Clinical outcomes following bone marrow transplantation in patients with sickle cell disease: A cohort study of US Medicaid enrollees

Objectives Bone marrow transplantation (BMT) is currently the only curative therapy available for patients with sickle cell disease (SCD), but clinical outcomes in routine care are not well understood. We describe the rates of vaso‐occlusive crises (VOCs), transplant complications, and mortality in...

Full description

Saved in:
Bibliographic Details
Published in:European journal of haematology 2021-02, Vol.106 (2), p.273-280
Main Authors: Mahesri, Mufaddal, Schneeweiss, Sebastian, Globe, Denise, Mutebi, Alex, Bohn, Rhonda, Achebe, Maureen, Levin, Raisa, Desai, Rishi J.
Format: Article
Language:English
Subjects:
Bone marrow
Bone marrow transplantation
Clinical outcomes
Cohort analysis
epidemiology
Graft-versus-host reaction
Medicaid
Mortality
pediatric hematology
Sickle cell disease
Transplants & implants
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Objectives Bone marrow transplantation (BMT) is currently the only curative therapy available for patients with sickle cell disease (SCD), but clinical outcomes in routine care are not well understood. We describe the rates of vaso‐occlusive crises (VOCs), transplant complications, and mortality in SCD patients after BMT. Methods A cohort study of SCD patients who underwent BMT was designed using US Medicaid claims data (2000‐2013). Results A total of 204 SCD patients undergoing BMT were identified with a mean (SD) age of 10.6 (7.3) years, with 52.9% male and 67.6% African American. The overall VOC rate was 0.99 per person‐year (95% CI: 0.91‐1.07) over a median follow‐up time of 2.1 years (IQR: 0.8‐4.3 years). A total of 138 (67.6%) remained free of VOCs. The mortality rate was 1.7 (95% CI: 0.9‐3.1) per 100 person‐years, transplant‐related complications occurred among 113 (55.4%) patients with an incidence rate of 38.2 (95% CI: 31.7‐45.9) per 100 person‐years, while 47 (23%) patients had GvHD with an incidence rate of 8.0 (95% CI: 6.0‐10.7) per 100 person‐years. Conclusion Two thirds of the BMT recipients remained VOC‐free over 2 years of follow‐up, but transplant‐related complications, including GvHD occurred with high frequency. This highlights a continuing unmet need for alternative curative interventions in SCD.
ISSN:0902-4441
1600-0609
DOI:10.1111/ejh.13546