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

Blood
Ruta BrazauskasMary Eapen

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
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
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

❮ Previous
Next ❯

Citations

Jan 21, 2021·Frontiers in Pediatrics·Lakshmanan Krishnamurti
Nov 27, 2020·The Lancet. Haematology·Josu de la FuenteUNKNOWN Paediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation

❮ Previous
Next ❯

Related Concepts

Related Feeds

Allogenic & Autologous Therapies

Allogenic therapies are generated in large batches from unrelated donor tissues such as bone marrow. In contrast, autologous therapies are manufactures as a single lot from the patient being treated. Here is the latest research on allogenic and autologous therapies.

Anemia

Anemia develops when your blood lacks enough healthy red blood cells. Anemia of inflammation (AI, also called anemia of chronic disease) is a common, typically normocytic, normochromic anemia that is caused by an underlying inflammatory disease. Here is the latest research on anemia.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.