Increased prevalence of clonal hematopoiesis in children with sickle cell disease

J Jessica Ulloa (1Human Genetics Program, Vanderbilt Genetics Institute, Vanderbilt University, Nashville, TN) K Kristin Wuichet (2Division of Genetic Medicine, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN) S Sara R. Rashkin (3Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN) Y Yash Pershad C Connor Shore (2Division of Genetic Medicine, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN) C Caitlyn Vlasschaert (4Department of Medicine, Queen’s University, Kingston, ON, Canada) M Mark Rodeghier (5Rodeghier Consulting, Chicago, IL) Y Yu Yao (Key Laboratory for Advanced Materials and Joint International Research Laboratory of Precision Chemistry and Molecular Engineering, Feringa Nobel Prize Scientist Joint Research Center, Frontiers Science Center for Materiobiology and Dynamic Chemistry, Institute of Fine Chemicals, School of Chemistry and Molecular Engineering) V Victor R. Gordeuk B Binal N. Shah (6Division of Hematology and Oncology, University of Illinois Chicago, Chicago, IL) C Clifford M. Takemoto (3Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN) S Santosh L. Saraf (1Comprehensive Sickle Cell Center, Division of Hematology and Oncology, Department of Medicine, University of Illinois Chicago, Chicago, IL) M Michael R. DeBaun (7Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN) M Mitchell J. Weiss A Alexander G. Bick

Abstract

Abstract Recent studies have reached opposing conclusions about whether clonal hematopoiesis (CH) is increased or decreased in patients with sickle cell disease (SCD). Given that CH is typically age-related, its presence in children with SCD could offer unique insights into early-life mutagenesis and disease-related stressors. We tested the primary and secondary hypotheses that children with SCD would have a higher prevalence of CH than age-, sex-, and race-matched children without SCD and that children with hydroxyurea would have a higher CH prevalence than children not treated with hydroxyurea. To address this, we conducted a cross-sectional study in 2 independent cohorts of children aged 0 to 18 years with SCD (N = 1025 and N = 1293, respectively) and a 2957-person matched comparison group. Using a highly sensitive, error-corrected sequencing assay capable of detecting CH at a variant allele frequency of ≥0.5%, we found that children with SCD have a significantly higher prevalence of CH than the comparison group (odds ratio [OR], 4.2; P = 7.4 × 10−13). In addition, CH was not associated with exposure to hydroxyurea therapy (OR, 0.76; P = .44).

Article Details

Journal Blood
Volume / Issue Vol. 147, Issue 13
Published March 26, 2026
Pages 1447-1455
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (15)

J

Jessica Ulloa

1Human Genetics Program, Vanderbilt Genetics Institute, Vanderbilt University, Nashville, TN

K

Kristin Wuichet

2Division of Genetic Medicine, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN

S

Sara R. Rashkin

3Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN

Y

Yash Pershad

C

Connor Shore

2Division of Genetic Medicine, Department of Medicine, Vanderbilt University Medical Center, Nashville, TN

C

Caitlyn Vlasschaert

4Department of Medicine, Queen’s University, Kingston, ON, Canada

M

Mark Rodeghier

5Rodeghier Consulting, Chicago, IL

Y

Yu Yao

Key Laboratory for Advanced Materials and Joint International Research Laboratory of Precision Chemistry and Molecular Engineering, Feringa Nobel Prize Scientist Joint Research Center, Frontiers Science Center for Materiobiology and Dynamic Chemistry, Institute of Fine Chemicals, School of Chemistry and Molecular Engineering

V

Victor R. Gordeuk

B

Binal N. Shah

6Division of Hematology and Oncology, University of Illinois Chicago, Chicago, IL

C

Clifford M. Takemoto

3Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN

S

Santosh L. Saraf

1Comprehensive Sickle Cell Center, Division of Hematology and Oncology, Department of Medicine, University of Illinois Chicago, Chicago, IL

M

Michael R. DeBaun

7Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN

M

Mitchell J. Weiss

A

Alexander G. Bick