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Reduced-Toxicity (BuFlu) Conditioning Is Better Tolerated but Has a Higher Second Transplantation Rate Compared to Myeloablative Conditioning (BuCy) in Children with Inherited Metabolic Disorders

Gupta, Ashish, Downey, Michael, Shanley, Ryan, Jennissen, Cathryn, Miller, Weston P., Lund, Troy C., Orchard, Paul J., Smith, Angela R.·2020·Biology of Blood and Marrow Transplantation
DOI 10.1016/j.bbmt.2019.11.014
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Research works printed but not linked: 23

  1. Gooley, 2010, Reduced mortality after allogeneic hematopoietic-cell transplantation, N Engl J Med, 363, 2091, 10.1056/NEJMoa1004383 · DOI
  2. Santos, 1983, Marrow transplantation for acute nonlymphocytic leukemia after treatment with busulfan and cyclophosphamide, N Engl J Med, 309, 1347, 10.1056/NEJM198312013092202 · DOI
  3. Tutschka, 1987, Bone marrow transplantation for leukemia following a new busulfan and cyclophosphamide regimen, Blood, 70, 1382, 10.1182/blood.V70.5.1382.1382 · DOI
  4. Prasad, 2008, Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes, Blood, 112, 2979, 10.1182/blood-2008-03-140830 · DOI
  5. Gooptu, 2018, A comparison of the myeloablative conditioning regimen fludarabine/busulfan with cyclophosphamide/total body irradiation, for allogeneic stem cell transplantation in the modern era: a cohort analysis, Biol Blood Marrow Transplant, 24, 1733, 10.1016/j.bbmt.2018.03.011 · DOI
  6. Bartelink, 2014, Fludarabine and exposure-targeted busulfan compares favorably with busulfan/cyclophosphamide-based regimens in pediatric hematopoietic cell transplantation: maintaining efficacy with less toxicity, Biol Blood Marrow Transplant, 20, 345, 10.1016/j.bbmt.2013.11.027 · DOI
  7. Harris, 2018, Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine, Blood Adv, 2, 1198, 10.1182/bloodadvances.2018016956 · DOI · Paper page
  8. Ben-Barouch, 2016, Busulfan fludarabine vs busulfan cyclophosphamide as preparative regimen before allogeneic hematopoietic cell transplantation: systemic review and meta-analysis, Bone Marrow Transplant, 51, 232, 10.1038/bmt.2015.238 · DOI · Paper page
  9. Lindemans, 2014, Impact of thymoglobulin prior to pediatric unrelated umbilical cord blood transplantation on immune reconstitution and clinical outcome, Blood, 123, 126, 10.1182/blood-2013-05-502385 · DOI
  10. Admiraal, 2016, Excellent T-cell reconstitution and survival depend on low ATG exposure after pediatric cord blood transplantation, Blood, 128, 2734, 10.1182/blood-2016-06-721936 · DOI
  11. Przepiorka, 1995, 1994 Consensus Conference on Acute GVHD Grading, Bone Marrow Transplant, 15, 825
  12. Filipovich, 2005, National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and Staging Working Group report, Biol Blood Marrow Transplant, 11, 945, 10.1016/j.bbmt.2005.09.004 · DOI
  13. Mohty, 2016, Revised diagnosis and severity criteria for sinusoidal obstruction syndrome/veno-occlusive disease in adult patients: a new classification from the European Society for Blood and Marrow Transplantation, Bone Marrow Transplant, 51, 906, 10.1038/bmt.2016.130 · DOI
  14. Kaplan, 1958, Nonparametric estimation from incomplete observations, J Am Stat Assoc, 53, 457, 10.1080/01621459.1958.10501452 · DOI · Paper page
  15. Gray, 1988, A class of K-sample tests for comparing the cumulative incidence of a competing risk, Ann Stat, 16, 1141, 10.1214/aos/1176350951 · DOI
  16. Fedele, 2014, Tolerability and efficacy of busulfan and fludarabine as allogeneic pretransplant conditioning therapy in acute myeloid leukemia: comparison with busulfan and cyclophosphamide regimen, Clin Lymphoma Myeloma Leuk, 14, 493, 10.1016/j.clml.2014.04.006 · DOI
  17. Lei, 2015, Busulfan plus fludarabine compared with busulfan plus cyclophosphamide as a conditioning regimen prior to hematopoietic stem cell transplantation in patients with hematologic neoplasms: a meta-analysis, Int J Clin Exp Med, 8, 12064
  18. Myers, 2005, Adenovirus infection rates in pediatric recipients of alternate donor allogeneic bone marrow transplants receiving either antithymocyte globulin (ATG) or alemtuzumab (Campath), Bone Marrow Transplant, 36, 1001, 10.1038/sj.bmt.1705164 · DOI
  19. de Lima, 2004, Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS, Blood, 104, 857, 10.1182/blood-2004-02-0414 · DOI
  20. Aldenhoven, 2015, Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines, Biol Blood Marrow Transplant, 21, 1106, 10.1016/j.bbmt.2015.02.011 · DOI
  21. Mallhi, 2017, Allele-level HLA matching impacts key outcomes following umbilical cord blood transplantation for inherited metabolic disorders, Biol Blood Marrow Transplant, 23, 119, 10.1016/j.bbmt.2016.10.019 · DOI
  22. Saito, 2008, Impact of T cell chimerism on clinical outcome in 117 patients who underwent allogeneic stem cell transplantation with a busulfan-containing reduced-intensity conditioning regimen, Biol Blood Marrow Transplant, 14, 1148, 10.1016/j.bbmt.2008.07.013 · DOI
  23. Aronovich, 2009, Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system, Mol Ther, 17, 1136, 10.1038/mt.2009.87 · DOI

Cited by

Metadata

Authors
Gupta, A., Downey, M., Shanley, R., Jennissen, C., Miller, W. P., Lund, T. C., Orchard, P. J., & Smith, A. R.
ORCID
Venue
Biology of Blood and Marrow Transplantation
Publisher
Elsevier BV
Published
2020-03
Volume
26
Issue
3
Pages
486-492
ISSN
1083-8791
DOI
10.1016/j.bbmt.2019.11.014
Cited by
21
References
2
Language
en
Licence
Funders
National Institutes of Health

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