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Clinical Trials

Phase-2 trial of an intensified conditioning regimen for allogeneic hematopoietic cell transplant for poor-risk leukemia

Bone Marrow Transplantation volume 46, pages 1256–1262 (2011)Cite this article

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Abstract

Patients with poor-risk leukemia have a high relapse rate despite allogeneic transplant. We report on the phase-2 trial of an intensified allogeneic transplant regimen whose aim was tolerable toxicity and durable remission. Study patients (n=30) had unfavorable first remission cytogenetics, progression from myelodysplasia or active disease due to induction failure or relapse. Conditioning was i.v. BU, targeted to a first-dose plasma area under the curve (AUC) of 700–900 μM min, VP-16 at 30 mg/kg of adjusted ideal body weight and fractionated TBI (FTBI) at 1200 cGy in 10 fractions. GVHD prophylaxis was CsA and mycophenolate mofetil. Regimen-related toxicities (Bearman) included grade II mucositis in 29 patients (97%) and grade III in one patient, grade II–III sinusoidal obstructive syndrome in 2 patients (7%), and grade 2–3 (CTC) skin toxicity in 8 patients (27%). The 30- and 100-day TRMs were 0 and 7% respectively. The median follow-up was 83.7 months (60.7–96.4) for surviving patients. The 5-year overall and disease-free survival was 40% for all patients. Cumulative 5-year relapse incidence (RI) was 23% and TRM was 37%. We have shown promising OS and RI in these poor-risk patients, who typically have few curative options.

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Acknowledgements

This study was supported in part by grants from the National Institute of Health, CA30206 and CA33572. We would like to acknowledge the dedicated nurses of the City of Hope Bone Marrow Unit for excellent care of our patients and support of medical research.

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Authors and Affiliations

  1. Department of Hematology/Hematopoietic Cell Transplantation City of Hope National Medical Center, Duarte, CA, USA

    A S Stein, M R O'Donnell, A P Nademanee, P M Parker, V A Pullarkat, D S Snyder, J C Alvarnas, S H Thomas & S J Forman

  2. Departments of Biostatistics, Pathology, Radiation Oncology, Pharmacology, City of Hope National Medical Center, Duarte, CA, USA

    T W Synold, A C Dagis, A Tsirunyan & J Y C Wong

  3. City of Hope–Kaiser Permanente, Los Angeles, CA, USA

    R T Spielberger

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  1. A S Stein
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Correspondence to A S Stein.

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Stein, A., O'Donnell, M., Synold, T. et al. Phase-2 trial of an intensified conditioning regimen for allogeneic hematopoietic cell transplant for poor-risk leukemia. Bone Marrow Transplant 46, 1256–1262 (2011). https://doi.org/10.1038/bmt.2010.295

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