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

Blood. 2020 Jul 30;136(5):623-626. doi: 10.1182/blood.2020005687.

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Sickle Cell / genetics
  • Anemia, Sickle Cell / mortality*
  • Anemia, Sickle Cell / therapy*
  • Blood Grouping and Crossmatching
  • Child
  • Child, Preschool
  • Female
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality*
  • Histocompatibility Antigens Class I / genetics
  • Humans
  • Male
  • Middle Aged
  • Progression-Free Survival
  • Risk Factors
  • Transplantation, Homologous / mortality
  • Treatment Outcome
  • Young Adult

Substances

  • Histocompatibility Antigens Class I