A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders

S. Shenoy, W. J. Grossman, J. DiPersio, L. C. Yu, D. Wilson, Y. J. Barnes, T. Mohanakumar, A. Rao, R. J. Hayashi

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113 Scopus citations


Bone marrow transplantation (BMT) benefits nonmalignant diseases but is limited by regimen-related toxicity, graft-versus-host disease (GVHD), donor availability, and graft rejection (GR). To overcome some of these barriers, we developed a new conditioning strategy for these patients. In total, 16 patients received Campath-1H (33/48 mg; days -21 to -19), fludarabine (150 mg/m2; days -8 to -4), melphalan (140/70 mg/m2; day -3), and transplant using related/unrelated stem cells. GVHD prophylaxis included cyclosporine/methylprednisolone for cord cells. Other recipients also received methotrexate. Risk factors for GR included multiple transfusions (6), low stem cell numbers (1), and immunologic/metabolic disorders (3). Donor engraftment was present in 14/16 recipients. Neutrophils (ANC >0.5 × 109/l) and platelets (>50 × 109/l) engrafted at a median of 13 and 24 days. Two patients died of Pseudomonas sepsis prior to engraftment, one of CMV disease, and another of intracranial hemorrhage. With median follow-up of 281 days (78-907), 12/16 are stable/improved, or cured. Acute GVHD was absent (n = 10) or mild and transient (grade 1-2 skin) (n = 4). There was no chronic GVHD. Toxicities were predominantly early infections within 100 days, and correlated with lymphopenia (CD4+ T and B cells). Stable engraftment and low incidence of significant GVHD, irrespective of age or stem cell source, make this reduced-intensity regimen attractive for nonmalignant disorders.

Original languageEnglish
Pages (from-to)345-352
Number of pages8
JournalBone Marrow Transplantation
Issue number4
StatePublished - Jan 1 2005


  • Campath-1H
  • Metabolic disorders
  • Nonmalignant
  • Reduced-intensity transplant regimen
  • Stem cell

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