期刊
BLOOD
卷 136, 期 5, 页码 623-626出版社
AMER SOC HEMATOLOGY
DOI: 10.1182/blood.2020005687
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资金
- National Cancer Institute [U24-CA76518]
- National Heart, Lung, and Blood Institute
- National Institute of Allergy and Infectious Diseases, National Institutes of Health
- Health Services Research Administration, Department of Health and Human Services [HHSH 250201200016C]
We developed a risk score to predict event-free survival (EFS) after allogeneic hemato-poietic 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.
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