Long Term Organ Stability and Outcome in Children and Adolescents With Sickle Cell Disease (SCD) Following Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation (SCT)
Year: 2011
Type: article
Abstract: Myeloablative (MA) SCT is curative of SCD, and children with stable donor engraftment no longer experience SCD symptoms (Walters et al, BBMT 2009), but quality of life may be diminished by MA SCT related organ toxicity. RTC regimens may potentially enhance preservation of organ function in patients with SCD. Between 8/27/2004 and 3/2/10, 18 (16M: 2F) patients with symptomatic SCD underwent RTC SCT. Conditioning was Busulfan (4mg/kg × 4d ≤ 4 yrs and 12.8mg/kg × 4d > 4 yrs), Fludarabine (30mg/m2 × 6d), and Alemtuzumab (2mg/m2 × 1d, 6mg/m2 × 2d, and 20mg/m2 × 2d).
Institutions: Columbia University, University of California, San Francisco
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2095997020