1. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease
- Author
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Mary Eapen, Ruta Brazauskas, Damiano Rondelli, Barbara Cappelli, Courtney D. Fitzhugh, Julie Kanter, Hai-Lin Wang, Jane S. Hankins, Julie A. Panepinto, John E. Wagner, Shalini Shenoy, Mark C. Walters, Eliane Gluckman, Graziana Maria Scigliuolo, John F. Tisdale, Joerg J Meerpohl, and Annalisa Ruggeri more...
- Subjects
Adult ,Male ,medicine.medical_specialty ,Allogeneic transplantation ,Adolescent ,Immunology ,Anemia, Sickle Cell ,Biochemistry ,Young Adult ,Risk Factors ,Internal medicine ,medicine ,Humans ,Transplantation, Homologous ,Progression-free survival ,Young adult ,Child ,Transplantation ,Framingham Risk Score ,Proportional hazards model ,business.industry ,Histocompatibility Antigens Class I ,Hematopoietic Stem Cell Transplantation ,Cell Biology ,Hematology ,Middle Aged ,medicine.disease ,Progression-Free Survival ,Sickle cell anemia ,Treatment Outcome ,Blood Grouping and Crossmatching ,Child, Preschool ,Population study ,Female ,business - 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. more...
- Published
- 2020
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