Hematopoietic stem cell transplantation for childhood myeloid malignancies after high-dose thiotepa, busulfan and cyclophosphamide (original) (raw)

Conditioning Regimens

Bone Marrow Transplantation volume 24, pages 947–952 (1999)Cite this article

Abstract

Seventeen children with advanced myeloid malignancies (induction failure, relapse, myelodysplasia, secondary AML, or CR >1) received thiotepa 750 mg/m2 i.v., busulfan 12 mg/kg or 640 mg/m2 p.o., and cyclophosphamide 120 mg/kg i.v. as a preparative regimen for allogeneic or autologous hematopoietic stem cell (HSC) transplantation. Of the 15 allogeneic transplants, eight were from matched siblings, one was from a mismatched sibling, and six were from unrelated donors. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine or tacrolimus and methotrexate. Regimen-related toxicity was common but tolerable, affecting mainly the skin and gastrointestinal tract. Three patients died early and were not evaluable for engraftment; engraftment occurred in the remaining patients. Nine patients with active disease at the time of transplant were evaluable for response; all achieved remission. With a median follow-up of 40 months (range, 10–71 months), nine patients are alive and disease-free. The 3-year actuarial event-free survival was 51% (95% confidence interval (CI) 27–76%). Seven patients died of transplant-related complications: infection (n = 4), chronic GVHD (n = 1), veno-occlusive disease, VOD, (_n_= 1) and pulmonary alveolar hemorrhage (n = 1). Only one patient had leukemia relapse and died. We conclude that the use of high-dose thiotepa, busulfan and cyclophosphamide is an effective conditioning regimen for childhood myeloid malignancies and may be tested in patients with less advanced disease (eg CR1).

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Authors and Affiliations

  1. Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
    LL Worth, D Petropoulos, SC Culbert, CA Mullen, WM Roberts & KW Chan
  2. Division of Pharmacy, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
    H Tran
  3. Division of Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
    D Przepiorka

Authors

  1. LL Worth
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  2. H Tran
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  3. D Petropoulos
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  4. SC Culbert
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  5. CA Mullen
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  6. WM Roberts
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  7. D Przepiorka
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  8. KW Chan
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Worth, L., Tran, H., Petropoulos, D. et al. Hematopoietic stem cell transplantation for childhood myeloid malignancies after high-dose thiotepa, busulfan and cyclophosphamide.Bone Marrow Transplant 24, 947–952 (1999). https://doi.org/10.1038/sj.bmt.1702016

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