Characteristics of Donor and Recipient Clonal Hematopoiesis in Nonmyeloablative Transplant for Sickle Cell Disease.
Sickle cell transplant recipients carry elevated cancer-risk mutations at baseline, highlighting the urgent need for monitoring during cellular therapy.
In a prospective NIH HCT cohort of 170 individuals, SCD recipients carried significantly elevated DNA damage response-mutant CH at baseline compared to non-SCD donors and showed post-HCT clonal expansion from multiple origins. Three fatal cases of TP53-mutant MDS/AML underscore the urgent need for CH monitoring in SCD patients receiving cellular therapy.
What the study was
- Study design
- Prospective cohort with matched donor controls and error-corrected DNA sequencing
- Population
- 98 sickle cell disease patients and 72 non-SCD donors undergoing HCT at NIH (2004-2023)
- Sample size
- 170
- Category
- Genomics/Precision Medicine
- Maturity
- Validated
- Journal
- Blood
Why it surfaced
Prospective NIH cohort characterizing DDR-mutant CH risk in SCD patients at curative HCT; identifies novel MDS/AML precursor pattern with immediate implications for monitoring and cellular therapy safety screening.
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