow therapeutic window and the average concentration of BU at steady state (Css) is critical for successful engraftment in children receiving BU as part of

the preparative regimen for allogeneic transplants. Sixteen patients with sickle cell disease (SCD) underwent allogeneic bone marrow transplant (BMT) from HLA-identical siblings. The

preparative regimen consisted of intravenous BU 0.8–1 mg/kg/dose for 16 doses, cytoxan (CY) of 50 mg/kg daily for four doses and equine anti-thymocyte globulin (ATG) 30 mg/kg daily for three

doses. BU levels were adjusted to provide a total exposure Css of 600–700 ng/mL. The median age at the time of transplant was 6.2 years (range 1.2–19.3). Fourteen (87%) patients required

1.3–9), the event-free survival (EFS) and overall survival (OS) are both 100%. We conclude that targeting of BU Css between 600 and 700 ng/mL in this regimen can result in excellent and

sustained engraftment in young patients with SCD. Access through your institution Buy or subscribe This is a preview of subscription content, access via your institution ACCESS OPTIONS

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FOLLOWED BY REDUCED-TOXICITY MYELOABLATIVE HAPLOIDENTICAL STEM CELL TRANSPLANTATION IN HIGHLY HLA-IMMUNIZED ADULTS WITH SICKLE CELL DISEASE Article 14 March 2024 LONG-TERM OUTCOMES BY BONE

MARROW B-CELL DEPLETION FROM THE R2W TRIAL OF BORTEZOMIB WITH CYCLOPHOSPHAMIDE AND RITUXIMAB IN WALDENSTRŐM MACROGLOBULINAEMIA Article 26 February 2024 PRE-TRANSPLANT MYELOID AND IMMUNE

SUPPRESSION, UPFRONT PLERIXAFOR MOBILIZATION AND POST-TRANSPLANT CYCLOPHOSPHAMIDE: NOVEL STRATEGY FOR HAPLOIDENTICAL TRANSPLANT IN SICKLE CELL DISEASE Article 15 September 2020 REFERENCES *

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administrative support. This project was supported by the Carolyn Perot Rathjen Chair in Pediatrics, Nashville, TN, USA (Haydar Frangoul, MD). AUTHOR INFORMATION Author notes * H Frangoul

and E Yang: Haydar Frangoul and Elizabeth Yang share senior authorship. AUTHORS AND AFFILIATIONS * Department of Pediatrics, Inova Children’s Hospital, Falls Church, VA, USA S Maheshwari *

Division of Hematology and Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN, USA A Kassim * Pharmacokinetics Laboratory, Seattle Cancer Care Alliance,

Seattle, WA, USA R F Yeh * Division of Hematology/Oncology, Department of Pediatrics, Monroe Carell Jr Children’s Hospital at Vanderbilt, Vanderbilt University Medical Center, Nashville, TN,

USA J Domm, C Calder, M Evans, B Manes, V Brown, R Ho & H Frangoul * Department of Pharmacy, Vanderbilt University, Nashville, TN, USA K Bruce * Department of Hematology and Oncology,

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Maheshwari, S., Kassim, A., Yeh, R. _et al._ Targeted Busulfan therapy with a steady-state concentration of 600–700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling

bone marrow transplant. _Bone Marrow Transplant_ 49, 366–369 (2014). https://doi.org/10.1038/bmt.2013.188 Download citation * Received: 16 January 2013 * Revised: 30 September 2013 *

Accepted: 10 October 2013 * Published: 09 December 2013 * Issue Date: March 2014 * DOI: https://doi.org/10.1038/bmt.2013.188 SHARE THIS ARTICLE Anyone you share the following link with will

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content-sharing initiative KEYWORDS * sickle cell disease * busulfan * pharmacokinetics * transplant