Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease

Academic Article

Abstract

  • Abstract We developed a risk score to predict event-free survival (EFS) after allogeneic hematopoietic cell transplantation for sickle cell disease. The study population (n = 1425) was randomly split into training (n = 1070) and validation (n = 355) cohorts. Risk factors were identified and validated via Cox regression models. Two risk factors of 9 evaluated were predictive for EFS: age at transplantation and donor type. On the basis of the training cohort, patients age 12 years or younger with an HLA-matched sibling donor were at the lowest risk with a 3-year EFS of 92% (score, 0). Patients age 13 years or older with an HLA-matched sibling donor or age 12 years or younger with an HLA-matched unrelated donor were at intermediate risk (3-year EFS, 87%; score, 1). All other groups, including patients of any age with a haploidentical relative or HLA-mismatched unrelated donor and patients age 13 years or older with an HLA-matched unrelated donor were high risk (3-year EFS, 57%; score, 2 or 3). These findings were confirmed in the validation cohort. This simple risk score may guide patients with sickle cell disease and hematologists who are considering allogeneic transplantation as a curative treatment relative to other available contemporary treatments.
  • Authors

    Published In

  • Blood  Journal
  • Digital Object Identifier (doi)

    Pubmed Id

  • 21392116
  • Author List

  • Brazauskas R; Scigliuolo GM; Wang H-L; Cappelli B; Ruggeri A; Fitzhugh CD; Hankins JS; Kanter J; Meerpohl JJ; Panepinto JA
  • Start Page

  • 623
  • End Page

  • 626
  • Volume

  • 136
  • Issue

  • 5