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 contempora...Continue Reading
References
Dec 31, 2005·The New England Journal of Medicine·Robert J AdamsUNKNOWN Optimizing Primary Stroke Prevention in Sickle Cell Anemia (STOP 2) Trial Investigators
Nov 10, 2009·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Andrea BacigalupoMary Horowitz
Mar 3, 2010·Blood·Charles T QuinnGeorge R Buchanan
May 4, 2010·The Journal of Pediatrics·Henrietta Enninful-EghanJanet L Kwiatkowski
Oct 14, 2010·Blood·Monica L HulbertMichael R Debaun
Feb 10, 2012·Blood·Russell E WareUNKNOWN SWiTCH Investigators
Jul 6, 2014·PloS One·Mark T GladwinUNKNOWN walk-PHaSST Investigators and Patients
Aug 21, 2014·The New England Journal of Medicine·Michael R DeBaunJames F Casella
Mar 17, 2015·Bone Marrow Transplantation·S J RotzJ A Little
Dec 17, 2015·Lancet·Russell E WareRobert J Adams
Jan 21, 2017·Haematologica·Poulami MaitraKenneth I Ataga
Jan 4, 2018·Blood Advances·Courtney D FitzhughJohn F Tisdale
Jan 4, 2018·Blood Advances·Michael R DeBaun, Ellen Wright Clayton
Dec 1, 2018·Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation·Josu de la FuenteAdetola A Kassim
Dec 12, 2018·Blood·Michael R DeBaunKenneth I Ataga
Mar 18, 2019·The Lancet. Haematology·Javier Bolaños-MeadeRobert A Brodsky
Sep 10, 2019·The Lancet. Haematology·Mary EapenJohn F Tisdale
Citations
Jan 21, 2021·Frontiers in Pediatrics·Lakshmanan Krishnamurti
Apr 9, 2021·Transplantation and Cellular Therapy·Madiha IqbalMohamed A Kharfan-Dabaja
Nov 27, 2020·The Lancet. Haematology·Josu de la FuenteUNKNOWN Paediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation
May 5, 2021·Bone Marrow Transplantation·Lisa V E OostenbrinkAlexander B Mohseny
Jul 19, 2021·Bone Marrow Transplantation·Tami D JohnKathryn Leung
Aug 2, 2021·Blood Reviews·Emanuela CimpeanuSeah H Lim
Aug 31, 2021·Transplantation and Cellular Therapy·Helen E HeslopMary M Horowitz
Dec 11, 2021·Hematology·Lakshmanan Krishnamurti
Dec 11, 2021·Hematology·Niketa Shah, Lakshmanan Krishnamurti