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

Research output: Contribution to journalArticle

113 Scopus citations

Abstract

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
Volume35
Issue number4
DOIs
StatePublished - Jan 1 2005

Keywords

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

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