Investigating Access to, and Impact of, Hematopoietic Cell Transplant on Morbidity and Mortality of Children with Sickle Cell Disease Open Access

Abstract

Background: Sickle cell disease (SCD) is associated with morbidity and midlife mortality. Hematopoietic cell transplant (HCT) is a curative therapy that can stabilize or prevent sickle-related organ dysfunction but can incur life-threatening complications. More information is needed about the long-term benefits of HCT over standard medical therapy for SCD.  

 

Objective: To compare long-term outcomes between patients with SCD who underwent HCT and those who did not and to explore barriers to HCT for patients with SCD who did not proceed to HCT.

 

Methods: This IRB-approved, retrospective, single institution, cohort study of patients with SCD with or without HCT, matched 1:2. Cases included all patients who underwent HCT between 2010 and 2018. Controls were randomly matched on age, sex, disease genotype, and disease severity using transplant date as the match timepoint. Kidney function was compared between the two groups at pre-HCT and at 1-, 2-, 3-, and 5-years post-HCT. The number of hospitalizations in the 5 years pre-HCT (averaged) and in the 1-, 2-, 3-, and 5-years post-HCT were compared between the groups using Wilcoxon signed-rank tests. Overall survival at 5-years post-HCT was compared between the groups using the Kaplan-Meier analysis.

 

Results: Fifty-eight patients who underwent HCT were matched with 116 controls who continued with standard medical therapy. The median age of the cohort was 8 years (IQR=5-11), and 53% of the cohort were female. While there were differences in available data in renal function between the groups at some timepoints, these data were not evaluable due to the proportion of missing data. Greater than 1-year post-HCT, unscheduled hospitalizations were significantly decreased in the HCT group compared to the non-HCT group (p<0.001). Two patients in the HCT group died during the study period compared to 1 patient in the non-HCT group (p=0.215).

 

Conclusion: After 1-year post-HCT, the frequency of unscheduled hospitalizations was significantly lower for children who underwent HCT. There was no difference in survival between the two groups. Our data support the need for further study of the impact of HCT on other organ outcomes and longer-term follow-up to better assess survival and organ function in children with SCD.

Table of Contents

A.   BACKGROUND……………………………………………………………………………………………9

B.   METHODS………………………………………………………………………………………………...15

C.   RESULTS………………………………………………………………………………………………..…22

D.  DISCUSSION…………………………………………………………………………………………….29

E.   REFERENCES…………………………………………………………………………………………...36

F.   TABLES/FIGURES…………………………………………………………………………………….41

About this Master's Thesis

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School	Laney Graduate School
Department	Clinical Research
Degree	M.S.
Submission	Master's Thesis
Language	English
Research Field	Health Sciences, Medicine and Surgery
Keyword	Pediatrics Hematopoietic cell transplant (HCT) Sickle Cell Disease (SCD)
Committee Chair / Thesis Advisor	Amita Manatunga, Emory University Jordan Kempker, Emory University
Committee Members	Kirsten Williams, Emory University Ross Fasano, Emory University

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