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Clinical Trial
. 2024 Aug 8;144(6):672-675.
doi: 10.1182/blood.2023023028.

Impact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease

Akshay Sharma  1 , Subodh Selukar  2 , Yu Bi  2 , Anthony Merlocco  3   4   5 , Cara E Morin  6 , Chris Goode  5 , Parul Rai  7 , Jeffrey A Towbin  3 , Jane S Hankins  7   8 , Stephen Gottschalk  1 , Brandon Triplett  1 , Jason N Johnson  2   3   4
Affiliations
Clinical Trial

Impact of hematopoietic cell transplantation on myocardial fibrosis in young patients with sickle cell disease

Akshay Sharma et al. Blood. .

Abstract

Serial cardiovascular magnetic resonance evaluation of children and young adults with SCD who underwent hematopoietic cell transplantation showed mean ECV, representing diffuse myocardial fibrosis, decreased 3.4% from baseline to 12 months posttransplantation. This trial was registered at www.clinicaltrials.gov as #NCT04362293.

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Conflict of interest statement

Conflict-of-interest disclosure: This content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

A.S. has received consultant fees from Spotlight Therapeutics, Medexus Inc, Vertex Pharmaceuticals, Sangamo Therapeutics, and Editas Medicine, is a medical monitor for the Conditioning SCID Infants Diagnosed Early (CSIDE) clinical trial for which he receives financial compensation, and has also received research funding from CRISPR Therapeutics and honoraria from Vindico Medical Education. A.S. is the St. Jude Children’s Research Hospital site principal investigator of clinical trials for genome editing of sickle cell disease sponsored by Vertex Pharmaceuticals/CRISPR Therapeutics (NCT03745287), Novartis Pharmaceuticals (NCT04443907), and Beam Therapeutics (NCT05456880). The industry sponsors provide funding for the clinical trial, which includes salary support paid to A.S.’s institution. A.S. has no direct financial interest in these therapies. S.G. is a coinventor on patent applications in the fields of cell or gene therapy for cancer, a member of the Scientific Advisory Board of Be Biopharma and CARGO, a member of the Data and Safety Monitoring Board of Immatics, and has received honoraria from TESSA Therapeutics within the past year. The remaining authors declare no competing financial interests.

Figures

Figure 1.
Figure 1.
Significant decrease in ECV from baseline to 1- and 12-month post-HCT. The gray points connected by gray lines are the individual values of the ECV at the baseline, 1-month, and 12-month time point for each participant. The genotype is denoted by solid lines with circles (HbSS) or dashed lines with triangles (HbSC). The red dots and red line represent the mean of these ECV values, and the red shaded area connects the 95% confidence intervals for the mean at baseline, 1 month after HCT, and 12 months after HCT. The gray rectangular area next to the vertical axis represents the normal range for ECV levels. The P value shown (.005) is from a likelihood ratio test from the linear mixed effects model to assess whether mean ECV differed from baseline at 1 and/or 12 months. The time point–specific paired t test P values are .061 and .002 for the 1- and 12-month comparisons with baseline, respectively.
See this image and copyright information in PMC

Comment in

References

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