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

doi:10.1182/blood.2020005687

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

Pediatrics - Blood/Marrow Transplant

Research output: Contribution to journal › Article › peer-review

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 5 1425) was randomly split into training (n 5 1070) and validation (n 5 355) 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 language	English (US)
Pages (from-to)	623-626
Number of pages	4
Journal	Blood
Volume	136
Issue number	5
DOIs	https://doi.org/10.1182/blood.2020005687
State	Published - Jul 30 2020

Bibliographical note

Funding Information:
The Center for International Blood and Marrow Transplant Research is supported by grant U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases, National Institutes of Health, and by the Health Services Research Administration, Department of Health and Human Services (HHSH 250201200016C).

Funding Information:
The Center for International Blood and Marrow Transplant Research is supported by grant U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases, National Institutes of Health, and by the Health Services Research Administration, Department of Health and Human Services (HHSH 250201200016C). The content is solely the responsibility of the authors and does not represent the official policy of the National Institutes of Health or the Health Resources and Services Administration or any other agency of the US government.

Publisher Copyright:
© 2020 American Society of Hematology. All rights reserved.

Access

10.1182/blood.2020005687

Fingerprint Dive into the research topics of 'Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease'. Together they form a unique fingerprint.

Cite this

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

Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. / Brazauskas, Ruta; Scigliuolo, Graziana M.; Wang, Hai Lin; Cappelli, Barbara; Ruggeri, Annalisa; Fitzhugh, Courtney D.; Hankins, Jane S.; Kanter, Julie; Meerpohl, Joerg J.; Panepinto, Julie A.; Rondelli, Damiano; Shenoy, Shalini; Walters, Mark C.; Wagner, John E.; Tisdale, John F.; Gluckman, Eliane; Eapen, Mary.

In: Blood, Vol. 136, No. 5, 30.07.2020, p. 623-626.

Research output: Contribution to journal › Article › peer-review

Brazauskas, R, Scigliuolo, GM, Wang, HL, Cappelli, B, Ruggeri, A, Fitzhugh, CD, Hankins, JS, Kanter, J, Meerpohl, JJ, Panepinto, JA, Rondelli, D, Shenoy, S, Walters, MC, Wagner, JE, Tisdale, JF, Gluckman, E & Eapen, M 2020, 'Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease', Blood, vol. 136, no. 5, pp. 623-626. https://doi.org/10.1182/blood.2020005687

Brazauskas, Ruta ; Scigliuolo, Graziana M. ; Wang, Hai Lin ; Cappelli, Barbara ; Ruggeri, Annalisa ; Fitzhugh, Courtney D. ; Hankins, Jane S. ; Kanter, Julie ; Meerpohl, Joerg J. ; Panepinto, Julie A. ; Rondelli, Damiano ; Shenoy, Shalini ; Walters, Mark C. ; Wagner, John E. ; Tisdale, John F. ; Gluckman, Eliane ; Eapen, Mary. / Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. In: Blood. 2020 ; Vol. 136, No. 5. pp. 623-626.

@article{7ac4322cbe674d34bde59f9f06acc08f,

title = "Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease",

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 5 1425) was randomly split into training (n 5 1070) and validation (n 5 355) 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.",

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

note = "Funding Information: The Center for International Blood and Marrow Transplant Research is supported by grant U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases, National Institutes of Health, and by the Health Services Research Administration, Department of Health and Human Services (HHSH 250201200016C). Funding Information: The Center for International Blood and Marrow Transplant Research is supported by grant U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases, National Institutes of Health, and by the Health Services Research Administration, Department of Health and Human Services (HHSH 250201200016C). The content is solely the responsibility of the authors and does not represent the official policy of the National Institutes of Health or the Health Resources and Services Administration or any other agency of the US government. Publisher Copyright: {\textcopyright} 2020 American Society of Hematology. All rights reserved.",

year = "2020",

month = jul,

day = "30",

doi = "10.1182/blood.2020005687",

language = "English (US)",

volume = "136",

pages = "623--626",

journal = "Blood",

issn = "0006-4971",

publisher = "American Society of Hematology",

number = "5",

}

TY - JOUR

T1 - Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease

AU - Brazauskas, Ruta

AU - Scigliuolo, Graziana M.

AU - Wang, Hai Lin

AU - Cappelli, Barbara

AU - Ruggeri, Annalisa

AU - Fitzhugh, Courtney D.

AU - Hankins, Jane S.

AU - Kanter, Julie

AU - Meerpohl, Joerg J.

AU - Panepinto, Julie A.

AU - Rondelli, Damiano

AU - Shenoy, Shalini

AU - Walters, Mark C.

AU - Wagner, John E.

AU - Tisdale, John F.

AU - Gluckman, Eliane

AU - Eapen, Mary

N1 - Funding Information: The Center for International Blood and Marrow Transplant Research is supported by grant U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases, National Institutes of Health, and by the Health Services Research Administration, Department of Health and Human Services (HHSH 250201200016C). Funding Information: The Center for International Blood and Marrow Transplant Research is supported by grant U24-CA76518 from the National Cancer Institute, the National Heart, Lung, and Blood Institute, and the National Institute of Allergy and Infectious Diseases, National Institutes of Health, and by the Health Services Research Administration, Department of Health and Human Services (HHSH 250201200016C). The content is solely the responsibility of the authors and does not represent the official policy of the National Institutes of Health or the Health Resources and Services Administration or any other agency of the US government. Publisher Copyright: © 2020 American Society of Hematology. All rights reserved.

PY - 2020/7/30

Y1 - 2020/7/30

N2 - We developed a risk score to predict event-free survival (EFS) after allogeneic hematopoietic cell transplantation for sickle cell disease. The study population (n 5 1425) was randomly split into training (n 5 1070) and validation (n 5 355) 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.

AB - We developed a risk score to predict event-free survival (EFS) after allogeneic hematopoietic cell transplantation for sickle cell disease. The study population (n 5 1425) was randomly split into training (n 5 1070) and validation (n 5 355) 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.

UR - http://www.scopus.com/inward/record.url?scp=85089126120&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85089126120&partnerID=8YFLogxK

U2 - 10.1182/blood.2020005687

DO - 10.1182/blood.2020005687

M3 - Article

C2 - 32518950

AN - SCOPUS:85089126120

VL - 136

SP - 623

EP - 626

JO - Blood

JF - Blood

SN - 0006-4971

IS - 5

ER -

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

Abstract

Bibliographical note

Access

Other files and links

Cite this