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

Ruta Brazauskas, Graziana M. Scigliuolo, Hai Lin Wang, Barbara Cappelli, Annalisa Ruggeri, Courtney D. Fitzhugh, Jane S. Hankins, Julie Kanter, Joerg J. Meerpohl, Julie A. Panepinto, Damiano Rondelli, Shalini Shenoy, Mark C. Walters, John E. Wagner, John F. Tisdale, Eliane Gluckman, Mary Eapen

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
Pages (from-to)623-626
Number of pages4
JournalBlood
Volume136
Issue number5
DOIs
StatePublished - Jul 30 2020

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    Brazauskas, R., Scigliuolo, G. M., Wang, H. L., Cappelli, B., Ruggeri, A., Fitzhugh, C. D., Hankins, J. S., Kanter, J., Meerpohl, J. J., Panepinto, J. A., Rondelli, D., Shenoy, S., Walters, M. C., Wagner, J. E., Tisdale, J. F., Gluckman, E., & Eapen, M. (2020). Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood, 136(5), 623-626. https://doi.org/10.1182/blood.2020005687